[Senate Hearing 106-413]
[From the U.S. Government Publishing Office]
S. Hrg. 106-413
STEM CELL RESEARCH, PART 3
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HEARINGS
before a
SUBCOMMITTEE OF THE
COMMITTEE ON APPROPRIATIONS UNITED STATES SENATE
ONE HUNDRED SIXTH CONGRESS
SECOND SESSION
__________
SPECIAL HEARINGS
APRIL 26, 2000--WASHINGTON, DC
SEPTEMBER 7, 2000--WASHINGTON, DC
SEPTEMBER 14, 2000--WASHINGTON, DC
__________
Printed for the use of the Committee on Appropriations
Available via the World Wide Web: http://www.access.gpo.gov/congress/
senate
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COMMITTEE ON APPROPRIATIONS
TED STEVENS, Alaska, Chairman
THAD COCHRAN, Mississippi ROBERT C. BYRD, West Virginia
ARLEN SPECTER, Pennsylvania DANIEL K. INOUYE, Hawaii
PETE V. DOMENICI, New Mexico ERNEST F. HOLLINGS, South Carolina
CHRISTOPHER S. BOND, Missouri PATRICK J. LEAHY, Vermont
SLADE GORTON, Washington FRANK R. LAUTENBERG, New Jersey
MITCH McCONNELL, Kentucky TOM HARKIN, Iowa
CONRAD BURNS, Montana BARBARA A. MIKULSKI, Maryland
RICHARD C. SHELBY, Alabama HARRY REID, Nevada
JUDD GREGG, New Hampshire HERB KOHL, Wisconsin
ROBERT F. BENNETT, Utah PATTY MURRAY, Washington
BEN NIGHTHORSE CAMPBELL, Colorado BYRON L. DORGAN, North Dakota
LARRY CRAIG, Idaho DIANNE FEINSTEIN, California
KAY BAILEY HUTCHISON, Texas RICHARD J. DURBIN, Illinois
JON KYL, Arizona
Steven J. Cortese, Staff Director
Lisa Sutherland, Deputy Staff Director
James H. English, Minority Staff Director
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Subcommittee on Labor, Health and Human Services, and Education, and
Related Agencies
ARLEN SPECTER, Pennsylvania, Chairman
THAD COCHRAN, Mississippi TOM HARKIN, Iowa
SLADE GORTON, Washington ERNEST F. HOLLINGS, South Carolina
JUDD GREGG, New Hampshire DANIEL K. INOUYE, Hawaii
LARRY CRAIG, Idaho HARRY REID, Nevada
KAY BAILEY HUTCHISON, Texas HERB KOHL, Wisconsin
TED STEVENS, Alaska PATTY MURRAY, Washington
JON KYL, Arizona DIANNE FEINSTEIN, California
ROBERT C. BYRD, West Virginia
(Ex officio)
Professional Staff
Bettilou Taylor
Mary Dietrich
Jim Sourwine
Ellen Murray (Minority)
Administrative Support
Kevin Johnson
Carole Geagley (Minority)
C O N T E N T S
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Wednesday, April 26, 2000
Page
Opening statement of Senator Arlen Specter....................... 1
Statement of Gerald Fischbach, M.D., Director, National Institute
of Neurological Disorders and Stroke, National Institutes of
Health, Department of Health and Human Services................ 2
Prepared statement........................................... 5
Statement of Allen M. Spiegel, M.D., Director, National Institute
of Diabetes and Digestive and Kidney Diseases, National
Institutes of Health, Department of Health and Human Services.. 4
Prepared statement........................................... 5
Opening statement of Senator Tom Harkin.......................... 10
Opening statement of Senator Harry Reid.......................... 11
Opening statement of Senator Patty Murray........................ 13
Statement of Hon. Sam Brownback, U.S. Senator from Kansas........ 15
Prepared statement........................................... 19
Statement of Frank Young, M.D., Ph.D., Former Commissioner, Food
and Drug Administration, Department of Agriculture............. 21
Statement of Mary Jane Owen, M.S.W., executive director, National
Catholic Office for Persons With Disabilities.................. 23
Prepared statement........................................... 26
Statement of Christopher Reeve, actor/director; chairman,
Christopher Reeve Paralysis Foundation......................... 35
Prepared statement........................................... 38
Statement of Jennifer Estess, actor/producer..................... 41
Statement of Lawrence B. Goldstein, Ph.D., professor, Division of
Cellular and Molecular Medicine, University of California, San
Diego School of Medicine; investigator, Howard Hughes Medical
Institute...................................................... 42
Prepared statement........................................... 45
Thursday, September 7, 2000
Opening statement of Senator Arlen Specter....................... 49
Opening statement of Senator Tom Harkin.......................... 51
Statement of Gerald D. Fischbach, M.D., Director, National
Institute of Neurological Disorders and Stroke, National
Institutes of Health, Department of Health and Human Services.. 52
Prepared statement........................................... 55
Statement of Allen M. Spiegel, M.D., Director, National Institute
of Diabetes and Digestive and Kidney Diseases, National
Institutes of Health, Department of Health and Human Services.. 54
Prepared statement........................................... 55
Statement of David A. Prentice, M.D., Ph.D., professor of life
sciences, Indiana State University............................. 61
Prepared statement........................................... 65
Statement of Micheline M. Mathews-Roth, M.D., associate professor
of medicine, Harvard Medical School............................ 67
Prepared statement........................................... 70
Thursday, September 14, 2000
Opening statement of Senator Arlen Specter....................... 81
Statement of Senator Tom Harkin.................................. 82
Statement of Senator Paul D. Wellstone........................... 83
Statement of Richard O. Hynes, Ph.D, director, Center for Cancer
Research, Massachusetts Institute of Technology................ 83
Prepared statement........................................... 85
Statement of Darwin J. Prockop, M.D., Ph.D, director, Center for
Gene Therapy, Tulane University Medical Center................. 87
Prepared statement........................................... 89
Statement of Ron Heagy, president and founder of the Life is an
Attitude Foundation............................................ 93
Statement of Russell Saltzman, pastor, Ruskin Heights Lutheran
Church, Kansas City, MO........................................ 95
Prepared statement........................................... 96
Statement of Anton-Lewis Usala, M.D., chairman/chief technical
officer, Encelle, Inc., Greenville, NC......................... 98
Prepared statement........................................... 100
Statement of Gina Gershon, actress............................... 110
Statement of Jennifer Estess, actor/producer..................... 111
Statement of Mary Tyler Moore, international chairman, Juvenile
Diabetes Foundation............................................ 113
Prepared statement........................................... 115
Statement of Michael J. Fox, Foundation for Parkinson's Research. 117
FEDERAL FUNDING FOR STEM CELL RESEARCH
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WEDNESDAY, APRIL 26, 2000
U.S. Senate,
Subcommittee on Labor, Health and Human
Services, and Education, and Related Agencies,
Committee on Appropriations,
Washington, DC.
The subcommittee met at 11:02 a.m., in room SH-216, Hart
Senate Office Building, Hon. Arlen Specter (chairman)
presiding.
Present: Senators Specter, Harkin, Reid, and Murray.
OPENING STATEMENT OF SENATOR ARLEN SPECTER
Senator Specter. Good morning, ladies and gentlemen. The
hearing of the Appropriations Subcommittee on Labor, Health and
Human Services, and Education will now proceed.
We are taking up a very important subject today, and that
is the issue of Federal funding for stem cell research. This is
the fifth hearing which the subcommittee is holding on this
very important subject, and we do it as a preliminary to action
on the Senate floor on this subject, which will be taken up in
the course of the next several weeks.
When the medical possibilities of stem cells was noted in
November of 1998, this subcommittee very promptly scheduled a
hearing in December of 1998 to take up the issue of the private
research which had been done showing that stem cells had
enormous potential for many diseases, Parkinson's, Alzheimer's,
amyotrophic lateral sclerosis, known as Lou Gehrig's disease,
possibly implications for heart disease.
And subsequent hearings were held, one on the subject of
the proprietary interest in these patents. A very major
question arises as to whether this is something which ought to
be in the private domain or ought to be in the public domain.
And we have had hearings which have taken up the ethical and
moral considerations. And there is a very profound debate which
has been undertaken on this very, very important subject.
My analysis has been that the use of discarded embryos
would not affect human life, that in in vitro fertilization,
there are many embryos taken, and if there is any possibility
of human life, I would be the first to oppose any use for
medical research if potential human life were to be involved.
My analysis and study has demonstrated that the discarded
embryos are not going to be used for human life, so it is not a
question of taking human life or the risk of taking human life,
but the potential for saving life. I believe that stem cell
research has the potential for a veritable fountain of youth.
And there are other views and other views must be taken
into account and will be considered as the Congress considers
the issue of whether the current ban on use of Federal funding
for embryonic research will take place.
The General Counsel for the Department of Health and Human
Services has rendered a ruling that Federal funding may be used
on the stem cells once extracted from the embryos, but not on
the embryos themselves. And there is a substantial body of
medical evidence which says that is not sufficient, that the
embryos really need to be used.
This issue has a corollary on fetal tissue which had been
banned for many years, and after extensive consideration, fetal
tissue is now used for medical research. There had been a
concern that the use of fetal tissue would encourage abortions,
and I think those fears were finally allayed. Senator Thurmond
was a key vote on that matter, and I think persuaded many in
the Senate when Senator Thurmond voted for the use of fetal
tissue for medical purposes in a very close personal matter
which his daughter having juvenile diabetes, given his deep
respect for human life, as I also have that deep respect and I
think we all do, but now fetal tissue is used for medical
research because the conclusion was reached that it does not
promote or encourage abortions.
We had inserted a provision in the appropriations bill last
fall to eliminate the ban on Federal funding, and when it
appeared that that would tie up the appropriations bill, we
removed that provision with the understanding reached with our
distinguished Majority Leader Senator Lott that the Senate
would take up the bill as a freestanding bill, which was
introduced in January by Senator Harkin and myself on
bipartisan support.
We will now proceed with our first panel: Dr. Gerald
Fischbach and Dr. Allen Spiegel. If you gentlemen would step
forward.
STATEMENT OF GERALD FISCHBACH, M.D., DIRECTOR, NATIONAL
INSTITUTE OF NEUROLOGICAL DISORDERS AND
STROKE, NATIONAL INSTITUTES OF HEALTH,
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Senator Specter. Dr. Fischbach was appointed Director of
the National Institute of Neurological Disorders and Stroke in
1998. From 1990 until 1998, he served as Director of the
Neurobiology Departments at the Harvard Medical School and
Massachusetts General Hospital. He has also been Professor of
Neurobiology and head of the Department of Anatomy and
Neurobiology at Washington University School of Medicine.
Thank you for joining us, Dr. Fischbach, and we look
forward to your testimony.
Dr. Fischbach. Thank you, Mr. Chairman.
In the next 2 or 3 minutes I would like to give you a brief
statement with a definition of stem cells, their general
promise, and then their promise in the area of my expertise in
brain science.
As you know, stem cells are unique in that they have a
capability of self-renewal. They can give rise to many cells of
the same type, but they also have the very special property of
giving rise to unique highly specialized cells, such as heart
cells, muscle cells, nerve cells, and pancreas cells under the
right conditions.
There is a hierarchy of stem cells. Some stem cells are
more limited in their capacity to proliferate and to give rise
to different types of cells than others. Much like seeds in the
woods, some seeds can give rise to a few trees with a limited
number of branches and potential avenues of growth, whereas
other seeds can populate a whole forest and can give rise to
trees with an enormously elaborate set of branches and arbors.
The pluripotent human stem cells, which we will discuss
further, are in the latter class according to current
scientific information. They have the broadest potential for
renewal and the broadest potential for specialization at the
same time.
Stem cells have enormous promise in four major areas. The
one that really brings us here today, that has ignited patient
advocacy communities and inspired scientists, is the ability of
stem cells to repair damaged tissue, to replace cells that have
become dysfunctional. And while Dr. Spiegel and I are here
representing two organs, the pancreas and the brain, I know you
realize that virtually every institute at the NIH has a deep
interest in stem cell research for replacing various tissues
throughout the body. The potential in this regard is exciting
and unlimited, and I think it is the reason Science magazine
named stem cells as the breakthrough of the year last year in
all fields, chemistry, physics, and biology.
Stem cells are also promising as a means for discovering
new drugs with modern new assays. They are very important for
understanding the mechanism of disease, and very recently it
has been found that they have enormous potential for delivering
medicines. They seem to track the path of disease cells and
deliver medicines right to the source.
There is nowhere, I believe, a more urgent need for the use
of stem cells than in these devastating neurodegenerative
disorders of the brain. Nerve cells in the brain, by and large,
are a non-renewable resource. When they are damaged or lost,
they cannot, with very, very rare exception, be replaced. In
previous years, there has been hope only for symptomatic, not
for therapeutic interventions. And there are cases now where we
can pinpoint deficits where stem cells have already had great
promise in animal models of human disease, replacing as you
have mentioned, dopamine neurons in Parkinson's disease, motor
neurons in ALS and spinal muscular atrophy, cholinergic neurons
in Alzheimer's disease.
But you have to realize that the brain is probably the most
complex structure in the known universe with over 100 billion
cells of great diversity. So, it is important to choose stem
cells that themselves have the potential for great diversity
and that can repopulate the needed environment. The complexity
of the brain requires team work, so in other disorders, in
repair after stroke, in repair after spinal cord injury, or in
general diseases such as multiple sclerosis, the full diversity
of the stem cell phenotype is needed.
Now, there are problems and there is a need to do
additional research. And I will end with this. These are very
important challenges that many of us feel are on the horizon.
We must understand how to regulate the proliferation of stem
cells. We must learn how to steer them into one branch of that
tree versus another, and we must really learn how to promote
their long-term survival once they are implanted. The hope is
that with Federal funding of the use of stem cells, we can
bring one of the the world's treasures, which I believe is the
American scientific community, funded by the NIH, to bear on
these problems.
Senator Specter. Thank you very much, Dr. Fischbach.
STATEMENT OF ALLEN M. SPIEGEL, M.D., DIRECTOR, NATIONAL
INSTITUTE OF DIABETES AND DIGESTIVE AND
KIDNEY DISEASES, NATIONAL INSTITUTES OF
HEALTH, DEPARTMENT OF HEALTH AND HUMAN
SERVICES
Senator Specter. We turn now to Dr. Allen Spiegel,
appointed Director of the National Institute of Diabetes and
Digestive and Kidney Diseases last year. Prior to his
appointment as Director, Dr. Spiegel had a number of positions
at the Institute, including Director of Intramural Research and
Chief of Metabolic Disease Branch. He holds an M.D. from
Harvard, a bachelors from Columbia.
Thank you for joining us, Dr. Spiegel, and we look forward
to your testimony.
Dr. Spiegel. Thank you, Mr. Chairman. I appreciate the
opportunity to appear before you today to discuss the promise
of research on human pluripotent stem cells.
The research holds great potential for treatment of many of
the diseases within the research mission of the National
Institute of Diabetes and Digestive and Kidney Diseases, but
I'd like to focus my remarks today on the treatment of type 1,
or juvenile, diabetes.
In type 1 diabetes, there is a lack of insulin due to
destruction by the body's own immune system of the insulin-
producing cells in the pancreatic islets. While treatment with
insulin is life-saving, it is not a cure. Our research has
shown that tight control of the blood sugar with insulin
treatment can delay or prevent the complications of diabetes,
such as blindness, and kidney failure, but tight control is
extremely difficult to achieve. In this regard, nothing I could
say would be as eloquent as the testimony you yourself heard
last year from the children who came to Washington as part of
the Children's Congress on Diabetes. The frequent needle sticks
and constant danger of low blood sugar they described are only
some of the difficulties they and their parents must endure
every day and night of their lives.
This is why scientists are working so diligently to find
ways to cure type 1 diabetes. Of the many approaches we are
pursuing toward this goal, the most promising, in my view, is
transplantation of the insulin-producing islet cells. For many
years, this approach had minimal success, but recent advances
in immunology research have led to innovative treatments that
effectively block islet transplant rejection. While the results
in humans are still preliminary, and there is certainly need
for wide replication before we can be sure, islet
transplantation offers the prospect of a real cure for type 1
diabetes.
PREPARED STATEMENT
The very real problem, though, is that the available supply
of pancreases for harvesting islets is completely inadequate
for the hundreds of thousands of patients with type 1 diabetes
who would be candidates for such transplants. It is here that
research on human pluripotent stem cells with their theoretical
ability to provide a limitless source of islet cells is so
important. Some have argued that we could achieve the same goal
by using adult pancreatic stem cells, and I agree that this
line of research must be vigorously pursued. But at this point,
we have no certainty that adult pancreatic stem cells can be
isolated in a practical way, nor that they can replicate to
provide sufficient numbers of islet cells for transplantation.
For this reason, it is vital that we simultaneously pursue
research on human pluripotent stem cells, which offer the
greatest promise of providing an adequate supply of islet cells
for treating and ultimately curing type 1 diabetes.
Thank you for your attention.
Senator Specter. Thank you very much, Dr. Spiegel.
[The joint statement follows:]
Joint Prepared Statement of Allen M. Spiegel, M.D. and Gerald D.
Fischbach, M.D.
Mr. Chairman and Members of the Subcommittee, we are pleased to
appear before you to discuss the promise of human pluripotent stem cell
research. Recent published reports on the isolation and successful
culturing of the first human pluripotent stem cell lines have generated
great excitement among scientists, patients and their families.
Research using human pluripotent stem cells holds enormous promise for
advances in the prevention, treatment, and diagnosis of a vast array of
diseases. Virtually every realm of medicine might be touched by this
innovation. Because of this enormous promise, NIH believes that this
research must proceed, as long as it is conducted ethically and
legally.
WHAT ARE STEM CELLS?
Stem cells are self-renewing and can give rise to the more
specialized cells of the human body, such as muscle cells, blood cells
and brain cells. They are best described in the context of normal human
development. When a sperm fertilizes an egg, the product is a single
cell that has the potential to form an entire organism. This fertilized
egg is a totipotent stem cell, which has the potential to develop into
a complete organism. In the first hours and days after fertilization,
this cell begins to divide into identical totipotent stem cells. Then,
approximately four days after fertilization, these totipotent stem
cells begin to specialize, forming a hollow sphere of cells called a
blastocyst. One part of the blastocyst is a cluster of cells called the
inner cell mass, which are the stem cells that will go on to form most
of the cells and tissues of the human body. These are pluripotent stem
cells, which are different than totipotent stem cells. Pluripotent stem
cells do not develop into a complete organism.
Recently, human pluripotent stem cells have been isolated from two
sources: the inner cell mass of human embryos at the blastocyst stage
and from fetal tissue obtained from terminated pregnancies. Because
these cells are capable of limitless division and self-renewal, they
can be maintained indefinitely in tissue culture, making them a vital
resource for research.
WHY ARE HUMAN PLURIPOTENT STEM CELLS IMPORTANT?
There are several reasons why the isolation of human pluripotent
stem cells might lead to better treatment, even cures, of many
diseases. At the most fundamental level, pluripotent stem cells could
help us to understand the complex events that occur during normal human
development. By identifying the mechanisms underlying routine cell
differentiation we hope to understand how disease-causing aberrations
occur. Another goal of this research would be the identification of the
factors involved in the cellular decision-making process that results
in cell specialization--why do some cells become heart cells, for
example, while other cells become liver cells? We know that turning
genes on and off is central to this process, but we do not know much
about these ``decision-making'' genes or what turns them on or off.
Some of our most serious medical conditions, such as cancer and birth
defects, are due to abnormal cell differentiation and cell division. A
better understanding of normal cell processes will allow us to further
delineate the fundamental errors that cause these often deadly
illnesses.
Human pluripotent stem cell research could also dramatically change
the way we develop drugs and test them for safety. While a limited
number of cultivated cell lines are currently available and provide
invaluable tools for drug development and testing, pluripotent stem
cells would allow expansion of this testing to more varied cell types.
For example, drugs could be tested first on particular cell lines to
determine toxicity, before they are tested in either animals or humans.
Although this would not replace testing in animals and in human beings,
it would streamline the process of drug development, and reduce
potential for harm in humans and animals. Only the drugs that are both
safe and appear to have a beneficial effect in cell line testing would
graduate to further testing in laboratory animals and human subjects.
Perhaps the most far-reaching potential application of human
pluripotent stem cells is the generation of cells and tissue that could
be used for ``cell transplantation therapies,'' which are aimed at
diseases and disorders resulting from the destruction or dysfunction of
specific cells and tissue. Although donated organs and tissues can
sometimes be used to replace diseased or destroyed tissue, the number
of people suffering from such disorders far outstrips the number of
organs and tissues available for transplantation. Pluripotent stem
cells, stimulated to develop into specialized cells and tissue, offer
real hope for the possibility of a renewable source of replacement
cells and tissue to treat a myriad of diseases, conditions, and
disabilities for which replacement tissue is in short supply. Examples
of these include neurological disorders, burns, heart disease,
osteoarthritis and rheumatoid arthritis.
HUMAN PLURIPOTENT STEM CELLS AND DIABETES RESEARCH
One of the best examples of the promise of this line of research is
in the treatment of Type 1 diabetes. Research on islet cell
transplantation and stem-cell biology offers compelling opportunities
for the development of new, innovative approaches for treating and
ultimately curing this disease.
Type 1 diabetes, often referred to as Juvenile Diabetes, is
characterized by the inability of the body to produce insulin, a
hormone necessary for glucose metabolism. This form of diabetes occurs
when the body's immune system attacks and destroys its own insulin-
producing beta cells in the islets of the pancreas. As a result of
inadequate insulin production, glucose does not enter cells as readily
as when insulin levels are normal. The standard treatment is to try to
control the glucose level with insulin injections. Insulin treatment
can sustain the patient's life, but not necessarily prevent the
devastating complications of type I diabetes, which include kidney
failure, blindness, amputation, heart attack and stroke. Clinical
trials have shown that these complications can be prevented or
significantly delayed by maintaining blood glucose levels as close to
normal as possible. However, precise blood glucose control is difficult
to achieve and requires multiple daily injections of insulin or use of
an insulin pump. These regimens are extremely challenging to follow,
especially for children and teenagers. In addition, one risk of such
precise blood glucose control is the development of dangerously low
blood sugars which could cause loss of consciousness, seizures or other
complications.
To address these problems, researchers are investigating
alternative approaches to restoring insulin-producing capacity,
including attempts to develop an artificial pancreas, whole pancreas
transplantation, and islet cell transplantation. Formidable
bioengineering problems attend development of an artificial pancreas,
and while researchers are working diligently to overcome them, a time
frame for success cannot be predicted. Whole pancreas transplantation,
while successful in some patients, is an extremely difficult surgical
procedure and it requires lifelong treatment with immunosuppressive
drugs that can have toxic side effects. This surgery is typically
performed only in adults, often in conjunction with a needed kidney
transplant for which immunosuppressive drugs would already be required.
But, the success rate for the survival of the transplanted pancreas is
much lower than the survival rate for the transplanted kidney.
Islet cell transplantation is a much simpler procedure than whole
pancreatic transplantation and has several potential advantages. Until
very recently, serious technical problems have been a major impediment
to rapid progress in islet transplantation research. These key
challenges have been: (1) to keep the body's immune defense system from
rejecting the transplanted islets; and (2) to ensure that there is a
sufficient supply of islet cells for transplantation. To date, only
about five percent of people with diabetes who have received
transplanted islets along with immunosuppressive drugs have been able
to stay off insulin longer than one year. Stem cell research offers the
potential to overcome these obstacles.
The renewed promise of islet cell transplantation derives from two
complementary research opportunities. The first is the development of
new methods for adjusting the immune system to keep the body from
rejecting transplanted islet cells. The second is the prospect that
stem cell research could ensure the needed supply of islet cells for
transplantation. Human pluripotent stem cells offer the greatest
promise of providing a limitless source of islet cells for treating and
curing type 1 diabetes. Together, these opportunities offer
unprecedented hope for curing type 1 diabetes, especially for children
and young adults whose disease has not yet progressed to the point of
debilitating complications.
HUMAN PLURIPOTENT STEM CELL RESEARCH AND THE NERVOUS SYSTEM
As significant as the promise of stem cells is for the treatment of
diabetes, the potential of stem cells for treating diseases of the
nervous system is equally impressive. It is startling to consider the
range of neurological disorders for which scientists are actively
investigating stem cell therapies in animal models. A partial list
would include classic neurodegenerative diseases such as Parkinson's
disease, Alzheimer's disease and amyotrophic lateral sclerosis (ALS);
acute insults of stroke, brain trauma and spinal cord injury; multiple
sclerosis and other demyelinating disorders; and inherited disorders
such as Tay-Sachs disease and Duchenne muscular dystrophy. It might be
possible to use stem cells to treat epilepsy and brain tumors. We have
only begun to understand the extraordinary range of possibilities that
stem cells present for treatment of these maladies.
The most obvious and exciting use of stem cells in neurological
disorders is to replace lost nerve cells. Many diseases destroy
particular types of nerve cells, and mature nerve cells cannot produce
new cells to replace those that are lost. Animal experiments have
demonstrated that the potential exists for coaxing stem cells to
specialize and replace the dopamine cells that are lost in the brain in
Parkinson's disease. A similar approach might apply to several other
neurological disorders. Stem cells, given appropriate control signals,
might specialize to replace the lost acetylcholine producing nerve
cells in Alzheimer's disease, to restore lost motor neurons in ALS, or
to produce inhibitory cells to help restrain electrical activity in
epilepsy.
Replacing lost nerve cells is only the beginning of the list of
possible therapeutic applications for stem cells. For some disorders,
such as multiple sclerosis, stem cells might replace supporting cells--
such as the glial cells, which provide the insulation necessary to
allow some nerves to conduct electrical impulses rapidly. Stem cell
strategies might be useful for correcting inherited defects. For
example, in disorders that devastate children's brains we might rely on
the ability of stem cells to migrate widely in the brain and supply the
vital missing enzyme that leads to early and tragic death from Tay-
Sachs disease. In addition, stem cells might regenerate the many
different kinds of complex brain tissue that are damaged as a result of
brain trauma or stroke. Transplanted stem cells might also supply
natural growth and survival chemicals to pave the way for regeneration
of remaining healthy neural tissue following spinal cord injury. Recent
findings suggest that stem cells might be harnessed to seek out and
destroy brain tumor cells that evade surgery or radiotherapy. The list
of possible applications of stem cells continues to grow as we learn
more about these cells.
FUTURE CHALLENGES
There is much to be done before these discoveries can be
incorporated into clinical practice. First, we must do the basic
research to understand the process by which human cells become
specialized, so that we can direct pluripotent stem cells to become the
type(s) of tissue needed for transplantation. For example, applying
basic knowledge obtained from research in developmental and stem cell
biology will enable the production of progenitor stem cells and the
rational design of cellular therapies for human diseases such as
diabetes. It is essential to underscore that studies of stem cells and
the genes that regulate their development could be important for the
development of ways to intervene in type 1 diabetes, and various
neurological conditions, even beyond their use in transplantation.
Second, before these cells can be used for transplantation, the
well-known problem of immune rejection must be overcome. Because human
pluripotent stem cells derived from embryos or fetal tissue would be
genetically different from the recipient, future research would need to
focus on modifying human pluripotent stem cells to minimize tissue
incompatibility or to create tissue banks with the most common tissue-
type profiles. In addition, just delivering cells to the appropriate
sites within the human body is an extremely difficult task. All of
these factors argue for intensified efforts to understand the basic
biology of pluripotent stem cells and, with due caution, to apply what
is learned towards the treatment of disease.
WHAT ARE THE LIMITATIONS OF ADULT STEM CELLS?
Recent findings have shown that even the adult human brain harbors
neural stem cells, and that these adult stem cells can respond to a
wide range of external and internal influences, such as learning,
stress, exercise, seizures, and trauma. In addition, if pancreatic stem
cells are ever isolated from adult tissue, it might be possible to
direct these cells to differentiate into islet cells. The
identification of adult pancreatic stem cells would open up entirely
new prospects, beyond transplantation strategies, for encouraging the
body's own stem cells to help repair damage.
It is important to note that scientists who are leading the way in
studying adult stem cells present compelling arguments why we must
pursue research on both pluripotent and adult stem cells. While some
stem cells are present in adults, there may not be an adult stem cell
for every type of cell in the body, and they may be present in only
minute numbers. In addition, they may be very difficult to isolate; for
example, in the case of adult neural stem cells, they may be confined
to certain regions of the brain that are not easily accessible. More
importantly, pluripotent and adult stem cells are not qualitatively
alike. Pluripotent stem cells have truly amazing abilities to self-
renew and to form many different cell types, even complex tissues, but
in contrast the full potential of adult stem cells is uncertain, and,
in fact, there is evidence to suggest they may be more limited. Unlike
pluripotent stem cells, the adult stem cells may be able to divide only
a limited number of times, which would limit their usefulness in the
production of adequate numbers of well characterized cells for reliable
therapies. Another issue is the question of how robust transplanted
adult cells may be or how vulnerable to disease processes. In light of
these limitations, it is important that we pursue research on both
pluripotent and adult stem cells simultaneously.
NIH GUIDELINES
Given the enormous promise of human pluripotent stem cells to the
development of new therapies for the most devastating diseases, it is
important that both privately and federally funded researchers have the
opportunity to pursue this promise. To this end, on December 2, 1999,
NIH published draft Guidelines in the Federal Register. NIH is
currently in the process of analyzing public comments and will publish
final Guidelines in the Federal Register. NIH will not fund human
pluripotent stem cell research until final Guidelines have been
published and an oversight process is in place.
CONCLUSION
Mr. Chairman, we appreciate the opportunity to discuss this
promising and extraordinary science and are pleased to respond to any
questions you may have.
Senator Specter. Dr. Fischbach, could you describe in lay
terms exactly what a stem cell is and how it works to cure or
prospectively cure, say, Parkinson's disease?
Dr. Fischbach. A stem cell is a cell that has the
capability of giving rise to many other types of cells. It is
like the stem at the root of a tree. It also can renew itself.
It can reproduce many times, but it also can, under appropriate
cues, give rise to one or another specialized cell.
Senator Specter. And illustratively, how would that cell
reproduce a cell which is deficient, causing someone to suffer
from Parkinson's?
Dr. Fischbach. The environment and cues, many of which are
known, can steer that cell into a pathway of differentiation,
as it is called, to produce a nerve cell that is deficient in
Parkinson's disease.
Senator Specter. So that it produces a nerve cell to
replace a deficient nerve cell in a person's body.
Dr. Fischbach. Yes.
Senator Specter. And it is the deficiency of that nerve
cell, for example, which causes Parkinson's.
Dr. Fischbach. That is exactly right.
Senator Specter. I have asked for a list of the ailments
where there is the potential for cure by stem cells. I would
like you to tell me if this is complete. Diabetes, Alzheimer's,
heart disease, muscular dystrophy, Parkinson's, spinal cord,
amyotrophic lateral sclerosis, stroke, multiple sclerosis, and
Tay-Sachs. Is there potential for curing all of those ailments
with stem cells?
Dr. Fischbach. I think there is great potential for curing
or certainly reducing the burden of those diseases to an
enormous degree.
Senator Specter. Taking Parkinson's, again for illustrative
purposes, we have had testimony that we may be within 5 to 10
years, 5 to 7 years of a cure of Parkinson's. What is the
expectation of accelerating, speeding up that process through
the use of stem cells?
Dr. Fischbach. I think that estimate, which I have also
heard and been very involved with, is very dependent on the use
of stem cells. That is certainly one of the main efforts that
will be undertaken to make the Parkinson's strategic research
plan successful.
Senator Specter. Dr. Fischbach--and, Dr. Spiegel, you may
want to comment on this as well--there have been suggestions
that there are alternative sources for stem cells besides
embryos: umbilical cords, a variety of other sources. Are there
other ways to get an adequate number of stem cells to do the
medical research which you have described?
Dr. Fischbach. I think this is a very important question. I
think we both would like to comment.
My strong feeling is there are other sources. The key
question is are they adequate? That is a matter for future
research. Stem cells have been discovered in many tissues.
Specifically, certain types of stem cells have been discovered
in the brain, but we do not know as much about them in terms of
how renewable they are and what the limits, and range of their
capabilities are. And as I said, we need to maximize both of
those.
My current reading of the literature is by neither one of
those criteria are they as adequate as embryonic stem cells.
SUPPLY OF STEM CELLS
Senator Specter. Dr. Spiegel, you had made the comment
about diabetes. What is your professional judgment as to
whether there would be an adequate supply of stem cells to work
on all of these terrible ailments if we do not utilize
discarded embryos?
Dr. Spiegel. As I indicated in my comments, I think this
would be tying one hand behind our back and tying the hands of
our most superb scientists who are eager to pursue research in
this area.
I indicated that I absolutely agree that we do need to
pursue vigorously the adult stem cell area. We convened
scientists and experts, from all over the country just this
past couple of weeks at the NIH to review stem cell research.
They were essentially unanimous in their feeling that we
vigorously need to pursue the human pluripotent stem cell in
addition to the adult stem cell question. This is particularly
true in areas such as the pancreas where, unlike the blood
system and unlike the intestinal system, for example, there is
as yet no definitive evidence for adult pancreatic stem cells.
Senator Specter. How many scientists were convened at the
meeting which you referred to?
Dr. Spiegel. On the order of 200, including some of the
really most superb scientists in the area of developmental
biology and stem cells.
Senator Specter. Well, my red light just went on, so I am
going to turn to our very distinguished ranking member, Senator
Harkin.
Opening Statement of Senator Tom Harkin
Senator Harkin. Thank you very much, Mr. Chairman. I
apologize for being a little late. I will just make a
statement. I know other people have been here before me, and I
will defer to them for their questions. Then I will come back
and ask my questions after them.
As you know, Chairman Specter and I have a very close
bipartisan partnership when it comes to medical research. We
both strongly believe that medical research holds great hope
for improving the health and well-being of millions of our
fellow Americans. I want to compliment and congratulate our
chairman, Senator Specter, for his very bold leadership on this
issue of biomedical research. It is a welcome breath of fresh
air amid some of the confusion and I think some of the
misinformation that has been bandied about on different aspects
of biomedical research, but especially the one that we are here
today to talk about and that is stem cell research. So, I just
want to thank you, Mr. Chairman, for your leadership on this
issue.
I am late because I was just at another hearing on defense.
You were there briefly and I knew you had to leave before I
did. We were talking about our defense structure, and I was
struck by the fact that here we are putting enormous amounts of
money in to protecting our country, keeping our country strong.
It occurred to me, as I was listening to the amount of money
that we are now asking for more missile defense and smart bombs
and things like that, that it came back to me a little known
fact, and that is in the last 5 years, we have spent more money
on military research and development than we have spent on all
biomedical research since the turn of the century. That shocks
a lot of people when I tell them that, but it is true. You can
add it up. I mean everything. I mean smallpox, diphtheria,
polio, cancer, everything that we have spent all this money on
since the turn of the century, we have spent more than that on
military R&D in the last 5 years. So, we have smart bombs and
we have smart missiles and we have a strong defense, and I am
thankful for that.
We need the smart bombs and the smart missiles to knock out
Parkinson's disease and ALS and spinal cord injuries and
juvenile diabetes, all of the ailments that I believe can be
overcome if we just really focus on research. So, when people
tell us under your leadership that we are putting too much
money in NIH, I always roll out that fact of what we have done
in the military and how little we are doing in medical
research.
In this area of stem cell research, it holds so much
promise. Of course, there are different areas of getting stem
cells: adult stem cells, embryonic, fetal. To say we are only
going to use one source is, as you said, Dr. Spiegel, to tie
one hand behind our backs. We need to unleash an army, unleash
an army of researchers all over this country to use this new
resource and to bring us forward in developing stem cell
research to the point where I believe it is going to take us.
No one can guarantee it. No one can guarantee what it is going
to lead to, but to blindly stop it now or to tie one hand
behind our back I think is basically to doom millions of
Americans and people around the world to perhaps lives that
they might not otherwise be leading.
So, I am grateful that you are here. I just wanted to make
that statement to say that we need to push the boundaries.
Again, we need the Federal guidelines according to the
Bioethics Commission and their findings and the statement they
came out with. As you probably have pointed out in your
testimony, private research is going on in this area. But to
the extent that it is done privately and done chaotically, it
is going to push back the time when we have the kind of
discoveries that would come if we did it in a regulated,
ethical manner. If we did it that way and we were able to guide
and direct this research, I believe the fulfillment of the
promise would come much more rapidly than if we just leave it
in a chaotic system out there with no supervision and no
ethical controls.
Mr. Chairman, thank you for affording me this time.
Senator Specter. Well, thank you very much, Senator Harkin.
Thank you for your good words and for our bipartisan
partnership.
Our practice is to take Senators in order except for the
ranking member because of the structure of the Senate. We turn
now to Senator Reid.
Opening Statement of Senator Harry Reid
Senator Reid. Mr. Chairman, I appreciate very much the
bipartisan tone that this subcommittee has set. It is easy to
talk in a bipartisan tone in a subcommittee hearing, but you
and Senator Harkin have done that on the Senate floor. An
example was the budget battle that we won on the Senate floor
and lost in conference, but I admire and respect the work that
you did on the budget.
I had, Dr. Fischbach, Dr. Spiegel, the good fortune this
morning to spend some time with Dr. Ruth Kirschstein, the
acting Director of NIH. We talked about a number of things, but
one of the things that we spoke about is her experience. She
talked about her work in the early 1950's when they knew they
were getting close to finding a cure--I do not know if ``cure''
is the right word, but a way to prevent polio. And growing up
in that era, I can remember that was the thing that we were
most afraid of. It was not the nuclear holocaust. We were
afraid of getting polio, all the young boys and girls. And she
said that they knew that they had in their sight a way to cure
polio.
We have talked this morning about diabetes, Alzheimer's,
Parkinson's, and a number of other things. Do either of you
gentlemen think that we have in sight a cure for some of these
diseases that we are so afraid of today: Parkinson's,
Alzheimer's? Half the money we spend in nursing homes in
America today is spent on those two diseases.
Dr. Fischbach. I think there is enormous hope for real
advances and in some of the cases, real cures in the sense of
stopping the progression of a disease. The analogy with polio
is an interesting one and quite stimulating to think about.
That was due to the infection by a specific organism and a
vaccine worked a miracle.
I think in the disorders I am most familiar with, the
complexity of the brain is a hurdle to overcome.
Senator Reid. And you made that very clear in your
testimony.
Dr. Fischbach. But I am as optimistic now as I have been
throughout my scientific career in the last 30 years that we
will be able to stop and in some cases reverse disorders of the
brain that are associated with loss of nerve cells.
Senator Reid. Dr. Spiegel, do you agree with Dr. Fischbach?
Dr. Spiegel. I would like to make two points in response to
your question.
First, I want to indicate, outside of the context of the
immediate subject, that we are not focused only on cures. Those
are incredibly important, but you mentioned the polio vaccine.
Prevention is critical, and we are very heavily focused on
prevention of many diseases, including type 1 diabetes.
TRANSPLANTATION
In terms of cures, the situation for some of the diseases--
I would say type 1 diabetes and liver failure--the situation is
particularly interesting with respect to transplantation.
Transplantation is a potential cure, as I indicated, for both
of these diseases, and with immunology advances, we have the
possibility of not just substituting one disease, complications
of treatment with immunosuppressive drugs, for another, but
rather of allowing these individuals to lead a relatively
normal life.
The problem, though, is that despite the best efforts of
increasing organ donation, there simply is an inadequate supply
to provide these cures. And this is where stem cell research is
so important. I do not want to over promise, and I think we
always have to be very cautious. I think Senator Harkin made
the point we cannot guarantee it. But certainly, if we do not
do the research, it will not happen. That is guaranteed.
Senator Harkin. That is guaranteed.
Senator Reid. Let me say that in biomedical research I
certainly do not want anyone's hands tied behind them. We have
scientists all over the United States and the direction that
they take and the grants that they write that are okayed by you
folks at NIH and the work that you do at NIH--I want no hands
tied behind anyone's back. I can speak, as someone who has a
pro-life voting record here in the Senate, that as far as I am
concerned, there are no holds barred on what you should be able
to do using embryos, any other place that you feel that you can
get stem cells. I am all behind you and I think that the work
that you have done has been exemplary and I look forward to
joining hands with the members of this subcommittee, the
members of the full committee, and the Senate in giving you all
the resources that you need to help people that really need
help.
Dr. Spiegel. I am certainly grateful for your support and
for that of the chairman and Senator Harkin. It has been
incredibly important throughout. We appreciate the support for
NIH in general and on this issue. I want to just articulate
that.
Senator Reid. Mr. Chairman, I have other obligations. I
wanted to come and make this brief statement. Again, it is a
great panel. I am sorry I cannot be here for the rest of it.
Senator Specter. Well, thank you very much, Senator Reid,
and thank you for your comments about the funding. I think it
is worth just a sentence or two to note that in the past 3
years, this subcommittee has taken the lead on increasing NIH
funding by more than $5 billion. It was at $13 billion, and in
the last 3 years, we have added some $5 billion. And we have
done that over the votes which we have not gotten extra funding
for, but we have established priorities. One of my frequent
statements is that the National Institutes of Health are the
crown jewels of the Federal Government. Some say the only
jewels of the Federal Government.
But there is nothing more important than health. Health is
number one. We have a very large group here today, as we have
had with juvenile diabetes and as we have had with cancer and
as we will have later in a couple of weeks with amyotrophic
lateral sclerosis. The public is very concerned about medical
research and about curing Parkinson's or Alzheimer's, these
dreaded diseases, or heart disease. And this subcommittee
intends to do what it can to get that done.
Senator Murray.
Opening Statement of Senator Patty Murray
Senator Murray. Well, Mr. Chairman, thank you very much for
having this hearing. I want to just personally thank you, Mr.
Chairman, for all the time and effort you have spent on this
particular issue. I think the work you have done has really
built a balanced and a fair hearing record on stem cell
research and I think has moved us in a very positive direction.
And I want to thank Senator Harkin as well for his work on
this and his excellent statement as well.
You know, my father had multiple sclerosis. He was
diagnosed when I was 15. He lived most of my life in a
wheelchair, and I know the personal hope that he had many times
and the disappointments he had throughout his lifetime.
This research, obviously, will not help him. He passed away
several years ago, but I know of thousands and thousands and
thousands of families who are counting on us to do the right
thing so that there is hope in their future.
