[Senate Hearing 107-127]
[From the U.S. Government Publishing Office]
S. Hrg. 107-127
STEM CELL RESEARCH
=======================================================================
HEARING
OF THE
COMMITTEE ON HEALTH, EDUCATION,
LABOR, AND PENSIONS
UNITED STATES SENATE
ONE HUNDRED SEVENTH CONGRESS
FIRST SESSION
ON
EXAMINING THE SCIENTIFIC AND ETHICAL IMPLICATIONS OF STEM CELL RESEARCH
AND ITS POTENTIAL TO IMPROVE HUMAN HEALTH
__________
SEPTEMBER 5, 2001
__________
Printed for the use of the Committee on Health, Education, Labor, and
Pensions
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COMMITTEE ON HEALTH, EDUCATION, LABOR, AND PENSIONS
EDWARD M. KENNEDY, Massachusetts, Chairman
CHRISTOPHER J. DODD, Connecticut JUDD GREGG, New Hampshire
TOM HARKIN, Iowa BILL FRIST, Tennessee
BARBARA A. MIKULSKI, Maryland MICHAEL B. ENZI, Wyoming
JAMES M. JEFFORDS (I), Vermont TIM HUTCHINSON, Arkansas
JEFF BINGAMAN, New Mexico JOHN W. WARNER, Virginia
PAUL D. WELLSTONE, Minnesota CHRISTOPHER S. BOND, Missouri
PATTY MURRAY, Washington PAT ROBERTS, Kansas
JACK REED, Rhode Island SUSAN M. COLLINS, Maine
JOHN EDWARDS, North Carolina JEFF SESSIONS, Alabama
HILLARY RODHAM CLINTON, New York MIKE DeWINE, Ohio
J. Michael Myers, Staff Director and Chief Counsel
Townsend Lange McNitt, Minority Staff Director
(ii)
C O N T E N T S
__________
STATEMENTS
Wednesday, September 5, 2001
Page
Specter, Hon. Arlen, a U.S. Senator from Pennsylvania and Hon.
James Langevin, a Representative in Congress from Rhode Island. 11
Thompson, Hon. Tommy G., Secretary, U.S. Department of Health and
Human Services, Washington, DC................................. 15
Prepared statement........................................... 21
Attachment................................................... 23
Melton, Douglas, Thomas Dudley Cabot Professor in the Natural
Sciences, and Chair, Department of Molecular and Cellular
Biology, Harvard University; Karen Hersey, Senior Counsel for
Intellectual Property, Massachusetts Institute of Technology;
James F. Childress, Edwin B. Kyle Professor of Religious
Ethics, University of Virginia; Father Kevin FitzGerald, Doctor
Lauler Professor for Catholic Health Care Ethics and Associate
Professor, Department of Oncology, Georgetown University; and
John P. Chute, M.D., Head, Hematopoietic Stem Cell Studies
Section, NIDDK, Navy Transplantation and Autoimmunity Branch,
Naval Medical Research Center.................................. 59
ADDITIONAL MATERIAL
Articles, publications, letters, etc.:
Alpha-1 Foundation........................................... 88
Don C. Reed.................................................. 89
Christopher C. Straub........................................ 90
Culture of Life Foundation................................... 92
(iii)
STEM CELL RESEARCH
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WEDNESDAY, SEPTEMBER 5, 2001
U.S. Senate,
Committee on Health, Education, Labor, and Pensions,
Washington, DC.
The committee met, pursuant to notice, at 9:40 a.m., in
room SD-106, Dirksen Senate Office Building, Senator Kennedy
(chairman of the committee) presiding.
Present: Senators Kennedy, Dodd, Harkin, Mikulski,
Jeffords, Wellstone, Murray, Reed, Edwards, Clinton, Gregg,
Frist, Hutchinson, Warner, and Collins.
Opening Statement of Senator Kennedy
The Chairman. Good morning. I would ask if our guests would
kindly be seated so that we can move along with the hearing. We
have an enormously important hearing today and some really
extraordinary witnesses, and we are here to listen and to
learn, and we are grateful to all those who have come to share
their experience and their knowledge with us in this hearing
this morning.
It is a privilege to convene this hearing on the important
issue of stem cell research. One of our greatest fears as human
beings is that 1 day, we will learn that we or a loved one have
cancer, Alzheimer's, diabetes, Parkinson's, heart disease, or
any number of dread and deadly illnesses. But every day,
thousands of Americans are stunned by such bad news. The phone
rings, the doctor is on the line, and lives are changed forever
by the awful news.
Stem cell research holds the greatest promise of hope for
millions of Americans who face these diseases. Research on
these tiny cells may mean that the next time a doctor gives the
bad news of a horrible disease, the doctor can also say that
these diseases are now curable.
So there is probably no more important work before this
Congress than to support stem cell research, to provide life
and hope to millions of Americans who would otherwise face
lives of struggle, disability, and even death.
President Bush has opened the door to Government funding
for this important area of health research, and we look forward
to hearing from Secretary Thompson about his plans and those of
the administration in moving forward on stem cell research.
The question before the Congress is whether the door is
open wide enough; whether the stem cell lines identified by the
administration are adequate and available for the research that
is needed now to save lives.