So, I really want to thank you, Mr. Chairman, Senator
Harkin, for your work on this, and hope that we can continue to
push the limits on this and find some resolution not only for
MS, for Parkinson's, Alzheimer's, for so many other exciting
opportunities that are out there.
I have listened to the people who oppose this, and I think
part of what we are hearing is that they have little
understanding of some of the bioethical requirements that NIH
has regarding stem cell research. If you could just take a
minute and outline for us what ethical standards you do adhere
to in this research, I would appreciate it.
Dr. Fischbach. Well, we can both take a shot at that. I
think the guidelines being proposed by the NIH address these
really awesome ethical issues in terms of ensuring informed
consent on the part of those who donate embryos, ensuring the
utmost care in the scientific use and complete dedication to
the full informed use of the data. They are complex and they
involve the use of the tissue and the circumstances under which
it can be used. It involves oversight to make sure that these
guidelines are followed. It involves review by scientists, lay
people on council, and NIH officials. So, I think they are a
rather complete examination of the legal, the ethical, and the
social issues, as well as the scientific issues involved.
Senator Murray. Dr. Spiegel?
Dr. Spiegel. The only point I would add is very much in
line with what the chairman had to say, and that is the
guidelines try to ensure to totally dissociate the issue of the
availability of embryos from the issue of the use of the stem
cells. Informed consent, as we have heard, no monetary
remuneration, nothing that would encourage this, in other
words, to totally dissociate the two issues and to allow real
public oversight and scrutiny of the way that these stem cells
would be used. I think that is what is vitally important.
Senator Murray. Thank you.
We all understand that without Federal standards and
guidelines the potential for abuse of this research is
significant. I think the recent front-page article in the
Washington Post made that point very clear.
From your opinion, if there is no Federal involvement in
there, what would the ramifications be?
Dr. Fischbach. My own opinion is that the Federal
involvement is critical for the ethical conduct of this type of
science. Science conducted behind closed doors is behind closed
doors, and we cannot regulate that at all or bring the most
disinterested parties to the table to think about the
ramifications.
Dr. Spiegel. Certainly the ethical issues are paramount,
but the other point we have alluded to several times. There is
a lot of work going on in the private sector, and at some
level, that is fine. But only with Federal funding, and in
addition, the oversight, are we going to really see all the
talented scientists who want to work in this area contribute
and give us their capabilities.
Senator Murray. Would there also be ramifications for who
would have access to the research and the results of that
research if the Federal Government is not involved?
Dr. Fischbach. At the present time, we do not have any
reason to believe there would be limited access. But this is a
very dynamic, rapidly moving field, and there is always the
potential for restriction of access. But we do not--perhaps Dr.
Spiegel would like to add--see any evidence at the present time
for restriction of access.
Dr. Spiegel. I will just leave it at that.
Senator Murray. All right. Thank you very much, Mr.
Chairman.
Senator Specter. Thank you, Senator Murray.
Before moving on to the next panel, just a comment about
the time table. It was on January 15 of last year that the
Health and Human Services General Counsel determined that the
use of Federal funds to support stem cell research did not
violate the appropriations ban. It was not until April 8 that a
meeting was held with public and private groups to discuss the
guidelines. On December 2, there were draft guidelines
published with the comment period for 60 days, which would have
run out in early February. That was extended to February 22.
I know there are a great many comments, but there needs to
be a sense of urgency to getting this done because every day is
a day lost which could be saving lives. This subcommittee has
placed a very high priority on this subject, having five
hearings, more than we have had on any other subject, because
of the importance of focusing public attention and getting it
moving. So, we would urge you to apply a sense of urgency here
and get it done.
Dr. Fischbach. I think we all absolutely agree with that.
Senator Specter. We will not hold you any longer, so you
can go back to your offices to get this done.
Thank you very much.
We will now turn to our second panel: Senator Sam
Brownback, Dr. Frank Young, and Ms. Mary Jane Owen.
In our previous hearings, we have heard from witnesses who
have been opposed to eliminating the Federal ban. In our last
hearing, we heard from Congressman Jay Dickey who opposes
Federal funding on embryos in stem cell research. We consider
it very important to have balance in the presentation of the
views so that all of the information can be available to the
Senate when we debate this matter and render our judgments and
views can be available to the American people.
STATEMENT OF HON. SAM BROWNBACK, U.S. SENATOR FROM
KANSAS
Senator Specter. Our lead witness here is Senator Sam
Brownback, elected to the Senate in 1996. It says to take the
place of Senator Dole. I do not know that anybody can take the
place of Senator Dole.
Senator Brownback. That is not possible.
Senator Specter. Senator Brownback and I agree on many,
many matters. We disagree on this one.
We share common roots. I was born and raised in Kansas and
sometimes like to think of myself as Kansas' third Senator.
Senator Brownback. We do too.
Senator Specter. I would have to put myself fourth behind
Senator Dole whose presence is still on the scene.
Senator Brownback serves on the Commerce, Science, and
Transportation Committee, as well as the Health, Education,
Labor, and Pensions Committee. He chairs the Subcommittee on
the Middle East for the Foreign Relations Committee where he
has been very active.
He grew up in Parker, Kansas, has a law degree from the
University of Kansas and his bachelor's from Kansas State.
Welcome, Senator Brownback, and we look forward to your
testimony.
Senator Brownback. Thank you, Senator Specter, Senator
Harkin, Senator Murray. Thank you for allowing me the
opportunity to speak in front of you this morning to testify. I
do not know that I would consider myself the lead witness of
this panel. You have a former FDA Commissioner and I think you
will hear from Ms. Owen a very clear statement as well.
Senator Specter. In the Senate, Senator Brownback, you are
the lead witness.
Senator Brownback. Well, you are kind.
I appreciate this opportunity. I have great respect for
your thoughts. I have a great respect for your heart on these
matters and on all matters, but on this one, we do disagree. I
will be highlighting why I believe this issue should be handled
another way, that we should be funding aggressively research in
the area of adult stem cell research.
I have supported this panel's efforts to double NIH funding
over a 5-year time period. I think that is critically
important, very important, that we fund the science.
I also think it is very important that we have a critical
ethical view as to what we are doing. At the center of this
debate, at the very center of this debate, is the real
question: Is the young human person or property? And that is at
the very center of what this debate is about, and I want to
articulate that further. But that is the center of what this
question is about.
We are here today to discuss some of the issues that have
been raised regarding Federal funding for human embryonic stem
cell research. My position on this is that Federal funding of
human embryonic stem cell research is illegal, is immoral, and
it is unnecessary. And I want to go into those three issues.
As this subcommittee is well aware, Congress outlawed
Federal funding for harmful human embryo research in 1996 and
has maintained that prohibition ever since. The ban is broad
based and specific. Funds cannot be used for--and I quote now
from the act--``research in which a human embryo or embryos are
destroyed, discarded, or knowingly subjected to risk of injury
or death.'' The intent of Congress is clear. If a research
project requires the destruction of human embryos, no Federal
funds should be used for this project.
NIH recently published its proposed guidelines to
circumvent this language. Marcy Wilder, who I might add was a
former legal director for the National Abortion Rights Action
League and now Associate General Counsel at the Department of
Health and Human Services, wrote a legal opinion that sought to
justify the research being proposed by the NIH.
Yet, despite this fig leaf of the HHS legal opinion, the
fact remains that this research is illegal. It is illegal for
this reason. The deliberate killing of a human embryo is an
essential component of the contemplated research, and without
the destruction of the embryo, the proposed research would be
impossible.
Now, despite the legal gyrations of HHS, this is a point
that is not lost on the National Bioethics Advisory Commission.
Although their conclusions are wrong in my estimation, NBAC
observes in their recommendations to the President the
inconsistencies of the HHS legal opinion and the NIH
recommendations. Accordingly, NBAC recommends an approach that
is at once both more honest and more heinous.
Which brings us to a discussion of the morality of this
legislation. The NBAC position is given legislative form in the
Senate bill that we are discussing today, S. 2015, currently
referred to the Health, Education, Labor, and Pensions
Committee on which I sit. Among other and perhaps more serious
policy changes, it would constitute a lifting of the ban on
human embryo research. In brief, the Stem Cell Research Act of
2000 seeks to allow Federal funding for researchers to kill
living human embryos. Under this bill, Federal researchers
would be allowed to obtain their own supply of living human
embryos which they would then be allowed to kill for research
purposes.
Now, the very act of harvesting cells from live human
embryos results in the death of the embryo. Therefore, if
enacted, this bill would result in the deliberate destruction
of human embryos.
This bill will even violate current Federal policy on fetal
tissue in my estimation, which allows harvesting of tissue only
after an abortion was performed for other reasons and the
unborn child is already dead. Under this bill, the Federal
Government will use tax dollars to incentivize the killing of
live embryos for the immediate and direct purpose of using
their parts for research.
Taxpayer funding of this research is problematic for a
variety of reasons. First among those concerns is that if
Congress were to approve this legislation, it would officially
declare for the first time in our Nation's history that
Government may exploit and destroy human life for its own or
somebody else's purposes.
Now, this research is also problematic because it would use
Federal tax dollars to allow the Government to procure and
therefore own a vast supply of living human embryos. Now, this
notion of ownership, particularly by the Federal Government, of
other human beings I believe is deeply disturbing.
The bill even allows Federal funding for destructive
research using embryos created by cloning so long as that does
not result in--quote from the legislation--``the reproductive
cloning of a human being.'' On the one hand, this is an
attempt, it seems to me, to authorize the critical issue of
human cloning when what is really needed is the continuation of
the full public debate on this point. On the other hand, this
approach recognizes that for the purposes of possible clinical
applications, particularly to avoid possible tissue rejection,
human cloning is the logical next step, or so-called
therapeutic cloning. This means that live embryos created by
researchers can be experimented on and destroyed but cannot be
allowed to survive to live birth. That is prohibited in the
legislation, which seems to create a new class of human beings
who under the law will simply not be allowed to live.
I think history has already taught us some important
lessons on separating human beings into different classes. The
Dred Scott case issue was on that point where Dred Scott held
that African Americans at that time ``had no rights which the
white man was bound to respect.''
Now, I am not suggesting at all that that is the intent of
the chairman or of the ranking member of this committee, but if
you look into what the effect of what would happen with this
legislation, I think we get terribly close to those same issues
that we have seen throughout our history, and it is deeply
troubling to me.
My final point is that the human embryonic stem cell
research is unnecessary, and this is a key point because I want
to see people healed, which is what the chairman is after,
which is what the ranking member is after. We want to see these
diseases no more hit our people or anybody else across the
planet. That is our heart and that is our objective, and on
that we all agree. That is why I am saying this is not
necessary. We can go on the areas of legitimate research into
adult stem cells which do not create the moral and ethical
difficulties that we do in human embryo stem cell research.
Dr. Young will testify more about what is taking place in
this area in the research now.
In the past, Congress has increased funding for NIH. New
advances in adult stem cell research, being reported almost
weekly, show more promise than destructive embryo research. And
I want to give just a few of these.
Just this past February, writing in the journal Neurons,
scientists at Children's Hospital in Boston announced that they
had successfully generated new brain cells in birds using adult
neural stem cells.
Writing in the March 20 issue of Nature Medicine,
University of Florida scientists reported that they reversed
insulin-dependent diabetes in mice by using adult pancreatic
stem cells. Their quote, ``The next step is to take this into
humans, they say.'' They have extracted and cultivated viable
adult brain stem cells from eight living human patients
undergoing surgery for other reasons.
Writing in the March 17 issue of Science, University of
Toronto researchers reported they found retinal stem cells in
the eyes of adult mice, cows, and humans, and have shown that
they can be used to produce new neurons presenting the
prospects of repairing or regenerating damaged retinas and
restored sight.
In April, Dr. Karen Obote and colleagues at Children's
Hospital in Boston reported at a meeting of the American
Association of Neurological Surgeons in San Francisco that they
can use adult neural stem cells to target brain tumors in mice.
The cells could be used to reduce or kill the tumors by
delivering new genes or carrying cancer drugs to where they are
needed.
Clearly we must continue to fight and help cure diseases
and to eliminate suffering. I have got a chart over here on
some of these areas that are just now coming out in the adult
stem cell area.
The other issue that they--we do not have the ethical/moral
problems. We do not have the problems of the immunity system
rejecting cells, if we use our own adult stem cells.
Mr. Chairman, ranking member of this committee, I have
great respect for your heart and your desire in here, and I
think on that we completely agree. And I have no question about
your motives in doing this. I just think we are crossing an
enormous issue here of looking at this human life as a piece of
property rather than as a person.
PREPARED STATEMENT
And we do not need to go there. We can address these issues
with adult stem cells. We can address these issues with
increased funding in NIH and increased funding in these key
areas that are showing so much promise right now to address
these terrible diseases. That is the way we can go together.
That is where our hearts can be pure and we can do the right
thing and feel good about it rather than accepting the
deliberate killing of one human innocent life in order to help
another, which has never been right throughout human history.
I thank you, Mr. Chairman, for your patience and your
willingness to hear me out on this point.
[The statement follows:]
Prepared Statement of Senator Sam Brownback
Thank you Mr. Chairman and members of the Subcommittee for the
opportunity to testify on this very important issue.
We are here today to discuss some of the issues that have been
raised regarding federal funding of human embryonic stem cell research.
My position is that federally funded human embryonic stem cell research
is illegal, immoral and unnecessary.
As this subcommittee is well aware, Congress outlawed federal
funding for harmful embryo research in 1996 and has maintained that
prohibition ever since. The ban is broad-based and specific; funds
cannot be used for ``research in which a human embryo or embryos are
destroyed, discarded or knowingly subjected to risk of injury or
death.'' The intent of Congress is clearif a research project requires
the destruction of human embryos no federal funds should be used for
that project.
The language of the ban is clear--as are the lessons from history.
The Nuremberg Code (from the trials of Nazi war criminals) states, ``No
experiment should be conducted where there is an a priori reason to
believe that death or disabling injury will occur.'' Also, the World
Medical Association asserts in the Declaration of Helsinki that, ``In
research on man, the interest of science and society should never take
precedence over considerations related to the well-being of the
subject.'' Further, ``concern for the interests of the subject must
always prevail over the interest of science and society.'' As well, it
is hardly necessary to note that it has already been proven
biologically that an embryo constitutes human life (MERCK MANUAL and
NBAC recommendation to the President, ``Ethical Issues in Human Stem
Cell Research'').
Pope John Paul II, quite independent of government law, and
particularly of our own ban on embryo research states, in his
encyclical, Evangelium Vitae, ``[The] evaluation of the morality of
abortion is to be applied also to the recent forms of intervention on
human embryos which, although carried out for purposes legitimate in
themselves, inevitably involve the killing of those embryos. This is
the case with experimentation on embryos, which is becoming
increasingly widespread in the field of biomedical research and is
legally permitted in some countries. Although ``one must uphold as
licit procedures carried out on the human embryo which respect the life
and integrity of the embryo and do not involve disproportionate risks
for it, but rather are directed to its healing, the improvement of its
condition of health, or its individual survival'', it must nonetheless
be stated that the use of human embryos or fetuses as an object of
experimentation constitutes a crime against their dignity as human
beings who have a right to the same respect owed to a child once born,
just as to every person.
``This moral condemnation also regards procedures that exploit
living human embryos and fetuses--sometimes specifically ``produced''
for this purpose by in vitro fertilization--either to be used as
``biological material'' or as providers of organs or tissue for
transplants in the treatment of certain diseases. The killing of
innocent human creatures, even if carried out to help others,
constitutes an absolutely unacceptable act.''
THE NATIONAL INSTITUTES OF HEALTH AND THE HHS
NIH recently published its proposed guidelines to circumvent the
congressionally imposed ban on destructive human embryo research.
Marcy Wilder, former Legal Director of the National Abortion Rights
Action League, and now Associate General Counsel at the Department of
Health and Human Services wrote a legal opinion that sought to justify
the research being proposed by the NIH. Yet despite the fig leaf of the
HHS legal opinion, the fact remains that this research is illegal. It
is illegal for this reason: the deliberate killing of a human embryo is
an essential component of the contemplated research; and without the
destruction of the embryo the proposed research would be impossible.
Despite the legal sophistry of HHS, this is a point that is not
lost on the National Bioethics Advisory Commission. Although there
conclusions are wrong, NBAC observes in their recommendation to the
President the inconsistency of the HHS legal opinion and the NIH
recommendations. Accordingly, NBAC recommends an approach that is at
once both more honest--and more heinous.
WHICH BRINGS US TO A DISCUSSION OF THE MORALITY OF THIS RESEARCH
The NBAC position is given legislative form in Senate Bill 2015. S.
2015, currently referred to the Health Education Labor and Pensions
Committee, on which I sit would, among other and perhaps more serious
policy changes, constitute a lifting of the ban on human embryo
research.
In brief, the ``Stem Cell Research Act of 2000'' seeks to allow
federal funding for researchers to kill living human embryos.
Under this bill federal researchers would be allowed to obtain
their own supply of living human embryos, which they would then be
allowed to kill for research purposes.
The very act of harvesting stem cells--or perhaps more accurately
constructing so-called embryonic stem cells--from live human embryos
results in the death of the embryo. Therefore, if enacted, this bill
would result in the deliberate destruction of human embryos.
This bill even violates current federal policy on fetal tissue,
which allows harvesting of tissue only after an abortion was performed
for other reasons and the unborn child is already dead. Under this
bill, the federal government will use tax dollars to kill live embryos
for the immediate and direct purpose of using their parts for research.
Taxpayer funding of this research is problematic for a variety of
reasons. First among those concerns is that, if Congress were to
approve S. 2015, it would officially declare for the first time in our
nation's history that government may exploit and destroy human life for
its own or somebody else's purposes.
This research is also problematic because it would use federal tax
dollars to allow the government to procure, and therefore ``own,'' a
vast supply of living human embryos. The notion of ``ownership,''
particularly by the Federal government, of other human beings is deeply
disturbing.
The bill even allows federal funding for destructive research using
embryos created by cloning, so long as this does not result in ``the
reproductive cloning of a human being.'' On the one hand, this is an
attempt to authorize the critical issue of human cloning by stealth;
when what is really needed is the continuation of the full public
debate. On the other hand, this approach recognizes that for the
purposes of possible clinical applications, particularly to avoid
possible tissue rejection, human cloning is the logical next step--so-
called, ``therapeutic cloning.'' This means that live embryos created
by researchers can be experimented on and destroyed, but allowing them
to survive to live birth is prohibited. The bill defines a new class of
human beings who, under the law, will simply not be allowed to live.
History has already taught us the lessons of separating human
beings into different classes. The Dred Scott case held that African-
Americans, ``had no rights which the white man was bound to respect.''
Today, 143 years later, it is precisely this same argument which is now
being used to legitimate the destruction of human embryos. It is the
contention of S. 2015, as well as the NIH, that human embryos do not
have rights which people, already born, are bound to respect.
HUMAN EMBRYONIC STEM CELL RESEARCH IS ALSO UNNECESSARY
There are legitimate areas of research which are showing more
promise than embryonic stem cell research and which do not create moral
and ethical difficulties. Dr. Frank Young, former FDA commissioner,
under Ronald Reagan has detailed some of the alternatives in his
testimony which you will hear later.
In the past, Congress has increased funding for NIH. New advances
in adult stem cell research, being reported almost weekly, show more
promise than destructive embryo research.
Just this past February, writing in the journal Neuron, scientists
at Children's Hospital in Boston announced that they successfully
generated new brain cells in birds using adult neural stem cells
(MSNBC, Feb. 23).
Also, writing in the March 2000 issue of Nature Medicine,
University of Florida scientists reported that they reversed insulin-
dependent diabetes in mice by using adult pancreatic stem cells. ``The
next step is take this into humans,'' they say. They add that they have
extracted and cultured viable brain stem cells from the hippocampus of
eight living human patients undergoing surgery for other reasons
(Reuters, February 28).
Writing in the March 17 issue of Science, University of Toronto
researchers reported that they found retinal stem cells in the eyes of
adult mice, cows and humans and have shown that they can be used to
produce new neurons, presenting the prospect of repairing or
regenerating damaged retinas and restoring sight (UniSci, March 17).
In April, Dr. Karen Aboody and colleagues at Children's Hospital in
Boston report at a meeting of the American Association of Neurological
Surgeons in San Francisco that they can use adult neural stem cells to
target brain tumors in mice. The cells could be used to reduce or kill
the tumors, by delivering new genes or carrying cancer drugs to where
they are needed (Reuters, April 10).
Clearly we must continue to fight to help cure disease and to
alleviate suffering. However, it is never acceptable to deliberately
kill one innocent human being in order to help another. When did it
become acceptable to use an evil means to pursue a good end, even a
great one? Doesn't the so-called good end actually become bad by using
bad means? If we manage the cure of some diseases and the betterment of
some aspects of bodily health by means that involve the killing of the
most defenseless and innocent of human beings, we will rightfully be
judged harshly by history as having sought some benefits at the expense
of our humanity and moral being. The twentieth century has already
taught these lessons--are we to ignore them at the beginning of this
century? Or to put it another way, as George Santayana once said,
``Those who cannot remember the past are condemned to repeat it.''
Thank you, Mr. Chairman.
Senator Specter. Well, thank you very much, Senator
Brownback for your testimony. We appreciate your sincerity and
your position, and we will have some questions on the morality
and the alternatives to human stem cells. But first we are
going to go to our other two panelists.
STATEMENT OF FRANK YOUNG, M.D., Ph.D., FORMER
COMMISSIONER, FOOD AND DRUG ADMINISTRATION,
DEPARTMENT OF AGRICULTURE
Senator Specter. I would like to call now on Dr. Frank
Young, Commissioner of the Food and Drug Administration from
1984 to 1989. Previously served as Deputy Assistant Secretary
for Health in the Bush administration and as Director of the
Department of Health and Human Services of Emergency
Preparedness. He was Dean of the University of Rochester
Medical School, a microbiologist by training. He now serves
also as pastor of adult ministries at the Fourth Presbyterian
Church in Bethesda, MD.
It is a 1-year anniversary, Dr. Young, since you testified
on April 26 last year. So, welcome back.
Dr. Young. Thank you very much, Mr. Chairman and the
distinguished Senator Harkin. I thank you both from the bottom
of my heart for your years of steadfast support of the National
Institutes of Health and biomedical sciences. I have had a
chance to work with both of you in the past and I know of your
genuine commitment. I only urge you on in the support of this.
As a pastor, it would be remiss of me not to remind all of
us that we are totally biodegradable. I have conducted more
memorial services than I would like to admit now. So, as we
look at curing disease, we have to recognize that we each at
one day will ourselves die. And it is with that perspective of
the role of research that I would like to put my comments
before you today.
I also want to thank you, Mr. Chairman, for the excellent
way in which you have held these hearings. You have brought
forth witnesses of all persuasions, a rare event in
Washingtonville. And I thank you for what you have done in that
way.
Two quotes might frame this, and I have brought for the
press table the copy of the Science issue, which I am sure you
have, if not, I can provide you with more of these.
One is cited in there by Rabelais in 1532 who stated,
``Science without conscience is but death of the soul.'' So, we
have to look at where and how we develop our advances.
And more recently Robertson stated in a California Law
Review--and I quote--``Science is not an unmitigated blessing.
It is expensive and its discoveries, like the Tree of Knowledge
in Eden, expands man's capacity for evil as well as good. More
knowledge is not a good in itself, nor is it necessarily
productive of net good. Society as the provider of resources,
the bearer of costs, the reaper of the benefits has an
overriding interest in the consequences of science and hence
the direction and routes that research takes,'' which you are
doing today.
I will just summarize my statement.
Senator Specter. That is fine, Dr. Young.
Dr. Young. There are these issues that I think are
critical.
First, we are dealing with utilitarian ethics. We are
focusing on the decision of whether or not to use the promise
of potential good for the end of a life that we know is
essentially here. And that decision, as the Senator said, is
not one to be taken lightly. So, as we focus our energy, it is
at what price is humanness in the 21st century.
My worry, as both a physician and a pastor, is the degree
of violence that we see on human beings. We just saw it the
other day at the National Zoo. We see it in Bosnia. We have
seen it in Kosovo. We have seen it in many places. And I
believe the sacredness of human life is at the very heart of
what we will be in the 21st century.
I would suggest that as we do research, we take research in
a cautious fashion and first of all do no harm. As the Senator
summarized, we are blessed with the capacity to have stem cells
in most every organ in the body, and it was also stated in the
former panel. These enable us to regenerate our skin, to be
able to have liver regeneration at times. These are very
important parts that are just being discovered now.
When I last testified before you, Mr. Chairman, we had very
little knowledge of adult stem cells. One of the most
interesting ones was one in mice recently where they were able
to take from bone marrow, the adult bone marrow, and isolate
stem cells in as few as 50 stem cells added to the animal with
liver disease was able to produce a change in symptomatology.
The stem cells from an adult have these advantages. One, it
does not carry the moral baggage. Two, they are readily
isolated particularly from bone marrow. Three, they can be
isolated and preserved from cord blood, and in fact, one
company now is advocating at a small fee the preservation of
cord blood stem cells for use of the individual later on.
Therefore, it is not a need to destroy embryos that are
``spare'' at this time.
Four, I would like to say that we have been lax in the
Federal Government in regulating in vitro fertilization
clinics. Some of the margins that we see now are related to
this very expensive therapy unregulated that does generate
these ``excess'' individuals. And if we are not careful, they
can easily regenerate and develop more of ``spare'' embryos.
Next, it is critical to realize that the immunological
capacity and also the relative ease of differentiating the
adult stem cell vis-a-vis the pluripotent stem cell from
embryos may provide an advantage. As the first panel said,
these are promises. Remember President Nixon led a war on
cancer in 1968. Unfortunately, we are still a ways from there.
Research is critical but research crossing a line of
destroying human life I believe borders on not only the
unnecessary but the immoral at this time.
I thank you, sir, for the privilege of testifying again.
Senator Specter. Thank you very much, Dr. Young, for your
testimony. Thank you.
STATEMENT OF MARY JANE OWEN, M.S.W., EXECUTIVE
DIRECTOR, NATIONAL CATHOLIC OFFICE FOR
PERSONS WITH DISABILITIES
Senator Specter. We now turn to Ms. Mary Jane Owen,
Executive Director for the National Catholic Office for Persons
with Disabilities since 1991. In 1986, Ms. Owen established the
Disability Focus, Incorporated which is an organization
promoting a disability perspective on all social policy and
advocating for the appointment of qualified people with
disabilities at all levels of Government and business. She
received her master's degree in social work from the University
of California at Berkeley.
We welcome you here, Ms. Owen, and if we may be of any
assistance to you on the hearing or anything else, let us know.
We look forward to your testimony.
Ms. Owen. Thank you. That is very kind of you to offer your
assistance.
I am so gratified to be here to be able to participate in
the testimony that you are hearing about this very critical
issue.
If I overstay my time in terms of talking, please forgive
me. It is not out of disrespect for the lights. As a blind,
partially hearing person, it is very hard to stop me when I get
going. So, I simply in advance say please give me warning if I
have extended----
Senator Specter. Ms. Owen, we will give you a little slack
here.
Ms. Owen. OK.
I am very concerned about this topic. I think that it is
completely unnecessary and immoral for us to use live embryos,
to use living human beings for research which is unnecessary
because as my two compatriots here on this panel have stated so
clearly, we have alternatives.
I hope that you will review my written testimony which has
been submitted to you. I think that so much of what I was
planning to say, in terms of the moral concerns, as well as the
alternatives, have already been very, very adequately covered
by them. But I do think that as a disabled person, as a person
who has been a national leader in this disability rights
movement since 1972, that I do want to share some of my
concerns as it relates to disability and cure.
The title that I gave my testimony involves the word
``frenzied.'' I think that we are involved in a frenzied
pursuit based on fear. As a matter of fact, I think one of the
members of the committee even referred to the fact that we fear
our own vulnerability.
You know, it was over 20 years ago that I first developed a
definition that has been accepted rather widely, and that is
that disabilities are the normal, expected, anticipated outcome
of the risks and stresses of the living process.
Now, I think that many of us do not accept that reality. We
are fragile creatures. I do not think that it is an error in
our Creator's planning. I think that our vulnerability is one
of the factors that causes us to be more civilized.
I see fear in my society almost every day. I give some
examples in my written testimony where people approach me and
say, I would rather be dead than live like you. I am blind. I
am partially hearing. I am a wheelchair user because of
neurological and spinal injuries. But I live a very successful
life because of the advances that we have made in treatment and
in rehabilitation.
Sometimes as an outpatient at the National Rehabilitation
Hospital, I am amazed at the lack of those services that are
available to young people currently entering that facility.
When I was there most recently, I was there for 6 months. That
does not happen anymore. Young people today are being turned
out of that facility in a matter of weeks. We need to fund the
kind of things that allow people with disabilities to truly
fulfill their potential.
Yes, I think we are terrified of disabilities. We are
terrified of disease.
It was suggested that possibly we were looking for a cure.
I think that realistically what we can expect is a postponement
of our mortality, and Dr. Young referred to that. We do not
cure our vulnerability because it exists, and I would submit
that it is a positive, not a negative.
When I travel--and I often travel alone. And it is obvious
to everyone, including you, Mr. Chairman, that I might need
assistance. It is amazing to me how, as I travel alone, as I
need help, as I need assistance, that people thank me when they
have been given the excuse to be civilized, to be interactive,
to be a part of a larger community.
I would further submit that this fear, this frenzy to
escape our destiny, which truly is what Dr. Young was referring
to--we are biodegradable. I would submit that our fear, our
frenzied fear, our dread, our abhorrence of our shared
vulnerability is what drives this pursuit of some way to
escape.
You know, as a child, I mentioned in my written testimony
that there was a Navajo rug that hung on the family home. There
is always a flaw in those beautiful Navajo rugs. Any of you
from New Mexico know this. And I remember as a child recovering
from meningitis, running my finger over the break in the
pattern and thinking this is profound. This is profound.
We are not manufactured like Ken and Barbie. We are not
uniform. You know, Ken and Barbie, if they come down the
production line and there is a flaw, they are pulled out. They
are no longer a part of the Mattel family. They are cast aside
into the bin for discards. Their essence is regenerated and
becomes a part of another Ken or Barbie. We, in contrast, are
created unique, separate from all else. We have gifts, we have
weaknesses. My weaknesses, your weaknesses, my strengths, your
strengths, each one of you, I would suggest that those
intertwined weavings create the strongest social fabric.
So, I would say I would beg you to stop and think. Is the
fear of disability so great in this Nation that we must choose
an immoral and unnecessary strategy to avoid recognition that--
yes, we do need to cure, we do need to treat, we do need to
find ways to prolong life. And I am fully in support of stem
cell research, and I would hope that NIH would continue to be
funded to the extent that it can pursue adult stem cell
research. Adult stem cell research.
You have heard and I read some of the same articles that
Senator Brownback referred to. They are so inspiring. Do I want
to see again? Do I want to hear as well as I used to? Do I want
to dance again? Yes, it would be okay. But please know that I
do not want those things at the cost of any living person. And
I consider live embryos to be people.
I want to just say one more thing in terms of my daughter.
My daughter was born with a 1 percent chance of survival. I had
heard as a young woman during the height of the push for
abortion that children the size of my daughter were simply
collections of cells. I watched her. I watched her tenacity. I
watched her desire to live. I watched every breath as she was
breathing it. If she had not had a pro-life doctor there who
spent 24 hours promoting her life, she would not have survived.
She struggled to stay alive. Every time I hear people talk
about the ease of utilization of live embryos, I am reminded of
the beauty of that tiny, tiny little body of my daughter. She
is a wonderful, brilliant, young woman today. I am so thankful
that there was no one in the corridor outside the delivery room
who might have grabbed her.
Senator Specter. Ms. Owen, your red light has been on for
quite a while now. If you could----
Ms. Owen. I am sorry.
Senator Specter. I say your red light has been on for a
while now.
Ms. Owen. Thank you for letting me go on. I appreciate your
indulgence.
Senator Specter. I know you have not observed, you cannot
see it, but we did want to hear you out. If you could wrap up.
Ms. Owen. I am finished.
PREPARED STATEMENT
I just want to say that immoral? Yes. Unnecessary? Yes. The
results from adult stem cell research is very exciting and I
hope that each one of you are keeping up with that research
possibility.
Senator Specter. Thank you very much, Ms. Owen.
[The statement follows:]
Prepared Statement of Mary Jane Owen
INTRODUCTION
First, I wish to thank all who have welcomed me to appear before
this important Committee to offer testimony on an issue with profound
social, medical, and most of all, moral implications.
I am Mary Jane Owen, the Executive Director of the National
Catholic Office for Persons with Disabilities, a national organization
charged with creating welcome and justice, within the church and the
total fabric of society, for over 12 million Catholics with
disabilities. This mission requires extensive travel, speaking and
writing, which I have been able to do despite--or possibly better--
because of being a blind, partially hearing woman with neurological and
spinal impairments which require use of a wheelchair.
Over a half century of my working life, I have been a psychiatric
social worker, a professor of social research, a federal administrator,
a free-lance consultant and a businesswoman. More importantly, I have
been a participant observer in the difficult decades of change and
progress as our society has created new options and opportunities for
people with disabilities. Remarkable changes have taken place,
including passage of the Americans with Disabilities Act, an effort in
which I worked, along with thousands of other disabled people in our
search for fuller participation within our society.
I am here to urge you: Do not, in the name of progress for disabled
people, certify or justify the destructive harvesting of human embryos
for stem cell research, a practice both immoral and unnecessary.
While disabled people are interested in cures, as well as better
tools for living, greater inclusion in society and other possibilities
which will improve our quality of life, we are not so desperate for
cures that moral considerations disappear.
Do not use our struggles and aspirations to justify an immoral
policy that will encourage the destruction of unborn babies. Instead,
direct researchers to use the many alternative sources of stem cells so
that this promising area of research may be developed in an ethically
defensible manner!
MORAL CONSIDERATIONS
Congress determined once, and I believe rightly, that harvesting
fetuses is wrong. There are many moral reasons for this. In addition to
the opposition to abortion itself, shared by many Americans, there is
also the conviction that human embryos and fetuses should not be
harvested lest they come to be seen as products for sale.
This is not an insignificant issue. The medical and biotechnology
revolution will be even more powerful in its implications than our
internet/information technology revolution. Medical and biological
technology can change our very identity as human beings. I do not
propose that we stop this revolution, but I am confident we must
carefully consider where we want to go and where we are being carried
by frenzied attempts to deny our shared vulnerability.
Technology, commerce and science are all pushing us inexorably in
the direction of regarding human beings as products. We need to
consider if we continue down this slippery slope, are we ready to
justify the creation of human beings for spare parts? We are forced to
ask this question today, for the possibilities lie just ahead.
Researchers are already harvesting fetuses, and their so-called utility
has already become part of the moral calculus of abortion. Now the
demand for live embryos for stem cell harvesting threatens to become
part of the moral calculus of fertility treatments.
Yes, medical progress is desirable. Yes, new research vistas
require new ethical guidelines--But I pray they will NOT involve a
repudiation of our past moral stance and move toward an exclusively
utilitarian set of ``ethical'' rules. We must carefully calculate what
is essential to human decency and then defend that essence, even
hedging it about with an extra margin of caution. We Americans disagree
about many things, but most of us consider the idea of harvesting
fellow human beings for the advantage of the few as abhorrent. Let us
respect that moral intuition and the traditional values upon which it
rests.
There are medical experts who will assure us we need not sacrifice
scientific progress because of our abhorrence of a utilitarian approach
toward human life.
THE ALTERNATIVE SOURCES OF STEM CELLS
It is clear that researchers should have access to stem cells. The
issue is: why embryonic stem cells? Some would claim that refusing to
subsidize this particular kind of stem cell research would unduly
hamper medical progress. However, there are many other sources of these
vital human tissues which give clear indication of their potential for
positive results. Some would even avoid the possibilities of rejection
which are inherent in the use of tissue not recognized by the host
body.
Exciting possibilities lie ahead in making use of self-contributed
stem cells, as well as those harvested as a result of necessary
surgical interventions, tissue contributed by consenting adults and the
by-products of natural births.
THE PUSH TO BETRAY THE UNBORN
Congress has already attempted to prevent federal funding for
harmful human embryo research, but now the issue has arisen again, and
some say it is urgent to allow and encourage the use of embryonic cells
in research. What has changed since that earlier decision? Yes,
research possibilities have opened up but every day we are learning
more about alternative sources of stem cells.
And so we are forced to ask ourselves why a civilized society would
seek to overcome its moral compunctions about harvesting the unborn. Is
it that our fear of disease, disability and mortality is so
overwhelming that we forget or deny what we know is morally correct? I
am suggesting the frenzied rush to harvest embryonic humans is based
upon an undue fear of human fragility and disabilities. We fear
anticipated pain. Yet we know that management of pain has made
tremendous strides in the past few years.
I witness society's fear and anxiety about disability every day. I
have often been amazed by strangers who approach me to confide that
they would rather be dead than become blind or use a wheelchair. Aside
from the bluntness of these remarks, what surprises me is that many
people can not imagine that I have a happy life. And yet I am as happy,
as successful and productive as anyone I know. It seems obvious to me
that their fears about their own future disabilities keep them from
seeing the reality of my life--and the possibilities for all people
with assorted disabilities.
In my lifetime I have also observed our medical system negate the
idealism of the Hippocratic Oath and move from viewing its services as
having ties to charity. Within a for-profit enterprise, the view that
some human lives are potential ``product'' may be tempting but it is
still immoral. The sacrifice of some human lives for the benefit of
others must be defined as illegal, as it has been in the past.
My view can be summarized thus: Many of my fellow citizens suffer
unduly from fears and frenzied anxiety about assorted disabilities and
fragilities.
A NEW UNDERSTANDING OF DISABILITY AND VULNERABILITY
Twenty years ago I proposed a new and positive definition of
disabilities which has been used by advocates in the intervening years:
Disabilities are the normal, expected and anticipated outcome of
the risks, stresses and strains of the living process itself.
The eventual outcome of the shared fragility of our bodies is the
development of physiological glitches at some point in the normal life
cycle. Disabilities are not something which happens only to the unlucky
few but is an event which takes place for us all. We may face this
eventuality before birth, in early life, during the height of our
productive years or at the end of life. When we view our shared
vulnerability with these conceptual lenses, we understand that the
principles of universal design should be considered as we modify our
environments, programs and institutions.
Broadly available medical services, rehabilitation techniques and
technology and the evolving expectations of millions of American
citizens with disabilities can revolutionize the future status of those
millions of Americans who are destined to experience physical,
cognitive and sensory limitations. Public recognition and support of
these alternatives can relieve the rampant anxiety evident in the
frenzied pursuit of ``cures'' at any moral cost.
Unfortunately, the picture of life with disabilities often seen in
the popular media and skillfully utilized by those segments of our
population seeking to deny the essential dignity and value of all human
life, are generating the current pressure to authorize destructive
harvesting of stem cells from embryos as essential in our struggle to
prolong our productive lives.
We are not manufactured like Ken and Barbie, expected to fit a
model of physical perfection; required to be uniform in all those
characteristics by which we might be judged as having value. We are not
fabricated of high impact plastic. When a single Ken or Barbie appears
on the production line with even a tiny flaw, the results are thrown
into the recycle bin. That member of the Mattel family will be broken
down into its component parts to be used again in the ongoing effort to
reach uniform perfection.
In contrast to that process, each of us was uniquely and
individually created. For those of us who are Christian, we believe
that this variety in abilities and strengths, disabilities and
weaknesses, in some mysterious way which we can not fathom, reveal some
essential part of our Heavenly Father. Other faith and cultural groups
confirm the normality of assorted ``imperfections.'' The beautiful
Navaho rug that hung on the wall of my childhood home was woven with
the traditional ``flaw'' which marked it as reflective of the
traditional belief that only the ``Creator'' was perfect. As I traced
that defect in perfection, it comforted me as I dealt with the minor
disabilities caused by an early bout of meningitis.
Not only are we created to be unique, we are created of extremely
fragile material. We may be born without disabilities, but we must
anticipate that at some point in our lives we will be forced to
recognize our shared vulnerability. That recognition can inspire us to
acknowledge our need for each other and for the Creator. It may also
move us to cherish those of our community who are in need of
assistance, medical services or rehabilitation.
Many of us unfortunately believe that disabilities are a cosmic
accident which we must correct. I agree that we must work to do what we
can to reduce or prevent disabilities, using morally defensible means.
Much progress has already been made in prevention, treating or even
curing a variety of disabling conditions. However, I would affirm we
would never completely eliminate the vulnerability of the human
organism nor would it be such a great blessing if we could. My personal
experience convinces me that it is by God's wisdom that the gift of
life comes in fragile earthen vessels. Many of the virtues we feel are
the best that humanity has to offer, such as love, faith, hope, mercy,
and courage, are associated directly or indirectly with our
vulnerability.
I suggest that we, as a nation, need to use these alternative
lenses through which to view human vulnerability and disabilities as we
re-compute our outmoded ``scientific'' formulae for assessing other
people's quality of life. We need you, members of the United States
Senate, to call for a nation-wide calming of the frenzied research
efforts based upon destroying future citizens, rather than endorsing
this national anxiety. The potential quality of any human life can not
be judged by outside authority.
THE POSITIVE ASPECTS OF HUMAN VULNERABILITY
I propose that the catalytic effect of our mutual need for each
other is a social positive which must not be lost for it fosters a
sense of the need for mutual aid and interaction within our
communities.
We no longer need to respond in the same way our ancient ancestors
did to the dangers rampant in their primitive world. Fearful avoidance,
the patterns of behavior called forth by fear that the weak and
vulnerable would fall prey to the wooly mammoth, is no longer
appropriate. Superior brawn, eyesight, hearing and speed are not the
only human characteristics essential today. Many of us sit at our
computers which meet the needs of those of our brothers and sisters
with the palsied movements of Parkinson disease; the paralyzed arms of
the quadriplegic, or which speak to the blind or accommodate the
confused efforts of the poor speller. Today these ``imperfect''
characteristics no longer need throw us into a panic or deep
depression. Our quality of life can remain quite satisfactory in spite
of those impairments which would have severely limited the lives of our
grandparents.
In my travels, I have observed that the intertwining threads of the
interaction between people are enhanced when we acknowledge our need
for assistance or help. Apparently, at this point in our history, we
seek ``excuses'' to be interactive and to discard our sense of complete
autonomy. This serves to refresh our sense of being an essential part
of a vital community. This interaction is a potent antidote to the
rampant alienation which threatens so many of our neighborhoods today.
ON A PERSONAL LEVEL
I carry the genes for the blindness which knocked me from my ivory
tower. And I may have passed that pattern of visual impairment to my
daughter. Growing up I was surrounded by elderly blind women, all of
whom were active in their communities, well educated for their time and
place and recognized for their abilities more than their limitations. I
was unprepared for the loss of my sight at the height of my rapid climb
up the academic ladder.
The two women closest to me both died with Alzheimer's disease. I
may be programmed for a similar experience. Awareness of that
possibility makes each day of intellectual exploration more precious
and pushes me to greater achievement.
The women who built the base upon which I exist would never have
wished for a single life of an unborn baby to be sacrificed so that
their physical or cognitive challenges might have been eliminated or
postponed. They were strong feminists in the model of Susan B. Anthony.
They saw abortion as anti-woman and recognized that overcoming
challenges as a characteristic to be admired, not avoided.
When I visited with my aunt, Naomi Harward, during the period when
her Alzheimer's disease was being diagnosed, one morning I found her in
tears. She had been widely recognized as a political force, having
headed up a campaign to recall Governor Meecham. She was the oldest
woman to appear on the cover of MS Magazine.
She said, ``I don't want to have Alzheimer's.'' She had watched her
sister, my mother, another widely recognized and strong feminist, live
with that condition for long years. We embraced as I reminded her that
none of us know what the future holds. I told her that at each step in
her life she had showed me the way to live gracefully and successfully,
no matter the challenges faced. That was a powerful moment in my life.
She died quietly and peacefully during the few moments when her beloved
grandson and his wife stepped out of her room. We felt she had chosen
that moment to slip away and join those parts of herself which had
already gone before.
She was so strongly opposed to abortion; the idea of harvesting
stem cells from defenseless unborn children would have aroused loud and
political protest, I am sure.
When my daughter was born, she had less than 1 percent chance of
survival based upon her prematurity. I'd been told that babies at her
stage of development were merely blobs of cells, not unique and
individually crafted tiny people. I shall never forget the power and
beauty of her tiny body; the fragility of life and her stubborn hold on
it. I could see the intensity of her desire to live as each breath was
aided; each beat of her heart monitored. That image continues to
inspire me and that tiny essence of human life comes to mind every time
I hear reference to the harvesting of stem cells from unborn children
as an essential medical practice.
My daughter graduated cum laude from Harvard twenty years later and
continues to be a joy and support. Thank God no one was lurking in
those sterile halls, awaiting the emergence of a frail and fresh
``product'' to be marketed for research or ``therapeutic'' purposes.
I have neurological impairments and have sustained spinal injuries,
and while I enjoy dancing in my wheelchair, it might be pleasant to do
so in what is considered a normal way. But be very clear in this: I am
deeply opposed to any gain in my sight, mobility, or even my hearing if
it was purchased at the cost of a single human life.
As I've already noted, I may face Alzheimer's disease at some point
in my future. But that eventuality is less frightening than a world in
which, as a matter of medical intervention, one life can be casually
eliminated in order to offer a few additional months of ``normality''
to another.
I've talked with and cried with hundreds of my colleagues within
the disability movement. We seek better funding for rehabilitation, not
a quick fix. I challenge you, members of the United States Senate and
all my fellow citizens, to create environments, fund current
rehabilitation programs and alter perceptions of human vulnerability
which frighten those who fear what their future might hold. We can
escape from the ancient demons which haunt nightmares.
The worse thing in life is not disability, or pain, or even death.
The worse thing I can imagine is to create a society which sees itself
as justified in treating other people as objects to be used or
discarded, as best fits the desires of the moment. I would not wish to
live in such a world. And the moral choices we make today will surely
shape our future. Even a ``small thing'' like the fate of an unborn
child can have great implications as we create that future for
ourselves and our children.
Senator Specter. Senator Brownback, when we deal with the
morality issue, I totally agree with you that that is the
principal concern, that we proceed in an ethical and in a moral
way. I would join you totally in your objection, abhorrence,
disdain for interfering with human life. But the embryos which
are to be used here for extracting stem cells are discarded.
They are not to be used. They have been prepared for in vitro
fertilization, and if they are not used for the research, they
will not be used at all. So, how do you deal with that fact
that no human life is to be taken?
Senator Brownback. Well, I think the issue you raise here,
what you are talking about is strangely familiar from our past
where we say these are going to discarded and die anyway, so
therefore, why not get some benefit out of them. That sounds
strangely like some of the things that happened in World War
II.
Senator Specter. Well, how so? These have been discarded.
It is not that they are going to be discarded. How would that
run afoul or be analogous to what happened in World War II?
Senator Brownback. Well, you are taking live human embryos
in this case and they will be extracted--their stem cells from
them. You had the Nazis in World War II saying, of these
people, they are going to be killed. Why do we not experiment
on them and find out what happens with these experiments? They
are going to die anyway.
Senator Specter. But they were living people unlike the
embryos.
Senator Brownback. These are living embryos. These are
living embryos. And they are being treated in this case as
property.
Senator Specter. But the people whom the Nazis experimented
with in the abhorrent way in the holocaust were living.
Senator Brownback. These are living embryos. You are taking
living embryos.
Senator Specter. Let me ask you, Senator Brownback, do you
oppose in vitro fertilization?
Senator Brownback. I have not thought through that one, and
I am not prepared here today to talk about that particular
issue. The issue in front of us is you have a live embryo.
Senator Specter. I raise the in vitro fertilization issue--
and you are correct. It is not before us. We are exploring the
matter today, but we are going to have an opportunity to
discuss it on the Senate floor at greater length.
But I raise the in vitro fertilization issue because there
are some who do object to that, and it is a consequence of in
vitro fertilization that these embryos are created. There might
be an argument that every one of these embryos is entitled to
life. But the process of in vitro fertilization is to have a
large group and then to use some but not to use others. So,
this is something we will be getting into.
But I understand your point of view.
Let me shift for just a moment to the question of----
Senator Brownback. If I could on that very point.
Senator Specter. Go ahead, sir.
Senator Brownback. Because you are seeing the courts now
across the country starting to discuss this issue of are these
embryos then person or property. And that is at the core of
what we are talking about here. We will hopefully have some of
the legal opinions of what are developing on this, but does
that not sound dangerously close to things that we have skirted
around in the past and dealt with in this body a number of
years ago?
And my point on all this is we do not have to go this way.
Let us go the way that I will join you--and I do not know if
you can ask for a level of funding on adult stem cell research
that I would not support because I think it is so promising for
its potential and ethical. But let us not go into this one that
we have so many of these questions that mankind has been around
for a long time.
Senator Specter. Well, I am going to come to that in a
moment.
Ms. Owen. Could I respond to your question?
Senator Specter. Not yet, Ms. Owen. You can but not quite
yet.
When you talk about a person--you cannot, Ms. Owen, because
I want to develop this thought with Senator Brownback which we
are discussing, but I will come back to you.
When you talk about a person and we have these concerns
about when life begins and you have conception and then
abortion--and I am personally very much opposed to abortion,
although I think it has to be a decision for the woman.
But when you talk about conception and whether there is a
person, that conceived entity is on its way to life. But that
is not the situation--just to pursue this for another step,
Senator Brownback--with the discarded embryo. A discarded
embryo is not on its way to life. I do not like the concept of
property as opposed to person. As we consider the issue of a
person where there is conception, that entity is on its way to
life. But I do have that distinction which I would be
interested in your comment about with respect to the discarded
embryo.
Senator Brownback. If it is a live embryo and it is
destroyed for this purpose, it is killed. And you have that
destruction that is taking place. And then in this case we are
even saying that destruction we will then do this with the
parts out of it. I cannot see that that is something that this
great Nation really wants to move along when we have another
option that is so promising in front of us. We just do not have
to go this way.
Senator Specter. Well, let us take up that option for just
a moment with you, Dr. Young. I note, in one of the
commentaries from your magazine that you made available to us,
the following statement with respect to adult stem cells. I had
a concern from other materials which I have studied that the
stem cells from adult tissue, matured cells are already
differentiated into a narrow type, range of stem cells. Those
cells cannot support the kind of research on a broad basis.
Quoting from the publication which you have made available
to us on page 1419, ``Adult stem cells have a drawback,
however, in that some seem to lose their ability to divide and
differentiate after a time in culture. This short life-span
might make them unsuitable for some medical applications.'' Do
you disagree with that limitation on adult stem cells?
Dr. Young. I think the jury is out in general. This is a
very well-balanced set of articles, and there are other
articles there which state that one of the liabilities of the
embryonic stem cells is that they differentiate in a more
uncontrolled fashion, whereas it is better to use adult
embryonic cells which would be able to differentiate more
unidirectionally. I think that further research has to be
undertaken.
But in the interim, I do believe that it is critical to
focus on the animal work with embryonic stem cells which can
yield a great deal of information and the work on human adult
stem cells, and then pause for a while.
If I could add one answer to the question that you asked
the Senator, I think it could be very enlightening. There was a
case a short time ago where one of the in vitro fertilization
clinics--it was about a year and a half ago--found that they
had an embryo, ``a spare embryo,'' that was left from a woman's
in vitro fertilization about 6 to 8 years ago. It was reported
in the Los Angeles Times that that woman then desired to have
that embryo implanted and gave birth to a twin, if you would, 6
to 8 years down the line. It goes in accordance with what the
Senator was saying.
My remark that I really think the Senate needs to address
is that of the regulation of in vitro fertilization. I have
supported in vitro fertilization, have counseled couples in our
church, and have worked with them on this area. But there does
not need to be ``the development of excess embryos.'' And there
does not need to be the concept of spare or embryos that are
about to be destroyed. I think that is one the Senate might
like to look into.
Senator Specter. Before turning to Senator Harkin, Ms.
Owen, you had a comment you wanted to make. Ms. Owen?
Ms. Owen. Yes. I would like to respond to your question
about the viability.
Senator Specter. Fine. We would be pleased to hear you.
Ms. Owen. You see, I think that one of the things that
possibly needs to be considered is that fact that we are in a
society where we are beginning to talk about the sale of body
parts. I think that when we begin to say it is all right to
have excessive numbers of embryos created, we are saying go
ahead, private industry, go ahead, NIH, go ahead and create
excess embryos not just for in vitro fertilization, but with
the idea--maybe it is behind closed doors--but with the idea we
can make a profit by selling these embryos. So, I do not think
it is just simply a few embryos that we are talking about
today. We are talking about the potential of creating hundreds
of embryos for research purposes.
Senator Specter. Thank you, Ms. Owen.
Senator Harkin.
Senator Harkin. Thank you very much, Mr. Chairman. I too
want to thank especially my colleague, Senator Brownback, with
whom I have a great working relationship and friendship. I know
how deeply you feel about this issue, and I respect that. I
respect it greatly. I respect all of you who have perhaps come
at this from a different viewpoint perhaps than I come at it
from.
But I believe that by working together in a rational,
reasonable way, I think that we can find pathways out of this.
I just want to make sure that we are all talking about the same
thing and that we are all knowing that the words we use--we
kind of agree upon what they mean. I think a lot of times maybe
both sides of an issue talk to themselves a lot and they do not
talk to each other enough to try to figure out some pathways
that we can move ahead.
To that extent, I just again wanted to reiterate what our
chairman said, that we are looking at the--without getting into
whether adult stem cells will do the job or not. No one knows.
OK, we do not know. We do not know. But we do know and
scientists do have a belief that at least in the pluripotent
stage of embryonic stem cells that they do hold great promise.
Again, there are no guarantees out there. The only guarantee is
that if we do not do it, possible cures, interventions and
preventions for a whole host of illnesses will be put off for
longer periods of time. That guarantee we do have.
Second, with regard to the issue of embryos--again, Sam, we
get in this whole debate about the Nazi Germany and that kind
of thing. But I think we do have to keep some perspective on
this. I put something on this piece of paper, and I will bet no
one out there can tell me what is on that piece of paper
because you cannot see it. But I took my pencil and I put a dot
on it. That is how big we are talking about. That is how big
the human embryo is that we are talking about. In fact, in most
cases you cannot even see it with the naked eye.
Now, again I am not a theologian, but to equate that with
the individual person that the Nazis were experimenting upon I
think is to stretch the meaning of humanness and what a human
being is.
Now, with regard to that little dot that is on the piece of
paper, there are, as the chairman said, leftover--and this is
where it gets to you, Dr. Young. There are leftover embryos of
this size, frozen, about 100,000, Sam. We estimate about
100,000 of these are frozen in liquid nitrogen right now that
are left over. I have been told by scientists that we probably
do not need any more than that. I mean, we do not need to go
out and get more stem cells and that. We have 100,000 embryonic
stem cells right now left over from in vitro fertilization.
Regardless of what you may think about in vitro fertilization,
they are there. They are frozen. The question is, what is going
to happen to them?
Dr. Young, you pointed you--and I read that article about
the woman that can back 6 years later. Well, again, that is
fine.
Now, the guidelines that we set up--I am going to just jump
ahead here a little bit. The guidelines that were proposed said
this. And this gets to you, Ms. Owen, about the sale. You were
talking about selling human parts and stuff. The guidelines
stated three things. We could only use embryonic stem cells
from excess in vitro embryos, ones that are there right now.
OK? Second, there must be no financial compensation. No one can
sell these. No one can pay for them. And third, they can only
be used with the informed consent of the donors, the two
parents, whoever that is. So, if there is someone who donated
that in vitro fertilization and they are deceased now, they
cannot give that informed consent. Only those who can give
their informed consent could use it. Those are the guidelines.
So, we have no need to create more than the 100,000 that we
have now, regardless of what you may think about whether we
should go on with in vitro fertilization or not. Those are
already frozen. They are there. The question for us I think,
Sam, and others is they are there. Are we going to keep them in
liquid nitrogen forever and ever and ever? I doubt it.
Senator Brownback. Well, if I could answer that.
Senator Harkin. Yes.
Senator Brownback. Because you are seeing now court cases
of people coming back that these have been frozen and kept. The
couple is divorced.
Senator Harkin. Right.
Senator Brownback. One wants to implant the frozen embryo,
the other does not.
Senator Harkin. Yes.
Senator Brownback. And the court is then wrestling with the
issue, who gets to decide, and are they person or property. The
court is wrestling with this now. Should we in legislation
say--because as I read this, it would appear to me we are
saying these are property.
Senator Harkin. We are saying that only with the informed
consent of the two donors, Sam.
Senator Brownback. Well, but I am saying what the court is
wrestling right now.
Senator Harkin. But you have two donors that are wrestling
with each other.
Senator Brownback. You do but the central question, the
central legal question is, is it person or property? The court,
in a line of decisions that I think is probably going to start
developing here, is wrestling with the same sort of thing they
did 100 years ago in this country.
And there is a Boston case where they are saying, well, it
is not really a person, it is not really property, it is an
entity with the potential for life is one lower court ruling.
Now, does that sound like saying somebody is three-fifths of a
person and not a whole as we have in our prior history as a
country? I think in this legislation, we are saying these are
property to be decided upon by these two people what happens to
this.
I do not think we are there at deciding that issue at this
point, and I do not think it is wise for us to, particularly
since we have got an option that we do not have to do this.
Senator Harkin. The option I think, Sam, as others have
pointed out, and as Science magazine, as scientist, after
scientist, after scientist will tell you there may be some
benefit to adult stem cells, but as far as they can determine
now, their life is very short. They are not eternal like the
pluripotent stem cells of embryos.
Senator Brownback. Pluripotent are not eternal. I do not
think you would say that those would be eternal masses either.
Senator Harkin. No. They can regenerate. They can
regenerate.
Dr. Young. They can also differentiate very rapidly.
Senator Harkin. But they can also regenerate too.
So, you can use adult stem cells. I am not saying we should
not use them. I am saying why close off all these doors that
researchers could use.
Again, Sam, keep in mind there are private entities out
there right now conducting this kind of stem cell research. It
is being done chaotically. It is being done without guidelines.
Who knows what is happening out there?
I think we have taken the responsible, reasonable approach.
We set up a Bioethics Commission. We said only from excess in
vitro embryos, no financial compensation, informed consent of
the donors. To me, you know, you are talking about these as
being spare and products for sale. They are not for sale. These
are not spare. They are there.
It seems to me the height of morality to say that in order
to help someone's life to prevent Alzheimer's or ALS or to
regenerate neurons, to help people with juvenile diabetes, it
seems to me the moral thing to do would be to use what we have
there in these in vitro cells that are left over, the 100,000,
to permit the kind of ethical guideline structure that we set
up so that scientists can use those to help make lives better.
To me that seems to me--again, I know we differ on this but I
hope you give me as much benefit as I give you approaching this
from a moral standpoint.
Senator Brownback. And I have stated that repeatedly. I do.
I do not think our hearts are any different of what we want to
get done.
Senator Harkin. But I think our position is just as moral
and as morally defensible as your position.
Senator Brownback. Well, I would question whether we want
to destroy one human life for another. And there is private
work going on. There are also 10 States that have banned the
destructive embryo research even privately funded. 10 States
have already done that. So, this would have a Federal stepping
in on top of what States are doing as well.
We do not have to go this route. I listed the research that
is taking place in the very areas that you are talking about
solving and the promising work. Would it be not only moral but
also wise to invest more in that area and focus our efforts
there that we can get there faster?
Plus, we have not talked about the immunosuppressant
problem that you are going to have if we develop these off of
bodies that are not the same as the body that you want to put
this new entity in. You do not have that issue that you have to
wrestle with. If you develop pancreatic cells out of an embryo,
you can have, and in likelihood will have, immunosuppressant
problems with the body rejecting that. Whereas, if you pull it
out of your own adult stem cells, you do not have that issue.
I think we have got a wise and moral route that we can all
go together on.
Senator Harkin. With adult stem cells, you have one door to
open. Maybe it will work. But using other embryonic stem cells,
you open up a whole host of other doors that may lead you to
the right path for the prevention and the cure of many of our
common ailments. That is all I am saying.
Thank you, Mr. Chairman.
Senator Specter. Thank you very much, Senator Harkin.
Thank you very much, Senator Brownback, Ms. Owen, Dr.
Young. Dr. Young, you remind us that we are biodegradable.
Maybe now be can be bio-regeneratable.
Dr. Young. I think for a short time that is possible.
Senator Specter. One of the heros of my youth was Ponce de
Leon who searched for the fountain of youth, and if we have it
here, let us look at it.
Stay tuned, ladies and gentlemen. This debate is not over.
We will be on the Senate floor where we have unlimited time to
speak, and on this issue, we will probably be using it.
Thank you very much, Senator Brownback.
Ms. Owen. Thank you.
Dr. Young. Thank you very much.
STATEMENT OF CHRISTOPHER REEVE, ACTOR/DIRECTOR;
CHAIRMAN, CHRISTOPHER REEVE PARALYSIS
FOUNDATION
Senator Specter. We now turn to our third panel. We have
Mr. Christopher Reeve, Ms. Jennifer Estess, and Dr. Lawrence
Goldstein.
We thank you very much, Mr. Reeve, specially for your
personal crusade on stem cells. We noted with America generally
and the world the traumatic experience you had with your
accident on horseback and the severing of your spinal column,
but you have come back to lead this crusade in a very
inspirational way. The current issue of Time magazine has your
comments and pays special tribute to what you have done. I know
that this is a matter of--well, it is of life concern to you.
It is that important. Possible regeneration of your spinal
column to enable you to fly again. So, we thank you for all of
your extraordinary work here, and we thank you for being
available to testify. We now turn the floor to you.
Mr. Reeve. Well, thank you very much, Mr. Chairman. I
appreciate the opportunity to be here to testify.
I would start by saying that in vitro fertility clinics
have been around since the 1950's, and as far as I am aware,
even though the Pope, of course, stated a position against it
and it is a sin for a Catholic to use in vitro fertilization,
there has not been an enormous outcry or protest. In other
words, these clinics have existed in relative peace for nearly
40 years, and I do not understand why all of a sudden there is
a huge issue about it, now that discarded embryos will be used
for research instead of just being thrown into the garbage.
A critical factor in the quality of life for present and
future generations will be what we do with human embryonic stem
cells. These cells have the potential to cure disease and
conditions ranging from Parkinson's and MS to diabetes, heart
disease, to Alzheimer's, Lou Gehrig's, even spinal cord
injuries like my own. They have been called the body's self-
repair kit.
Their extraordinary potential is a recent discovery. And
much basic research needs to be done before they can be sent to
the front lines in the battle against diseases. But no obstacle
should stand in the way of responsible investigation of their
possibilities. To that end, the work should be turned over to
the Federal Government through the National Institutes of
Health. That will avoid abuses by for-profit corporations,
avoid secrecy and destructive competition between laboratories,
and ensure the widest possible dissemination of scientific
breakthroughs. Human trials should be conducted either on the
NIH campus or in carefully monitored clinical facilities.
Now, fortunately, stem cells are readily available and
easily harvested. In fertility clinics, women are given a
choice of what to do with unused fertilized embryos: they can
be discarded, donated to research, or frozen for future use.
Under NIH supervision, scientists should be allowed to take
cells only from women who freely consent to their use for
research. One very important factor is that this process would
not be open-ended. Within 1 to 2 years, a sufficient number
could be gathered and made available to investigators. So, for
those reasons, the ban on federally funded human embryonic stem
cell research should be lifted as quickly as possible.
Again, why has the use of discarded embryos for research
suddenly become such an issue? Is it more ethical for a woman
to donate unused embryos that will never become human beings or
to let them be tossed away as so much garbage when they could
help save thousands of lives?
Now, treatment with stem cells has already begun. They have
been taken from umbilical cords and become healthy red blood
cells used to cure sickle-cell anemia. Stem cell therapy is
also being used against certain types of cancer. But those are
cells that have significantly differentiated; that is, they are
no longer pluripotent or capable of transforming into other
cell types. For the true biological miracles that researchers
have only begun to foresee, medical science must turn to
undifferentiated stem cells. We need to clear the path for them
as quickly as possible.
Now, controversy over the treatment of certain diseases is
nothing new in this country. Witness the overwhelming
opposition to Government funding of AIDS research in the early
1980's. For years, the Government tossed this issue around as a
political football until a massive grassroots effort forced
legislators to respond. And today NIH is authorized to spend
approximately $1.8 billion annually on new protocols, and the
virus is largely under control in the United States.
Now, at this point I wish to submit a letter of support
from four theologians representing the Protestant, Catholic,
Jewish, and Islamic faiths. They say:
Our opinions about embryonic stem cell research reflect
several different religious perspectives: Jewish, Catholic,
Protestant and Islam. While they do not represent a single
voice from these religious communities, they do offer a
collective belief that is based on similar views about certain
moral and ethical questions.
According to our religious beliefs, all human life must be
protected. However, they also indicate that there is a
significant difference between an embryo suspended in liquid
nitrogen that will never be implanted inside a womb and an
unborn child who is already in the womb.
Our religious beliefs also stress the importance of
compassion. Thus, we support embryonic stem cell research
because it would use these frozen or otherwise discarded
embryos to help ease the suffering of those with catastrophic
diseases such as diabetes, Parkinson's, cancer, Alzheimer's,
heart disease, osteoporosis and arthritis.
As theologians, we put a great deal of importance upon the
need for ethical standards in biomedicine. Currently, stem cell
research is being conducted solely in the private sector, where
it is not subject to the important guidelines developed by the
NIH, parameters that reflect critical input from ethicists and
theologians. However, the guidelines would only come into
effect with Federal funding, which is another reason we support
moving forward with the research under NIH. In addition,
Federal funding would not only speed the discovery of cures,
but ensure infrastructure is in place that will guarantee
ethical conduct and maximize the benefit to society.
Our religious beliefs tells us that embryonic stem cell
research is worthy of Federal support for millions across the
country who are suffering from diseases. We hope you will join
us and support stem cell research through the NIH.
We look forward to your support for this research, as the
lives of millions are counting on you.
And this is signed by Rabbi Elliot Dorff, Ph.D., who is
Professor of Philosophy at the University of Judaism. It is
signed by Nancy J. Duff, Ph.D., Associate Professor of
Theological Ethics at Princeton Theological Seminary. It is
signed by a name I cannot pronounce: Abdulaziz Sachedina. I
cannot say this. Anyway, Islamic Ph.D., Department of Religious
Studies at the University of Virginia. And in my opinion, most
importantly, by Margaret Farley, Ph.D., who is Professor of
Christian Ethics at the Yale University Divinity School, and
she is a Catholic.
PREPARED STATEMENT
Finally, I wish to enter into the record a list of over 100
disease groups, clinicians, foundations, universities and
medical schools, all of whom are supportive of me and my
advocacy for stem cell research. You will see that in
attachment 2.
But while we prolong the stem cell debate, millions
continue to suffer. It is time to harness the power of
Government and go forward. Thank you.
[The statement follows:]
Prepared Statement of Christopher Reeve
WE MUST PURSUE RESEARCH ON EMBRYONIC STEM CELLS.
With the life expectancy of average Americans heading as high as 85
to 90 years, it is our responsibility to do everything possible to
protect the quality of life of the present and future generations. A
critical factor will be what we do with human embryonic stem cells.
These cells have the potential to cure diseases and conditions ranging
from Parkinson's and multiple sclerosis to diabetes and heart disease,
Alzheimer's, Lou Gehrig's disease, even spinal-cord injuries like my
own. They have been called the body's self-repair kit.
Their extraordinary potential is a recent discovery. And much basic
research needs to be done before they can be sent to the front lines in
the battle against disease. But no obstacle should stand in the way of
responsible investigation of their possibilities. To that end, the work
should be funded and supervised by the Federal Government through the
National Institutes of Health (NIH). That will avoid abuses by for-
profit corporations, avoid secrecy and destructive competition between
laboratories and ensure the widest possible dissemination of scientific
breakthroughs. Human trials should be conducted either on the NIH
campus or in carefully monitored clinical facilities.
Fortunately, stem cells are readily available and easily harvested.
In fertility clinics, women are given a choice of what to do with
unused fertilized embryos: they can be discarded, donated to research
or frozen for future use. Under NIH supervision, scientists should be
allowed to take cells only from women who freely consent to their use
for research. This process would not be open ended; within one to two
years a sufficient number could be gathered and made available to
investigators. For those reasons, the ban on federally funded human
embryonic stem cell research should be lifted as quickly as possible.
But why has the use of discarded embryos for research suddenly
become such an issue? Is it more ethical for a woman to donate unused
embryos that will never become human beings, or to let them be tossed
away as so much garbage when they could help save thousands of lives?
Treatment with stem cells has already begun. They have been taken
from umbilical cords and become healthy red cells used to cure sickle-
cell anemia. Stem cell therapy is also being used against certain types
of cancer. But those are cells that have significantly differentiated;
that is, they are no longer pluripotent, or capable of transforming
into other cell types. For the true biological miracles that
researchers have only begun to foresee, medical science must turn to
undifferentiated stem cells. We need to clear the path for them as
rapidly as possible.
Controversy over the treatment of certain diseases is nothing new
in this country: witness the overwhelming opposition to government
funding of AIDS research in the early 1980's. For years the issue was a
political football until a massive grass-roots effort forced
legislators to respond. Today, the NIH is authorized to spend
approximately $1.8 billion annually on new protocols, and the virus is
largely under control in the United States.
In conclusion, I wish to submit a letter of support from four
theologians representing the Protestant, Catholic, Jewish and Islamic
faiths (see Attachment 1).
And finally, I wish to enter into the record a list of over 90
disease groups, clinicians, foundations, universities and medical
schools, all of whom have endorsed my testimony (see Attachment 2).
While we prolong the stem cell debate, millions continue to suffer.
It is time to harness the power of government and go forward.
Attachments.
Attachment 1
October 12, 1999.
Hon. J. Dennis Hastert,
House of Representatives,
Washington, DC.
Dear Representative Hastert: Our opinions about embryonic stem cell
research reflect several different religious perspectives: Jewish,
Catholic, Protestant and Islam. While they do not represent a single
voice from these religious communities, they do offer a collective
belief that is based on similar views about certain moral and ethical
questions.
According to our religious beliefs, all human life must be
protected. However, they also indicate that there is a significant
difference between an embryo suspended in liquid nitrogen that will
never be implanted inside a womb, and an unborn child who is already in
the womb.
Our religious beliefs also stress the importance of compassion.
Thus, we support embryonic stem cell research because it would use
these frozen or otherwise discarded embryos to help ease the suffering
of those with catastrophic diseases such as diabetes, Parkinson's,
cancer, Alzheimer's, heart disease, osteoporosis and arthritis.
As theologians, we put a great deal of importance upon the need for
ethical standards in biomedicine. Currently, stem cell research is
being conducted solely in the private sector, where it is not subject
to the important guidelines developed by the National Institutes of
Health (NIH)--parameters that reflect critical input from ethicists and
theologians. However, the guidelines would only come into effect with
federal funding, which is another reason we support moving forward with
the research under NIH. In addition, federal funding would not only
speed the discovery of cures, but ensure infrastructure is in place
that will guarantee ethical conduct and maximize benefit to society.
Our religious beliefs tell us that federal funding of embryonic
stem cell research is worthy of federal support for millions across the
country who are suffering from diseases. We hope you will join us and
support stem cell research through the NIH.
We look forward to your support for this research, as the lives of
millions are counting on you.
Sincerely,
Rabbi Elliot Dorff, Ph.D.,
Professor of Philosophy,
University of Judaism
Margaret Farley, Ph.D.,
Professor of Christian
Ethics, Yale University
Divinity School
Nancy J. Duff, Ph.D.,
Associate Professor of
Theological Ethics,
Princeton Theological
Seminary
Abdulaziz Sachedina, Ph.D.,
Department of Religious
Studies, University of
Virginia
______
Attachment 2
Editor's note: This is a list of over 100 disease groups,
clinicians, foundations, universities and medical schools, all of whom
are supportive of Christopher Reeve and his advocacy for stem cell
research. This group of supporters was gathered with the assistance of
the American Society for Cell Biology. For more information, please
contact Tim Leshan at 301-530-7153 or at [email protected].
AIDS Action
Alliance for Aging Research
Alpha One Foundation
ALS Association
American Association for Dental Research
American Association for the Advancement of Science
American Association for the Study of Liver Diseases
American Association of Anatomists
American Association of Immunologists
American Autoimmune Related Diseases Association
American College of Obstetricians and Gynecologists
American Dental Education Association
American Foundation for AIDS Research
American Gastroenterological Association
American Medical Association
American Parkinson Disease Association
American Pediatric Society
American Physiological Society
American Society for Biochemistry and Molecular Biology
American Society for Cell Biology
American Society for Clinical Nutrition
American Society for Microbiology
American Society for Pharmacology and Experimental Therapeutics
American Society for Reproductive Medicine
American Society of Hematology
American Society of Human Genetics
American Society of Pediatric Hernatology/Oncology
Americans for Medical Progress
Association for Research in Vision and Ophthalmology
Association of American Medical Colleges
Association of American Universities
Association of Independent Research Institutes
Association of Medical School Pediatric Department Chairs
Association of Professors of Medicine
Association of Subspecialty Professors
Bay Area Bioscience Center
Biophysical Society
Boston University Medical Center
Canavan Research Fund
Cancer Treatment Research Foundation
Candlelighters Childhood Cancer Foundation
Cedars-Sinai Medical Center
Christopher Reeve Paralysis Foundation
Coalition of Advocates for Research on the Eye (CARE)
College of American Pathologists
Cooley's Anemia Foundation
Coriell Institute for Medical Research
Council of Graduate Schools
Endocrine Society
Federation of American Societies for Experimental Biology
Foundation Fighting Blindness
FRAXA Research Foundation
Friends of the National Institute of Dental and Craniofacial Research
Genetics Society of America
Hadassah
Harvard University
International Foundation for Anticancer Drug Discovery
International Longevity Center--USA, Ltd.
International Myeloma Foundation
Interstitial Cystitis Association
Jeffrey Modell Foundation
Johns Hopkins University
Joint Steering Committee for Public Policy
Juvenile Diabetes Foundation International
Kidney Cancer Association
Lankenau Medical Research Center
Lombardi Cancer Center
Medical College of Wisconsin
Medical University of South Carolina
Memorial Sloan-Kettering Cancer Center
Mount Sinai School of Medicine
Myasthenia Gravis Foundation of America
National Alliance for Eye and Vision Research
National Alopecia Areata Foundation
National Association for Biomedical Research
National Association of State Universities and Land-Grant Colleges
National Childhood Cancer Foundation
National Coalition for Cancer Research
National Health Council
National Psoriasis Foundation
Oncology Nursing Society
Paralyzed Veterans of America
Parkinson's Action Network
Parkinson's Disease Foundation
Patients' Coalition for Urgent Research (CURe)
Prevent Blindness America
Project A.L.S.
The Protein Society
PXE International
Radiation Research Society
Research!America
Research Society on Alcoholism
Research to Prevent Blindness
RESOLVE, National Infertility Association
Sjogren's Syndrome Foundation
Society for Pediatric Research
Society for Women's Health Research
The Genome Action Coalition (TGAC)
Treatment Action Group (TAG)
University of California, San Diego School of Medicine
University of Minnesota
University of Rochester Medical Center
University of Washington
University of Wisconsin-Madison
UPMC Health System
Washington University School of Medicine
Weill Medical College of Cornell University
Senator Specter. Thank you very much, Mr. Reeve, for that
very impressive testimony. We see your enthusiasm and your
determination, notwithstanding your own personal situation, and
we will work with you to try to get the research necessary to
revitalize the spinal column.
We will make a part of the record, Mr. Reeve, the long list
of groups, clinicians, foundations, universities, and medical
schools which support stem cell research, which you have
provided to us, together with the letter which you made
available, and the record will be able to handle all those
names we cannot pronounce.
Mr. Reeve. Thank you.
STATEMENT OF JENNIFER ESTESS, ACTOR/PRODUCER
Senator Specter. We turn now to Ms. Jennifer Estess, who
helped form the off-Broadway theater company, Naked Angels. She
is the Executive Producer of a CBS original movie based on her
life, scheduled to air early next year. She received her
bachelor's degree in fine arts from the New York University and
is now President of the Amyotrophic Lateral Sclerosis
Foundation which she organized in 1998 after being diagnosed
with ALS in 1997 at the age of 35.
We thank you very much for joining us, Ms. Estess, and look
forward to your testimony.
Ms. Estess. Thank you.
Good morning, Chairman Specter, Senator Harkin, and members
of the committee. I also want to thank Christopher Reeve for
inviting me here today to speak with him. It is an honor. And I
want to thank you for inviting me to speak today.
I am Jennifer Estess. I have ALS, also known as Lou
Gehrig's disease, which is always fatal.
As a little girl growing up in America, I had great plans.
I dreamed of falling in love, getting married, and raising a
family. I dreamed of making a difference.
As a teenager, I learned from my sisters that hard work,
commitment, and family would make my dreams a reality.
As a woman of 35, my American dream was starting to come
true. I was a producer living in New York City. I was working
12-hour days and loving it. I was a healthy and strong young
woman looking for Mr. Right and ready to take the next step.
Then one day I started to feel tired and run down. I
thought it was the flu. But weeks passed and the feeling did
not go away. A city block seemed like a mile. Stairs were like
mountains. And suddenly I was having trouble holding my 3-year-
old nephew in my arms.
It was like a scene out of a bad movie when the neurologist
turned to me and said, you have ALS. In 2 to 5 years, you will
lose the ability to walk, to talk, to swallow, to breathe. You
will die. There is no medicine I can give you.
That first year ALS took my legs, the second my hands, my
arms. Now I sit before you unable to breathe freely, but my
mind is fine and it is alert. And I am still dreaming.
The day of my diagnosis, I asked the neurologist why, if we
could transplant hearts and lungs, could we not also replace
damaged motor neurons, the cells destroyed in ALS? The
neurologist shook his head. He told me, the cell transplants
were science fiction.
Three years later I am here to say that we may have
discovered a way to replace the cells that carry messages from
the brain to the muscles.
Project ALS, which I built with my sisters and my friends,
has assembled and funded a Dream Team of scientists. In the
last 9 months, these scientists have produced stunning evidence
that neural stem cell replacement can replace damaged motor
neurons. These new cells may one day allow me to do the things
I miss so much like brushing my hair, laughing out loud, and
holding a cup of coffee.
The evidence now shows that neural stem cell replacement is
our best hope for treatment and cures, not only for ALS, but
for Alzheimer's, Parkinson's, stroke, multiple sclerosis, and
spinal cord injury affecting over 10 million Americans today. I
am sure each of you will be touched by one of these devastating
diseases.
I believe it is upon us as brothers, sisters, fathers,
mothers, Americans to join us in this growing family that will
solve these problems. I also believe that stem cell research
will be the answer for millions of people, babies, children,
and adults, who will be blind-sided as I was. And I am here
because I do not want anyone else to have to go through this.
Make no mistake. ALS is a national disaster. We must land
at the heart of this disaster. I hope that Congress, the
National Institutes of Health, and the Food and Drug
Administration will join Project ALS and me in pursuing the
safest, shortest distance between stem cells and the patients
who desperately need them.
Each day I speak from inside my body which has now become a
prison. I am still here and dreaming of an America that will
protect the right to life, liberty, and the pursuit of
happiness for all.
Thank you so much.
Senator Specter. Ms. Estess, we are very grateful to you
for appearing here today. We understand the difficulties that
you have described. Your voice is loud and clear and we hear
the message. When you appear and Mr. Reeve appears and we have
your circumstances in mind and know that you are representative
of many, many people in America and in the world whose lives
may be saved by stem cell research, it provides the kind of
public understanding that I think is going to be necessary to
change the law and activate this kind of National Institutes of
Health funding which is the source of the real hope to deal
with ALS, to deal with spinal cords, and to deal with heart
disease, and to deal with Parkinson's.
When you look at Senator Harkin and me and say that we will
have the same problems, we could tell you some stories of our
own. So, we thank you.
Ms. Estess. Thank you.
STATEMENT OF LAWRENCE B. GOLDSTEIN, Ph.D., PROFESSOR,
DIVISION OF CELLULAR AND MOLECULAR
MEDICINE, UNIVERSITY OF CALIFORNIA, SAN
DIEGO SCHOOL OF MEDICINE; INVESTIGATOR,
HOWARD HUGHES MEDICAL INSTITUTE
Senator Specter. Dr. Goldstein, we now turn to you,
Professor of Pharmacology at the Division of Cellular and
Molecular Medicine, investigator of the Howard Hughes Medical
Institute at the University of California, Ph.D. from the
University of Washington. You testified before the subcommittee
at one of our early hearings in January of last year. We thank
you for joining us and we appreciate your focus on the issue as
to adult stem cells versus embryonic stem cells.
Dr. Goldstein. And related topics, yes.
Mr. Chairman, members of the subcommittee, I am Lawrence
Goldstein. I am here today as a representative of the American
Society for Cell Biology. The society represents 10,000 basic
biomedical researchers most of whom work in our Nation's
leading research universities and institutes.
I am a professor now in the Department of Cellular and
Molecular Medicine at the University of California, San Diego
School of Medicine and an investigator in the Howard Hughes
Medical Institute. Before moving to San Diego, I was a
professor in the Department of Cellular and Developmental
Biology at Harvard University for 10 years. My research focuses
on the roles of protein motors in neuronal function and
neurodegenerative disease.
It is a pleasure to be here again today and it is a
particular honor to be here with Ms. Estess and Mr. Reeve who
have been such articulate advocates for biomedical research. My
oral remarks will be a shorter version of my written remarks,
so they will not be identical.
Senator Specter. Your full written statement will be in the
record, Dr. Goldstein.
Dr. Goldstein. Thank you, Senator.
I want to thank you, Senator Specter and Senator Harkin,
for your leadership in ensuring that the NIH is funded
sufficiently to pursue the most promising basic and clinical
research opportunities. One such opportunity is human embryonic
stem cell research. S. 2015, the Stem Cell Research Act of
2000, which you have introduced, would allow federally funded
scientists to not only use, but also to derive human embryonic
stem cell lines from embryos that are in excess of clinical
need and that would be donated with appropriate informed
consent from in vitro fertilization clinics. These cell lines
would be used for research purposes with the goal of developing
new therapeutic strategies to treat devastating human disease.
The American Society for Cell Biology stands firmly in
support of this bill. We believe that permitting peer-reviewed,
Federal funds to be used for this research is our best
assurance that the research will be of the highest quality and
benefit and performed with proper ethical oversight and public
input.
There are five supporting points I want to make.
First, as you have heard, human embryonic stem cells have
enormous potential research and therapeutic value because they
appear to be capable of generating many, if not all, of the
cell types that make up a human organism. Research work over
the past 20 years using mouse embryonic stem cells has
demonstrated that these cells by themselves cannot form an
adult organism, but they can differentiate into any adult cell
type. Most important, mouse embryonic stem cells have been used
in a variety of ``proof of therapeutic principle'' experiments
in several animal models of human disease. For example, these
cells appear to be able to produce neural progenitors that can
repair spinal cord damage and reconstitute various types of
brain cells and neurons. If reproducible with human embryonic
stem cells, we may be able to treat Parkinson's disease,
Alzheimer's disease, and other diseases such as ALS. We may be
able to produce bone marrow cells to treat cancer and other
diseases and pancreatic cells to alleviate diabetes. In fact,
we may be on the verge of a new era of medicine, one in which
cell therapy could help restore normal function to a variety of
affected tissues.
Second, some have argued that so-called adult stem cells
derived from adult tissues are of equivalent promise, less
ethically compromised, and should therefore be pursued
exclusively. But it is far too early to know if adult stem
cells have the same potential as embryonic stem cells, whether
they can be harvested in sufficient quantities to treat
disease, and whether they can grow indefinitely as can
embryonic stem cells. In fact, it is likely to take years to
find out if adult stem cells will be useful for treating many
diseases that may be treatable sooner with embryonic stem
cells.
Senator Specter. When you say years to find out, Dr.
Goldstein, could you give us your judgment on approximately how
long? Because we are really looking at an alternative here and
the duration is a relevant factor in so many people not being
treatable in the interim.
Dr. Goldstein. There are many different claims about how
long it will take to do these things. Adult stem cells could
give us something in a year. It could be 10 years. Embryonic
stem cells have shown, I think, greater promise in some of the
animal models, and we could be looking at something in 2 to 3
years.
But we cannot work with one hand tied behind our back, as
was so eloquently pointed out by Senator Harkin and by others.
And, of course, if we delay with diseases that prove to be
treatable with adult stem cells, we risk unnecessary delays for
patients such as Ms. Estess who may die or endure needless
suffering while the promise is being tested. Thus, it is
critical that we not prohibit or hinder research in any of
these areas.
The third point I want to make is that the Stem Cell
Research Act ensures that important avenues of medical research
will not be restricted to the private sector. Precluding
federally funded scientists from using human embryonic stem
cells would effectively bar the majority of the Nation's most
qualified researchers from pursuing scientific opportunities
most likely to lead to rapid advances.
In fact, past experience has proven that broad access to
breakthrough discoveries increases the likelihood of novel and
innovative extensions. The molecular biology revolution, as I
am sure you know, which is responsible for enormous social,
economic, and medical advances is the product of innovations
and discoveries made by thousands of highly qualified and
creative federally funded scientists who developed new
techniques and applications.
Fourth, an important feature of S. 2015 is that it will
allow federally funded investigators to derive human embryonic
stem cell lines with appropriate ethical safeguards. This is
important because we know that a variety of poorly understood
factors cause embryonic stem cells to lose their capacity to
differentiate into all possible cell types. This loss of
capacity may be caused by growth conditions, derivation
conditions, or other variables of handling or storage. Enabling
individual publicly funded investigators to derive cell lines
using a variety of conditions is the best way to learn what
conditions are critical to generate therapeutically useful
cells.
Fifth and finally, there are serious ethical implications
to not proceeding. Thus, we have heard many well-meaning
ethical concerns about the sacrifice of embryos to prepare stem
cells. However, the embryos in question will be legally
destroyed in any case, and I have to ask, is it ethical to
literally throw away one of our best opportunities to help our
friends, our parents, and our children, many of whom will
suffer and die if we do not find suitable therapies?
PREPARED STATEMENT
In closing, we have before us an unprecedented scientific
opportunity to engage in a noble effort to develop new forms of
medicine. That opportunity offers hope to the many millions of
our citizens who rely on the shared stewardship of our
scientists and our political leaders to enable science's
achievements to relieve all people of the burden of serious
disease.
I want to thank you for your courage in providing
leadership on this most important, indeed, life or death issue
and for inviting my testimony today.
[The statement follows:]
Prepared Statement Lawrence S. B. Goldstein
Mr. Chairman and members of the Subcommittee: I am Lawrence
Goldstein. I am here today as a representative of the American Society
for Cell Biology. The Society represents 10,000 basic biomedical
researchers throughout the United States and the world, most of whom
work in our Nation's leading research universities and institutes. It
is my pleasure to appear before you again and it is a particular honor
to be here with Mr. Reeve who has been such an articulate and effective
spokesperson on behalf of biomedical research.
I am a Professor in the Department of Cellular and Molecular
Medicine at the University of California, San Diego School of Medicine.
I am also an Investigator of the Howard Hughes Medical Institute.
Before moving to San Diego I was a Professor in the Department of
Cellular and Developmental Biology at Harvard University for 10 years.
My research focuses on the molecular and genetic analysis of protein
motors and their roles in neuronal function and neurodegenerative
disease.
I want to thank you, Senator Specter and Senator Harkin, for
ensuring through your leadership of this important subcommittee that
the NIH is funded sufficiently to pursue the most promising basic and
clinical research opportunities. One such opportunity is embryonic stem
cell research. S.2015, ``The Stem Cell Research Act Of 2000'' which you
have introduced, would allow federally-funded scientists to not only
use, but also to derive embryonic stem cell lines for research purposes
with the goal of developing new therapeutic strategies to treat
devastating human disease. The American Society for Cell Biology stands
firmly in support of this bill.
Just over a year ago, a milestone in biomedical research was
achieved when human embryonic stem cell lines were obtained by growing
cells from the inner cell mass of early stage human embryos. This
discovery catalyzed a serious debate on Capitol Hill about whether
federal funds should be used to support further research in this area.
At issue is whether the merits of public funding and the dreadful
burden of disease balance concerns about the origin of these special
cells. Because of its great potential to treat disease and alleviate
human suffering, the American Society for Cell Biology along with many
other scientific organizations and societies have expressed strong
support for Federal funding of this important research.
The American Society for Cell Biology supports The Stem Cell
Research Act Of 2000 for four major reasons:
First, research work over the past 20 years using mouse embryonic
stem cells has demonstrated the promise of embryonic stem, or ES, cells
for basic research and potential disease therapy. These cells by
themselves cannot form a mouse, but they can differentiate into any of
the cell types that comprise a mouse. Mouse ES cells have been used to
elucidate many important aspects of normal mouse embryology and
development, but, most important, mouse ES cells are currently being
used in a variety of ``proof of therapeutic principle'' experiments in
several animal models of human disease. For example, these cells appear
to be able to produce neural progenitors that can repair spinal cord
damage and reconstitute brain cells that produce the chemicals that
control cognition, motion and sensory perception. If reproducible with
human ES cells we may be able to treat Parkinson's disease and
Alzheimer's disease. We may be able to produce bone marrow cells to
treat cancer and other hematopoietic diseases, and pancreatic cells to
alleviate diabetes. In fact, we may be on the cusp of a new era of
medicine, one in which cell therapy could restore normal function to a
variety of affected tissues.
It is important to note that some have argued that so-called
``adult stem cells'', derived from adult tissues are of equivalent
promise, less ethically compromised, and should therefore be pursued
exclusively. But, notwithstanding important advances in the field of
adult stem cell research, it is far too early to know if adult stem
cells have the same potential as embryonic stem cells, whether they can
be harvested in sufficient quantities to treat or cure disease, and
whether they can grow indefinitely as can ES cells. For example, for
juvenile diabetes, there is little reliable evidence at present that
adult stem cells could be used for treatment, and thus embryonic stem
cells are the best near-term candidates for therapy. Furthermore,
embryonic, fetal and adult stem cells are very different from each
other. It is not at all clear that they will prove to be
interchangeable or equally receptive to manipulations that would make
them useful for therapy. Thus, it is likely to take years to find out
if adult stem cells will be useful for treating many diseases that may
be treatable sooner with embryonic stem cells. For diseases that prove
not to be treatable with adult stem cells, we risk unnecessary delays
for patients who may die or endure needless suffering while the promise
of adult stem cells is being tested. It is critical that we not
prohibit or hinder research in any of these areas.
Second, there is great medical need and urgency for stem cell
research. To understand the need for rapid research progress with human
pluripotent stem cells, one need look no further than many common, and
often fatal, diseases such as cancer, heart disease and kidney disease.
These diseases are treatable in whole or in part by tissue or organ
transplants, but there are persistent and deadly problems of rejection
and a woefully inadequate supply of suitable donor organs and tissues.
In addition, the grim arithmetic of most organ transplants requires
those who are seriously ill to wait for the tragic accidental death of
another person so that they may live. Worse, for juvenile diabetes and
many other diseases, there is not even a suitable transplantation
therapy or other cure. Unless we use federal funds for all aspects of
human pluripotent stem cell research new treatments for these
conditions may be delayed by years, and many who might otherwise have
been saved will surely die or endure needless suffering.
Third, The Stem Cell Research Act of 2000 ensures that important
avenues of medical research will not be restricted to the private
sector. We agree with the National Bioethics Advisory Commission that,
``relying on cell lines that might be derived exclusively by a subset
of privately funded researchers who are interested in this area could
severely limit scientific and clinical progress.'' The effect of
precluding federally-funded biomedical from such work will effectively
bar the majority of the Nation's most prominent and most-qualified
researchers from engaging in this critical research. Excluding these
publicly funded investigators will close off scientific opportunities
to those most qualified to make rapid and dramatic advances towards
using stem cells for the treatment of disease.
We also know that a variety of poorly understood factors cause
embryonic stem cells to lose their capacity to differentiate into all
possible cell types. This loss of capacity may be caused by growth
conditions, derivation conditions, or other variables of handling.
Enabling individual publicly-funded investigators to derive cell lines
using a variety of conditions in their own laboratories is the best
route to finding out what conditions are critical to generate useful
cells for therapeutic purposes. In the future, it is likely that cells
prepared in one's own laboratory will have been derived, stored, and
maintained in ways that maximize their potency for particular uses,
whereas cells obtained from commercial sources are likely to be of
unknown genetic background and history and therefore less useful for
some important studies.
Federal funding is also the best way to guarantee that stem cell
therapies are developed with the greatest consideration of the public
good. Left to the private sector alone, stem-cell derived treatments
may only be pursued for diseases that commercial companies project to
yield the largest profit if treated. Thus, market forces could create a
situation where deadly, but less widespread, diseases are ignored.
In fact, past experience has proven that allowing greater and more
diverse access to breakthrough discoveries increases the likelihood of
novel and innovative extensions. The Molecular Biology Revolution,
which is responsible for enormous social, economic, and medical
advances is the product of innovations and discoveries made by
thousands of highly qualified and creative individual scientists who
developed new techniques and applications. Our world would be very
different today had this vital technology been restricted to just a few
investigators.
Fourth and finally, the American Society for Cell Biology supports
The Stem Cell Research Act of 2000 because it guarantees that this
important biomedical research will proceed with the highest possible
ethical standards, and with sensitivity to the public concern about the
origins of these cells. The National Biomedical Ethics Advisory
Commission held numerous hearings and debates on this issue and
solicited input from the public, scientific experts, and religious
leaders from different faiths. After careful deliberation, the National
Biomedical Ethics Advisory Commission concluded that it would be
ethically permissible to prepare stem cell lines from embryos that had
been obtained in the course of in vitro fertilization procedures, but
were deemed by the donors and the physician to be in excess of the
clinical need for the intended procedure: that is, those destined to be
ethically and legally discarded.
The National Biomedical Ethics Advisory Commission also recommended
specific regulatory and oversight procedures to ensure that the
creation and use of human stem cells would meet the highest ethical
standards. In fact, the draft guidelines developed by the NIH to govern
use of embryonic stem cells specify similar criteria to those suggested
by the National Biomedical Ethics Advisory Commission. Analogous
procedures are wisely specified in the Stem Cell Act of 2000. These
guidelines include complete separation between those who conduct
research and those who donate stem cells, thus preventing any potential
effort to develop embryos for the purpose of research. For federally
funded research, this bill would also prohibit the use of embryos that
are purchased or sold, and will continue to ensure that human embryos
are not created for research purposes.
It is important to keep in mind that banning federal funding for
human pluripotent stem cell research will not eliminate it. Such
research will proceed in private industry and in other countries. This
fact prompts serious concern that such work could be conducted in
secret, without benefit of ethical regulation or public debate. Thus,
prohibiting Federal funding will de facto prohibit public involvement
and input into the future of this important field. Permitting peer-
reviewed federal funds to be used for this research, combined with
public oversight, is our best assurance that research will be of the
highest quality and performed with proper ethical oversight and public
input. Federal funding of this research will require the scientific
community and the government to work together to establish an
appropriate set of rules for this research. These rules will ensure the
advancement of critical medical research and maintain respect for
public sensibilities.
There are also serious ethical implications to not proceeding.
Thus, while I am acutely and personally aware of the well meaning
ethical concerns that have been expressed about the sacrifice of
embryos to prepare stem cells, I am also cognizant that the embryos in
question will be legally and ethically destroyed in any case. We must
then ask: Is it ethical to literally throw away the opportunity to
allow all people to benefit from the demise of these embryos? How can
we justify not pursuing every reasonable means of finding cures for our
friends, our parents, and our children, who will suffer and die if we
do not find suitable therapies?
In thinking about the answers to these questions, I am reminded of
the many parallels between our debate today about the potential use of
human embryonic stem cells to treat disease and past debates about
organ transplants and the proper treatment of donor families. The
famous heart transplant surgeon Dr. Christian Barnard, stated it best
in response to questions he received about the ethics of heart
transplantation. He said: ``Would it not be immoral to bury a heart
when we have the ability to save a life?'' We submit that the answer is
the same for human embryonic stem cells. Ethics, scientific
opportunity, and medical need can surely be balanced.
In closing, we have before us an unprecedented scientific
opportunity to engage in a noble effort to develop new forms of
medicine. That opportunity offers hope to the many millions of our
citizens who rely on the shared stewardship of our scientists and our
political leaders to enable science's achievements to relieve all
people of the burden of serious disease.
Thank you for your courage in providing leadership on this most
important--indeed, life-or-death--issue, and for inviting my testimony
today.
Senator Specter. Thank you very much, Dr. Goldstein.
It is now 1 o'clock and we have a news conference scheduled
at 1:00 so that there may be broader coverage to what Mr.
Christopher Reeve and Ms. Jennifer Estess have to say to
America and the world. Your testimony has been eloquent, and I
do not think that any questions would lead to embellishing it.
I have never done this before, but I am going to invite
people--I understand this is being carried live on C-SPAN I--to
write to me and/or your own Senators as to whether you think
there ought to be Federal funding used for stem cell research.
You can write to me, Arlen Specter, United States Senate, in
Washington, D.C., and I will get it. Or if you choose to write
to your own Senator or Senators, send me a copy. I think it
would be interesting to see what the response is.
This is a very controversial issue and there are many
people who feel very strongly about the factors which have been
testified to here today. Like the issue of fetal tissue, it has
become embroiled in another debate. We have heard three
witnesses testify in opposition to your views, Mr. Reeve, Ms.
Estess, and Dr. Goldstein, in opposition to my views. I think
the balance of authority is that the embryos are discarded and
will not be used, as Dr. Goldstein puts it, an ethical question
in discarding them and in not using them.
But this matter is going to be on the Senate floor and
Senators pay attention to constituents. So, let us hear from
you because we will post the tally on the website.
Dr. Goldstein, do you want the last word?
Dr. Goldstein. I would, if that is OK with you, Senator.
Senator Specter. I am not guaranteeing you will have the
very last word, but go ahead.
Dr. Goldstein. I would not presume.
I just want us to pose the scenario that Senator Brownback
and others have suggested where we stop embryonic stem cell
work and work exclusively on adult stem cells for 5 years, say.
So, what happens if in 5 years we find out that adult stem
cells are not going to do the job? What do we tell Mr. Reeve
and Ms. Estess at that time? Sorry, we have to start over?
There may not be another opportunity. I cannot accept that
conclusion.
Senator Specter. Well, you raise a good point. You know
that stem cells from embryos will work, and that is what we
have to make happen.
Ladies and gentlemen, we thank you for coming today. It is
a very large group here in the hearing room. Would you keep
your seats please? Keep your seats until Mr. Reeve and Ms.
Estess have a chance to move out of the room. Then we will be
assembling in the outer corridor for the news conference.
Thank you all very much, Mr. Christopher Reeve, Ms.
Jennifer Estess, Dr. Lawrence Goldstein.
CONCLUSION OF HEARING
Thank you all very much for being here. That concludes our
hearing. The subcommittee will stand in recess subject to the
call of the Chair.
[Whereupon, at 1:04 p.m., Wednesday, April 26, the hearing
was concluded, and the subcommittee was recessed, to reconvene
subject to the call of the Chair.]
STEM CELL RESEARCH
----------
THURSDAY, SEPTEMBER 7, 2000
U.S. Senate,
Subcommittee on Labor, Health and Human
Services, and Education, and Related Agencies,
Committee on Appropriations,
Washington, DC.
The subcommittee met at 9:35 a.m. in room SD-124, Dirksen
Senate Office Building, Hon. Arlen Specter (chairman)
presiding.
Present: Senators Specter, Gorton, and Harkin.
Opening statement of Senator Arlen Specter
Senator Specter. Good morning, ladies and gentlemen. We
will be proceeding with this appropriations Subcommittee on
Labor, Health and Human Services and Education, and I am
delighted to see our witnesses arriving. Traffic in Washington
today is unusually bad, I am advised.
Do not pause in the audience, Dr. Fischbach and Dr.
Spiegel. Move right up into the chairs which were occupied
yesterday by executives from Firestone and Ford, where the
Appropriations Transportation Subcommittee, which I am a member
of, had a hearing, and I want you to know that our question for
you today will be much more difficult and tough than they were
for the officials from Ford and Firestone.
We have convened this session of the subcommittee to
further our inquiry into stem cells. This is the sixth hearing
which the subcommittee will have held on this very important
subject going back to December of 1998, within a few weeks
after the stem cell opportunities were disclosed. It was
apparent at that time that stem cells had enormous potential as
a veritable fountain of youth to replace cells in the human
body which were diseased, and to solve many serious problems,
many serious diseases.
With some of the results now showing enormous progress on
Parkinson's and progress on Alzheimer's and on spinal cord and
on a wide range of medical problems, this sub committee felt a
special responsibility to undertake this issue because it was
in an appropriations bill in the mid-nineties where there was a
prohibition against using Federal funds for stem cell research.
There has been an interpretation by the General Counsel for
the Department of Health and Human Services that Federal funds
may be made on stem cell research once stem cells have been
extracted from embryos, but Federal funds may not be used to
extract these stem cells from embryos, and that still leaves a
very wide area of restriction on Federal research.
Let me note the arrival of my distinguished colleague from
Iowa, Senator Harkin. It is nice to see you. We have an ongoing
partnership which has survived the political process, as he
says from time to time. When Democrats control the Senate he
chairs this subcommittee. When Republicans now control, I
chair, and we both learned a long time ago that if you want to
get something in Washington you have to be willing to cross
party lines.
We have maintained our approach on a nonpartisan,
bipartisan basis, and I think the results show, such as our
funding for the National Institutes of Health, where we have
increased the funding very, very materially.
The three times of stem cells which are possible for
extraction are embryonic stem cells, fetal tissue stem cells,
and adult stem cells. Without going into the details, it is
apparent scientifically, and we will hear from the experts on
this today, that adult stem cells do not have the potential
which is present with the embryonic stem cells.
The issue has been raised as to the propriety of using
embryos, and we have had in our hearings the leading opponents
from both the House and the Senate who have testified, and we
propose to present the full picture, and in addition to the
hearing today we are going to be having a hearing next week,
because we expect this issue to come to the Senate floor for a
vote.
The subcommittee report had included last year a change in
this prohibition on Federal funding for stem cell research, and
we removed it at full committee last year because it had the
potential for tying up our appropriations bill and our Majority
Leader, Senator Lott, agreed to bring the issue to the floor as
a freestanding bill, which now will be taken up sometime this
month, so we have had the full range of hearings.
In my opinion the issue on stem cells is very much like the
issue on fetal tissue, where there was a lot of controversy,
pro-choice versus pro-life, and then it was finally established
to the satisfaction of, I think, almost everyone that use of
fetal tissue would not encourage abortions, but it would only
be discarded fetal tissue.
The matter with stem cells I think is highly analogous.
These are embryos which are not going to be used. They have
been created for in vitro fertilization, but they are to be
discarded, so the issue boils down to whether they will be
discarded and have no use, or whether they can be used to save
lives, and I think that is a balance which comes out in favor
of savings lives.
But there is serious debate on this subject, and this
subcommittee will pursue it as far as we can to set the stage
at having a very meaningful floor debate, and I think the
Senate vote will be very, very important not only on this
subject but on the future of medical research as we have this
opportunity for a veritable fountain of youth.
Simultaneously with my red light going on--I have a little
trouble understanding, frankly, why there are lights on for the
chairman, but there were, so I will abide by them.
Now I would yield to my distinguished colleague, the
Ranking Member, Senator Harkin.
Opening Statement of Senator Tom Harkin
Senator Harkin. Thank you very much, Senator Specter. It is
good to be back with you here in this subcommittee and back in
the Senate after our brief break period. I want to thank you
for your determined and continued leadership on a broad variety
of health-related issues, especially, as you mentioned,
leadership on the continued funding for the National Institutes
of Health.
I am pleased and honored to be a partner with you in those
efforts. As Senator Specter said, we have a close bipartisan
relationship in regards to medical research. We both belief in
the strong potential that medical research holds for improving
the lives of our fellow Americans and again I want to thank
Senator Specter for his leadership on those issues, but more
specifically on this issue that confronts us today.
I am pleased that we have the opportunity to hear from our
panelists on the issue of the final NIH guidelines for the
conduct of embryonic stem cell research. These guidelines are a
critical step forward in the pursuit of medical breakthroughs,
and I believe they will help lead to new therapies and
treatments that could save or improve the lives of countless
Americans.
I look forward to discussing both the scientific and the
ethical impact of this research with our panelists today. Quite
frankly, we are in a war, a war against cancer, spinal cord
injury, and Parkinson's and diabetes and Alzheimer's and a host
of other diseases and disorders which rob so many people and
their loved ones of health.
These enemies are in many ways more fierce than those
encountered in any military operation in our Nation's history.
The death toll from just one of these, cancer, over the past 10
years exceeds the casualties for all of the military wars in
our history. In fact, there have been nearly five times as many
American casualties in the war against cancer in the past 10
years than all of the military wars fought since the American
Revolution.
In World War II we left no stone unturned to gain victory.
In the Persian Gulf War we developed the smart bombs that could
go down Main Street and take a left on Elm and go into the
window of Apartment Number 25. We developed these smart bombs.
Well, we should have the same attitude when it comes to our
wars against disease.
We should leave no stone unturned, and that is why the NIH
guidelines and the ability to move forward with this research
are so important. I believe we are at the crossroads of what
could be a smart bomb against many diseases, and the key to
victory, and we should pursue it.
Now, we will hear, I know, testimony as to whether we
should use embryonic stem cells or just use adult-derived stem
cells. Well, again my feeling on this is that all avenues
should be pursued. I have often said that basic research is
like having a number of doors that are closed. If you have 10
doors closed and you open one, chances are, your odds are, what
about 10 to 1 that you are going to find something, but you
open all 10 doors your odds are pretty good that you are going
to make the breakthrough, and so rather than just going down
one pathway I think we need to go down a number of different
pathways.
I believe the NIH guidelines are an important step forward
so that scientists have a procedural and ethical framework to
pursue this research in an ethical and sound manner.
Again, I just would close by saying that stem cell research
holds a lot of hope, a lot of potential for millions of people
around the world who are sick and in pain, and I think it is
wrong for us to prevent or delay our world-class scientists
from building on the progress that has been made. If we are
going to win this war, we have to pursue every path.
Thank you very much, Mr. Chairman.
Senator Specter. Thank you, Senator Harkin, and thank you
for your cooperation and your leadership on this subcommittee
on these very important issues.
STATEMENT OF GERALD D. FISCHBACH, M.D., DIRECTOR,
NATIONAL INSTITUTE OF NEUROLOGICAL
DISORDERS AND STROKE, NATIONAL INSTITUTES
OF HEALTH, DEPARTMENT OF HEALTH AND HUMAN
SERVICES
Senator Specter. The focus of today's hearing is to be on
the National Institutes of Health guidelines which were
published very recently, August 25, for research involving
human embryonic stem cells, and our first witness is the
distinguished Director of the National Institute of
Neurological Disorders and Stroke, a position which Dr. Gerald
Fischbach has held since 1998.
Prior to that time, he served as chairman of the
Neurobiology Department at the Harvard Medical School and the
Massachusetts General Hospital, past president of the Society
for Neuroscience, a member of the National Academy of Science,
a very, very distinguished research scientist, representative
of the caliber of the National Institutes of Health.
Dr. Fischbach, welcome. You can skip the traffic problems
and go right to the guidelines.
Dr. Fischbach. Thank you, Mr. Chairman and Senator Harkin.
I am pleased to be here today, and actually relieved to be here
on time, but pleased to be here to discuss the use the medical
promise of human pluripotent stem cells and the new NIH
guidelines released a couple of weeks ago, which are designed
to ensure that such cells are used in an ethical and legal
manner.
Stem cells indeed are revolutionary tools, perhaps the most
exciting development in biomedical science in my career, at
least dealing with the nervous system. They are exciting for
many reasons but they do, as very few other therapies do, offer
the possibility of reversing the course of disease, of actually
curing disease in addition to controlling symptoms.
This for the first time offers the possibility of reversing
disease processes and replacing damaged parts in the body, and
the promise is due to two properties of stem cells. One, they
can proliferate, and they can multiply and produce a large
supply of these tools and cells. Second, they can be, under
proper coaxing, forced to differentiate. That is, to develop
into mature cells that actually can function as missing parts
in various organs.
Now, there are many, many uses of stem cells. One of them
certainly will be in the area of novel tools for drug
discovery. There is some evidence that they will be the vehicle
that will enable breakthroughs in gene therapy, and they
certainly will teach us a great deal about the fundamental
biology of development and the pathogenesis of disease.
But perhaps the most striking aspect of step cell biology
which has attracted most attention is the ability to transplant
them into the body, and this is what has caused such great
excitement, and if I can for a moment speak about the nervous
system of the brain, with which I have been particularly
involved, this is a vital, vital property of these cells and
the nervous system.
With rare exception our nerve cells are nonrenewable
resources. When a nerve cell dies in our brains it is simply
not replaced, or not replaced easily. With rare exceptions
cells do not divide in the brain, so when they degenerate they
are lost. Therefore, the ability to replace cells is extremely
important and major advances have been made, as you said,
Senator Specter, in Parkinson's disease and a number of other
disorders.
In Parkinson's disease, we know exactly where the lesion
starts. We know the type of nerve cell that degenerates
initially, and it has been possible, using tissue, fetal
tissue, to replace these degenerating cells and to reverse the
symptoms of Parkinson's disease in animal models and in first
studies in human trials. This offers great promise, and
challenges us to do more research on stem cells and to replace
the fetal tissue implants with a much more controllable and
useful source.
But it does not stop there. Stem cells have been used
recently in treatment of sequellae of stroke, ALS, spinal cord
injury, tumors of the nervous system, and strangely enough,
because it is widely disseminated, even in multiple sclerosis.
Now, there is a hierarchy of stem cells. I think the
consensus of scientific opinion is that cells proliferate and
differentiate when isolated from the adult nervous system, but
there is a common view that cells proliferate more plentifully,
and they have a wider range of choices, if taken from the fetal
or embryonic tissue, so we would like to discuss that
hierarchy.
Now, much remains to be learned. Although I have emphasized
the promise, much research needs to be done about how we can
control the proliferation of these cells and how we can urge
them to differentiate in one half or another, and how we can
prevent overproduction of cells.
Now, we recognize that there are difficult ethical issues
involved in formulating policies in dealing with human
embryonic stem cells, which in my view are the most promising
at the present moment of all the stem cells isolated to date,
and therefore the NIH has formulated guidelines. They have
proceeded slowly and in public.
This committee has had several public hearings. The NIH
developed a working group from the Advisory Council to the
Director which included scientists, ethicists, patients,
patient advocates, lawyers, and laymen, and that resulted in
the guidelines which we are prepared to discuss, and I want to
thank you for the opportunity to present this statement, and I
would be prepared to answer questions that may arise.
Senator Specter. Thank you very much, Dr. Fischbach. Before
calling on Dr. Spiegel, I would like to yield to our
distinguished colleague, Senator Slade Gorton, for an opening
statement if he has one.
Senator Gorton. Please go ahead.
STATEMENT OF ALLEN M. SPIEGEL, M.D., DIRECTOR, NATIONAL
INSTITUTE OF DIABETES AND DIGESTIVE AND
KIDNEY DISEASES, NATIONAL INSTITUTES OF
HEALTH, DEPARTMENT OF HEALTH AND HUMAN
SERVICES
Senator Specter. All right, then. We turn to Dr. Allen M.
Spiegel, appointed Director of the National Institute of
Diabetes and Digestive Kidney Diseases last year, and prior to
that appointment Dr. Spiegel held significant positions in the
Institute, including Director of Intramural Research, and Chief
of Metabolic Disease, an M.D. from Harvard and a B.A. from
Columbia. Thank you for joining us, Dr. Spiegel, and we look
forward to your testimony.
Dr. Spiegel. Thank you very much, Mr. Chairman, and
Senators Harkin and Gorton. I appreciate the opportunity to
appear before you today with Dr. Fischbach on behalf of my
colleagues at the National Institutes of Health to discuss the
promise of research on human pluripotent stem cells, research
that will be funded under the recently released NIH guidelines.
The guidelines prescribe procedures to enhance both the
scientific and ethical oversight of this important area of
research. As described in our written statement, the guidelines
specify the documentation and assurances that must accompany
requests for NIH funding for research utilizing human
pluripotent stem cells. These include cells that were derived
in the private sector from human embryos that were created for
fertility treatment, were frozen, and are in excess of clinical
need. They also set out specific conditions for the use of
pluripotent stem cells derived from fetal tissue.
These guidelines will encourage openness, help make certain
that researchers can make use of these critical research tools,
and help assure public access to the practical medical benefits
of research using these tools.
You have heard from Dr. Fischbach of the potential
applications of research on human pluripotent stem cells to
developing treatments for neurological disorders. In fact, this
research holds great potential for leading to new cell
therapies for many other types of disease, such as those
involving the heart and the liver.
Perhaps one of the best examples of the promise of this
line of research is in the treatment of Type 1 diabetes, or
juvenile onset diabetes. This form of diabetes is characterized
by the inability to produce insulin, the hormone necessary for
sugar metabolism. Type 1 diabetes occurs when the body's immune
system attacks and destroys its own insulin-producing islet
cells in the pancreas. The standard treatment is to try to
control the sugar level with insulin injections. This is
exceedingly difficult even in adults, and much more difficult
in children with the disease. Transplantation of insulin-
producing islet cells, in theory, offers a much better approach
to controlling sugar levels.
In a recent published study, seven patients receiving islet
transplants became completely independent of the need for
insulin injections. NIH is currently funding a multi-center
trial of the protocol used in this study to determine if the
same high rate of success can be achieved in a larger number of
patients. A successful outcome to this trial, as exciting as it
would be for patients with Type 1 diabetes, and for their
families, will only serve to underscore the limitation in the
supply of islets available for transplantation compared with
the need.
While many approaches to address the islet supply problem,
including research on adult stem cells, should be and are being
vigorously pursued, human pluripotent stem cells offer the
greatest promise of providing a limitless source of islet cells
for transplantation in patients with Type 1 diabetes.
PREPARED STATEMENT
The release of the NIH guidelines now allows us to fund
this research that could lead to a cure for Type 1 diabetes and
for other serious diseases for which there is no satisfactory
current treatment.
Thank you, Mr. Chairman. Dr. Fischbach and I would be
pleased to respond to questions you and the other members may
have.
[The joint statement follows:]
Prepared Joint Statement of Gerald D. Fischbach and Allen M. Spiegel
Mr. Chairman, Senator Harkin, and Members of the Subcommittee, I am
Dr. Gerald Fischbach, Director of the National Institute of
Neurological Disorders and Stroke. I am accompanied by Dr. Allen
Spiegel, Director of the National Institute of Diabetes and Digestive
and Kidney Diseases. We are before you again to discuss one of the most
exciting areas of biomedical research: the enormous potential of human
pluripotent stem cells to treat and cure debilitating and deadly
diseases.
Only two years ago, researchers discovered and isolated these
primordial cells--whose existence in humans had been theorized but
never proven--the precursors of most of the other cells in the body.
Their unique properties of self-renewal and the ability to
differentiate into the full spectrum of other cell types make them
ideal candidates for repairing and replacing tissues and organs ravaged
by disease. New more effective treatments, and maybe even cures, might
be developed for juvenile-onset diabetes, Parkinson's Disease, spinal
cord injury, ALS or Lou Gehrig's disease, Alzheimer's Disease and many
other brain disorders. There is similar potential for the treatment of
cancer and heart disease. Virtually every realm of medicine and human
health might benefit from this innovation. Stem cell research could
alleviate a great deal of human suffering.
Chairman Specter, you and Senator Harkin, in particular, have
encouraged the National Institutes of Health (NIH) to invest our
resources in stem cell research in order to pursue its enormous
opportunities. Patients who are suffering from the most deadly and
disabling diseases have also asked that NIH fund this promising arena
of research. We believe that federal funding would encourage openness,
stimulate more discoveries and translate the promise of this research
into practical use more quickly, efficiently, and effectively--and with
procedural safeguards.
Recognizing that the ethical issues related to this research
required careful consideration, NIH and the Department of Health and
Human Services were committed to developing Guidelines to help ensure
that pluripotent stem cell research funded by NIH would be conducted in
a legal and ethical manner. In drafting the Guidelines, the NIH sought
the advice of scientists, patients and patient advocates, ethicists,
clinicians, lawyers, the National Bioethics Advisory Commission, and
members of Congress. Draft Guidelines were published in the Federal
Register last December. The NIH reviewed and considered all comments in
preparing the final Guidelines.
We are pleased to inform you that, on August 25, NIH published
final Guidelines for research using human pluripotent stem cells. NIH
is prepared to begin receiving applications immediately. As soon as the
oversight process is in place, NIH will be in a position to fund such
research. We expect broad interest from researchers seeking funding for
pluripotent stem cell research, and we are hoping to begin funding this
research as soon as possible. For procedural reasons, the soonest that
awards could likely be made is early next year.
WHAT ARE STEM CELLS?
Human pluripotent stem cells are a unique scientific and medical
resource. They can develop into most of the specialized cells and
tissues of the body, such as muscle cells, nerve cells, liver cells,
and blood cells, and they are self-renewing, making them readily
available for research, and potentially, for treatment purposes.
Scientists derived these unique cells from human embryos and from fetal
tissue.
WHY ARE HUMAN PLURIPOTENT STEM CELLS IMPORTANT?
There are three reasons why the isolation of human pluripotent stem
cells is so important to science and the future of public health.
First, pluripotent stem cells could help us to understand the
complex events that occur during human development.
Second, human pluripotent stem cell research could also
dramatically change the way we develop drugs and test them for safety
and efficacy. Rather than evaluating safety and efficacy of a candidate
drug in an animal model of a human disease, these drugs could be tested
against a human cell line that had been developed to mimic the disease
processes. This would not replace whole animal and human testing, but
it would streamline the road to discovery, and ensure that only the
safest drugs are tested in humans.
Third, and perhaps the most far-reaching potential application of
human pluripotent stem cells, the generation of cells and tissue could
be used for ``cell transplantation therapies.'' Such therapies are
aimed at diseases and disorders resulting from the destruction or
dysfunction of specific cells and tissue. Although donated organs and
tissues can sometimes be used to replace diseased or destroyed tissue,
the number of people suffering from such disorders far outstrips the
number of organs and tissues available for transplantation. Pluripotent
stem cells, stimulated to develop into specialized cells and tissue,
offer real hope for the possibility of a renewable source of
replacement cells and tissue to treat a myriad of diseases, conditions,
and disabilities for which replacement tissue is in short supply.
Examples of these include neurological disorders (including spinal cord
injuries and ALS), diabetes, burns, heart disease, and arthritis.
REQUIREMENTS OF THE GUIDELINES
The Guidelines prescribe procedures to ensure that NIH-funded
research in this important arena is conducted in an ethical and legal
manner. They specify the documentation and assurances that must
accompany requests for NIH funding for research utilizing human
pluripotent stem cells. These Guidelines will encourage openness, help
make certain that researchers can make use of these critical research
tools, and help assure public access to the practical medical benefits
of research using these cells. The Guidelines accomplish these goals in
the following ways.
First, the Guidelines help ensure that embryos will not be created
for the purpose of deriving human pluripotent stem cells to be used in
NIH-supported research. Investigators seeking NIH funds are required to
provide documentation that the human pluripotent stem cells were
derived from frozen embryos that were created for the purpose of
fertility treatment and that were in excess of clinical need. They
require a clear separation between the fertility treatment and the
decision to donate embryos for this research. In addition, the donation
of the human embryos must be made without any restriction regarding the
individual who may be the ultimate recipient of the cells for
transplantation. Similarly, researchers wishing to use human fetal
tissue to derive stem cells must demonstrate that they are in
compliance with all applicable laws and regulations. The Federal
statute applicable to NIH-funded fetal tissue transplantation research
also includes provisions creating a separation between the decision to
terminate a pregnancy and the decision to donate fetal tissue for
research.
Second, the Guidelines ensure that individual choosing to donate
embryos cannot receive any inducements, monetary or otherwise. The
Guidelines detail specific elements that must be included in the
informed consent to help ensure that potential donors receive
sufficient information to allow them to decide whether or not to donate
human embryos for this type of research. The Guidelines require review
and approval by an Institutional Review Board (IRB) to ensure that
consent was informed, voluntary, and meaningful.
Third, the Guidelines require accountability on the part of the
researcher. Detailed documentation must be submitted to NIH to
demonstrate compliance. For example, the grantee institution must sign
an assurance that the research to be conducted is in compliance with
the Guidelines, and that the institution will maintain documentation to
support the assurance. The researcher/grantee institution must submit a
sample informed consent document, with patient identifier information
removed, a description of the informed consent process, and
documentation of IRB review.
Fourth, the Guidelines specify types of research that the NIH will
not fund. For example, NIH will not fund any research that seeks to
derive pluripotent stem cells from human embryos, research utilizing
pluripotent stem cells that were derived from human embryos created for
research purposes, or any research that seeks to derive or utilize stem
cells from embryos that were created using somatic cell nuclear
transfer (cloning technology).
Fifth, the NIH has designed an oversight process that will provide
an extra level of protection, above and beyond standard peer review of
grant applications, to ensure that researchers have complied with the
Guidelines. A newly-created NIH working group called the Human
Pluripotent Stem Cell Review Group (HPSCRG) will review documentation
submitted by researchers demonstrating that they are in compliance with
the Guidelines. Members of the HPSCRG will subsequently make
recommendations to its parent committee, the Center for Scientific
Review Advisory Committee. NIH will not fund research or allow existing
funds to be used for research using human pluripotent stem cells
derived from human embryos or human fetal tissue until the required
compliance documentation receives HPSCRG review and approval of the NIH
Center for Scientific Review Advisory Committee. Continued compliance
with the Guidelines will be a term and condition of the NIH award.
HUMAN PLURIPOTENT STEM CELLS AND DIABETES RESEARCH
One of the best examples of the promise of this line of research is
in the treatment of Type 1 diabetes. Research on islet cell
transplantation and stem-cell biology offers compelling opportunities
for the development of new, innovative approaches for treating and
ultimately curing this disease.
Type 1 diabetes, often referred to as juvenile diabetes, is
characterized by the inability of the body to produce insulin, a
hormone necessary for glucose metabolism. This form of diabetes occurs
when the body's immune system attacks and destroys its own insulin-
producing islet cells in the pancreas. As a result of inadequate
insulin production, glucose does not enter cells as readily as when
insulin levels are normal. The standard treatment is to try to control
the glucose level with insulin injections.
Transplantation of insulin-producing islet cells is an alternative
approach to controlling glucose levels. In a recent study, seven
patients receiving islet transplants became completely independent of
the need for insulin injections. NIH is currently funding a multi-
center trial of the protocol used in this study to determine if the
same success can be achieved in a larger number of patients. A
successful outcome to this trial, as exciting as it would be for
patients with Type 1 diabetes, will only serve to underscore the
limitation in the supply of islets available for transplantation
compared to demand. While many approaches to address the islet supply
problem, including work on cell bioengineering and adult stem cells are
being vigorously pursued, human pluripotent stem cells offer the
greatest promise of providing a limitless source of islet cells for
treating and curing Type 1 diabetes.
HUMAN PLURIPOTENT STEM CELL RESEARCH AND THE NERVOUS SYSTEM
As significant as the promise of stem cells is for the treatment of
diabetes, the potential of stem cells for treating diseases of the
nervous system is equally impressive. The most obvious and exciting use
of stem cells in neurological disorders is to replace nerve cells lost
to disease or injury. Many diseases destroy particular types of nerve
cells, and mature nerve cells cannot produce new cells to replace those
that are lost. Animal experiments have demonstrated that the potential
exists for coaxing stem cells to specialize and replace the dopamine
cells that are lost in the brain through Parkinson's disease. A similar
approach might also apply to several other neurological disorders.
Human pluripotent stem cells, given appropriate control signals, might
specialize to replace the lost acetylcholine producing nerve cells in
Alzheimer's disease, to restore lost motor neurons in ALS, or to
produce inhibitory cells to help restrain electrical activity in
epilepsy.
Replacing lost nerve cells is only the beginning of the list of
possible therapeutic applications for stem cells. For some disorders,
such as multiple sclerosis, stem cells might replace supporting cells,
such as the glial cells, which provide the insulation necessary to
allow some nerves to conduct electrical impulses rapidly. In addition
to their potential in replacement therapy, stem cells can provided
nutritive factors that might prevent the loss of nerve cells in the
first place. Stem cell strategies might be useful for correcting
inherited defects. For example, in disorders that devastate children's
brains, we might rely on the ability of stem cells to migrate widely in
the brain and supply the vital missing enzyme that leads to early and
tragic death from Tay-Sach's disease. In addition, stem cells might
regenerate the many different kinds of complex brain tissue that are
damaged as a result of brain trauma or stroke. Transplanted stem cells
might also supply natural growth and survival chemicals to pave the way
for regeneration of remaining healthy neural tissue following spinal
cord injury. Recent findings suggest that stem cells might be harnessed
to seek out and destroy brain tumor cells that evade surgery or
radiotherapy. The list of possible applications of stem cells continues
to grow as we learn more about these cells.
CONCLUSION
Mr. Chairman, we appreciate the opportunity to discuss this
promising and extraordinary science and are pleased to respond to any
questions you may have.
Senator Specter. Thank you very much, Dr. Spiegel. As you
men have testified, I have made notes of the kinds of diseases
which could be cured by stem cells and I have noted the
following, and I would like to ask you if I am correct and if
there are others, and the follow-up question is going to be,
how many Americans, in ball park figures, are affected by these
diseases?
Parkinson's, amyotrophic lateral sclerosis, strokes, spinal
cord, tumor, multiple sclerosis, heart, liver, diabetes? Is
that the total? How about the impact on cancer, if any?
Dr. Fischbach. Cancer should be included in that list.
There is evidence for stem cells homing to tumors to deliver
toxins to tumor cells.
Senator Specter. Could you give a ball park figure as to
how many Americans suffer from these ailments?
Dr Spiegel. I will address that with an example. For liver
disease, we need to understand that liver transplantation is a
cure for liver failure caused by hepatitis viruses, by
autoimmune diseases, by many other conditions.
Thousands of Americans die every year awaiting liver
transplants. Therefore, an alternative to the insufficient
supply of cadaveric livers is vital, and thousands of American
lives could be saved if cell therapy for liver disease could be
developed as an alternative to liver transplantation.
Senator Specter. We may have a short answer. Senator Harkin
has anticipated this issue and has listed these categories, and
I would be interested in your response to put it into the
record beyond the hearsay document which Senator Harkin has
handed me: cardiovascular disease, number of persons affected,
58 million; autoimmune, 30 million; diabetes, 16 million;
osteoporosis, 10 million; cancer, 8.2 million; Alzheimer's, 4
million; Parkinson's, 1.5 million; severe burns, .3 million,
spinal cord injuries, .25 million; birth defects, 150,000 per
year, total of 28,400,000.
Does that sound about right to you, Dr. Fischbach?
Dr. Fischbach. Yes, it does, and if you are listing the
number of people affected by those disorders, that does sound
right, certainly.
Senator Specter. That is about half of America.
Senator Harkin. Well, heart disease gets about half the
people in America.
Senator Specter. Well, that is quite an impressive list.
Thank you, Senator Harkin.
With respect to the opportunities for adults themselves, I
know you have already testified that the adults themselves are
not as well-directed, and we are going to have another panel in
opposition to your views, but could you elaborate, Dr.
Fischbach, on why adult stem cells do not eliminate the need
for stem cells from embryos, as you earlier generalized?
Dr. Fischbach. That is actually the perfect way to state
it, and I think we can both respond to that. I believe that
stem cells have been discovered in adult tissues, actually
quite surprisingly in the brain, and that they do have the
capacity to proliferate, and to differentiate. Indeed, they may
even switch identities, and cells from other organs, from the
blood, have some promise of forming nerve cells in tissue
culture, but I think these are early reports and the conversion
to a neuronal phenotype is not complete, and the overwhelming
evidence is that they do not generate sufficient numbers of
nerve cells to be useful in the brain.
So I think the way you phrased it is exactly right. It
would not be responsible of us to use these cells in place of
embryonic stem cells, which at the present time most of the
advances I described are using embryonic stem cells, and that
is where the lead effort is right now, and the most promise.
Senator Specter. Well, could you specify which of these
enumerated diseases have promise with the embryonic stem cells
and which do not with the adult stem cells?
Dr. Fischbach. It is hard to say do not, because the
research is such that there can be, and we are hoping for
further breakthroughs in the use of stem cells from adult
tissues, but the embryonic stem cells have already been used in
the treatment of Parkinson's disease, stroke and some aspects
of spinal cord injury, and adult stem cells have not.
Senator Specter. My red light has just gone on. I will
yield to Senator Harkin.
Senator Harkin. Thank you, Mr. Chairman. Just a couple of
follow-ups. I expect many scientists have been anxious to get
started on this research, and waiting for these guidelines for
sometime now. Do you have any idea how many researchers have
already told NIH that they want to apply for funding?
Dr. Fischbach. I do not think we have those numbers in yet.
I have seen one report that one of the developers of embryonic
stem cells has had 150 requests the day after the stem cells
were published, but I do not have the numbers for NIH receipt.
Senator Harkin. A number of privately funded researchers
are already working on stem cells. What will the impact on the
field be once NIH starts funding this research, since it is
already being done in the private sector? Tell us what, once we
move ahead with these guidelines, assuming we do, what will be
the impact on the field?
Dr. Spiegel. I can comment in part relating to the previous
question. Our institute now funds several dozen investigators
who have expressed interest in working on these human
pluripotent stem cells, particularly with reference to the
pancreatic islets and the liver cells.
We support a very large portfolio of research currently on
adult stem cells. We are very vigorously supporting research in
that area. These same researchers, recognizing the incredible
potential of the human embryonic stem cells and pluripotent
stem cells from fetal tissue, are eager to be able to do this.
I think the impact will be enormous, because--although some
private sector investigators are working in that field--we need
to harness the entire brain power the NIH is capable of
mobilizing.
Dr. Fischbach. I think it will make the research more open,
more public, more published, and more diverse, and better.
Senator Harkin. And since there are guidelines now for the
private sector, I was told one day it was sort of like the Wild
West, there is no real guidelines, and while the guidelines
promulgated by NIH are not forced on the private sector, these
are not mandatory on private researchers, is it your
understanding that once NIH publishes guidelines like this it
sort of--because NIH is so big and the funding comes to NIH
that the private researchers then would follow and sort of come
under the umbrella of these guidelines? Would that be your
understanding?
Dr. Fischbach. Senator, that is one reason I am proud to be
at the NIH. I think the NIH sets the standard for the field and
for the world, and I think you behave differently at some risk,
and I think the guidelines will influence industry. Certainly
the guidelines specify we will fund research using embryonic
stem cells derived from human embryos only according to strict
regulations and rules, and that will have a big influence.
Senator Harkin. Again, for the lay person's mind like mine,
would you again, as much as you can in lay person's terms,
discuss the news that came out over the summer about the
finding that they could use adult bone marrow cells, stem
cells, that could differentiate into other cell lines, and once
that came out some people said to me, well then, you do not
need to use embryonic any more now we have this, and I think
there is some confusion out there, because I talked to a lot of
people and they said, well, you do not need the embryonic. We
now know that we can take bone marrow cells, adult stem cells,
and they can differentiate into, I think it was liver--
neurological cells.
So please, explain what this is all about.
Dr. Fischbach. Again, we can both take a shot at that.
My understanding of that paper is that it is an important
observation in that it does show that bone marrow, which we
have known for 40 or 50 years has diverse developmental
potential, and some of those cells--remember, bone cells are
turning over all the time in our body, unlike nerve cells.
So there are stem cells in bone marrow to replace the
marrow, especially during the war on radiation injury and
marrow transplants, and this report pointed out that when the
cells are treated in a certain way in tissue culture, that a
few of the cells would begin to express--in other words, begin
to manifest certain proteins that are characteristic of nerve
cells, and they look a little bit like nerve cells under the
microscope, but there is no demonstration yet that this will be
a realistic supply of cells yet, and there is no demonstration
that these cells will become the type of nerve cells that are
needed. It is like an existence proof. Something can happen,
but we do not know how far down the road they will go to be
useful.
So while it is an interesting first step, I cannot tell
whether it is 1 year, 5 years, 10 years, or a generation before
those cells will be truly clinically useful, so I think it
should not preclude--I do not agree with the statement that we
have shown this, therefore you do not need human embryonic stem
cells.
Dr. Spiegel. Since you asked in lay terms, let me try a
sports analogy. We are talking in terms of human pluripotent
stem cells capable of playing any position on a team, and
having a limitless supply, a bench strength, that has
incredible depth.
In terms of the adult stem cells, there is new evidence
that they may not have the high degree of versatility of human
pluripotent stem cells. If we use a baseball analogy, adult
stem cells may be like a utility infielder who can play a
couple of positions in the field, but it is not necessarily
good at all positions. Much more research needs to be done to
see whether adult stem cells can really play every position in
the sense of combating mutiple diseases. They do not have the
same bench strength.
In embryonic stem cells from mice, there are several cell
lines that are replicating, that is, dividing endlessly in many
labs and being used in limitless supply. I think that is a
critical difference.
When we think in terms of our goal, which is the treatment
of disease and alleviating suffering, we have to think in terms
of the range of disease possibility. The embryonic human
pluripotent stem cells can address every one of these diseases.
The adult stem cell research may, and we hope it will, be
successful in certain areas, but not necessarily every area.
Senator Specter. There is a vote in process right now on
the Senate floor, to be followed by another vote, and we are
going to take a very brief recess. We will return as promptly
as we can, and at that time we will turn to the next panel of
distinguished witnesses, Dr. Prentice and Dr. Roth, and we
would ask you, Dr. Fischbach and you, Dr. Spiegel, to stay, and
after we hear the testimony from those expert witnesses we may
want to have some interchange, and so we stand in recess for
just a few minutes.
The subcommittee will resume.
Although both Senator Harkin and I have expressed our views
on this subject by introducing legislation to remove the
prohibition so that there may be funding on stem cell research,
we have both had many contacts from constituents who have a
different point of view, and whatever our personal views may
be, the subcommittee considers it indispensable to hear all
sides, and earlier in our hearings we had heard the leading
opponents of stem cell research both in the Senate and in the
House as witnesses before this subcommittee, and now we turn to
two distinguished witnesses who are opposed to stem cell
research, Dr. David A. Prentice and Micheline M. Mathews-Roth.
Dr. Mathews-Roth, if you would step forward.
STATEMENT OF DAVID A. PRENTICE, M.D., Ph.D., PROFESSOR
OF LIFE SCIENCES, INDIANA STATE UNIVERSITY
Senator Specter. We will hear first from Dr. Prentice,
professor of life sciences, Indiana State University, and an
adjunct professor of medicaid and molecular genetics for
Indiana University School of Medicine. He also serves as
science fellow for United States Senator Sam Brownback, who had
testified earlier in opposition to stem cell research at one of
our prior hearings.
Dr. Prentice is a founding member of Do No Harm, the
Coalition of Americans for Research Ethics. He received his
bachelor of science in cellular biology and his Ph.D in
biochemistry from the University of Kansas, parenthetically,
Dr. Prentice, my home State. We welcome you here and look
forward to your testimony.
Dr. Prentice. Mr. Chairman, thanks for this opportunity to
appear before you today to talk about this extremely important
issue, stem cell research. Your colleague, Senator Brownback,
has stated previously his position that federally funded human
embryonic stem cell research is illegal, immoral, and
unnecessary, and others have discussed at length the arguments
related to illegal and immoral.
My point today would be to talk at some length about the
unnecessary aspect, whether we should actually be doing human
embryonic stem cell research, so I would like to spend some
time talking about that, because I feel at times the statements
that have been made are somewhat misleading.
Mr. Chairman, because embryonic stem cells are derived from
very early embryos in the continuum of human development, they
should have the ability to form virtually every tissue needed
in the body. It is for this reason that many are anxious to see
these cells used for possible clinical applications.
You are also well aware that production of human embryonic
stem cells does require the destruction of human embryos. There
are some scientific reasons why human embryonic stem cells may
not be as desirable as adult stem cells. One is simply the fact
that tissues generated by these cells will cause transplant
rejection, the same as any normal organ transplant will require
the use of toxic immunosuppressive drugs, possibly for the
lifetime of the patient.
Now, one possible solution might be to clone the patient,
destroy that embryo, essentially the patient's own twin to
divide the cells, but this complicates the debate even more
with an additional controversial technique.
Another possible problem may be control of differentiation
of embryonic stem cells to form the desired tissue, rather than
other tissues, or possibly even a tumor.
Senator Specter. What was it you just said, Dr. Prentice,
your last point?
Dr. Prentice. Another problem might be precise control of
the differentiation of the embryonic stem cell so that we get
the desired tissue.
Now, one other concern that has come up in terms of
embryonic stem cells, these have been termed pluripotent, able
to form most, not necessarily all stem cell types, but actually
published reports indicate that they are totipotent, able to
form all tissues of the developing human, or possibly even an
integral human embryo itself.
The publications regarding human or primate embryonic stem
cells and their derivation note that, unlike mass embryonic
stem cells, human and primate embryonic stem cells can form not
only the tissues that become part of the human body, but also
trophoblast tissue.
Now, you are probably well aware that it is the trophoblast
tissue that surrounds what become the embryonic stem cells that
allows the embryo to implant into the uterine wall and nurtures
early development. This is the tissue layer of the embryo that
is destroyed when the embryonic stem cells are derived.
Now, reformation of this tissue layer in cultures of
embryonic stem cells could actually lead to reformation of
complete human embryos in culture, able to survive if implanted
into a womb. This means actually the possible production and
destruction of thousands of new humans in culture created with
Federal funds, which would be a direct violation of Federal law
and NIH's own guidelines. Image of a virtual embryo body shop
springs to mind.
If we could turn to my main goal here, talking about the
ethical alternative of adult stem cells, there have been a
number of statements critical of this alternative and their
abilities, and I am somewhat mystified by these statements, Mr.
Chairman, because they seem to have been made without a
thorough reading of the scientific literature, especially
within the last 1 to 2 years.
For example, it has been stated that embryonic stem cells
will be in clinical use before adult stem cells, but to date
there have been no publications regarding the current clinical
use of human embryonic stem cells. The statement was made
earlier about clinical use for Parkinson's and stroke, but
those particular uses used transplants of fetal brain tissue,
not embryonic stem cells.
I have, and I will be submitting for the record, a number
of references regarding current clinical uses, including
cancer, lupus, various other immune diseases, corneal scarring.
Another criticism that has been leveled is that there are
only a few types, but more and more we are finding there are
many different types of adult stem cells, and these essentially
are pluripotent, just as has been claimed for the embryonic
stem cells.
In fact, in June of this year, a group in Sweden performed
an experiment with mice using adult neural stem cells, showing
these cells are pluripotent. They repeated an experiment done
in 1984 using mouse embryonic stem cells, an experiment, I
might add, which has not been done and ethically should not be
done with human embryonic stem cells.
The group stated that these studies suggest stem cells in
different adult tissues may be more similar than previously
thought, and perhaps in some cases have a developmental
repertoire close to embryonic stem cells.
I realize I am short on time, Senator, and I will be
introducing----
Senator Specter. Your red light is on, but you may proceed.
Dr. Prentice. Thank you, sir. If I could just hit a few of
the high points, as we say, others have said adult neural stem
cells can be stimulated to grow even while still within the
brain if given sufficient signal. One report notes adult neural
stem cells can be multiplied in culture and are, quote, similar
to human embryonic stem cells, end quote.
Other adult stem cells have been identified in muscle, and
just 2 days ago a report in the Journal of Cell Biology
announced purification of adult stem cells from the muscle of a
mouse model of Duchenne's muscular dystrophy. These cells were
introduced intravenously injected back into the dystrophic
mice, and resulted in muscle regeneration and partial
restoration of dystrophon expression in the mice.
Diabetes, one of the leading killers in the United States,
previous witnesses mentioned the use of adult stem cells from
cadavers, and this is a very promising technique, but obviously
the patient could use their own stem cells. This would be a
distinct advantage.
In March of this year, adult pancreatic stem cells from
mice were used to reverse diabetes in these animals. These
animals are a model of Type I, or juvenile diabetes. After
treatment the mice no longer needed insulin shots to survive.
Other criticisms are that the adult stem cells are hard to
access. They do not multiply well in culture. In August,
researchers showed that human adult bone marrow stem cells can
take up a new job description, if you will, and be changed into
neurons. The report notes that these grow readily in culture,
are readily accessible, and provide a renewable population.
In July, two groups, one in the United States and one in
the U.K., found human adult bone marrow stem cells could form
liver and they noted, we could avoid problems with current
liver transplants where the patient's body rejects the foreign
organ. Another one said, this would suggest maybe we do not
need any type of fetal stem cell at all, that our adult bodies
continue to have stem cells that can do this stuff, perhaps not
precise scientific language, Senator, but I think they make the
point that we do seem to have within our bodies these cells,
pluripotent adult stem cells, which can take care of organ and
tissue regeneration.
Some have urged research on both types of stem cell should
go forth, but the President's own National Bioethics Advisory
Commission has said that because human embryos deserve respect
as a developing form of human life, destroying them is, quote,
justifiable only if no less morally problematic alternatives
are available for advancing research, close quote.
Senators, the scientific literature overwhelmingly
demonstrates that adult stem cells are already fulfilling the
goals only hoped for with embryonic stem cells, making
destruction of human embryos completely unjustifiable.
Mr. Chairman, essentially, and we are really talking about
a human rights issue, it has been said that these human embryos
targeted for destruction to derive embryonic stem cells will
die anyway, so we should get some good out of them, possibly to
benefit those with life-threatening diseases.
PREPARED STATEMENT
If we follow this path of devaluing human life and
sacrificing one set of lives for the potential benefit of
others, how long might it be before those patients with life-
threatening diseases will hear the same phrase? They are going
to die anyway. Let us get some good out of them. Whom will we
choose to assign value, and who will dare to make those
choices? I implore you not to follow this path.
I thank you once again for this opportunity to discuss this
extraordinarily important topic with you, and I will be pleased
to respond to any questions you might have.
[The statement follows:]
Prepared Statement of David A. Prentice
Mr. Chairman and Members of the Subcommittee, I thank you for this
opportunity to appear before you today to talk about this extremely
important issue of stem cell research. Your colleague, Senator
Brownback, has stated previously his position that federally funded
human embryonic stem cell research is ``illegal, immoral, and
unnecessary''. Others have discussed at length the arguments related to
the ``illegal'' and ``immoral'' aspects, but not enough has been said
regarding the reasons that it is ``unnecessary'', and in fact it would
appear at times that some of the information which has been brought
forth is misleading. Therefore, I would like to spend some time with
you today discussing this aspect in terms of an ethical alternative to
embryonic stem cells, the so-called ``adult stem cells'', and the
scientific information which indicates that this alternative actually
does make destruction of human embryos completely unnecessary.
Mr. Chairman, while I am certain that you and the other Members of
this Subcommittee are by now well aware of the basic information
regarding stem cells, for the benefit of those in the audience who are
relatively new to this debate I would like to give just a brief
background. A stem cell is a cell that can proliferate with almost
unlimited potential, maintaining a pool of growing and dividing cells,
with the added ability that some of the daughter cells can
differentiate into specific cell types. Thus, a stem cell allows for
replenishment of itself while providing for specific functional tissues
needed by the body. Stem cells have been known for many years, such as
the blood stem cells in our bone marrow which continually replenish the
red and white cells and platelets in our bloodstream. Embryonic stem
cells (ES cells) have been isolated from mice for 20 years. In the fall
of 1998, there were two reports on isolation of human embryonic stem
cells; in Dr. John Gearhart's laboratory at Johns Hopkins, the workers
actually isolated embryonic germ cells from early human fetuses after
elective abortion, while Dr. James Thomson's laboratory in Wisconsin
isolated true embryonic stem cells from human embryos a few days old.
Because embryonic stem cells are derived from embryos quite early in
the continuum of human development, they should have the ability to
form virtually any tissue needed in the body. It is for this reason
that many are anxious to use these cells for possible clinical
applications in repair of damaged or diseased tissues. As you are also
well aware, part of the debate revolves around the requisite
destruction of the human embryos for derivation of these embryonic stem
cells.
There are, however, some scientific reasons why use of these human
embryonic stem cells is less desirable than use of adult stem cells.
One is simply the fact that tissues generated by these cells will face
transplant rejection, as would any normal organ transplant, and require
use of toxic immunosuppressive drugs, perhaps for the lifetime of the
patient. While one possible solution to this problem would be to clone
the patient and destroy that embryo, the patient's ``twin'', to derive
the cells, this complicates the debate even more with an additional
controversial technique. Another possible problem may be control of the
differentiation of these embryonic stem cells such that they form the
desired tissue, and not other tissues or even a tumor.
One concern and potential problem is that while these embryonic
stem cells have been termed ``pluripotent'', able to form most but not
all cell types, published reports indicate that they are actually
``totipotent'', able to form all tissues of the developing human, or
even the integral human embryo itself. The publications regarding human
or primate embryonic stem cell derivation note that, unlike mouse
embryonic stem cells, human and primate embryonic stem cells can form
not only tissues which become part of the human body, but also
trophoblast tissue. Trophoblast is the tissue layer of the early embryo
that allows it to implant into the uterine wall, forms part of the
placenta, and essentially nurtures early development. It is this layer
which is removed in the destruction of the early embryo. Re-formation
of this tissue layer in cultures of embryonic stem cells could lead to
re-formation of complete human embryos in culture, able to survive if
implanted into a womb. This means the possible production and
destruction of thousands of new human embryos in culture, created with
Federal funds, a direct violation of Federal law and NIH's own
guidelines. The image of a virtual ``embryo body shop'' springs to
mind. This concern certainly impacts the debate again in terms of the
legal and moral aspects.
But now turning to the main topic, the ethical alternative of adult
stem cells. Let me note for those unfamiliar with the terminology that
these cells reside not only in adults but in all of our bodies from
birth, one rich source being cord blood from the umbilical cords of
newborns. There have been a number of statements critical of this
alternative and the abilities of adult stem cells to fulfill the
promise of tissue repair. I am somewhat mystified by these statements
which malign the capabilities of adult stem cells, and can only assume
that the statements have been made without a thorough reading of the
scientific literature, especially within the last 1-2 years.
For example, it has been stated that embryonic stem cells will be
in clinical use before adult stem cells. To date, there have been no
publications regarding the current clinical use of human embryonic stem
cells. However, human adult stem cells have already been used
successfully for several clinical treatments. There is a wealth of
references on the use of stem cells as an adjunct in the treatment of
numerous types of cancer, including brain tumors, ovarian cancer,
various solid tumors, multiple myeloma, breast cancer, and non-
Hodgkin's lymphoma. The adult stem cells are purified from bone marrow
or circulating blood, preferably from the patient, and after treatment
to destroy the cancer cells the patient is given back their own
purified stem cells (an ``autologous'' transplant.)
Adult stem cells have also been used successfully to treat several
autoimmune diseases such as multiple sclerosis, systemic lupus,
juvenile rheumatoid arthritis, and rheumatoid arthritis, as well as
anemias. A report in August noted one of the first uses of an adult
neural stem cell line for treatment of stroke. This summer there were
two reports of the use of corneal stem cells to restore vision to
patients with corneal scarring in which normal corneal transplants
would not work. Again, in most of the cases, the patient's own adult
stem cells were used. Adult stem cells have been used to treat children
with a condition which leads to bone and cartilage deformities. And, in
April of this year, ``adult'' bone marrow stem cells were used in what
seems to be the first successful example of human gene therapy, in
which infants with a severe immunodeficiency seem to have been cured by
giving them their own stem cells with the defective gene replaced.
One of the common criticisms leveled against adult stem cells is
that there are only a few types, and they are not pluripotent, lacking
the range of ability to differentiate into all tissues which is claimed
for embryonic stem cells. In point of fact, human embryonic stem cells
also have not been shown at this time to be able to generate all
tissues in the body; nonetheless, it is presumed that they have
pluripotent potential.
For adult stem cells, however, in June of this year a group in
Sweden performed an experiment with mice using adult neural stem cells
which shows that these adult cells are pluripotent. The study
replicates an experiment done in 1984 using mouse embryonic stem cells,
an experiment which has not been done-and ethically should not be done-
with human embryonic stem cells. The Swedish group's results confirm
that adult neural stem cells are pluripotent-the cells were able to
participate in formation of heart, lung, intestine, liver, nervous
system, muscle, and other tissues. The authors state that ``--these
studies suggest that stem cells in different adult tissues may be more
similar than previously thought and perhaps in some cases have a
developmental repertoire close to that of ES cells.''
Other reports note that adult neural stem cells can be stimulated
to grow even while still within the brain if given sufficient signal.
Several sites with the human brain have been found to contain neural
stem cells, and that these cells can be used in animals to repair
spinal cord damage and other neural damage. One report notes that these
human adult neural stem cells can be multiplied in culture, established
as continuous cell lines, and are ``similar to human embryonic stem
cells.'' The authors of this study also note that ``The fact that this
revolutionary strategy uses autologous neuronal material means that it
has all of the advantages of biosafety, histocompatibility, and
neurophysiological efficiency. Furthermore, it does not raise the
ethical and moral questions associated with the use of embryonic or
heterologous material.''
Other adult stem cells identified include those in muscle, and
these cells also appear capable of reprogramming to form different cell
types including blood, bone, and cartilage. Just 2 days ago a report in
the Journal of Cell Biology announced purification of adult stem cells
from muscle of a mouse model of Duchenne's muscular dystrophy. The
cells were intravenously injected back into the dystrophic mice, and
resulted in muscle regeneration and partial restoration of dystrophin
expression in the mice. Transplantation of these cells engineered to
secrete a bone protein resulted in the cells changing from muscle to
bone cells, and accelerated healing of a skull defect in mice.
Diabetes is one of the leading killers in the United States. There
have recently been reports of successful treatment using adult
pancreatic stem cells from cadavers. Again, if a patient could use
their own stem cells, there would be a distinct advantage. In this
respect, in March of this year adult pancreatic stem cells from mice
were used to reverse diabetes in these animals, employing their own
stem cells. After treatment, the mice no longer needed insulin shots to
survive.
Other criticisms of adult stem cells are that they are hard to
access, and that they do not multiply well in culture. One of the most
versatile and accessible adult stem cell populations is in bone marrow.
In August researchers showed that these human adult bone marrow stem
cells can take up a ``new job description'' and be changed into
neurons. The authors noted that this source could provide ``a virtually
limitless supply'' of nerve cells. According to reports, the cells
``grow rapidly in culture'', ``are readily accessible'', and ``provide
a renewable population.'' They also add that ``Autologous
transplantation overcomes the ethical and immunological concerns
associated with the use of fetal tissue.''
In July, two groups, one in the U.S. and one in the U.K., found
that human adult bone marrow stem cells could form liver. In
considering the promising potential of these stem cells, one researcher
noted, ``We could avoid problems with current liver transplants where
the patient's body rejects the foreign organ.'' Another said ``This
would suggest that maybe you don't need any type of fetal stem cell at
all-that our adult bodies continue to have stem cells that can do this
stuff.''
Others have found that human adult bone marrow stem cells can be
reprogrammed to form bone, cartilage, muscle, and fat cells. You might
ask, ``Why would I want fat cells?'' Such tissues have significant
applications in reconstructive surgery. Numerous reports in the last
year demonstrate the significant proliferative capacity of adult stem
cells in culture when given the proper signals.
And one more note on the tremendous potential of adult stem cells
in terms of tissue engineering and reconstructive repair. There are now
numerous reports where adult stem cells have been seeded onto polymer
matrices. Testing these constructs in a sheep model system, autologous
adult heart cells have been used to form heart valves and aorta.
Some have urged that research on both adult and embryonic stem
cells should go forth. However, the President's own National Bioethics
Advisory Commission has said that because human embryos deserve respect
as a developing form of human life, destroying them ``is justifiable
only if no less morally problematic alternatives are available for
advancing research.'' Senators, the scientific literature
overwhelmingly demonstrates that adult stem cells are already
fulfilling the goals only hoped for with embryonic stem cells, making
destruction of human embryos completely unjustifiable.
Mr. Chairman, respected Members of this Subcommittee, it has been
said that these human embryos targeted for destruction to derive
embryonic stem cells will die anyway, and so we should get some good
out of them, possibly to benefit those with life-threatening diseases.
If we follow this path, devaluing human life and sacrificing one set of
lives for the potential benefit of others, how long might it be before
those patients with life-threatening diseases will hear the same
phrase-"they're going to die anyway, let's get some good out of them?''
To whom will we choose to assign value, and who will dare to make those
choices? I implore you not to follow this path.
Thank you once again for this opportunity to discuss this
extraordinarily important topic with you, and I would be pleased to
respond to any questions you might have.
STATEMENT OF MICHELINE M. MATHEWS-ROTH, M.D., ASSOCIATE
PROFESSOR OF MEDICINE, HARVARD MEDICAL
SCHOOL
Senator Specter. Thank you very much, Dr. Prentice. We will
come to questions as soon as we hear from Dr. Mathews-Roth, who
is an associate professor of medicine at Harvard Medical
School, and an associate physician at Brigham Women's Hospital.
She performs basic science and clinical research and
photobiology. Her clinical studies focus on developing
photoprotective treatments for photosensitivity diseases. Dr.
Mathews-Roth received her M.D. from New York University.
Thank you for joining us, Dr. Mathews-Roth, and we look
forward to your testimony.
Dr. Mathews-Roth. I just want to say I am not here as a
representative of either the Brigham or Harvard Medical School,
but as a physician who deals with patients with a genetic
disease that is called erythropoietic protoporphyria. We call
it EPP because nobody wants to say erythropoietic
protoporphyria, and I am also concerned about the ethics
involved in embryonic and fetal stem cell research.
I did leave a copy of my full testimony. I will try and
summarize it. I also would like to leave two sets of clippings
that describe some of the adult stem cell work as an appendix
to my testimony, if that is okay.
Senator Specter. Dr. Roth, your full statement will be made
a part of the record, as will the articles you referred to,
without objection.
Dr. Mathews-Roth. I think it is crucial for people and
legislators alike to realize the only way to obtain embryonic
stem cells is, as Dr. Prentice was saying, is to literally tear
apart growing human embryos, and I brought a picture. this is
the zona palusa, which sort of holds everybody together. This
is the trophoblast layer that Dr. Prentice was talking about.
These little guys here are the inner cell mass, or stem cells
that people are interested in getting, so literally you have to
break these guys apart to get it.
Now, what we all know is that the embryological fact is
that we do know when a new life begins, and that is at
fertilization, when egg and sperm join. Now, stem cells are not
embryos, but as Dr. Prentice was pointing out, the human ones
seem to be a little bit more totipotent than mouse embryo
cells, but remember, you have to break apart an embryo to get
the stem cells, and this is really the ethical objection, so I
think we all have to ask ourselves, do we really want to allow
the deliberate killing of even one of the youngest members of
our species to help sick members of our own species also?
It is a tough ethical question, but again, can we really do
this because we know ethically that a good end can never
justify using a bad means, or an evil means to attain it.
The fact that the embryos which would be used for stem cell
harvest are spares and would die or be destroyed anyway still
does not justify killing them for their stem cells. I think
there is a big difference between that and between people who
sign organ donation cards. They make the informed decision. I
have decided after I die a natural death, or am killed in an
accident, or whatever, then I will donate my organs.
There is a heck of a lot of difference between that and
deliberately killing a growing human to get organs, and again
also I am concerned with the problem that Dr. Prentice
mentioned of rejection, immunological rejections. What do we
suggest? Adult stem cells, really emphasizing research on adult
stem cells.
And also I would add, which I sort of forget to put in my
testimony, the use of embryonic tissues from spontaneous
abortions or miscarriages, I was talking to a physician, or a
Ph.D. who does this kind of work, and yes, if you establish a
network and let hospitals know that you are interested in doing
this work, and there is no ethical problem here, these babies
have died a natural death, their parents can say yeah, just
like to a child or somebody who is killed in an accident, you
may use the organs.
These are potential forms, or potential sources also of
adult, quote-unquote, if you want to call them that, stem
cells. Well, I guess the term is really fetal stem cells, so
no, we are not leaving patients in the lurch. We are saying,
look, there is so much promise for adult stem cells that really
the research should be emphasizing those.
There is mounting evidence of the versatility of these
adult stem cells that may make the use, as Dr. Prentice was
saying, of fetal cells unnecessary, and really, I agree with
this statement--well, actually it is the NBAC statement, if
there was another morally and scientifically acceptable
alternative, use it. Do not use the embryological stem cells.
I want to quote--I was going to, and I have in my testimony
several examples, as Dr. Prentice did, of the use of adult stem
cells. I just really want to quote one of those in a little bit
more detail. ``Adult bone marrow stem cells such as the marrow
stromal cells have proven to be extremely versatile, as
Woodberry, et al., in a paper describing the transformation of
marrow stromal cells into nerve cells''--and I believe Dr.
Fischbach referred to that, and so did Dr. Prentice.
He says the marrow cells are readily accessible, overcoming
the risk of obtaining neural stem cells from the brain, and
provide a renewable population. Autologous transplantation,
that is, using the patient's own cells, overcomes the ethical
and immunological concerns associated with the use of fetal
tissue.
Moreover, marrow stromal cells grow rapidly in culture,
precluding the need for immortalization, and immortalization, I
believe, adds a little foreign virus to the cells, and
differentiates into neurons exclusively with the use of a
simple protocol, and other scientists, Allison et al. reports
that human hematopoietic stem cells can differentiate into
liver cells in the tissues, in the livers of people receiving
bone marrow transplant, so if they can do it in people
receiving bone marrow transplants, they can also do it in
people who need help with their liver.
The exciting thing about these bone marrow stem cells is
that they are able to be transformed in all of the three
original embryonic layers, the ectoderm--and this is the
example for that, is the nervous tissue--mesoderm, muscle, fat,
and bone cells, and endoderm, example is the liver cells, and
it is just a matter of time that more things will be developed
about them.
In my testimony I was going through the four objections
that the NIH guidelines had suggested to liver cells, or to use
of adult cells, so I am sort of briefly going through that. One
of their objections is valid. They said that in diseases caused
by genetic defect, the defect would also be present in the
patient's stem cell.
However, this problem can be solved by ex vivo gene
therapy. That is gene therapy to cells that you take out of the
person's body, then you put them back in, adding the normal
gene to bone marrow stem cells which are then treated to get
them to develop into, say, whatever needed cells you need, like
liver cells or muscle, fat, or bone, or nervous tissue.
Successful gene therapy to bone marrow cells has also been
reported by French workers, who cured a severe combined
immunodeficiency disease in children with gene therapy to the
bone marrow, and I am happy to tell you my colleagues and I are
also studying gene therapy using bone marrow cells. We have
cured the mouse model of the genetic disease that I am
studying, erythropoietic protoporphyria with ex vivo gene
therapy.
Again, the advantage of that is that you--and obviously we
are hoping to eventually develop it for people. The advantage
of that is, if you take the person's own stem cells, treat
them, cure them with gene therapy, put them back into the
patient, they are not going to get rejection and it is going to
work.
So in summary, I just want to say the most ethical and
scientifically appropriate steps for the Government to take
concerning stem cell research are first to revoke the
guidelines and to continue the ban on research on embryonic
stem cells, and two, I would say also to impose a ban on
research on stem cells obtained from induced aborted fetuses,
because there is a danger of their potential of encouraging
abortion to obtain these cells, and we have heard stories about
doctors, well, sort of suggesting to patients, why don't you
wait a week or two, or something like that.
I think there are problems here. However, on the other
hand, approving research on fetal cells derived from
miscarriages, and I think it is somewhat of a myth to say that
this is not practical, having done this the Government must
strongly encourage and fund research in adult stem cells as
well as research on umbilical cord blood and placental stem
cells. Although these latter ones may cause immunological
problems they are probably still worth looking at.
So that the full potential of adult stem cells and possibly
these other forms, certainly the fetal stem cells from aborted,
from miscarried fetuses can be really looked at and explored to
find out just how versatile these cells really are, and this
way I think we are not deserting our patients, but we are doing
things that would be ethically acceptable to everyone.
PREPARED STATEMENT
I would hate to have to tell my patients that, boy, I have
to destroy a human life before I can treat you, and I do not
think we have to do that. I think work on adult stem cells will
avoid that awful problem.
Thank you, sir.
[The statement follows:]
Prepared Statement of Micheline M. Mathews-Roth
Dr. Mathews-Roth is an Associate Professor of Medicine at the
Harvard Medical School, and a Physician at the Brigham & Women's
Hospital, in Boston, Massachusetts. She performs clinical and basic
research on the genetic disease, erythropoietic protoporphyria.
[NOTE.--The views presented here are Dr. Mathews-Roth's--she is NOT
acting as a spokesperson for either the Harvard Medical School or the
Brigham & Women's Hospital.]
The stated purpose of the Guidelines for Research Using Human
Pluripotent Stem Cells issued by the National Institutes of Health is
to ``establish procedures to help ensure that NIH-funded research in
this area is conducted in an ethical and legal manner''. However these
guidelines, as presently written, are ethically very questionable.
Since the guidelines deal with the beginnings of individual human
lives, and thus an issue of great importance, American society cannot
tolerate ethical and scientific errors concerning this issue. It is
crucial for people to understand that the only way to obtain embryonic
stem cells is to literally tear apart growing human embryos. Although
the Guidelines state that ``NIH funds may not be used to derive human
pluropotent stem cells from human embryos'' (though funds can be used
to derive stem cells from fetal tissue), nevertheless, these guidelines
are allowing scientists to let the youngest members of our species,
even though their bodies consist of just a few cells, to grow for a few
days, and then deliberately kill them by breaking them apart to obtain
their stem cells. My understanding is that the bill, S. 2015 is similar
in intent to the Guidelines, but would remove the restriction about
deriving the stem cells from embryos. Good ethics hold that a good end
can never justify evil means to attain it. Certainly killing a growing
human (no matter what its age) is not an ethical means to obtain even a
most desirable end such as curing disease. The fact that the embryos
would die or be destroyed anyway does not justify the act of killing
those whom we all know are the youngest members of our species. The
Guidelines and S. 2015 condone the attitude of ``they are going to die
anyway, so why not use them?'': this attitude applies to the use of
stem cells derived from fetal tissue as well as from embryos. This
willingness to destroy the youngest of our species is a logical
continuation of the ``slippery slope'' this country got on when it
condoned killing of somewhat older members of our species by allowing
abortion on demand. It is doubtful that the ``safeguards'' proposed in
the Guidelines will totally prevent the abuses they are meant to
prevent. The more ethical thing to do is to prohibit the production of
embryonic or fetal stem cells.
In addition to the basic ethical problem with the Guidelines (and
with S. 2015), there are scientific problems with the use of both
embryonic and fetal pluripotent stem cells which may make their use
unnecessary, which the Guidelines do not sufficiently address: the
first is the use of adult stem cells instead of embryonic or fetal stem
cells, and the second is the problem of immunological rejection of
fetal/embryonic stem cell grafts by their recipients.
The Guidelines, in their responses to public comments that the ban
on the use of embryonic stem cells remain in force and emphasis be
placed on research of adult, placental and umbilical cord stem cells
instead, seem to ignore the mounting evidence in the current scientific
literature of the versatility of adult stem cells, which may make the
use of embryonic stem cells unnecessary, and avoid the ethical problems
of working with embryonic/fetal stem cells. Rather than simultaneously
pursuing all lines of research, the most ethical thing to do is to
thoroughly study adult/placental/umbilical cord stem cells to determine
their full potential, and continue the ban on the study of embryo/fetal
stem cells until this work is done.
The Guidelines state four reasons for not banning embryonic stem
cell research in favor of work on adult stem cells (``Scope of
Guidelines and General Issues'' section, Paragraph 4; ``Respondents . .
. adult tissues.'') These reasons are not totally accurate in view of
what is presently known about adult stem cells. Following are some
examples from the recent medical literature to illustrate this point.
(1) The Guidelines state that adult stem cells may have more
limited potential than embryonic stem cells, and that stem cells for
all cell and tissue types have not yet been found in the adult human.
On the contrary, after reviewing some other studies, Clarke et al.
state that ``. . . these studies suggest that stem cells in different
adult tissues may be more similar than previously thought and perhaps
in some cases have a developmental repertoire close to that of ES
[embryonic stem] cells'' (Science, vol. 288 page 1660, June 2, 2000).
It is important to remember that studies on adult stem cells have not
been going on for a particularly long time--much further progress can
be expected with future studies. The guidelines mentioned that cardiac
or pancreatic islet stem cells had not been found. It may not be
necessary to find specific organ stem cells--there is evidence that
bone marrow stronal cells or cells like them may be present in the
heart and could be converted into heart muscle-like cells (see page 233
of Kessler & Byrne, Annual Review of Physiology, vol. 61, page 219,
1999) and also that ductal structures of the adult pancreas contain
stem cells that differentiate into insulin-producing cells in an animal
model (Ramiya et al., Nature Medicine, vol. 6, page 278, 2000).
(2) The Guidelines claim that stem cells of adults are only present
in minute quantities, are difficult to purify and may decrease with
age. Such a generalization should not be made. Bone-marrow stem cells
such as marrow stromal cells have proved to be extremely versatile:
recent research has demonstrated their ability to develop into neurons
as well as other tissue cell types such as muscle, fat, bone, and even
liver cells: and marrow stromal cells are easily obtainable and can be
made to multiply in large numbers. As Woodbury et al., in a paper
describing the transformation of marrow stromal cells into nerve cells,
point out: ``The marrow cells are readily accessible, overcoming the
risks of obtaining neural stem cells from the brain, and provide a
renewable population. Autologous transplantation [i.e. using the
patient's own cells] overcomes the ethical and immunological concerns
associated with the use of fetal tissue. Moreover, marrow stromal cells
grow rapidly in culture, precluding the need for immortalization, and
differentiate into neurons exclusively with use of a simple protocol''
(Journal of Neuroscience Research, vol. 61, page 364, 2000). These
authors' statement seems to refute the Guidelines' expressed concerns
about ease of isolation and obtaining sufficient quantities of cells.
Additionally, Allison et al. report that human hematopoietic stem cells
can differentiate into hepatocytes (liver cells) in the livers of
people receiving bone marrow transplants (Nature vol. 406, page 6793,
2000). The exciting thing about these bone-marrow stem cells is that
they are able to be transformed into all of the three original
embryonic layers, ectoderm (represented by nervous tissue), mesoderom
(muscle, fat and bone cells) and endoderm, (liver cells).
(3) The Guidelines correctly state that in diseases caused by a
genetic defect, the defect would also be present in the patient's stem
cells. This is true, but this problem can be solved by ex vivo [outside
the body] gene therapy, adding the normal gene to bone marrow stem
cells, which are then treated to get them to develop into the needed
cells. Successful gene therapy to marrow cells has already been
reported by French workers who have cured severe combined
immunodeficiency disease wirth gene therapy to the bone marrow
(Cavazzana-Calvo et al., Science, vol. 288, page 669, 2000). My
colleagues and I are also studying gene therapy using bone marrow stem
cells for the treatment of a genetic disease: we have cured the mouse
model of a human disease called erythropoietic protoporphyria with
``ex-vivo'' gene therapy (Pawliuk et al., Nature Medicine, vol. 5, page
768, 1999).
(4) The Guidelines also claim that there is evidence that the stem
cells from adults may not have the same capacity to multiply as do
younger cells. This does not seem to be true, since at least in the
case of bone-marrow stem cells, adults can be successful bone marrow
donors (Anderlini et al., British Journal of Hematology, vol. 97,
p.485, 1997).
The Guidelines do not mention the main problem with the use of
embryonic and fetal stem cells, that is the rejection of the grafts by
the recipients. This serious immunological problem is the same as is
encountered when people receive lung, kidney, heart or liver
transplants from non-identical twin donors, necessitating the use of
immunosuppressive drugs for the rest of the recipient's life. This
immunological problem will not occur if stem cells from the person's
own body are used--an important value of adult stem cells, derived from
the recipient, and a strong reason in favor of exploring the full
potential of adult stem cells.
In summary, the most ethical and scientifically appropriate steps
for the Government to take concerning stem cell research are to: (1)
revoke the ``Guidelines'' and to continue the ban on research on
embryonic stem cells, and (2) impose a ban on research on stem cells
obtained from aborted fetuses (in the latter case because of the
potential of encouraging abortion to obtain these cells). Having done
this, the Government must strongly encourage and fund research on adult
stem cells, as well as research on umbilical cord blood and placental
stem cells (although these latter two groups may cause immunological
problems), so that the full potential of all of these other kinds of
stem cells for generating tissue cells of various kinds can be fully
detennined.
Senator Specter. Thank you very much, Dr. Mathews-Roth.
Dr. Prentice, Dr. Mathews-Roth, supported the use of stem
cells from fetal tissue except where there were induced aborted
fetuses. Am I citing your testimony correctly, Dr. Roth?
Dr. Mathews-Roth. Yes, and emphasize no induced abortions,
just spontaneous.
Senator Specter. I said that, you excluded that, but
otherwise fetal tissue. Dr. Roth has confirmed my statement
that it is appropriate to use those stem cells. Do you agree
with that, Dr. Prentice?
Dr. Mathews-Roth. Excuse me, could I say one thing? I think
that that research has been restricted to marrow stromal cells.
I would not be in favor of using the germ line cells from the
germ line fetal tissue.
Senator Specter. But there is fetal tissue you would
approve?
Dr. Mathews-Roth. I would say bone marrow stromal cells or
other organ cells from the spontaneously aborted fetuses.
Senator Specter. But there is a form of stem cell from
fetal tissue that you would approve of?
Dr. Mathews-Roth. Yes.
Senator Specter. Dr. Prentice, do you agree with that?
Dr. Prentice. As far as I understand it, Senator, that
would be an ethically as well as already legally acceptable
choice, to use these spontaneously aborted fetuses.
Senator Specter. Well, I raise the issue because for a long
time there had been a total objection to the use of fetal
tissue at all, and that was on the basis that once you open the
door, a slippery slope, you start to use fetal tissue even if
they are from circumstances where the abortion was not made, it
was going to happen anyway, so that at least so many people who
had objected to the use of fetal tissue for some time have
really reversed their position.
Senator Thurmond, interestingly, a very strong pro-life
Senator, a model of conservatism, was against the use of fetal
tissue, and then in his own family his daughter had juvenile
diabetes, and Senator Thurmond testified many years ago before
stem cells came up on the juvenile diabetes issue, and then he
has testified now in favor of stem cells, but on the use of
fetal tissue, when Senator Thurmond changed his vote it went
from about 40 Senators in favor of using fetal tissue to about
80, and I raise that issue because of the potential
similarities, where there had been an objection raised to fetal
tissue, which has abated at least in some circumstances, as Dr.
Roth would approve, and you concur, Dr. Prentice.
Dr. Prentice. Senator, if I might comment, that source,
even though it might be acceptable, might still face some of
the problems of the embryonic stem cell sources that we have
mentioned, because unless we are using cells from the patient
themselves, their own adult stem cells, we are still going to
run this risk of immunological rejection.
The other difference between those arguments is, if we are
using fetal tissue at that point, by current Federal
regulations the embryo is already dead, to derive embryonic
stem cells, we must actually wilfully destroy the embryo, so
there is a difference.
Senator Specter. Well, let us take that point up, because
that is really a core argument which has been made here, and
Dr. Roth is very emphatic about destroying a human life, but
where you have these embryos which have been created for in
vitro fertilization and they are to be discarded, and the
choice is just having them discarded, or using stem cells which
may be derived from them to save lives, what is the balance of
argument on destroying a human life when the issue arises as to
what is the quality of a human life in the embryo, if any.
I understand your point about aging people. Are you going
to kill them to help others? I do not think that follows, and
the testimony that Senator Brownback advanced about the analogy
to the Holocaust, aside from the issues of sensitivity, is just
not, at least in my judgment, very sound, but come to grips
with the destruction of a human life. When these embryos--how
do you categorize them as a human life, and how do you justify
letting them be destroyed collaterally by being discarded when
there could be a use made?
I notice you nodding, Dr. Prentice, and we will turn to you
first. Well, we can go to the ladies first. Dr. Roth, you go
first.
Dr. Mathews-Roth. Again, it was what I was trying to say
before, between the difference of organ donation and taking a
life of an embryo. I think there are some things that we just
ethically should not do, and I think that these extra embryos,
if you cannot find adoptive parents for them that are willing
to use them because they cannot have children--and there have
been some reports of women applying to IVF clinics and saying,
hey, have you got any spare embryos, if you can get permission
from the original parents I would like them because my husband
and I cannot do it, or whatever--I thing ethically the best
thing to do is let them die a natural death if you want to put
it that way.
In spite of the fact that a good end would occur, I still
think the means of destroying these embryos, of letting them
grow for a few days and ripping them apart to take these stem
cells, that is a bad means. I do not think ethically you can
justify it.
Senator Specter. Dr. Roth, you referred to consent from the
parents. Are you suggesting that if you had consent from the
parents it would be appropriate to use the embryos?
Dr. Mathews-Roth. I still would have a big problem with
that, because it is direct killing. There is a difference when
a child dies in an accident or of diabetes, or of overwhelming
infection that we cannot cure, and this child dies a natural
death, then I do not think there is a problem, if the organs
obviously are suitable, for a parent to say, sure, take my
child as an organ donor, or if there is a miscarriage, take my
child as an organ donor, but with these embryos you have got to
kill them, literally destroy them.
Senator Specter. Dr. Prentice, let us turn to you on the
issue of human life, quality of human life contrasted with
saving so many other lives.
Dr. Prentice. Actually, Senator, I am not sure that, from
my own perspective, I could justify even discarding those
embryos. I know that is a very--it is a sticky issue.
Senator Specter. You cannot justify what, sir?
Dr. Prentice. That I could justify discarding the embryos,
either, and I realize, so what do we do with all of those
embryos, and perhaps that is something that Congress itself
should be looking at in terms of the IVF industry.
Senator Specter. Well, what would the alternative be?
Dr. Prentice. I am not certain, sir, until we can get them
adopted, or what the answer might be.
Senator Specter. Well, they have to be carried to term. Are
you suggesting that the Government would compel someone, a
woman to carry them to term?
Dr. Prentice. I do not think we should be in the business
of compelling anyone to carry these embryos to term or destroy
them, either way.
Senator Specter. Well then, what would you do?
Dr. Prentice. I think it would have to be perhaps some sort
of an adoption program, but that is not really the focus of the
issue. The focus----
Senator Specter. I agree with you, but you brought it up,
so I wanted to explore it if you care to. What would you do
with them, if not discard them? If there were another
alternative to discarding them, I think you have a very valid
point.
This is a very sensitive and very difficult issue, when
human life begins, and I understand your sincerity in saying
that human life begins when you have a human embryo, and the
next step up is if you have human life, what is the quality of
life, but if you can structure having them carried to term so
that you have a baby after birth, then I think we would be in a
very different realm. I would have a very different view.
I would not in any way suggest sacrificing a child who has
been born for what benefit there may be to others from the
child's body, but where you have an embryo which is going to be
discarded, then it is a different matter. If you would care to
pursue an alternative to what you do as opposed to discarding,
I would be interested in hearing you.
Dr. Prentice. I have not really thought through the issue,
Senator, but as we mentioned, one possibility might be some
sort of adoption program.
Senator Specter. How would you structure an adoption
program?
Dr. Prentice. I am not really certain. I have not really
had time to think through the issue. It has really just come
up.
Senator Specter. Well, in order to get to adoption, you
have to have a child born.
Dr. Prentice. Well, no, sir.
Senator Specter. Excuse me, Dr. Roth.
Dr. Mathews-Roth. You have to find a mother to carry it.
Senator Specter. That is what I was saying. You and I agree
on this now, I want it noted.
Dr. Mathews-Roth. One way you could avoid--through the
adoption business is for IVF clinics to publicize the fact that
they have got extra embryos up, quotes, for adoption, and if
infertile couples, neither of whom are able to--the eggs of the
women, she does not have enough eggs, or the man has no sperm,
if they want to adopt one of these embryos fully made,
implanted into a uterus, then this is possible. This is a way
to go, and it is a matter of getting the news out there that
these are available, but I personally find no way of justifying
destroying an embryo even to help another person, I think
ethically.
Senator Specter. I am advised by staff there are 100,000
frozen embryos in excess of what people propose to use by
carrying to term.
Dr. Mathews-Roth. I have also heard from IVF people that
the tendency has been in the past, up until now, to make many
more than actually needed. They seem now to want not to do this
so much, so in the future there may not be that many extra
embryos. I do not know what the figure is of infertile people
who would like to adopt embryos.
Senator Specter. Dr. Roth, I wrote down one of your
statements, that if there is a moral or ethical alternative
then we should not use embryos. Do I quote you accurately?
Dr. Mathews-Roth. I guess that was my paraphrase of what
the NBAC was trying to say, that if there was a morally
acceptable alternative to using fetal embryo, or fetal stem
cells, then we should use it.
What we are both proposing, of course, is adult stem cells,
and I am going one step further and saying hemopoietic stem
cells, or possibly solid organ stem cells from spontaneously
miscarried fetuses.
Senator Specter. The contention has been raised about adult
stem cells being adequate to handle these issues, and if adult
stem cells are not adequate, would you then be willing to use
embryonic stem cells?
Dr. Mathews-Roth. I do not think so, not if it requires
killing. That is not good. I want to get away from killing
people, even little, eensy-weensy tiny people. That is one of
the reasons I do not like abortion, either. Abortion is direct
killing.
Senator Specter. I want to be sure we have an adequate
exploration of your views, and these green, yellow, and red
lights are only guideposts, and the red light for Dr. Prentice
was on, he noted, and I said go ahead, and yours was on a
while, and I have questioned you fairly extensively, far beyond
my customary time.
I would like now to have Dr. Fischbach and Dr. Spiegel come
back with you, and I would like to have a discussion among the
four of you scientists, and keep your seat, Dr. Prentice and
Dr. Roth. They will surround you.
Dr. Fischbach, you have heard the testimony of Dr.
Prentice, then Dr. Roth, about the potential applications for
adult stem cells, and Dr. Prentice has been considerate enough
to bring a chart along to illustrate his point. What is your
view of what Dr. Prentice has said about the use of adult stem
cells?
Dr. Fischbach. There were several things mentioned, and I
think Dr. Spiegel and I both might comment. I think the notion
that stem cells in bone marrow might be useful for replacing
nerve cells is a hope we all share, and I think it is in the
future.
The publication referred to demonstrated a very first step,
a small first step, and that for a few hours in tissue culture
cells from bone marrow appear to have some of the properties of
nerve cells. It will take years to translate that into a useful
therapy. I believe that studies of human, of embryonic stem
cells are much closer to that goal now, but I think both
efforts should be pursued vigorously.
Senator Specter. Are there things embryonic stem cells can
do that adult stem cells cannot do?
Dr. Fischbach. I do not know any case where an adult stem
cell derived from an adult animal has formed a nerve cell that
can make and release dopamine, and can reverse the behavioral
defect of Parkinson's disease. That may change with time, with
years, but I feel differently about patients dying with
devastating neurological disorders. I do not think we have that
time to wait.
Senator Specter. Dr. Prentice, would you care to comment on
Dr. Fischbach's statement?
Dr. Prentice. Mr. Chairman, I am not aware of currently a
dopaminergic adult neural stem line, but there was a report in
this month's Experimental Neurology where neurons were
generated from adult stem cells, neural stem cells which formed
functional glutamenergic and gatamenergic synapses in culture,
so there is evidence the adult stem cells can form functional
nerve connections and functional types of synapses in culture.
There have also been examples where in rat and mouse models
the adult stem cells have been transplanted back into the
animal and actually formed normal synapses in the animal.
Senator Specter. Dr. Spiegel, would you care to comment on
this issue, as to overall, but in part responding to what Dr.
Prentice has said?
Dr. Spiegel. I will not comment on the aspect of these
cells because I am not an expert in that area.
Senator Specter. Well, before you leave it, would you care
to comment on the issue of neuronal cells, Dr. Fischbach?
Dr. Fischbach. I think we shifted grounds in midstream from
bone marrow to neural stem cells. I think the adult neural stem
cells have promise, but I do not think they have yet reformed
function to the extent of correcting a neurological defect.
Senator Specter. Dr. Spiegel, and Dr. Mathews-Roth, if you
want to make a comment, go ahead.
Dr. Mathews-Roth. I just want to say I think the bone
marrow stromal cells are much more plastic than the bone marrow
hemopoietic regenerative stem cells.
Senator Specter. Dr. Fischbach, for the record, would you
respond or care to respond? I will study this later and try to
figure it out.
Dr. Fischbach. I think one way to approach this is, there
are many types of cells in bone marrow. Some cells are more
germinal in the sense that they can give rise to many other
types of cells. I agree that there are different types of cells
in the bone marrow, and some have potential as stem cells for
therapy.
Senator Specter. Do you think we should impose a
requirement on Senators before being permitted to vote on this
issue, that they know something about it, like these complex
matters you are advancing here today, and you do not have to
answer that, doctor. Dr. Spiegel, you were right in the middle
of a response when we had a couple of interruptions. You have
the floor.
Dr. Spiegel. Thank you, Mr. Chairman. I am not aware of a
single report in which either bone marrow stromal cells or
hematopoietic cells formed pancreatic islet cells. I am clearly
aware of a report, for example, in ``Nature Medicine'' of mouse
embryonic stem cells forming pancreatic beta cells and curing
diabetes.
There are also reports of a diabetes breakthrough using
adult stem cells from the pancreas, and this is what Dr.
Mathews-Roth was showing as a clip. This is work we vigorously
support, from the University of Florida, in which adult
pancreatic stem cells were transplanted into mice with
diabetes, with some positive effect.
There are also efforts to isolate human pancreatic ductile
cells at the Joslin Diabetes Clinic. However, the numbers
generated were clinically insufficient by two orders of
magnitude, that is, they were only one-hundredth of the amount
necessary for any kind of clinical utility. In the final
analysis, as Senator Harkin said, we really need to pursue each
of these avenues of embryonic and adult stem cell research to
have the greatest possibility of success.
Dr. Fischbach. I want to underline that, if I can, that
these involve quantitative statements. The question is not, can
this happen, but the real issue is, can this happen in ways
that we can control and make clinical use of, and I think that
sets embryonic stem cells apart at the current time.
Senator Specter. Dr. Mathews-Roth, would you like to make a
concluding statement?
Dr. Mathews-Roth. I would just say----
Senator Specter. I am going to give each of you an
opportunity to conclude here. Your turn comes first, Dr.
Mathews-Roth.
Dr. Mathews-Roth. I guess my concern is that there are some
things that ethically we should not do. I am not convinced that
it is going to take a terribly long time to get adult stem
cells up to speed, because they have not been worked on as long
as fetal stem cells, and I really think for the ethics
involved, for the idea of yeah, it is okay to kill these little
guys, even if they are very teeny, tiny humans, I think that is
wrong, and that is why I think that ban should continue.
Look at adult stem cells. Look at stem cells, organ stem
cells, maybe pancreatic ductile stem cells from spontaneously--
spontaneously aborted, that is the medical terminology for
miscarriages, miscarried babies may be a way to go, from
stillborn children may be a way to go, but do not kill to do
this.
Senator Specter. Dr. Spiegel, would you care to make a
concluding statement?
Dr. Spiegel. The issue of transplant rejection and immune
suppression has been raised, and this is a vigorous area of
research that we are pursuing. This research is essential
because of the many organ transplants we are already doing, and
I am happy to say that there is really significant progress and
potential success, which could address this issue.
As a physician, scientist, and public servant, I have tried
to illuminate the terms of the debate, and to offer an
objective view about the science. I hope I have contributed to
that.
Senator Specter. Thank you, Dr. Spiegel.
Dr. Prentice, would you care to make a concluding
statement?
Dr. Prentice. Thank you, Mr. Chairman. In the end, in a
real sense, the question we are grappling with here is not a
scientific question, whether adult or whether embryonic is the
correct way to go, the only way to go and so on. I think you
have heard evidence on both sides of the promise of both
particular types of stem cells, but in the end this is really a
moral and ethical question which you and the other Senators are
going to have to grapple with, and it is my opinion that it has
never been acceptable to sacrifice one set of human lives for
the benefit of others.
Thank you, sir.
Senator Specter. Thank you, Dr. Prentice. Dr. Fischbach,
would you like to sum up?
Dr. Fischbach. Senator, I think these are exciting times.
Everything we have talked about today has been published or
discussed for the first time in the last 2 years, most of it in
the last year, and we are faced with issues of extraordinary
promise, and revolutionary times.
I think these ethical issues are profound, and I think that
is the very purpose of the NIH guidelines is to allow work to
go forward under strict regulation and debate, and to allow it
to proceed.
I would say that every time society has closed down avenues
of investigation people have suffered, and I think the
guidelines try to address that issue.
Senator Specter. Thank you very much, Dr. Fischbach, Dr.
Mathews-Roth, Dr. Prentice, Dr. Spiegel. I believe this has
been a very productive hearing, unusually so. I think it is
always useful when we have people who are knowledgeable
advocate their positions, and especially when there is the kind
of friendly but adversarial opposition that we have had here
today.
I think it has been very constructive, and I know my
colleagues in the Senate are very much concerned about this,
and we will be studying the record so we can understand it,
because a good bit of what has been testified to here today
requires some definitions to work it through, but the exchanges
I think have been very helpful in that regard.
CONCLUSION OF HEARING
Thank you all very much for being here, that concludes our
hearing. The subcommittee will stand in recess subject to the
call of the Chair.
[Whereupon, at 11:20 a.m., Thursday, September 7, the
hearing was concluded, and the subcommittee was recessed, to
reconvene subject to the call of the Chair.]
STEM CELL RESEARCH
----------
THURSDAY, SEPTEMBER 14, 2000
U.S. Senate,
Subcommittee on Labor, Health and Human
Services, and Education, and Related Agencies,
Committee on Appropriations,
Washington, DC.
The subcommittee met at 9:33 a.m., in room SH-216, Hart
Senate Office Building, Hon. Arlen Specter (chairman)
presiding.
Present: Senators Specter, Harkin, and Reid.
Also present: Senator Wellstone.
Opening statement of Senator Arlen Specter
Senator Specter. Good morning, ladies and gentlemen. The
hour of 9:30 having arrived, the Subcommittee on Labor, Health
and Human Services, and Education for the Appropriations
Committee will now proceed with this hearing, which is the
seventh in a series of hearings on stem cells.
When the dramatic breakthrough occurred on stem cell
research in November of 1998, this subcommittee undertook these
hearings to focus on the prohibition which exists in the law
saying that the Federal Government, the National Institutes of
Health, may not engage in research to derive stem cells from
embryos.
The potential for stem cells is virtually limitless, so we
are told by the research community and by the people who are
studying this subject. The stem cells pose a veritable fountain
of youth, with already some tremendous inroads on Parkinson's
Disease, prospects on Alzheimer's and stroke, potential for
heart ailments and very vast potential in what we have found in
a series of hearings which we have already conducted.
There are those who object to stem cell research on
ethical, moral, perhaps religious grounds, with the contention
that the embryo is a living being. We have invited people on
both sides of the issue to testify so that we can have all of
the positions presented, and that the Senate can be in the best
place to make an informed judgment.
We have had leading opponents from the Senate and the House
of Representatives testify before this subcommittee, and today
we have a very distinguished panel of witnesses. Dr. Richard
Hynes, director of the Center for Cancer Research from
Massachusetts Institute of Technology, Dr. Darwin Prockop,
director of the Center for Gene Therapy from Tulane University;
in opposition we have Mr. Ron Heagy, president and founder of,
quote, Life is an Attitude Foundation, Mr. Ron Saltzman, pastor
of the Ruskin Heights Lutheran Church in Kansas City, Missouri,
and Dr. Anton-Lewis Usala, chairman and chief technical officer
for Encelle.
We have had assurances from the Majority Leader that the
legislation which Senator Harkin and I have introduced will be
called for a vote this month, so we are accelerating our
efforts to present all facets of the issue.
We also have for our third panel Mr. Michael J. Fox, Ms.
Mary Tyler Moore, Ms. Jennifer Estess, and Ms. Gina Gershon,
and as frequently is the case, we have called on people who are
well-known to the public, because when, candidly Michael J. Fox
and Mary Tyler Moore testify about their ailments and the
opportunity for stem cell research illustratively to cure
Michael J. Fox from Parkinson's, that is a point of really
great emphasis.
That is a brief summary of my formal opening statement
which will be put in the record without objection, and now I
yield to my distinguished colleague, Senator Harkin, Ranking
Democrat for the subcommittee.
Statement of Senator Tom Harkin
Senator Harkin. Thank you very much, Mr. Chairman, and my
thanks to our distinguished panelists who are here today. As
you said, Mr. Chairman, this is our seventh hearing on the
issue of stem cell research. I want to commend you, Mr.
Chairman, for the seriousness with which you take our
responsibility over this important controversial issue. These
hearings are part of a necessary dialogue on both the promise
and the ethical implications of stem cell research.
Two years ago, researchers discovered and isolated the
human pluripotent stem cells, the precursor of almost all of
the cells in the human body, and demonstrated the ability to
self-renew and differentiate into many different cell types.
As we have learned, they hold unprecedented potential for
the treatment and cure of the terrible diseases and conditions
that strike too many of our family members and friends, and so
we are on, I think, the cusp of some really exciting research
and discovery. No one knows what the potential for this is, but
almost all of the scientists I have spoken to indicate that
there could be tremendous, tremendous potential for the
interventions and cures of some of our most disabling diseases.
There have been, obviously, ethical and moral
considerations involving the use of these cells. For a couple
of years the NIH, along with the commission, the President's
Commission on Ethical Standards have been working to set up
guidelines.
On August 23, NIH issued the final guidelines, and I
believe these guidelines will permit the research to advance
and move ahead within, I believe, fairly stringent and
comprehensive ethical guidelines, and for the record I want to
once again say what those are, because I keep hearing about how
people are going to sell these cells, and they are going to
patent, they are going to make money, and all that kind of
stuff. Let me just read again what the guidelines are.
NIH will not fund the actual derivation of pluripotent stem
cells.
NIH funds can be used only if the stem cells were derived
from embryos in excess of need that were created as part of in
vitro fertilization procedures.
Providers of stem cells cannot make a profit from supplying
them to federally funded researchers.
And last, providers of stem cells must provide
documentation of informed consent by the donors.
Those are the ethical guidelines. NIH will begin accepting
grant applications for projects sometime early next year. Our
legislation is pending and, as the chairman said, we hope that
we can get it up before we leave here this year.
So that is just again the shortened part of my statement,
Mr. Chairman, and again I want to thank you for your tremendous
leadership on this very, very important issue.
Senator Specter. Senator Wellstone has joined us this
morning. We welcome him here and would invite him to make
whatever opening comments he makes here today.
Statement of Senator Paul D. Wellstone
Senator Wellstone. Mr. Chairman, I appreciate your
courtesy, and I know we need to get going with this hearing, a
very important hearing. Let me just say to both Senator Specter
and Senator Harkin that I come here to just show my strong
interest and support for your work. I have done a lot of work
with the Parkinson's community, but this legislation and your
effort extends way beyond just one community. It is terribly
important to many, many people, many, many families, many
Americans, and I am here just as an indication of the strong
support for your work.
I thank you.
Senator Specter. Senator Wellstone, at a previous hearing
we made a tabulation, and stem cell research could affect 125
million Americans, just about half of the people in this
country with a variety of ailments, which it may be able to
deal with.
STATEMENT OF RICHARD O. HYNES, Ph.D, DIRECTOR, CENTER
FOR CANCER RESEARCH, MASSACHUSETTS
INSTITUTE OF TECHNOLOGY
Senator Specter. We turn now to Dr. Richard Hynes, director
of the Center for Cancer Research at Massachusetts Institute of
Technology since 1991. He serves as president of the American
Society for Cell Biology, and is a member of the National
Academy of Sciences. Dr. Hynes received his M.A. in
biochemistry from Cambridge University in England, and his Ph.D
in biology from MIT. Welcome, Dr. Hynes, and in accordance with
our standard practice the green light starts when you do, and
it signifies 5 minutes, the yellow light gives you a minute,
and the red light says stop.
The floor is yours.
Dr. Hynes. Thank you, Senator. I appreciate greatly the
invitation to appear before you today, and I am here
representing the American Society of Cell Biologists, 10,000
biomedical researchers, and many of our other colleagues in the
biomedical research community, and I am here to explain to you
and to your colleagues why our organization feels strongly that
the full potential of human embryonic stem cell research must
be realized.
On behalf of the society I want first to extend my deepest
appreciation to you, Chairman Specter, to Ranking Member
Harkin, and to members of the subcommittee for your visionary
and courageous commitment to investment in biomedical research
at the NIH. This funding is vitally important to allow our
Nation's scientists and clinicians to exploit the tremendous
opportunities offered by the current revolution in biomedical
science in order to enhance public health.
I want to stress today that embryonic stem cell research is
among the most promising of these new opportunities. We
understand the ethical concerns that some have raised about
this research, but we respectfully submit that appropriately
regulated research on human embryonic stem cells can be
conducted while taking these concerns into account.
We believe that this research will undoubtedly lead to new
ways to treat disease and disability. Embryonic stem cells will
allow creation of new healthy tissue to replace damaged or dead
tissue. Examples include bone marrow for the treatment of
cancer and sickle cell anemia, pancreatic cells for the
treatment of diabetes, neuronal cells for the treatment of
Parkinson's, Alzheimer's, and various brain and spinal
disorders, and I could make a much longer list, but we do not
have the time.
We are not alone in our conviction that this invaluable
research must go forward. Attached to my testimony is a
statement signed by 17 organizations around the country,
including the American Medical Association, the Michael J. Fox
Foundation for Parkinson's Research, the Juvenile Diabetes
Foundation, the Christopher Reeve Paralysis Foundation, and the
American Association for Cancer Research, among many others.
I also want to reiterate the support of the American
Society for Cell Biology for the Stem cell Research Act of 2000
which you have introduced, that would allow not only the user
but also the derivation of embryonic stem cell lines.
Some have argued that human embryonic stem cell research is
illegal, unnecessary, and immoral. We respectfully disagree on
all counts. On the contrary, we believe that it would be
immoral not to pursue this great opportunity to improve the
quality of life.
The charge of illegality is unfounded. As you know, various
appropriations bills have restricted embryo research for
several years, but these bills are silent on the use of
embryonic stem cells. Such cells are not embryos, and they
cannot independently develop into embryos.
The NIH guidelines set specific criteria governing the
derivation of the stem cells, as Senator Harkin just pointed
out, requiring informed consent, precluding payment, and
prohibiting the creation of embryos for research purposes.
Cells used for research must be derived solely from embryos
generated for fertility treatments and in excess of clinical
need. Such embryos would otherwise be discarded.
Previously, the use of embryonic stem cells was restricted
to private and commercial entities which were not accountable
to the public. NIH funding of stem cell research will allow
Federal Government to exercise control over the standards for
the use of stem cells and will encourage public input.
Second, critics argue that embryonic stem cell research is
unnecessary because stem cells from adult tissues may be
equally effective. I regret that this claim is ill-informed and
misleading. Recent reports on adult stem cells are indeed
encouraging, but this line of research is in its very early
stages, and far from definitive at this point. We know little
about the availability of adult stem cells, their
differentiation, or their potential for prolonged maintenance
outside the body.
While we strongly support continued research on adult stem
cells, it is far too early to conclude that they will be as
effective in treating and preventing disease as embryonic stem
cells seem certain to be. If we hold up the progress on
embryonic stem cells we may be very much at a loss in a few
years time.
PREPARED STATEMENT
Finally, we believe it would be immoral not to pursue this
embryonic stem cell research within the appropriate regulatory
oversight, because it has such enormous potential to save human
lives and to mitigate human suffering. I submit that if the
issue is morality, using embryonic stem cells for life-saving
research is greatly preferable to discarding them. I, along
with many others, believe we should take advantage of the
enormous life-saving potential of the thousands of embryos
currently frozen and destined for destruction.
So I thank you for the opportunity to testify before you,
Mr. Chairman, and I would be pleased to answer any questions.
[The statement follows:]
Prepared Statement of Richard O. Hynes
Mr. Chairman and members of the Subcommittee: I am Richard Hynes,
Professor of Biology at the Massachusetts Institute of Technology,
where I am also an investigator of the Howard Hughes Medical Institute
and Director of the Center for Cancer Research. I am a member of the US
National Academy of Sciences and of the Institute of Medicine. I am
here today as President of the American Society for Cell Biology. The
Society represents 10,000 basic biomedical researchers, most of whom
work in our Nation's leading research universities and institutes. It
is my great pleasure to appear before you to explain why our
organization feels that it is so important that the full potential of
human embryonic stem cell research be realized.
On behalf of the American Society for Cell Biology, I wish first to
extend my deepest appreciation to you, Chairman Specter, to Ranking
Member Harkin and to the members of the Subcommittee for the visionary
and courageous commitment you have made through your investment in
biomedical research at the NIH. We are extremely grateful that you have
embraced the goal of doubling the NIH budget over five years and that
we are halfway towards reaching that goal. This funding is vitally
important to allow our nation's scientists and clinicians to exploit
the tremendous opportunities offered by the current revolution in
biomedical research to enhance the health of the American public.
We believe deeply that this investment in biomedical research will
be most effectively used if embryonic stem cell research is included
among the innovative methods used to develop treatments and preventions
for disease. We understand the ethical concerns that some have raised
about this research but we respectfully submit that appropriately
regulated research on human embryonic stem cells can be conducted while
taking into account those concerns. The Guidelines recently released by
the National Institutes of Health enable federally funded scientists to
conduct research using pluripotent human embryonic stem cell lines. We
believe that this research will undoubtedly lead to new ways to treat
disease and disability. Embryonic stem cells will allow the creation of
new, healthy tissue to replace damaged or dead tissue, such as bone
marrow for the treatment of cancers, sickle cell anemia and
thalassemis; pancreatic cells for the treatment of diabetes, and
neuronal cells for the treatment of Parkinson's disease, Alzheimer's
and various brain and spinal cord injuries and disorders. The prospects
offered by this research are analogous to, but will likely far surpass,
the benefits realized by organ transplantation over recent decades.
We do not stand alone in our determination that this invaluable
research must go forward. Attached to my testimony is a statement
signed by 70 American organizations, including the American Medical
Association, the Michael J. Fox Foundation for Parkinson's Research,
the Juvenile Diabetes Foundation International, the Christopher Reeve
Paralysis Foundation, the American Association for Cancer Research, and
the Federation of American Societies of Experimental Biology which I
respectfully request be submitted for the record.
I also want to reiterate the support of the American Society for
Cell Biology for S. 2015, ``The Stem Cell Research Act of 2000'' which
you have introduced, that would allow federally-funded scientists not
only to use, but also to derive embryonic stem cell lines for research
purposes.
Some have argued that human embryonic stem cell research is
``illegal, unnecessary and immoral.'' We respectfully disagree on all
counts. On the contrary, we believe that it would be immoral not to
pursue this great opportunity to improve the quality of human life.
First, the charge that the NIH has acted illegally is unfounded. As
you well know, the Labor, Health & Human Services and Education
Appropriations bills have restricted embryo research for the last
several years, but these bills are silent on the use of embryonic stem
cells. These cells are not embryos and they cannot independently
develop into embryos. The NIH Guidelines prohibit the use of NIH funds
to create embryos for experimental purposes and they set specific
criteria governing the sources from which embryonic stem cells can be
obtained. These guidelines require the informed consent of the donors,
preclude any possible direct benefit to such donors, and prohibit the
creation of embryos for research purposes. Cells used for research must
be derived solely from embryos generated for fertility treatments and
in excess of clinical need. Such embryos would otherwise be discarded.
A critical element of the NIH Guidelines is that the federal government
will oversee the use of embryonic stem cells. Heretofore, this valuable
resource was available exclusively to private and commercial entities,
which were not accountable to the public. By funding human embryonic
stem cell research, the federal government may exercise control over
standards for use of stem cells. This provision will facilitate open
debate and encourage public input into the appropriate uses of this
important scientific opportunity.
Second, critics argue that embryonic stem cell research is
unnecessary because stem cells derived from adult tissues may be used
with equal effectiveness. I regret that this claim is ill-informed and
misleading. Scientists are indeed guardedly encouraged by recent
reports of plasticity of some adult stem cells, but this line of
research is in its very early stages and far from definitive. We know
little about the availability of adult stem cells, their
differentiation, or their potential for prolonged maintenance outside
the body. While we strongly support continued research on adult stem
cells, it is far too early to conclude that they will be as effective
in treating and preventing disease as embryonic stem cells seem certain
to be. If embryonic stem cell research were to be halted based on that
hope, it is entirely possible that years would pass before scientists
determine whether or not adult stem cells are of equivalent value.
During those years embryonic stem cell research can an should be
pursued in parallel, to the great benefit of many of our fellow
citizens. This possibility was emphasized in a letter to Chairman
Specter in May from some of this Nation's leading researchers
investigating adult stem cells who stated: ``We are . . . dismayed that
our research . . . is being used as a justification to hinder or
prohibit research with embryonic stem cells. It is simply incorrect to
use the future promise of adult stem cell research as an argument that
embryonic stem cell research is not critical and essential.'' Again, I
respectfully request that you submit their letters for the record.
Finally, given these facts, we believe it would be immoral not to
pursue embryonic stem cell research, within the appropriate regulatory
oversight mandated by the NIH Guidelines, because this research has
enormous potential to save human lives and to mitigate human suffering.
The embryos in question would be obtained from in vitro fertilization
clinics only from those in excess of clinical need. I submit that, if
the issue is morality, using embryonic cells for potentially life-
saving research is greatly preferable to discarding them. Surely we
should take advantage of the enormous life-saving potential of the
thousands of embryos that are currently frozen and destined for
destruction?
Great Britain has recognized the value of stem cell research,
having strongly recommend the use of embryonic stem cells, and is now
considering enabling publicly funded researchers to establish new
embryonic stem cell lines. Other European countries are moving in the
same direction. I do not believe that the Europeans are less moral or
ethical than we. I also do not believe that they are less sensitive to
the sanctity of life. I do believe that they have acted appropriately
to enact by law the generation of new sources for stem cells in order
to save lives and reduce suffering of the living and I believe we
should do the same in this country.
In conclusion, The American Society for Cell Biology strongly
endorses the NIH Guidelines which will enable federally-funded
scientists to pursue embryonic stem cell research. We also endorse S.
2015, ``The Stem Cell Research Act of 2000''. We feel that, for the
sake of humanity, studies using all forms of stem cells--embryonic,
fetal and adult--should be pursued vigorously. We owe it to all those
who are suffering to explore all possible avenues that could lead to
the prevention of, and remedies for, disease.
I thank you for the opportunity to testify before you, Mr.
Chairman. I would be pleased to answer any questions.
Senator Specter. Well, thank you very much, Dr. Hynes. You
point out that there is no law prohibiting Federal funding on
the stem cells once they have been extracted from the embryos,
which I think is an important distinction to make clear, as you
have, but the existing prohibition is on the use of Federal
funding to take the embryos themselves and to extract the stem
cells.
Once they have been extracted by somebody else, then
Federal funding may be used, and I think the point you make on
the discarding of the embryos is very important. The embryos
were created for in vitro fertilization, and those which are
not used will be discarded.
When we dealt with the issue of fetal tissue many years
ago, objections were raised that fetal tissue would promote
abortions, and finally that argument was discarded, but I think
it is important to focus on those couple of additional factors
at this juncture.
STATEMENT OF DARWIN J. PROCKOP, M.D., Ph.D, DIRECTOR,
CENTER FOR GENE THERAPY, TULANE UNIVERSITY
MEDICAL CENTER
Senator Specter. Now we would like to turn to the testimony
of Dr. Darwin Prockop, who as of March of this year was
appointed director of the Center for Gene Therapy at Tulane
University Medical Center. Prior to that time, he was director
of gene therapy at Hanneman University School of Medicine.
Dr. Prockop discovered the gene defect that causes brittle
bone disease, and his most recent work involves successfully
converting stem cells taken from adult bone marrow into nerve
cells. He received his M.D. from the University of Pennsylvania
and his Ph.D in biochemistry from George Washington University,
so I guess we have to call you doctor, Dr. Prockop.
The floor is yours, sir.
Dr. Prockop. Thank you very much, Senator Specter. It is a
real privilege to appear before this subcommittee.
I think I have been asked to speak to you today because
Tulane Center for Gene Therapy is focusing on using adult stem
cells, and developing these cells as a way of treating a number
of diseases.
These adult stem cells were discovered over 20 years ago,
the ones we are dealing with, and have been shown to have a
number of remarkable qualities. One is that they can be easily
obtained from a patient, and that means that we can use these
cells from the same patient who is going to be treated without
the use of embryonic tissues or cells. Another feature is that
we can grow them very rapidly in the laboratory. The technical
consequence of that is, we do not need viruses to work with
these cells.
The third and most remarkable feature of these cells is
that they can become many of the hundreds of kinds of cells one
finds in the human body, and they seem to be part of us, as a
kind of repairing tissue, so for example, a few years ago we
discovered that if one takes these cells from an experimental
animal and puts them into a vein of the same animal, the cells
go to many, many tissues, and they seem to take part in the
repair of those tissues. They form the new cells of those
tissues.
So, for example, some of the cells go to bone and become
new bone cells. The implication of that observation for therapy
of bone disease is very broad. In fact, Drs. Edwin Horowitz and
Malcolm Brenner and others at St. Jude's Hospital, with whom we
are collaborating now, have recently reported some remarkable
results in children with very brittle bones, children whose
bones are so brittle they break a rib rolling over in bed.
Some of the cells, after infusion in animals, go to lungs,
and so the possibility is there of using these cells to repair
the lung in cystic fibrosis.
Some of the cells go to cartilage and joints. The
possibility is there of using these cells to treat the damaged
cartilage and joints that occurs as a result of arthritis.
Some of the cells go to muscle, particularly if the muscle
is damaged, so the possibility is there of using the cells to
treat muscular dystrophy.
In fact, at this point we are not quite sure what the whole
spectrum is of diseases that could be treated. We cannot rule
out the possibility of using these cells for diabetes, for
heart disease, and kidneys. We simply do not know.
But one of the most remarkable features of these cells is
that when you put them in the brain of an experimental animal,
they become new brain cells. The discovery was made in our
laboratory by Dr. Asum Azizi and Donald Phinney some 2, 3 years
ago. It is extended now by the work of Ira Black at Robert Wood
Johnson Medical School, and two other investigators at other
medical schools.
They will become, in fact, some new brain cells, not all
possible brain cells, and those observations have led us and
others now to pursue the possibility of using these cells to
treat Parkinsonism. In fact, we published a few months ago some
very promising results in a rat model for Parkinson's.
We are also thinking about using the cells to treat
Alzheimer's disease, and stroke, even multiple sclerosis and
spinal cord repair, so the prospects here are really very
exciting.
But I would like to close by making two points. Yes, all
these things have been documented, the things I have just said.
The possibility is there of using adult stem cells to treat
these diseases, and we do not need therefore to use fetal
tissues, and we do not need to put any foreign cells into a
patient, and also we probably do not need to use a virus.
But the second point I would like to stress is, we are not
there yet. We have a very long way to go. For example, we are
working very hard in using these cells, adult stem cells for
Parkinsonism, but we are at least 2 years, maybe 3 years away
from the first trial in patients.
PREPARED STATEMENT
We need a lot of further work, we need continuing support
from the National Institutes of Health, other sources such as
the Louisiana State Consortium for Gene Therapy. We have to
build up the data we need from animal experiments before we can
try that. I think it would be a grave mistake to say, because
of the work we have done and others have done with adult stem
cells, one should now stop the kind of work with fetal tissues
and cells as called for under the NIH guidelines.
Thank you very much.
[The statement follows:]
Prepared Statement of Darwin J. Prockop
Good Morning: My name is Darwin Prockop. I am currently the
Director of the Center for Gene Therapy of Tulane University Health
Sciences Center in New Orleans, Louisiana. I have an M.D. degree, a
Ph.D. degree and two honorary doctoral degrees. In addition, I am a
member of the National Academy of Sciences and the Institute of
Medicine. I have published over 400 scientific articles. My work has
been continuously supported by the National Institutes of Health. In
addition, the Tulane Center for Gene Therapy that I now head is
supported by funds from Tulane University, the Columbia Healthcare
Association and the State of Louisiana Consortium for Gene Therapy.
Our Tulane Center for Gene Therapy is carrying out research on a
special class of adult stem cells that we think can potentially be used
for the treatment of a number of devastating diseases. The adult stem
cells we are using were first discovered 20 years ago, and we now know
they have a number of truly remarkable features. One remarkable feature
is that they can easily be obtained from a small sample of bone marrow
from the same patient who is to be treated. Therefore if our therapies
work, we will not need fetal tissues nor will we need to introduce
foreign cells into a patient. Another important feature of the cells is
that we can grow them rapidly in the laboratory. A technical
consequence of this fact is that we can introduce new genes without the
use of a virus. But the cells are of special interest primarily because
they can change into a large number of the many different types of
cells found in the human body. For example, we discovered several years
ago that if the cells are isolated from the bone marrow of an animal,
and then injected back into the blood stream of the same experimental
animal from which the cells were obtained, they travel to a number of
different organs and tissues. Moreover, when they reach a given organ
or tissue they become new cells of the same kind that are normally
found in that organ or tissue. In effect, the cells can repair and
rejuvenate the organ or tissue.
These features of the cells raise some exciting possibilities for
using them in the therapy of diseases. For example, after infusion into
a vein, some of the cells go to bone and become new bone cells.
Therefore, the cells can potentially be used to treat diseases of bone
such as osteoporosis. In fact, Drs. Edwin Horowitz, Malcolm Brenner and
others at St. Jude's Children's Hospital In Memphis have recently
reported promising results with the cells in treating children with
severe brittle bone disease. Some of the cells go to lung and become
lung cells. Therefore, they can potentially be useful in treating
diseases of the lung such as cystic fibrosis. Some of the cells go to
joints and cartilage and become cells of joints and cartilage.
Therefore, they can potentially be used to treat diseases of the joint
such as arthritis. Some preliminary data suggest that in the future the
cells may also be useful in treating additional diseases such as
diabetes, heart disease and kidney disease. One of the most dramatic
observations about such cells is that they can become new cells of the
brain. The first observations here were made one and two years ago in
my laboratory primarily by Dr. Asum Azizi and Dr. Donald Phinney. In
the past several months these observations have been extended by Dr.
Ira Black and his associates at the Robert Wood Johnson Medical School
and by research groups at two other medical schools. Because the cells
can become some of the cells of the central nervous system, my
laboratory and others are currently trying to see if they can be used
to treat disease of the central nervous system such as Parkinsonism,
Alzheimer's disease, stroke, spinal cord injury and multiple sclerosis.
In fact, we have recently published some promising results using the
cells in a rat model of Parkinsonism.
In closing I would like to make two general statements. One is,
yes, it is true that if the work that my laboratory and many others are
now doing continues to be successful, it will open the possibility of
using stem cells from adults, or even from the patient himself or
herself, to treat a large number of terrible diseases. If successful,
the therapies will not use any fetal tissues and probably will not use
any viruses.
However, I would like to stress the second very important point: we
are not there yet. We have a long way to go. In my estimation, it will
be at least two years before the first adult stem cells can be tested
in patients with diseases such as Parkinsonism. It will probably be
much longer for testing the cells in patients with the other diseases I
have mentioned. We simply cannot be certain in advance which therapies
will work and which will not. We need several years of hard work and we
need continuing support from sources such as the National Institutes of
Health and other sources such as the Louisiana State Consortium for
Gene Therapy and the Columbia Healthcare Association that are currently
supporting the Tulane Center for Gene Therapy. In my opinion, it would
be a serious mistake to stop all research on human embryonic cells and
tissues because of the exciting discoveries my laboratory and others
have recently made about adult stem cells. We are simply not ready for
a moon shot-like strategy in which we place all our bets on adult stem
cells. I think it would be a mistake to tell the millions and millions
of people whose lives are being destroyed by these terrible diseases
that they to wait three, four, five years or even longer until we see
the results obtained with adult stem cells before we even begin doing
research on the other kinds of tissues and cells that may cure their
diseases. I myself would be extremely sorry to see this sub committee
or any other group decide that because of the work we and others have
done on marrow stem cells, the kinds of research called for in the new
NIH guidelines should be stopped.
Senator Specter. Thank you, Dr. Prockop.
Picking up on one of your last statements about the
prospects for having stem cells utilized in humans in
Parkinson's, would you elaborate first of all why--2 to 3 years
is pretty good. We press with some frequency the people who
appear here, especially those from NIH, where Senator Harkin
and I have led the way for very material increases. In the last
four appropriations periods we have increased the funding by
some $7 billion, so that the current projection for next year
will be in excess of $20 billion. What do you anticipate in 2
to 3 years with respect to stem cells and Parkinson's?
Dr. Prockop. Well, I can give you the best case scenario.
If our experiments now being conducted in animals come out
right in terms of efficacy, in toxicity, we think we may be
able to begin the very first clinical trials in patients with
Parkinsonism in 2 or 3 years. That is our best case scenario.
Senator Specter. Why not sooner, Dr. Prockop?
Dr. Prockop. Senator Specter, we have to be safe about
this. We have to be sure we are not going to do more damage
than we are going to help the patients.
Senator Specter. What sort of experimentation do you
anticipate between now and 2 to 3 years, when you are using the
stem cells on people who have Parkinson's?
Dr. Prockop. First we have to go to animal models of the
disease, and there are models in rats and monkeys that are
pretty close to what people suffer from in this disease, and we
have to do the tests there. It is not a test one does for a day
or a week. One has to do the treatment and then wait out 3
months, or 6 months, or 9 months to be certain there is not
some unfortunate consequence of what you are doing. It takes
that, and one does not go from small animals to the more
expensive tests in monkeys until one has those.
Senator Specter. All right. You move to 2 or 3 years. You
make the use of stem cells with people who have Parkinson's. Do
you have a projection as to what your expectation is once that
treatment starts, how long it would take from there, how long
from there to cure Parkinson's?
Dr. Prockop. Well, we have to go through the FDA
regulations on this, and it turns out that is a new chapter.
The stage of stem cells in people is a new chapter for the
kinds of regulations the Federal Drug Administration should set
up, and a beginning dialogue has already started between people
working with stem cells and the FDA.
What do you need in the way of safety and tests of efficacy
before it is appropriate to go to a patient? It would just be
terrible, and there are many examples in medicine, as you know,
where a drug that looks very good on preliminary tests has some
disastrous results in the first few patients.
Senator Specter. Well, you are going to have to qualify
with the Food and Drug Administration, understandably so,
important precautions, but my question goes to, do you have a
projection as to how long it will take to cure Parkinson's?
Dr. Prockop. Senator Specter, I am an optimist, all right.
One has to be an optimist to do research. I think it is
somewhere in the order of 4 or 5 years. I am saying it is 2 or
3 years for the first few patients, carefully controlled
studies and a very carefully controlled environment. I am
hoping it goes fast after that.
Senator Specter. I appreciate your optimism, and there are
many people hanging on, really hanging on your every word,
people who have Parkinson's. One of my constituents from
Pittsburgh has an hourglass. Whenever I see him he turns it
upside down to remind me that every hour is slipping away, and
that if something is not done soon it will be too late for him,
so these time parameters are closely watched.
Dr. Prockop. These, as you know, are very difficult
judgments, when you can make the jump from the laboratory to
the patient. It is not an easy decision, particularly when
introducing something so new as stem cells into people.
Senator Specter. Dr. Hynes, you had started to say that
there would be a longer list than the itemized diseases which
you thought could be dealt with by stem cells. Let me move to
the issue of cancer, which is your specialty. What is your
thinking about the potential for stem cells on cancer?
Dr. Hynes. Well, a third of the people in this room are
going to get cancer at some point in their lives, and so a
large proportion of the American public needs treatment for
this disease.
Senator Specter. How many of the people in the room did you
say would get cancer?
Dr. Hynes. 1 in 3. We are making some progress on that, but
we desperately need new approaches, and the application of stem
cells to variations on bone marrow stem cell transportation is
certainly one of the opportunities that I think is going to be
very valuable--the replenishment of tissues that are damaged
during therapy, the replacement of tissues that have to be
removed.
These stem cells are likely to be able to give rise to a
large proportion of the tissues of the body, as are adult stem
cells. I think that we need to work on both of them, and I
think the opportunities from both of them approaching cancer
are considerable.
Senator Specter. You said it would take a long time to list
all of the ailments which might be affected by stem cells. I
think it is worth the time. Tell us what you have in mind.
Dr. Hynes. Well, let me give you several more. For
instance, replenishment of heart muscle tissue. We know from
studies of embryonic stem cells in mice that one of the easy
cells to derive from those is functional beating heart muscle
cells, so that is a source of cells that could be used to
repair damaged heart tissue. That will take a while, obviously.
It is a complicated issue, but I think it is a definite
prospect.
Multiple sclerosis, the loss of the cells that ensheathe
neurons, glial cells. Glial cells can be derived from embryonic
stem cells, as can neurons, and so I already mentioned the
neuronal diseases that affect the neurons themselves, but the
accessory cells such as glia are also damaged in many diseases.
Juvenile diabetes, the ability to make islet cells that
make insulin, that one we are not so close to, I do not think,
because I do not think there is so much evidence yet as to how
to derive those cells from any source, but I think there is a
very good chance that that will come about, so that is three
more examples for you.
Senator Specter. OK. Thank you very much.
I yield now to my distinguished colleague, Senator Harkin.
Senator Harkin. Mr. Chairman, you have covered all the
points that I think need to be covered here. I just want to
thank you two witnesses for being here. I just want to make it
clear as to sort of the summation of what your testimony is,
and that first of all, am I right in assuming that you both
feel that the ethical guidelines that are published by NIH are
sound and will permit the Federal funding of stem cell research
within good, ethical and moral guidelines? Do you feel that
they are strong guidelines?
Dr. Hynes. I firmly believe that, that they separate the
decisions about the embryos themselves from the science that
goes forward from them, and I think that is a clear, clean
separation. I think the ethical concerns are important and have
been addressed by these guidelines.
Senator Harkin. Dr. Prockop.
Dr. Prockop. I think the guidelines are really a very good
plan for us to move ahead.
Senator Harkin. Second, it is both your positions that--
especially you, Dr. Prockop. You said that in my opinion it
would be a serious mistake to stop all research on human
embryonic cells and tissues because of the exciting discoveries
in my laboratory, in other words, recently made about adult
stem cells. We are simply not ready for a moon-shot-like
strategy in which we place all our bets on adult stem cells.
So your position is that the research ought to go forward
on all the fronts. Is that basically what you are saying?
Dr. Prockop. Absolutely. Absolutely, Senator Harkin. I
really think it would be very unwise to do anything else at
this time.
Senator Harkin. Is that your feeling, too, doctor?
Dr. Hynes. That is absolutely my opinion, too. Each of
these approaches is likely to produce useful insights. They
will not be identical, they will be complementary, and we
should proceed on all fronts, adult, fetal, and embryonic stem
cells.
Senator Harkin. Thank you both very much.
Senator Specter. Well, thank you very much, Dr. Hynes and
Dr. Prockop. We appreciate the work you are doing. It is avant
garde. This subcommittee and the full Congress is committed to
research help. We have put in this year's budget $2.7 billion
increase in NIH funding, which was on top of $2.3 billion last
year, which had been reduced to $2.2 billion on an across-the-
board cut. We put in $2 billion the year before. We put in
almost a billion the year before that, so the total is right at
$8 billion in increases because of our determination to see
this kind of phenomenal research advance, and the scientific
community has performed magnificently, and the Congress would
like to, if not perform magnificently, at least perform a
supporting role, so we thank you.
Dr. Hynes. Well, Senator, we thank you very much for your
support, and we will do our best to make good use of it.
Senator Specter. Thank you. I call our next panel, Dr.
Ronald Heagy, Mr. Russell Saltzman, Dr. Anton-Lewis Usala.
STATEMENT OF RON HEAGY, PRESIDENT AND FOUNDER OF THE
LIFE IS AN ATTITUDE FOUNDATION
Senator Specter. Our first witness, Dr. Ronald Heagy, is
president and founder of the Life is an Attitude Foundation. He
became a quadriplegic at the age of 17 when he was injured
while surfing. He is a motivational speaker and the author of
his autobiography, Life is an Attitude.
In 1995, he received the Disabled Oregonian of the Year
Award, and he received his master's degree in social work from
San Diego State University.
Mr. Heagy, we welcome you here. Let the record show that
Mr. Heagy appears in a wheelchair with a microphone close to
his lips and his hands.
Mr. Heagy. A chin-operated wheelchair.
Senator Specter. Thank you very much, and the floor is
yours.
Mr. Heagy. Thank you very much, and I appreciate this
opportunity as a citizen of America and a resident of Oregon. I
went through quite an adventure to get here, and the natives
from the West Coast bring greetings to you.
I have accomplished a number of things. My disability
occurred as a result of my choice. We know that choices and
consequences occur, and I ended up at 17 in a wheelchair,
paralyzed from the neck down, a quadriplegic on a ventilator
machine. Thank God I am no longer on a ventilator machine. I
had a choice to make, whether I go on in life or I give up, and
I chose to go on. I learned how to write with a pencil in my
mouth, a stick in my teeth.
I type, and I entered college. I was placed on welfare,
started out with no income, had no support in college. I worked
hard without the State support a lot of people assume that
folks like myself get. I am interested in seeing some of those
programs be developed, of course.
I accomplished a master's degree at Seneca State
University, and went out to try to get a job. I went through 20
different interviews, did not get a job, so I started my own
job, became a speaker. I spoke at my own high school, my first
speech, and I have spoken to 2 million high school students in
this Nation. I just finished speaking yesterday to 1,000 middle
school children, and my emphasis is on life, and here I think
we have an issue that focuses not on life.
Of course, I hear everyone make assumptions, and there are
opinions, and mine is an opinion, but we have no idea what this
can do, we have no idea the ramifications that this sends out
to our country.
Who determines the quality of life? My life did not begin
until I ended up in a wheelchair. Prior to this, I had a
scholarship to play at Oregon State, I was a proud and arrogant
person, lived for myself. I am now building camps across
America for kids with disabilities to go out and enjoy the
forest and the trees. I encourage them to be all that they can
be, and I have seen the effect that a disabled person can have
on others, including myself.
You look at my web site, go over on .com, read my
guestbook, I have thousands of entries from students, high
school students across the Nation who have been encouraged not
to commit suicide. I could read them here, but for the sake of
time--if someone chooses to live as a result of my disability,
I think it is encouraging to me to continue on.
Life happens. Sometimes life dumps on you. You have got a
couple of choices, get bitter or better, get negative or
positive, and there is more than just Parkinson's here, and I
know that we are all talking about some celebrity status, and I
do not hold a celebrity status, but I do hold an opinion that a
lot of my little disabled friends also hold.
I am opposed to this on moral grounds, No. 1. No. 2, it
forces financial support from taxpayers that may have
opposition to this, who they would have no choice but for their
dollars to go towards this.
No. 3, what happens if we do find a cure using this type of
procedure? Am I going to be forced to take the cure? If you say
to me, well, we are not going to support you through medicare
or medicaid any more because we have a cure for you, and I say,
I cannot take it because it came through this procedure that I
do not believe in, that is not just for me. That could be
millions of Americans. There are 61 million Americans with
disabilities that I am aware of, and it sounds like the numbers
have changed, and not every one of them are on Prozac.
There is life after disability, and I visited last week
here to speak before some of the Federal Government leaders
encouraging them. They asked me, they paid me to come speak to
encourage them. I visited the Holocaust--I think this is the
beginning of the end, and I am opposed to it.
The other day I had a young man tell me at a school that he
appreciated me talking about life, because there was so much
shooting in America. What am I going to tell the kids--I am
going to Denver, Colorado next week. What am I going to tell
them, that we are deciding to take life, any form of life for
the purpose of bettering somebody else's life?
Isn't that exactly what these students are doing? I am
trying to talk against that. If you get in my way, I am going
to take you out, because my life would be better if you were
not here. This is just a--it is appalling to myself, and I
would appreciate this country considering other means in order
for folks like myself to be bettered, and I believe that my
quality of life is determined by my attitude and not my
circumstance.
Thank you.
Senator Specter. Well, thank you very much, Mr. Heagy, for
your comments, your testimony, your approach to the subject. We
turn now to Pastor Russell Saltzman of the Ruskin Heights
Lutheran Church in Kansas City, Missouri. Pastor Saltzman has
served in other parishes in Omaha, Chicago, Charleston, South
Carolina. In 1995 he was diagnosed with diabetes, so has some
special insights. He received his master's in divinity from
Trinity Lutheran Seminary in Columbus, Ohio.
Welcome, Pastor Saltzman, and we look forward to your
testimony.
STATEMENT OF RUSSELL SALTZMAN, PASTOR, RUSKIN HEIGHTS
LUTHERAN CHURCH, KANSAS CITY, MO
Mr. Saltzman. Thank you, Mr. Chairman, Senator, for the
opportunity to appear before this subcommittee. I count it as a
privilege. I once worked for a Member of Congress and I know
the energy and the time you bring to this work, and how
difficult your decisions sometimes are, and you are to be
thanked for your efforts.
This is my second visit to the Capitol grounds since I left
Washington, and I left January 20, 1973, and that was the day
Richard Nixon was inaugurated for his second term. I like
pointing this out. I left Washington and he had to resign
later. My wife hates that joke. Evidently she is right.
Mr. Saltzman. I am here appearing as a person with diabetes
to testify against the use of human embryonic stem cell
research. First, though, I will reveal something of myself. I
am the adopted child of Harry and Lola Saltzman. My parents,
who live yet in the house my father built, and where I was
raised in Olathe, Kansas, and since I am an adopted child you
might guess accurately that the circumstances of my conception
were not ideal.
When I was conceived in the summer of 1946 I was an
unplanned and an unwanted pregnancy. My parents, birth parents,
were members of the same family. In fact, they were step-
siblings, and very possibly my conception was the result not
only of step-sibling incest but step-sibling rape.
There is no question in my mind, given the circumstances
today, that my birth mother would have been urged to accept
abortion, and very likely would have sought one as the means of
solving the dilemma I represented. I am unable to look at these
issues in any light except that of my origin, and when I say
that appearing here is a privilege, I hope I also convey my
sense of the miraculous, for had my conception occurred after
1972 I would not be here at all.
And suddenly it comes to mind that, having been aborted,
the fetal parts that were once me might have become research
material for somebody's investigation into the very disease I
have come here to discuss, so I say at the outset, this is a
terrible thing we undertake in these discussions, not only
because the matter touches me so personally, but also because I
know our common origin, our core humanity that links us one to
another, whatever our stage of development of maturity. We all
spring once from an act of union between egg and sperm. We all
once were human embryos. We all once were fetuses, quickening
in our mother's wombs. We are all each human life.
We may hope that all of us were conceived in love, but in
my case that matters to me not at all whether I was conceived
in love or in violence. What is important for me is the fact
that I am here in the first place, and my existence, by itself,
has some considerable consequence for other people, not least
for my seven children, two of whom are adopted.
I suffer from diabetes, and since my diagnosis I have
learned that the burden of chronic illness is a genuine burden,
and I have experienced the progression of this illness from a
time when simple diet alterations controlled it to the point
now where I require oral and insulin injections, and it is the
chronic part that constitutes the real burden, knowing I will
never be rid of it, and yet I cannot consent to stem cell
therapy, and what I find disturbing about this is that no
technique at present has produced a pancreatic cell.
It comes to a question, is the human embryo human life, or
is it a mere bit of research material, and if it is mere
research material, then why should any human life at any stage
of development, yours or mine, carry any special privilege?
But if the embryo is human life, then we should have in
place some restraint that cautions the strong against using the
weak for their own purposes.
PREPARED STATEMENT
If a cure for diabetes and a host of other ailments require
the production and destruction of human embryos, then I beg you
to consider the possibility that some diseases are better than
their cure.
Thank you.
[The statement follows:]
Prepared Statement of Russell E. Saltzman
Thank you, Mr. Chairman and Senators, for the opportunity to appear
before this subcommittee this morning. I count it as a privilege, I
once worked for a Member of Congress and I know the energy and the time
you bring to this work and how difficult your decisions sometimes are,
and you are to be thanked for your efforts. This is my first visit to
the Capitol grounds since I left Washington on January 20, 1973. That
was the day Richard Nixon was inaugurated for his second term. I don't
want to make too much of it, but I do enjoy pointing out that I left
Washington and the president had to resign.
I am here as a person with diabetes to testify against the use of
human embryonic stem cell research. But I shall first reveal something
of myself. I am the adopted child of Harry and Lola Saltzman, my
parents who live yet in the home where I was raised in Olathe, Kansas.
Since I am an adopted child, you might guess, accurately, that the
circumstances of my conception were not ideal. In the summer of 1946, I
was an unplanned, unwanted pregnancy. My birth parents were members of
the same family. In fact they were step-siblings. Very possibly my
conception was the result not only of step-sibling incest, but step-
sibling rape.
There is no question in my mind--given the circumstances current
these days--that my birth mother would have been urged to accept
abortion and very likely would have sought one as the means of solving
the dilemma I represented. I am unable to look at abortion in any light
except those of my origin. When I say that appearing here is a
privilege, I hope I also convey my sense of the miraculous, for had my
conception occurred after 1972, I would not be here at all.
And suddenly it comes to mind that--having been aborted--the fetal
parts that were once me might have become research material for
somebody's investigation into the very disease I have come here to
discuss.
So at the outset, I say it is a terrible thing we undertake in
these discussions, not only because the matter touches me so
personally, but also because I know our common origin, the base
humanity that links us one to another, whatever our stage of
development or maturity. We all once sprang from an act of union
between egg and sperm. We all once were human embryos. We all once were
fetuses quickening in our mothers' wombs. We are all, each, human life.
We may hope that all of us were conceived in love, but in my case that
matters not at all. Whether I was conceived in love or in violence,
what is important for me is the fact that I am here in the first place.
My existence by itself has some considerable consequence for other
people, not least for my seven children, two of whom are adopted.
I suffer from diabetes. Since my diagnosis in 1995, I have learned
that the burden of a chronic illness is a real burden. I have
experienced the progression of this illness from a time when simple
diet alterations controlled it, to the point now where I require both
oral medication and insulin injections. It is the chronic part that
constitutes the real burden, knowing I shall never be rid of it,
knowing my life will always be governed by diet and injection
schedules, and knowing, too, that my death probably will be the result
of some diabetic complication. When I say I wish I did not have it, I
am saying there is almost anything I would do to get rid of it.
Almost.
The prospect of stem cell therapy derived from human embryonic
research--involving the destruction of a human embryo--touches me in a
most profound way. I would never consent to any treatment for my
diabetes that directly or indirectly came about as the result of
destroying a human embryo. What I find disturbing about this incessant
rush to harvest stem cells from embryos is the fact that no researcher
to date has been able to develop a pancreatic cell from the techniques
presently used, this while there are several promising avenues of
research that do not involve destruction of a human embryo.
Most recently, I have learned about investigations by Canadian
researchers that employed pancreatic islet cells from cadavers. The
technique successfully eliminated insulin-dependence of several
diabetics who received the procedure. The procedure is subject to
further trials and it must be nuanced in application. But this holds
greater promise for a diabetic cure than anything else I have heard
about--and islet cell transplant is ethically neutral. It has no moral
implications associated with it. Yet, we here in the United States seem
in a rush to use what is arguably the most ethically objectionable
method available, while other morally neutral medical technologies
virtually are at hand. The President's own National Bioethics Advisory
Commission has said that because human embryos deserve respect as a
developing form of human life, destroying them ``is justifiable only if
no less morally problematic alternatives are available for advancing
research.'' The fact is, those alternatives exist.
It comes to a question. Is the human embryo human life, or is it a
mere bit of research material? If it is mere research material, then
why should any human life at any stage of development--yours or mine--
carry any special privilege? But if the embryo is human life, then we
should have in place some restraint that cautions the strong against
using the weak for their own purposes.
I would commend to your reading Aldous Huxley's Brave New World.
Writing in 1933 Huxley, with astonishing prophetic foresight, created a
world of genetic clones and what he called ``decanted babies.'' All
this arose because in the world of his novel, the human embryo was
merely research material. He worried that science was being twisted all
around. Where once, as with the sabbath, science was made for Man, he
foresaw a time when Man would be made for science. In Huxley's
fictionalized world the process that turned science around was
methodical and deliberate, and without moral regard. In our own world,
the process going on is less tidy but no less deliberate, and, I fear,
with equally little moral regard.
If a cure for diabetes and a host of other ailments require the
production and destruction of human embryos, then I beg you to consider
the possibility that some diseases are better than their cure.
Senator Specter. Thank you very much, Pastor Saltzman.
We will have questions when we conclude the full panel, and
we now turn to the third and final member of the panel, Dr.
Anton-Lewis Usala, founder, chairman, and chief scientific
officer for Encelle, a biomedical device transplantation
company located in Greenville, North Carolina. He received his
medical degree from Jefferson Medical College in Philadelphia.
Welcome, Dr. Usala. The floor is yours.
STATEMENT OF ANTON-LEWIS USALA, M.D., CHAIRMAN/CHIEF
TECHNICAL OFFICER, ENCELLE, INC.,
GREENVILLE, NC
Dr. Usala. Senators, I come as a medical scientist, a
person who has had diabetes 41 of my 42 years, and a concerned
citizen of the United States. I was asked to present the
approach my company has developed for the treatment of type I
diabetes and other diseases states that require replacement of
nonfunctioning tissues.
The premise of our approach is to replace as
physiologically as possible the insulin-producing cells that
have been destroyed in children with diabetes. This effort
requires large numbers of replacement cells, as there are
approximately 1 million patients with type I diabetes in the
United States, and since only several thousand human pancreases
are available to provide a resource for these cells, developing
a method for preventing rejection of animal-derived cells has
become the focus of our effort.
Our most promising approach to solving the problem of
cross-species transplantation has involved culturing tissue
from pig pancreases and proprietary hydrogel material derived
from pig skin. The cells in this material are then injected in
a diabetic animal without using any immunosuppression. The
material is a copolymer designed to simulate the structure of
connective tissue present during the first few months of fetal
development.
Connective tissue is the scaffolding the cells attach to,
and during phytogenesis the connective tissue is quite
different in appearance than later in life.
Stem cells are attractive as a therapy because they are
responsive to embryonic signals and are thereby differentiable.
That is, they can differentiate into many different kinds of
tissue. Our biopolymer material has been used for its ability
to directly simulate host tissue differentiation, regeneration
and repair without the need for stem cell transplantation.
Last Thursday, the Food and Drug Administration authorized
the commencement of human clinical trials utilizing our
material in the treatment of diabetic foot ulcers. We have
demonstrated that a single injection of the material induces
complete reconstruction of chronic and acute skin wounds in
rabbits, dogs, and pigs. This includes reconstruction of skin
architecture that is normally only made during fetal
development. Blood vessels, hair follicles, and dermal
structures are integrated in a scarless healing after a single
injection of the material.
Replication of specific tissue requires cells to receive an
enormous number of specific signals. What defines a human life
is cellular mass able to produce and integrate this enormous
number of sequences, and this occurs shortly after
fertilization. The cascade of specific cellular differentiation
begins and continues throughout the adult life of the person.
It can be argued when the reasoning of the fetal organism
begins. It cannot be argued when it is human.
Cells from a human embryonic donor can be induced to this
degree. Severed from the intricate and specific environment in
which they are made, totipotent or pluripotent cells require
the signals from thousands of sources. While knowledge would
undoubtedly be gained from this research, this is different
from development of a treatment, and there are different
approaches that do not require using human embryonic cells.
Whether or not our approach will induce recreation of
complex tissue structures in human patients will be known very
shortly. I hope what we have observed in preclinical studies
will also be the case in patients with diabetic foot ulcers. If
so, variations on this approach will be researched by others
for the ability to induce other types of tissue repair such as
nerves, utilizing the patient's own regenerative capabilities.
I am 42 years old, and have had diabetes 41 years. I have
seen the therapy for diabetes dramatically change over this
time. Prior to the manufacture of disposable needles, which
appeared when I was 8 years old, I recall my mother sharpening
reusable hypodermic needles on a stone to make the injections
less painful. During my childhood, adolescence and young
adulthood, the medical community nearly unanimously concurred
that blood sugar control made no difference in the development
of diabetic complications, and thousands of children with
diabetes died prematurely as a result.
Clearly, embryonic stem cell research will require enormous
resources to define a usable path to treatment, if such a path
exists. While many respected scientists are understandably
enthused about the possibilities, human embryonic stem cells
may not provide a viable path, and as the path adopted for
treatment of diabetes for 40 years, just as this treatment was
also not viable.
Senators, I am not sure that my experience gives me any
more legitimacy in my views than any of the other 1 million
patients with diabetes or any other conditions, but the more
fundamental issue is not whether embryonic stem cells will
provide a better path for a cure. It is whether or not this
line of research should be entertained by society.
All societies are based on law, even unjust societies. The
difference between a just and an unjust society is the premises
it accepts to begin establishment of legal precedents. The
United States, in my view, is a uniquely just society because
of the first 10 amendments to its Constitution. These
amendments for the first time in human history established a
system where the rights of the individual supersede the
perceived right of the State, implicitly defining the
individual as the most valued entity within the society.
History has amply demonstrated the ghastly consequences
when Government arbitrarily defines what constitutes human
life. The law is based on precedent, and once the United States
allows the individual human embryo to be sacrificed for a
perceived greater good, the greatest defense for the rights of
individuals will have been eroded. For the first time, the
perceived right of the Government will supersede the right of
the individual.
PREPARED STATEMENT
The pain and suffering endured by those with disease must
be addressed, and the means to providing solutions encouraged.
The decision to allow human embryonic stem cell research,
however, must not be made solely on the basis of its utility in
curing disease. It must be considered in the context of its
potential to initiate an insidious disease in our society's
values and its potential to erode the foundation that the
United States provides for all individuals. Alternative methods
and cures must be pursued on both scientific and societal
grounds.
Thank you.
[The statement follows:]
Prepared Statement of Anton-Lewis Usala
Senators: I come as a medical scientist, a person who has had
diabetes 41 of my 42 years, and a concerned citizen of the United
States. I was asked to present the approach my company has developed
for the treatment of Type I diabetes, and other disease states that
require replacement of non-functioning tissues. The premise of our
approach is to replace, as naturally and physiologically as possible,
the insulin producing cells that are destroyed in children with
diabetes. This effort would require large numbers of replacement cells,
as there are approximately one million patients with Type I diabetes in
the United States. Since only several thousand human pancreases are
available to provide a source of these cells, developing a method of
preventing rejection of animal derived insulin producing cells has
become the focus of our effort.
Our most promising approach to solving the problem of cross species
transplantation has involved culturing tissue from pig pancreases in a
proprietary hydrogel material derived from pig skin. The cells in this
material are then injected in a diabetic animal, without using any
immunosuppression. The material is a co-polymer designed to simulate
the structure of connective tissue present during the first few months
of fetal development. Connective tissue is the scaffolding that cells
attach to, and during fetogenesis, the connective tissue is quite
different in appearance than later in life.
My hypothesis was that during embryogenesis, undifferentiated cells
must first bind to this connective tissue in order to properly
communicate, integrate, and mature with surrounding cells. If this were
true, then perhaps allowing mature cells to bind to a similar
connective structure would induce de-differentiation, allowing them to
receive local signals from the area in which they were transplanted.
This de-differentiation apparently reduces the ability of the patient
to recognize the tissue as foreign. When we have placed pig pancreatic
cells in our biopolymer material, and injected them into either the
muscle or liver of diabetic dogs, the animals' diabetes control is
greatly improved. Furthermore, we have demonstrated that the pig tissue
survives, and integrates within the dog tissue, without the need for
immunosuppressive drugs. This is not yet a cure, but does demonstrate
the ability to keep foreign tissue alive and partially functional
without the need for either human embryonic tissues or
immunosuppression.
Stem cells are attractive as a therapy because they are responsive
to embryonic signals and are thereby ``differentiable'' into many
different kinds of tissue. Our biopolymer material has been used for
its ability to directly stimulate host tissue de-differentiation,
regeneration and repair, without the need for stem cell
transplantation. Last Thursday, the Food and Drug Administration
authorized the commencement of human clinical trials utilizing our
material in the treatment of diabetic foot ulcers. We have demonstrated
that a single injection of the material induces complete reconstruction
of chronic and acute skin wounds in rabbits, dogs, and pigs. This
includes reconstruction of skin architecture that is non-nally only
made during fetal development. Blood vessels, hair follicles, and
dermal structures are integrated in a scarless, rapid healing after a
single injection of the material. While the complete mechanism of
action is not known, we believe these findings result from the
following:
Upon injection, the biopolymer binds to the patient tissue with
which it comes in contact. Upon binding to this fetal-like connective
tissue biopolymer, the host cells de-differentiate into a fetal shape,
resembling pluripotent mesenchymal tissue. Over time, we have observed
these de-differentiated cells apparently differentiating into specific
structures required in the wound areas. Thus, the host cells are again
receptive to local signals that enable re-creation of specialized
structures after binding to the biopolymer. Thus far, the material is
limited in its ability to induce recreation to skin and related
structures.
Replication of specific tissue requires cells to receive an
enormous number of specific signals. What defines a human life is the
cellular mass that is able to produce and integrate this enormous
number of sequences, that occurs shortly after fertilization. The
cascade specific cellular differentiation begins, and continues
throughout the adult life of the person. It can be argued when the
reasoning of the fetal organism begins; it cannot be argued when it is
human.
While the developing embryo is equipped to orchestrate the timing
of these millions of signals, it is not at all clear how taking
totipotent or pluripotent cells from a human embryonic donor can be
induced to this degree. Severed from the intricate and specific
environment in which they are made, totipotent or pluripotent cells
require the signals from thousands of sources. While knowledge would
undoubtedly be gained from this research, this is different from
development of a treatment, and there are different approaches that do
not require using human embryonic cells.
Whether or not our approach will induce recreation of complex
tissue structures in human patients will be known shortly. I hope what
we have observed in animal studies will also be the case in patients
with diabetic foot ulcers. If so, variations on this approach will be
researched by others for the ability to induce other types of tissue
repair, such as nerves, utilizing the patient's own regenerative
abilities.
Often, the highly regarded scientific and medical community will
take a position based on their collective interpretation of data that
is incorrect. Differing opinions are not heard, because they are
outside the mainstream thinking. In the treatment of diabetes, this has
had chilling results. I am 42 years old, and have had diabetes 41
years. Prior to the manufacturing of disposable needles when I was 8
years old, I recall my mother sharpening the re-useable hypodermic
needles on a stone to make the insulin injection less painful. During
my childhood, adolescence, and young adulthood, the medical community
nearly unanimously concurred that blood sugar control made no
difference in the development of diabetic complications. In 1968, the
life expectancy of a child who developed diabetes before the age of ten
was approximately 30 years, as most would die of kidney failure. When I
was 10, it seemed to me that nature keeps blood sugars within a very
tight range for some reason, and it accomplishes this by releasing
insulin from the pancreas whenever a person eats food. Shortly
thereafter, I began to surreptitiously take injections of fast acting
insulin before or immediately after eating. My intent was to try to
replicate nature by providing insulin when it was required to normalize
blood sugar after eating.
I sit here today able to talk about the mistakes. Most of the
children I grew up with that have diabetes are no longer alive, as
their physicians practiced the state of the art medical doctrine, which
was that control of blood sugar was not important. Throughout my
medical school training, this is what was taught. It wasn't until the
early 1990's that the medical community finally reversed its view,
after hundreds of thousands of children had died from complications of
poorly controlled diabetes. Clearly, embryonic stem cell research will
require enormous resources to define a useable path to treatment--if
such a path exists. While many respected scientists are understandably
enthused about their possibility, human embryonic stem cells may not
provide a viable path, as the path adopted for treatment of diabetes
for forty years was also not viable.
Senators, I am not sure that my experience gives me any more
legitimacy in my views than any of the other one million patients with
diabetes. But the more fundamental issue is not whether embryonic stem
cells will provide a better path for a cure or not; it is whether or
not this line of research should be entertained in our society.
All societies are based on law, even unjust societies. The
difference between a just and an unjust society is the premises it
accepts to begin establishment of legal precedents. The United States,
in my view, is a uniquely just society because of the First Ten
Amendments to its Constitution. These Amendments, for the first time in
human history, established a system where the rights of the individual
supercede the perceived right of the State, implicitly defining the
individual as the most valued entity within the society.
History has amply demonstrated the ghastly consequences when
government arbitrarily defines what constitutes human life. I am not
suggesting that those who want to use human embryonic tissue are of the
same mind. However, the law is based on precedent, and once the United
States allows the individual human embryo to be sacrificed for a
perceived greater good, the greatest defense for the rights of
individuals will have been eroded. For the first time, the perceived
right of the government will supercede the right of the individual. The
pain and suffering endured by those with disease must be addressed, and
the means to providing solutions encouraged. The decision to allow
human embryonic stem cell research, however, must not be made solely on
the basis of its utility in curing disease. It must be considered in
the context of its potential to initiate an insidious disease in our
society's values, and its potential to erode the foundation of the
United States' protection of all individuals. Alternative methods and
cures must be pursued, on both scientific and societal grounds.
Senator Specter. Thank you very much, Dr. Usala.
I will begin the questioning with you, Mr. Heagy, and you
obviously have undergone great trauma, and interestingly make
the comment that your life began on the day when you were
disabled, and you talk about the quality of life, and Dr. Usala
talks about sacrificing human embryos.
What we are considering here in a sense is the availability
of stem cells from embryos to cure very horrible illnesses,
Parkinson's, Alzheimer's, stroke, perhaps cancer, and we have
the embryo, which Dr. Usala defines as human embryos, but what
is your evaluation of the quality of life, on the assumption
that the human embryo is living, or with the conclusion that
the human embryo is living, with the ability of those stem
cells to cure so many people from dreaded diseases?
Mr. Heagy. I think the difference in my opinion would be
federally funding programs and the ramifications of the future
that we do not have control of. At this point we all are here,
but eventually we will all die and new people will come
forward, and this I think is the beginning of some serious
decay in respect to human life.
Senator Specter. The embryos are going to be destroyed, as
we all know, if they are not used in in vitro fertilization,
and being discarded, they will not live, again assuming life,
and how do you evaluate their being discarded and the quality
of life which they have as embryos contrasted with the good
that may be done to people who have Parkinson's or other
diseases?
Mr. Heagy. I believe that that is another issue that this
Nation has determined was an okay procedure, and it is a
different subject. We are talking about a new set of laws, a
new set of procedures, and utilizing that and funding that
through taxpayer's dollars. I am not opposed to research. I am
not opposed to walking again. I am just opposed to the process
that we are discussing here and the funding source, and the
good people in this Nation that have serious negative
implications to this process. Forcing them to fund this is--it
goes against what I know about America.
Senator Specter. Let me ask the same essential question,
Dr. Usala. You talk about sacrificing human embryos. To what
extent would you say that you have a human embryo, given the
status of the embryo in in vitro fertilization and the
consequence of its being discarded, so it either--it ceases to
exist, or cannot help people who have horrible illness, what do
you think?
Dr. Usala. I think you have crystallized, Senator, the
dilemma. I think that some of what I have learned is from the
result of not elective, but research that was done on
abortuses, and so some of what we were able to do to stimulate
the regeneration, and I am talking total regeneration and
reconstruction, was coming from that kind of, taking an infant
that was prematurely born and died, we were able to get some
knowledge from that.
But Senator, what I am concerned about is the paradigm
shift that is occurring here. We all know what happens once the
precedent is that, well, taking a human embryo and using it for
research is okay. What I am very much concerned about is the
paradigm shift and its effect on the United States
Constitution. I am very, very concerned about that.
Senator Specter. You say you think it is okay to use the
human embryo? You are concerned about what that may lead to?
Dr. Usala. What we know it will lead to, Senator.
Senator Specter. Well, what do you know it will lead to?
Dr. Usala. Well, for instance, for in vitro fertilization
they can create embryos and----
Senator Specter. I am going to go a little over my 5
minutes here, because I want you to have a full opportunity to
set forth your viewpoints here.
Dr. Usala. Thank you, Senator. What we know will happen is
that with in vitro fertilization, if we have these embryos,
science is an all-consuming fire. What will happen is, maybe a
few more embryos will be fertilized so that we could give the
embryos to justifiably sound science for development.
Senator Specter. So you think they will be creating embryos
for scientific research.
Dr. Usala. It will lead to that, if we state as the
precedent that the use of these embryos in this way is in and
of itself okay.
Senator Specter. But if we could certify, guarantee that
embryos would not be created except for genuine purposes of in
vitro fertilization, and it is a medical decision as to how
many you need to create and what you will do with them, but you
do not create them specifically for scientific research, if
that could be done, would that satisfy you?
Dr. Usala. It cannot be. I am a medical scientist, and I
know what happens. What happens is, as I say, science is an
all-consuming fire, and the need to know is all-consuming, and
what will happen is, well, let us say we need to do,
arbitrarily, 10 embryos to have a reasonable success at in
vitro fertilization. What will happen, I assure you, is that
for medical reasons is they will say, well, we should probably
do 20, because that way we can get a better feel for this. It
will happen, Senator, there is no doubt. It cannot be
legislated.
Senator Specter. Well, the guidelines prohibit it.
Specifically, quote, the ban on Federal funding involves the
prohibition of Federal funding for creation of a human embryo,
or embryos for research purposes, but I understand your point
of view, and we are very interested in it.
Pastor Saltzman, you and I have the same home State. I grew
up in Russell, Kansas.
Mr. Saltzman. We were at opposite ends.
Senator Specter. Not quite. Russell is about half-way
across the State, and Olathe, of course, is in the eastern part
of the State. When you were 1 year old I was graduating from
high school, so I have seniority on you. I want you to
understand that.
Mr. Saltzman. I am happy to hear it.
Senator Specter. Pastor Saltzman, you talk about, if
aborted you might have had your fetal tissue as part of someone
else. Do you oppose the use of fetal tissue for medical
purposes?
Mr. Saltzman. By and large, yes. I think the present system
we have is subject to far too much abuse. We have had examples
in the Kansas City area of tissue extraction companies playing
things with the fees and such as that.
Senator Specter. Tissue extraction companies, fees?
Mr. Saltzman. Yes.
Senator Specter. Well, there had been the concern expressed
by many that use of fetal tissue would promote abortions. That
concern has pretty much receded. We had an interesting vote in
the Senate when Senator Thurmond, who is a very strong
conservative and strong right-to-life, had opposed the use of
fetal tissue, and then his daughter had juvenile diabetes, so
he testified in favor of use of fetal tissue, and instead of 40
Senators favoring fetal tissue, the number jumped to 80, and
parenthetically it is worth observing that Senator Thurmond
believes in the use of stem cells.
Mr. Saltzman. If you are speaking of fetal material derived
innocently----
Senator Specter. Yes.
Mr. Saltzman. Spontaneous miscarriage, that removes the
moral dilemma for me.
Senator Specter. Well, but suppose you have fetal tissue,
or however you designate it, terminology, from an abortion
which is being done not to create fetal tissue but for many
other reasons why they are performed, do you object to the use
of that tissue?
Mr. Saltzman. I do.
Mr. Heagy. Could I make a comment on that, Senator?
Senator Specter. We would be interested in your views on
the life or quality of life of embryos. You said we were all
once embryos, but the general pattern is that we are embryos
having been created by in most cases by a wife and a husband
and then carried to term, as you and I were, but what about
these embryos which are frozen, created for purposes of in
vitro fertilization and then----
Mr. Saltzman. I think if you are asking about their
disposition----
Senator Specter. Disposition, quality of life----
Mr. Saltzman. I think that is a separate issue, and it
needs to be addressed separately. What I do know is that we
should not use human life for the benefit of ourselves. If
these embryos are going to be discarded anyway--I know the
argument goes--why not go ahead and use them? We have prisoners
on death row that are going to be executed anyway. Why not get
some use out of them before they are dead?
All of this cheapens, I think, human life and our respect
for it. We have decided that some may be used by others for
their own purposes. Is this what we are saying?
If the embryo was human life, as the President's own
advisory bioethics commission said, if the embryo is human
life, then it deserves the respect owing to human life, and the
commission also said then that when it comes to research in
these areas we need to find the least morally objectionable
method to advance our research, and that is the President's own
commission, and this is all I am saying, too.
Senator Specter. Well, it is a matter of where you draw the
line. If you have convicts who are serving life sentences----
Mr. Saltzman. Where do you draw the line? Where do you make
the distinction? The convict is bigger and the embryo is not?
Senator Specter. Well, I would draw the line that a convict
is a living person, and although he may have the death penalty,
or she may have the death penalty, they may have a DNA test
tomorrow and they may be exonerated, but at any rate, they are
living people, and this comes back to the issue of the quality
of life which is articulated of the embryo and the contrast as
to what can be done by way of saving a lot of people from
greater diseases.
Senator Harkin--Pastor Saltzman, I did not want to cut you
off if you want to say something more.
Senator Harkin. Thank you, Mr. Chairman. I have listened
closely to your line of questioning and the answers and I
again--I do not think anyone should--I know Senator Specter
well, we know each other well. I do not think anyone should get
the misimpression that we do not wrestle with the ethical and
moral implications of what this is all about.
We push very hard a couple of years ago, 2, 3 years ago to
get the bioethics commission. We had them here to testify to
get them to move on this, to examine in depth the ethical and
moral implications of this, and to give us some
recommendations--I am not an ethicist. There are people that
understand these things much better than I do. You, for
example--but try to get as much of an input as possible into
this to advise us on what would be--if there is an ethical and
moral way of doing this, and if there is, how? Well, they came
back and said yes there is, and here is how, and they put these
guidelines out, and I guess we get back to the point that--the
dividing point on this seems to me to be whether or not one--
you see, I think if one is opposed to in vitro fertilization as
a procedure, just totally opposed to that.
Then I can understand how logically from that one might say
that the use of any of these embryos from that is immoral. If
the first act is immoral, the other acts are immoral, so I
guess we have to first find out whether or not the person
thinks in vitro fertilization is an ethical or moral act.
Second, then it seems to me that once you have in vitro
fertilization, you have got all these frozen embryos--I guess
we have got 100,000, I guess, that are frozen now in nitrogen,
liquid nitrogen--that they are going to be discarded. They are
just not going to be kept forever and ever and ever and ever.
They are going to be discarded.
So if they are going to be discarded, is it more ethical
and moral, then, to use these in a method that might reduce
human suffering, prolong life, make our quality of life better?
Is it more ethical and moral to do that than it is to discard
them? That seems to me where we are coming, where we are sort
of at the balance point here.
Mr. Saltzman. Let me pose a question to you, Senator, if I
may. We have 100,000 frozen embryos at present on hand,
roughly.
Senator Harkin. Yes.
Mr. Saltzman. We use those up. We go ahead with our stem
cell research, and we use those up. We need more. Are we not
stimulating the production of human embryos for the purpose of
experimentation? We are not going to stop at 100,000.
Senator Harkin. Well, let me just say this. No, under the
guidelines, again, unless you just say the guidelines are
worthless, but I do not think they are worthless. They are
saying that----
Mr. Saltzman. Can they be amended? They will be.
Senator Harkin. Well, it says here that providers of stem
cells cannot make a profit. You cannot sell them, and you have
to have informed consent.
For example, if I am a donor through in vitro
fertilization, I say no, you cannot use those embryos for that,
you cannot use them. You have to have the informed consent of
the donors themselves, and they have to be derived in excess of
those needed for in vitro fertilization, so if someone goes in
for in vitro fertilization, embryos are created, one is
implanted, and that embryo develops into a human being and a
child, then you have the other embryos that are frozen, I mean,
how can you say you are producing the embryos for research when
in fact it is a couple going in for in vitro fertilization?
They are not going in saying, we want to donate for research.
They are donating it for in vitro fertilization.
Mr. Saltzman. Why would the fertility clinic--I presume the
Federal money would be spent paying the fertility clinic for
the embryos that have been donated, is that not so?
Senator Harkin. I am not--say that again. What is that?
Mr. Saltzman. When a couple goes in, they make 10 embryos,
they use one, there is 9 left over, the Federal Government will
pay the fertility clinic for the 9 embryos, will they not?
Senator Harkin. I do not think so.
Mr. Saltzman. No?
Senator Harkin. Two things. Under the guidelines, they
cannot pay for them. They can pay for the cost of
transportation, storage and transportation, but they cannot
make a profit from them. They cannot sell them. Second, the
guidelines specifically require the separation of the donation
and the generation.
Mr. Saltzman. I think that is a bookkeeping thing, you
know.
Senator Harkin. Well, let me ask you this. Are you opposed
to in vitro fertilization?
Mr. Saltzman. Personally?
Senator Harkin. Well, I do not know, I mean personally,
philosophically, ethically, morally, are you opposed to it?
Mr. Saltzman. No. I believe that is morally neutral. It
raises a host of questions afterwards, though, and that is why
we are having these discussions today. What do you do with
these embryos that are left?
Senator Harkin. Well, that is right. I keep saying that is
the balance point.
Mr. Saltzman. I do not know what to do with them. I know
what not to do.
Senator Harkin. Well, do you think they should--I mean, if
they are going to be destroyed, I mean, again, what do you do?
Mr. Saltzman. I cannot--we are going to die, all of us.
Senator Harkin. We are all going to die, that is true.
Mr. Saltzman. But you know, I once said, just because we
say this thing is going to happen anyway does not allow us to
do another thing first.
Senator Harkin. Well, again I think I can understand if you
were opposed to in vitro fertilization, then I can see it, but
if you are for it, then you have got to say, okay, what do you
do with these left-over--and if you have strict guidelines that
separate it out, you cannot pay for them, you cannot generate
it for that purpose, it seems to me that--to me, anyway, that
the morally and ethically responsible thing would be to say how
can--if there is a promise, in terms of research under ethical
guidelines, to use them to alleviate human suffering, that is
where I come down. I come down on that side of it, I guess.
Mr. Saltzman. You are familiar with the old argument, does
good actually derive from evil means? It does not. I believe
that human embryonic research, the production and use of human
embryos for those purposes----
Senator Harkin. But we are not doing that. These are in
excess of in vitro fertilization. I keep saying that we are not
producing them for this purpose. The guidelines are very, very
specific on that.
Well, obviously we can go on and discuss this for a long
time.
Mr. Saltzman. Have you ever read Brave New World, by Aldous
Huxley?
Senator Harkin. Sure, when I was in college, and I read it
once again later on.
Mr. Saltzman. Read it again--three times. He wrote it in
1933. He first saw genetic cloning in what he called decanted
babies.
Senator Harkin. Yes.
Mr. Saltzman. What he worried about then is what we need to
worry about today. He foresaw a time, as with the Sabbath, when
science was made for man and instead we have come to a day when
man is being made for science, and somehow things have been
twisted all around.
Senator Harkin. I do not agree with that. I mean, I do not
think that is where we are at this point. I happen to think
that our scientists are working to try to find interventions
and cures for diseases and illnesses that affect humankind.
There may be other new illnesses and diseases that arise in the
future that we do not even know about, and we are going to
continually need to do the research necessary to try to
intervene on those.
I guess I am not a fatalist. I do not believe in, I guess--
maybe because of my religious background, I do not believe in
predestination. I do not believe in--I am not a fatalist. I do
not believe that just because a person has had an accident, or
because they were born a certain way, or have an illness, that
that is how it must be forever and ever. If there are medical
ways of intervening to relieve suffering or to make a life more
whole, I think that is what we ought to be about.
I think that is the humane, moral approach that we ought to
be about as a society. It is not demeaning human life. I think
it is enhancing human life and making human life better. Of
course we are all going to die, but I do not think that it is a
necessary lot in life that people have to suffer from
debilitating diseases. If that is so, we never would have cured
polio. If that is so, we would not have called smallpox, or the
other things that we have overcome. Now we are looking at other
things that we can overcome.
Mr. Saltzman. No one seated here is opposed to research.
All we are saying is there are less ethically objectionable
methods to do the same kind of research.
Senator Harkin. Well, I do not know. I mean, obviously I
think--I agree we ought to do the adult stem cell research, as
Dr. Hynes and Dr. Prockop said before, but that does not mean
we should do that and not do the other ones. I think we ought
to advance on all these fronts.
But I am taking a lot of time to engage in a polemic
exercise here, and I apologize to you for it, but it is an
important aspect, and it is something that we wrestle with all
the time.
Mr. Saltzman. I have enjoyed the exchange. Thank you.
Senator Specter. Thank you very much, Senator Harkin. I
think it is very useful to spend extra time on those who are
opposed to stem cell research, we have a complete record and
give every opportunity to state your position, because we put
on the record that we have introduced legislation in favor of
research, but in so doing we want to be sure that these
hearings are conducted in a very fair and evenhanded way to
give you a full opportunity to express all of the opposing
views.
Senator Harkin. I am sorry, I have one other thing if you
do not mind, Mr. Chairman. I was so involved with Dr. Saltzman.
Mr. Heagy said something that I wanted to respond to. If
you know anything about my background, you know I worked for a
long time in the area of disability rights, American
Disabilities Act, which I was the chief sponsor of. You made a
mention of something about, does this mean that we are going to
back off on support for community services and things for
people with disabilities. I just want to answer that, and the
answer is absolutely not.
In fact, Senator Specter and I are both sponsor and
cosponsor of the MCASSA bill--I can never remember what it
means--Medicaid Community Attendance Services and Supports Act,
which reforms medicaid to enable and empower people with
disabilities to decide exactly how they want their support
services delivered, in a community setting, at home, with
personal attendance services, or in an institutional setting.
Mr. Heagy. In my State I--yes. For 18 years of my life I
received in-home support services so that I did not have to be
in a nursing home, that I could go out and try to make a life
for myself, but it has been recently that we have implemented
an initiative for disabled folks to go to work and continue to
get those benefits, because I require 24-hour care, which I
would have to make $100,000 a year just to be able to afford my
caretakers.
Senator Harkin. I just wanted to respond that we are not
backing off of that.
Mr. Heagy. I appreciate that, but there is a whole lot that
we needed to do to get here. There was hardly any
transportation. I mean, I had to roll from the hotel to here,
and the fact that these services are not accessible, it is just
difficult. There are issues in life that we need to address and
spend more time, and the money that we are spending.
And I just wanted to ask concerning, real quick, the frozen
embryos are potential human life right now. If you farm them
out and let them die, then they are dead, correct, and
prisoners, and like--what we were discussing, yes, they have a
voice right now, but once they are executed they are dead, so
why can we not take their parts. It is the same thought process
in my mind, and do we seek permission from the folks that gave
up those 100,000 frozen embryos to see if they morally agree
with this? You are talking about the future, but what about
where those came from? Are those donors okay with this?
Senator Specter. Mr. Heagy, I am advised that consent has
been obtained from the donors, that we have some 100,000 unused
embryos, and the NIH guideline requires informed consent from
the donors, including the statement that embryos or fetal
tissue will be used to derive stem cells, so that that has been
obtained.
We have been joined by Senator Reid, who is a member of the
subcommittee. Senator Wellstone has rejoined us, but let us
turn to Senator Reid now, and Senator Wellstone, we will come
back to you if you wish to ask questions.
Senator Reid. Mr. Chairman, thank you very much. I have to
be at the Senate when it opens, so I thank you very much for
allowing me to say just a few words. I first of all am
continuing to be the biggest cheerleaders that you and Senator
Harkin have. I greatly appreciate the work that you have done
in this area. This hearing is also part of the ongoing work
that you are doing, and I cannot give you enough accolades for
the work that you are doing and have done.
Let me just say, briefly, that I come at this hearing and
the work that you have done in this area not because of
statistics and philosophy, but because people that I know that
I have hope that work that we are doing here and work being
done in the scientific community will help them.
Steve Ragazio is a young man who has Lou Gehrig's disease.
He is the president of the largest utility we have in Nevada,
and every day he gets up his condition is worse. I hope that
the work that is being done is going to help Steve, that he can
play with his children and do things that he could do a few
months ago. He cannot do it now.
I hope that Molly Singer, who is a little 11-year-old girl
that I have known since she was a baby, who is a twin, we can
do something about her diabetic condition as a result of the
work that is being done because of the emphasis that you,
Senators Specter and Harkin, have focused on this issue, and
the money that has come from this subcommittee to allow
research to continue.
So we have made progress. Again, I am not here to
philosophize. I am here to help Molly and Steve get well, and I
hope that we can do that.
Senator Harkin. Thank you very much.
Senator Reid. I will ask permission of the committee to be
excused to go to the Senate. We are opening at 11 a.m.
Senator Specter. Thank you very much for joining us,
Senator Reid, and thank you very much for those very
complimentary----
Senator Reid. The one thing I did want to say, also I want
to thank Michael J. Fox, Jennifer Estess, and Mary Tyler Moore.
It is wonderful that they are willing to put their celebrity
status on the line to help us with this project.
Senator Specter. I agree with you, Senator Reid.
Senator Wellstone.
Senator Wellstone. Fine, Mr. Chairman. I thank the panel. I
came in at the very end, so I do not have the context. I am
just here right now. Thank you.
Senator Specter. We are going to take a very brief recess.
Thank you very much for coming in, Mr. Heagy, Pastor Saltzman,
and Dr. Usala. Your testimony is very helpful in putting on the
record the articulated views in opposition to stem cell
research, which have been very cogently stated, so thank you.
The hearing will now resume, and we call Ms. Gina Gershon,
Ms. Jennifer Estess, Ms. Mary Tyler Moore, Mr. Michael J. Fox.
We very much appreciate your coming in, and to comment briefly
on a statement I made at the outset of the hearing, when people
understand the impact of these illnesses, when Americans
understand the impact of these illnesses on people they know,
there tends to be a greater public awareness and more public
support, so after hearing from the scientists, and after
hearing from people who were in opposition on a variety of
grounds to stem cell research, we do thank you for joining us.
We have Jennifer Estess who is coming in, but in the
interim we will proceed with the testimony of Ms. Gina Gershon,
who has enjoyed a wide-ranging career in film, television, and
on the stage. She most recently was seen in the Academy Award-
nominated film, The Insider with Al Pacino, and for the past 20
years Ms. Gershon has been a close friend to Ms. Estess.
Ms. Gershon met Ms. Estess while studying for her bachelor
of arts degree in New York University. We are going to be
joined here by Ms. Estess momentarily. Let us wait just a
moment or two. She is coming through the main door because of
the logistics of the situation.
While Ms. Jennifer Estess is coming in, and we thank her
for joining us again today, she is president of the project,
amyotrophic lateral sclerosis, which she founded in 1998 after
being diagnosed with the disease in 1997 at the age of 35.
Since that time, she has been working with the corporate
entertainment communities to help raise funds to find a cure
for ALS, also known as Lou Gehrig's disease. She was the
producing director of the off-stage Broadway theater company
Naked Angels, and is currently the executive producer of CBS
movie based on her life, scheduled to air early next year.
This is Ms. Estess' second appearance before the
subcommittee, having testified last time with Mr. Christopher
Reeves back in April 26 of this year.
Ms. Gershon, we turn to you. We have lights, with the green
one indicating 5 minutes, the yellow 1 minute, and the red,
stop, so you may proceed.
STATEMENT OF GINA GERSHON, ACTRESS
Ms. Gershon. Honored chairman, distinguished members of the
committee, I want to first thank you for giving me the
opportunity to speak to you today on behalf of my friend,
Jennifer Estess, and her work with Project ALS.
Jennifer and I met as freshmen in college, and we both
spent our twenties in New York City planning our futures and
building a theater company together. In a group of passionately
independent young people, it was always inevitably Jennifer who
took it upon herself to focus our work, who, despite our
energetic and often conflicting ambitions always managed to
keep the bigger picture in sight for us.
So it was no surprise to me, or any of us who knew her,
that when she was diagnosed with ALS, when she was told that
she had, at most, 5 years to live, Jennifer instinctively
turned her attention from her own condition to the larger goal.
She was scared. We were all scared, but once again she asked us
to look beyond ourselves to help her find a cure.
That search led her and her sisters to the stem cell
research being conducted independently at some of the Nation's
leading research institutions. She brought these doctors
together, encouraged them to share their research, and asked
how she could help. When they said they needed more money, she
and her sisters and friends created Project ALS and began
raising money, over $4 million so far, almost all of which has
gone directly to equipment and research, and that research has
already produced amazing results.
As you know, ALS is a singularly destructive disease,
striking randomly, methodically paralyzing its victims muscle
by muscle until they can no longer breathe. It now seems
possible that embryonic stem cells have the potential to stop
or even reverse that process. We do not know for certain, but
to not find out, to not get every possibility of finding out,
seems like an agonizingly cruel decision.
While there are some people here today who feel that some
diseases are better than the cure, I am positive that many
people would feel that a cure is much preferable to having the
disease. People should be allowed to have that choice.
Mr. Chairman, members of the committee, I cannot pretend to
be an expert on this subject. It is complex and controversial.
I know that. My friend here is dying, and I have to do
everything I can to help her, and to help her find a cure, if
not in her lifetime, perhaps in our own.
Thank you.
Senator Specter. Well, thank you very much, Ms. Gershon.
Amyotrophic lateral sclerosis is certainly a dreaded disease.
It came into very sharp public view with Lou Gehrig, who was
stricken with amyotrophic lateral sclerosis and disabled, and
died shortly thereafter.
Ms. Estess, we appreciate your being with us again, and we
look forward to your testimony.
STATEMENT OF JENNIFER ESTESS, ACTOR/PRODUCER
Ms. Estess. Thank you so much. I also wanted to thank Gina,
and I feel honored to be on the panel with Michael and Mary,
and good afternoon to Chairman Specter and the members of this
committee. I want to thank you for inviting me to come back to
speak to you again.
My name is Jennifer Estess, and I have ALS, a disease which
is always fatal. 5 months ago I came here to tell you about my
story, my work, and to ask for your help. Since that time,
while we wait, I have come to rely on this ventilator. That is
because ALS is destroying the muscles I use to breathe.
Three years ago I was a healthy woman who worked hard and
who dreamed of starting a family. Today, I cannot walk, brush
my own hair, or hold my nieces or nephews. But although my body
has deteriorated, I am proud to report that stem cell research
funded by Project ALS has progressed with stunning results.
When I was first diagnosed, I turned to the medical
community for help. They told me that I was beyond the reach of
science and medicine. There was not one drug to slow or stop my
disease. A cure was unimaginable. But we now know about stem
cells and their amazing medical potential.
We know that they instinctively migrate to the area of
injury in the brain and spine. We know that they transform into
healthy new cells. We know that they may eventually repair the
damaged nervous system. Every indication from our distinguished
team of doctors is that there will be a treatment, even a cure
for ALS, that stem cell therapy will work on humans, but we
need your help.
We need your funding. We need your funding to capitalize on
the tremendous success that Project ALS and other organizations
have realized in the private sector. We need Government control
so that no one will abuse stem cell research, and we need the
Federal Government to ensure that this life-saving work is
carried out in the safest, most responsible way.
Since we last met, I have taken what would have previously
been a vacation to the beaches of Southern California. As you
might imagine, it was quite a trick for me to get to the shore.
It took literally a team of people, my sisters, my friends, my
health aides, to carry me to the water's edge. I lay on a beach
chair as they lifted me up, and we all moved as a group into
the water, and it struck me that the only way we will ever beat
ALS is to work together like this to further the promise of
stem cells, because we are running out of time.
Millions of Americans with ALS, Parkinson's, Alzheimer's,
are running out of time. The children who love them are running
out of time.
Mr. Chairman, I know that good science makes no promises
and I am under no illusion that the potential rewards of this
research will save my life, but while I am still able to
breathe, I cannot sit silently as the hope for millions of
other Americans stalls or stagnates or dies, and so although I
am still unaccustomed to asking for help, I am urging you today
to pass the Stem Cell Research Act of 2000.
On behalf of Project ALS and the millions of Americans
living and dying with ALS, Parkinson's, Alzheimer's, and
childhood brain diseases, I implore you to move this
legislation forward with a renewed sense of urgency, to let
this opportunity not slip away, to let us save our friends, our
families, and ourselves now, because we can.
Thank you very much.
Senator Specter. Thank you very much, Ms. Estess. We
appreciate you coming back today. We know it is very difficult
for you to do that, and since you were here in April our
subcommittee came forward with a recommendation of $2.7 billion
of additional NIH funding. That is the largest amount it
increased. The year before we recommended $2.3 billion, the
year before $2 billion, and the year before that, a billion,
which are vastly increased sums over what had been done.
So within the last four appropriations cycles we have added
some $8 billion to NIH funding on top of $12 billion, so you
can see that kind of an increase, we are determined to double
the research funding, and when the stem cell issue was
disclosed in November of 1998, Senator Harkin and I introduced
legislation so that we could use the Federal funding to extract
stem cells from embryos. There has been a ruling that Federal
funding may be used on the stem cells once they are extracted,
but it is very important not to have the limitation on the
Federal funding to extract the stem cells, so we are moving
forward and anticipate a vote on it in the Senate yet this
month.
So we appreciate your coming in and providing this
testimony, and we not only hear you, but we have acted on it.
Ms. Estess. Thank you, sir.
Senator Specter. Our next witness is the distinguished star
of radio, stage and screen, Ms. Mary Tyler Moore, who has been
an active advocate for more than a decade, to my personal
knowledge, about the time Senator Harkin was made a
subcommittee member, honorary subcommittee member at least,
probably best known for her television roles in the Dick Van
Dyke Show and the Mary Tyler Moore Show, received an Emmy in
1992 for her role in Stolen Babies, and was nominated for an
Oscar in Ordinary People. Broadway honored Ms. Moore with a
special Tony for Whose Life Is It Anyway.
She has lived with diabetes for over 30 years, and has
worked to raise public and congressional awareness for this
disease. For the last 16 years she has served as the
international chairman of the Juvenile Diabetes Foundation, and
last week I saw her leading an entourage on the first floor of
the Hart Senate Office Building on her lobbying ways, and--did
she come to see you then, Tom? Me neither.
Ms. Moore, you have the floor.
STATEMENT OF MARY TYLER MOORE, INTERNATIONAL CHAIRMAN,
JUVENILE DIABETES FOUNDATION
Ms. Moore. Thank you, Mr. Chairman. Senator Harkin, Mr.
Chairman, members of the subcommittee, I thank you for the
opportunity today to discuss stem cell research, an issue that
could have enormous impact in the fight for a cure for
diabetes.
I am here today as the international chairman of the
Juvenile Diabetes Foundation, JDF, an organization whose
mission is to find a cure for diabetes and its complications, a
disease that affects a total of 16 million Americans, and costs
more than $100 billion per year.
Since its founding in 1970, JDF has grown to become the
largest private not-for-profit supporter of diabetes research
in the world. This year, JDF will fund up to $120 million worth
of diabetes research, with all of this funding coming from
privately raised resources.
I want to thank the subcommittee for its extraordinarily
strong support for Federal funding for the National Institutes
of Health. I vividly recall several years ago my joining many
of you in a kick-off press conference for the bipartisan effort
to double the budget of the NIH, and that would be over 5
years. Well, I am so appreciative of that work, and been so
glad to see that you have brought us to the almost end of that
5-year period.
However, despite the increases in research funding, our
mission to find a cure for diabetes may not happen unless
scientists are free to use funds to explore some of the most
promising avenues leading to a cure. Stem cell research is an
integral part of finding a cure for this disease.
Many of you know that I have had juvenile diabetes for more
than 30 years. The disease alone is always difficult to live
with. Trying to balance blood sugar levels and insulin
injections is hard enough, but adding to that the severe
complications of diabetes makes things frightening. I know too
well the fears that arise from this part of the disease, and I
know as well as anyone that insulin is not a cure.
In many ways I have been more fortunate than others who
have diabetes. While I have almost faced blindness due to the
diabetic retinopathy, I am still able to see. While I have
faced the possibility of amputation, I am still able to walk.
But things could have turned the other way, as they have for
millions of Americans, and the future of any person with
diabetes is always uncertain.
But Mr. Chairman, there is evidence of a cure that is
within our grasp. Earlier this year, in a study at the
University of Alberta, Canada, 12 individuals with juvenile
diabetes received a successful transplantation of insulin-
producing cells, and they no longer require insulin.
The transplants involve a minimally invasive injection
procedure which does not involve surgery. The cells are placed
into the portal vein. They then migrate to the liver, where,
even though they are not in the pancreas, they take root and
produce sufficient insulin, and almost perfect control of blood
sugar.
However, one of the major drawbacks of this study is that
two cadaver pancreases are needed for each transplantation, and
fewer than 2,000 are available each year. With millions of
Americans having diabetes, including nearly 2 million with
juvenile diabetes, you can see that even if this procedure is
perfected, as we hope it will be, there are not nearly enough
organs to cure everybody.
Stem cell research offers a tremendous amount of hope for
this research. Scientists believe that one day stem cells will
develop into any human tissue or organ. When scientists are
able to specialize these cells to become insulin-producing
islet cells, lines could be developed to produce an unlimited
number of insulin-producing cells. This would effectively solve
the supply issue.
Well, I do understand that some people have heartfelt
concerns about this research. I believe that, given the
safeguards developed within the National Institutes of Health,
and considering that the embryos and fetal tissue used in this
life-saving science would be destroyed, I believe that stem
cell research should be pursued vigorously and is appropriate
for Federal funding.
PREPARED STATEMENT
Mr. Chairman, every year thousands upon thousands of
children in this country are diagnosed with juvenile diabetes,
and one American dies from diabetes every 3 minutes. We owe it
to these children and the 16 million Americans with the disease
to pursue all promising research avenues, including stem cell
research, within the ethical framework established by the
Federal Government.
I thank you for hearing this plea, and I will to the best
of my ability answer any questions that you might have.
[The statement follows:]
Prepared Statement of Mary Tyler Moore
Mr. Chairman, Senator Harkin, and members of the Subcommittee,
thank you for the opportunity today to discuss stem cell research, an
issue that could have an enormous impact in the fight to find a cure
for diabetes.
I am here today as International Chairman of the Juvenile Diabetes
Foundation (JDF), an organization whose mission is to find a cure for
diabetes and its complications, a disease that affects a total of 16
million Americans and costs more than $100 billion per year. Since its
founding in 1970, JDF has grown to become the largest private, non-
profit supporter of diabetes research in the world. This year, JDF will
fund up to $120 million worth of diabetes research, with all of this
funding coming from privately raised resources.
And I want to thank the Subcommittee for its extraordinarily strong
support for federal funding for the National Institutes of Health. I
vividly recall several years ago joining many of you in a kick off
press conference for the bipartisan effort to double the budget of the
NIH over five years. I am so appreciative of the work that you have
done so far to get us almost through year three of this effort.
However, despite these increases in research funding, our mission
to find a cure for diabetes may not happen unless scientists are free
to use federal funds to explore some of the most promising avenues
toward a cure. And stem cell research is an integral part of finding a
cure for the disease.
Many of you know that I have had juvenile diabetes for more than
thirty years. The disease alone is always difficult to live with--
trying to balance blood sugar levels and insulin injections is hard
enough--but adding to that the severe complications of diabetes makes
things much scarier. I know too well the fears that arise from this
part of the disease. And, I know as well as anyone that insulin is not
a cure.
In many ways I have been more fortunate than others who have
diabetes. While I have almost faced blindness from diabetic
retinopathy, I am still able to see. While I have faced the possibility
of amputation, I am still able to walk. But, things could have turned
out differently for me as they have for millions of Americans, and the
future of any person with diabetes is always uncertain.
However, Mr. Chairman, there is hope for a cure. In fact, earlier
this year, in a study at the University of Alberta, Canada, seven
individuals with juvenile diabetes received a successful
transplantation of insulin producing cells and no longer require
injections of insulin.
The transplants involve a minimally invasive injection procedure
which does not require surgery. The cells are placed into the liver
through the portal vein. The cells then migrate to the liver where,
even though they are not in the pancreas, take root and produce
sufficient insulin and almost perfect control of blood sugar. However,
one of the major drawbacks of this study is that two cadaver pancreata
are needed for each transplantation, and less than 2,000 are available
each year. With millions of Americans with diabetes, including nearly
two million with juvenile diabetes, you can see that even if this
procedure is perfected--as we hope it will--there are not nearly enough
organs to cure everyone.
However, stem cell research offers a tremendous amount of hope for
this research. Scientists believe that one day stem cells will develop
into any human tissue or organ. When scientists are able to specialize
these cells to become insulin-producing islet cells, cell lines could
be developed to produce an unlimited number of insulin-producing cells.
This would effectively solve the supply problem.
While I do understand that some people have heartfelt concerns
about this research, I believe that given the safeguards developed
within the National Institutes of Health's recently released
guidelines, and considering that the embryos and fetal tissue used in
this research would be destroyed if not donated to this life-saving
science, I believe that stem cell research should be pursued vigorously
and is appropriate for federal funding.
Mr. Chairman, every day, 35 children in this country are diagnosed
with juvenile diabetes, and one American dies from diabetes every three
minutes. We owe it to these children and the 16 million Americans with
the disease to pursue all promising research avenues, including stem
cell research, within the ethical framework established by the federal
government.
I would be happy to answer any questions, and I would like to
introduce Dr. Marc Hurlbert from the Juvenile Diabetes Foundation, if
you have any scientific questions about this research.
Senator Specter. Thank you very much, Ms. Moore, for your
very poignant and moving testimony. When you talk about what
could happen to you with diabetes, blindness or amputation,
that is really laying it on the line, and when you talk about
somebody dying every 3 minutes, 2 people have died since we
started your introduction, and that is why there are many of us
who are so determined to make use of embryos which will be
discarded anyway----
Ms. Moore. Exactly.
Senator Specter [continuing]. To save lives and stop human
suffering.
We now turn to Mr. Michael J. Fox, who has had a
spectacular career, first as Alex B. Keaton on the television
series, Family Ties, later in a number of movies, including
Back to the Future, and most recently on television again in
the highly acclaimed Spin City. This past week, Mr. Fox won an
Emmy award for best actor in a comedy series for his work in
Spin City.
He was diagnosed with Parkinson's in 1991 at the age of 30,
and since announcing his retirement from Spin City, he has been
devoting his time to the Michael J. Fox Foundation for
Parkinson's Research, and I might comment that he even came to
the Republican convention in Philadelphia.
Mr. Fox. I was in Los Angeles as well, sir.
Senator Specter. Well, when you--Senator Harkin mentions
that you hit them both. I was not at the other one. What was
the other one, did you say?
Mr. Fox. As you said, sir, it is a nonpartisan problem that
will take a bipartisan solution.
Senator Specter. Well, we are bipartisan here, as the
Democrat and Republican representation on the podium suggests,
but here again, spectacular work, and from time to time
questions are raised about celebrities appearing at
congressional hearings.
In fact, it is even more than questions. There is some
pretty severe criticism about it, but we make no apologies,
because we need public awareness of issues like stem cells,
which could cure Parkinson's, or amyotrophic lateral sclerosis,
or Alzheimer's, and when public attention is focused on Michael
J. Fox because the American people know you, Michael, it is a
great use of your talent and celebrity status to let people
know what is going on and get public support for this kind of a
measure.
So we thank your for your time and your efforts, and we
know how debilitating the illness is, and we are just very
hopeful that soon--we had the testimony from the scientists
this morning--in 2 or 3 years there will be ready for use with
people, and that you will be back on ABC TV--I do not want to
say that in derogation of the other networks, which are here,
but back--well, you choose where you go back. We just want you
back.
Mr. Fox. I will be open to all offers.
Senator Specter. The floor is yours, Mr. Fox.
STATEMENT OF MICHAEL J. FOX, FOUNDATION FOR PARKINSON'S
RESEARCH
Mr. Fox. Thank you, Mr. Chairman and Senator Harkin. Thank
you for inviting me to testify, and good morning.
It is hard to believe that--I should say thank you to my
fellow panelists. I am honored to be in your company, and let
us hope it is for the good.
It is hard to believe an entire year has passed since I
first appeared before the subcommittee and addressed the need
to increase Federal funding for Parkinson's research. I am
grateful for that opportunity to speak on behalf of the
Parkinson's community about the reality of living with this
disease.
It was during a hearing in September 1999 that Senators
provided exciting testimony as to just how close we may be to a
cure for Parkinson's. In pursuit of that cure, I am back this
year to lend my voice and that of the Michael J. Fox Foundation
for Parkinson's Research to support Federal funding for
pluripotent stem cell research, including that research covered
by the NIH guidelines announced on August 23.
I do not intend to become a professional witness. I am not
a politician, nor I am a doctor, nor a research scientist. You
do not need me to explain embryonic stem cell research or its
medical applications, so what does qualify me to be at this
table?
The answer is simple. I am one of a million involuntary
experts on Parkinson's disease in the United States battling
its destructive nature as we wait for a cure. We need a rescue,
and the country should know it.
I am also here because I am a guy with PD who happens to be
on TV. Because of that, many people have felt comfortable
reaching out to me. By now, many of you have heard my story,
but you have not heard this story, about a 38-year-old senior
editor whose PD caused her to lose her job at a publishing
house, plunging her from New York's middle class into poverty.
She is now forced to exist on medicare and SSDI benefits, which
are nearly consumed by her monthly medication costs alone.
Nor have you heard about my new friend, a former lawyer,
now living on disability, who corresponds with me regularly.
Two weeks ago his friends and family watched in horror as he
disappeared into stony immobility while waiting for a
prescription delivery that had been delayed. This demonstrated
dramatically just how tenuous normalcy is.
And you have never heard about Brenda, a 53-year-old former
computer specialist. Recently her drugs failed to kick in, and
she found herself frozen in the bathtub with no one to help
her. She remained there for hours until enough medication had
reached her brain to allow her to crawl out of the tub. By this
time she was suffering a panic attack and could not speak. She
could only reach her computer to contact friends for help. Her
biggest regret, she says now, was that CNN was not there to
provide live, up-to-the-minute coverage of her predicament.
None of these people mind that I get more attention than
they do. They simply say that if I get a shot at this
microphone, that I start talking, so here I am again.
For 2 years, you have had a parade of witnesses,
scientists, ethicists, theologians of every school, and some
celebrities, discussing every nuance of stem cell research. You
have given time to all sides of the issue, including a few very
vocal opponents, but the consistent and inescapable conclusion
is that this research offers the potential to eliminate
diseases, literally save millions of lives.
So while I applaud your fairness, I cannot help but say
respectfully, enough. It is time to act on what we have
learned. Sadly, we have lost 2 years' progress towards a cure.
Further delay will come at a high price. That is why I am back
before this committee today. Every day funding is delayed means
that a person with Parkinson's is getting closer to total loss
of independence, or slipping slowly toward the progressive
inevitability of this disease. These delays have real human
consequences that are measured in human suffering and loss of
life.
The pioneers in stem cell research, Drs. Gerhardt,
Thompson, and West, told this committee in December 1998 that
Parkinson's would be one of the first diseases to benefit from
the use of stem cells. Still researchers have testified that it
will take at least 3 years from the time they received funding
to the development of the first stem cell therapies for
Parkinson's.
Even at the fastest possible pace under the newly released
NIH guidelines, the first scientists to receive Federal funding
will not begin working until late next year, which all means
that we are still years away from our rescue. Please, help us
to not wait any longer than we have to.
I am not here solely to represent the benefits of stem cell
research for Parkinson's patients. There are many other
promising applications, from heart disease, to blindness, to
Alzheimer's, to burn victims, to cancer, to HIV/AIDS, to
stroke, to autism, to deafness, to schizophrenia, to diabetes,
to MS and ALS.
Stem cell research could also help those with spinal cord
injuries. My friend Christopher Reeve sends his regrets that he
could not be here today to emphasize the urgency that we both
feel. His testimony has been submitted for the record, but I
would like to share a few of his words with you now.
Since its inception, a fundamental principle of our
Government has been to respond to the needs of people. Now, a
major scientific breakthrough has given us the opportunity to
uphold another principle of our Government, to do the greatest
good for the greatest number of people. I am referring, of
course, to the recent discovery of the miraculous discovery of
stem cells.
Now, those of us who are privileged to testify before our
representatives, though suffering individually from specific
conditions, have the unique opportunity to speak on behalf of
an entire population, both at home and in every corner of the
global village, who suffer from almost every conceivable form
of debilitation.
Thank you, Christopher.
The NIH is ready. Extensive and meticulous guidelines for
stem cell research have been written and approved. We urge you
to not let politics interfere and needlessly delay this
critical research. For 2 years, I have been watching this
debate. To me, this is not theoretical. There are real
consequences here. As I said earlier, I do not profess to be an
ethicist, but I do consider myself a good and decent man, a
loving father and husband. I would not claim any benefit that I
believe was made possible by harm done to another person. That
is not the reality here.
I am not a political scientist, either, but I have an
immigrant's love for my country, and I am humbled to
participate in this process. I see a need for our politicians
to act on our behalf, and for them not to politicize a
wonderful medical advance.
So that is what I ask of the people who are touched by this
issue. Those afflicted and affected, every patient, as well as
every mother, father, brother, sister, grandchild, uncle, aunt,
teacher, coach, friend, or neighbor of a person with
Parkinson's, in addition to the even wider circle of those with
related illnesses and their families and loved ones, which
ultimately include every person in this country, you all have a
stake in the outcome, and I ask those of you who are watching
today to join me in the conversation, study the record, visit
the NIH web site, add your voice. These good men and women are
your representatives. Ask them to tell you where they stand.
I am confident that the vast majority of you would want
this research funded, and quickly. I see in these cells a
chance for a medical miracle. The Government has done its work.
We ask you now to release our tax dollars so the scientists can
do theirs.
I thank you.
Senator Specter. Thank you very much, Mr. Fox, for that
very compelling testimony.
Mr. Fox. That red light is very----
Senator Specter. The red light was on, but it has been on
on other occasions for longer periods of time with people not
stopping talking, and it is OK. It is a parameter and a
guideline. It is not absolute.
You made a statement, Mr. Fox, saying, quote, you would not
claim any benefit that did any harm to another person, and that
leads me to the critical question on this issue, which will be
up for a vote, and the Congress, the Senate, decides questions
for public policy, and we have to make decisions weighing the
arguments on one side, and weighing the arguments on the other
side, and the argument on one side in opposition to Federal
funding for taking stem cells from embryos is that we are
dealing with a human entity, which is alive.
At the same time, it is acknowledged that the 100,000 stem
cells which are now being discarded with the consent of the
donors will not be used for any purpose, but will be discarded,
and any semblance of life will be ended.
On the other side, those argue that with 125 million
Americans being affected by the long list of diseases,
thousands with Parkinson's, thousands with ALS, what would your
message be to the United States Senate as to how you balance
the contention of destroying an embryo, which is living but
soon to be discarded, with the benefit to people who suffer
from Parkinson's, as you do.
Mr. Fox. Well, I think obviously that aspect of it is part
of a grave debate, which there are passionate opinions and
feelings on either side, and it is a debate that in a sense
does not relate specifically to this, but I will address it. As
you said, those cells that are undifferentiated, pluripotent in
the sense that they have not been assigned to be things, they
do not know what they want to be, they are very early in
development, they are pluripotent in the sense that they can be
anything, are being destroyed.
It is a separate argument, why they are being destroyed,
whether they should be destroyed, and like I said, there are
finer minds than mine that can debate that. The fact is, they
are being destroyed, they are being wasted, and the potential
to change the health of this country and this world is
something that it just seems to me the loss of that in the face
of this potential is great, and it is a chance to do good and
to do well.
Senator Specter. Thank you very much, Mr. Fox.
Let me turn to you next, Ms. Estess, since you suffer from
amyotrophic lateral sclerosis, and as you commented, and as is
well-known, it is always fatal, a question of how long, medical
research I have seen, 2 to 7 years. What would your message be
to the Senators who will be saying aye or nay on the contrast
between the contention of embryos being alive, but soon to be
discarded, contrasted with what might be done to save your
life?
Ms. Estess. I think that the focus is not that, it is our
responsibility as Americans to protect each other, and Michael
said while he was speaking with you that every single person in
this room is going to be touched by one of these illnesses, and
I also think that often we do not want to look at things that
are destroying lives, such as ALS and Parkinson's and diabetes
that Mary spoke about.
I think it is our responsibility as Americans to push this
forward so we can get treatments to these people so they can be
with their families and loved ones.
Senator Specter. Ms. Moore, same question. It is the
critical issue, the choice between the embryos, where the
contention is made that they are living, although there is
agreement they will be discarded, contrasted with healing and
saving lives.
Ms. Moore. The embryos that are being discussed, according
to science, bears as much resemblance to a human being as a
goldfish, I think makes the answer clear. We are dealing with
flesh and blood people now who feel pain, feel fear, feel
debilitation, and our obligation is to those who are here.
Senator Specter. Very cogently and movingly stated.
Ms. Gershon, we will give you the last word. It is a pretty
hard act to follow.
Ms. Gershon. Well, I think Mary just said it. I mean, that
is perfect. All I know is, when I look at, you know, my friend
and people that I know, suffering, to have the choice to help
them or not help them, there should be no question.
Senator Specter. Easy choice.
Ms. Gershon. Easy choice. To me that is pro-life. Let these
people live.
Senator Specter. The pro-life position is to let Ms. Estess
live.
Ms. Gershon. Yes.
Senator Specter. And others.
Senator Harkin. And others. Thank you, Mr. Chairman. I just
want to thank all of you for not only being here today, but for
your continuing efforts to get the public more knowledgeable
about what we are about.
I could not help, Mary, when you were talking about
resembling--I did this once before. I held up a piece of paper.
Can you tell me what is on that piece of paper?
Ms. Moore. You have got to be joking. No.
Senator Harkin. There is a little, teeny little pencil dot
that I put on there that you cannot even see. That is the size
of the embryos we are talking about. I think a lot of people
get confused, thinking an embryo is something, almost like a
fetus or something like that, fully developed fetus. We are
talking about something less than the size of a pencil dot----
Ms. Moore. Yes.
Senator Harkin [continuing]. That contains the cells, the
undifferentiated cells that Michael Fox was talking about, so
somehow to equate this with a fully developed human being I
think is stretching credulity quite a bit.
Anyway, I just thank you, because we need your help. I
think the bill that Senator Specter has authored, I am
cosponsor of, that we have the assurances of the Majority
Leader that we will have a vote on it----
Senator Specter. That is correct, this month.
Senator Harkin [continuing]. Before we leave here this
month. I do not know the outcome of that vote. I believe we
have the votes. I hope we have the votes. But your being here
today and your continued efforts in the public realm to just
focus attention on this, to bring it out of the shadows and put
the sunshine on it, does more to help us get this through than
anything else. I am consciously optimistic that we can get it
passed, and I am even more than consciously optimistic that in
the next few years we are going to see tremendous breakthroughs
using stem cells in addressing these illnesses and diseases, so
again I just thank you very much for your efforts.
Senator Specter. Thank you, Senator Harkin, and thank you,
Ms. Gershon, Ms. Estess, Ms. Moore, and Mr. Fox.
Mr. Fox. Thank you.
CONCLUSION OF HEARING
Senator Specter. Thank you for being here. That concludes
our hearing. The subcommittee will stand in recess subject to
the call of the Chair.
[Whereupon, at 11:43 a.m., Thursday, September 14, the
hearing was concluded, and the subcommittee was recessed, to
reconvene subject to the call of the Chair.]
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