Today our committee will hear from an outstanding group of
leaders in national policy, in science and ethics, and in the
law. Their testimony will be a useful guide to the committee
and to Congress as we consider action in this important area.
We must make certain that stem cell research can fulfill
its vast potential to improve the health and relieve the
suffering of millions of Americans. President Bush has
recognized the great value of this research with his recent
decision allowing Federal funds for the work. But many in the
scientific community are concerned that the President's
decision establishes restrictive conditions on this critical
research and will delay development of cures for dread diseases
for many years, at the cost of countless lives and immeasurable
suffering.
Failure to seize this unprecedented medical opportunity
would be a tragic betrayal of the hopes and dreams of millions
of patients who expect us to do all we can to develop these new
cures.
The President has said that his policy, which limits
federally-funded research to cell lines in existence before
August 9, will make more than 60 cell lines available to
researchers and that these cells will be adequate to conduct
all needed research. But this conclusion is hardly clear.
Scientists question whether many of these stem cell lines
will actually be usable and available. The President's
limitation gives monopoly power to only 10 organizations that
will now control the supply of stem cells. Most of the existing
stem cell lines would not meet Federal guidelines for safety if
they were to be used in actual clinical work with human
patients. These lines may deteriorate and become unusable in
just a few years.
New and more effective techniques for deriving stem cells
may be developed but could not be used in federally-funded
research under the President's guidelines.
The questions about the President's policy are serious
questions, and they deserve serious answers because the lives
and health of millions of patients and their families are at
stake. It would be unacceptable to offer these patients and
families the promise of effective stem cell research but deny
them the reality of it.
Our committee today begins its oversight of stem cell
research as it evaluates the need for legislation to assure
vigorous, ethical stem cell research. I hope that our hearing
will contribute to the understanding of these important
scientific and ethical issues, and I look forward to the
testimony of our witnesses and the comments of our colleagues.
Senator Gregg?
[The prepared statement of Senator Kennedy follows:]
Prepared Statement of Senator Kennedy
It's a privilege to convene this hearing on the important
issue of stem cell research. Today, our committee will hear
from an outstanding group of leaders in national policy, in
science, ethics and the law. Their testimony will be a useful
guide to the committee and to Congress as we consider action in
this important area.
In recent years, remarkable progress has taken place in
medical science. Advances in basic understanding of biology and
the application of that knowledge to clinical practice have
saved the lives and preserved the health of countless
Americans. Many conditions that once caused disability or death
can now be treated and even cured. Yet despite this continuing
progress, the lives of far too many Americans are darkened by
illness.
Parkinson's Disease robs thousands of senior citizens of
the power to control their own movements. Alzheimer's Disease
impairs the mental abilities of far too many older Americans.
Diabetes condemns children to a lifetime of insulin injections
and the ever-present risk of coma or death. Spinal cord
injuries confine thousands of our fellow citizens to
wheelchairs. Cancer and heart disease exact an extraordinary
toll of premature death on millions of Americans and their
loved ones.
But for each of these disorders--and for many other serious
illnesses--there is the promise of help and hope from a
revolutionary new medical development. Stem cells may 1 day
liberate children with diabetes from their dependence on
insulin injections--restore the damaged brains of patients with
Parkinson's Disease or Alzheimer's Disease, allow the paralyzed
to leave their wheelchairs, regenerate hearts damaged by heart
attack or coronary artery disease, and provide new cancer
treatments.
This week, we have seen yet another example of the
remarkable power of stem cells. Scientists at the University of
Wisconsin announced that they have been able to turn stem cells
into blood cells.
We must make certain that stem cell research can fulfill
its vast potential to improve the health and relieve the
suffering of millions of Americans.
President Bush has recognized the great value of this
research with his recent decision allowing Federal funding for
this important work. But many in the scientific community are
concerned that the President's decision establishes restrictive
conditions on this critical research and will delay development
of cures for dread diseases for many years--at the cost of
countless lives and immeasurable suffering. Failing to seize
this unprecedented medical opportunity would be a tragic
betrayal of the hopes and dreams of the millions of patients
who expect us to do all we can to develop these new cures.
The President has said that his policy, which limits
federally funded research to cell lines in existence before
August 9th, will make more than 60 cell lines available to
researchers and that these cells will be adequate to conduct
all needed research. But this conclusion is hardly clear.
Far fewer than 60 stem cell lines may actually be
available. The National Institutes of Health combed the globe
to find stem cell lines in far-flung laboratories--but it now
appears that many of these cell lines are untested, unproven
and may have uncertain medical value. Even many of the
researchers who developed these lines have said that many of
them have not been proven to actually be stem cell lines.
Of those that are truly stem cell lines, virtually
none have undergone the rigorous safety testing needed to
assure that they will be safe to transplant into patients. Most
of the stem cell lines appear to have been mixed with cells
from laboratory mice. Yet, just 2 weeks ago, the Department of
HHS issued strict new safety guidelines for clinical use of
human cells that had been mixed with animal cells. There is
little evidence that existing cell stocks would meet these
stringent criteria.
Even cell lines that are safe and usable may not
be available, because patent claims and legal restrictions may
impede access to them. I know that Secretary Thompson has been
working to resolve these difficulties, but many legal experts
are skeptical that researchers will have sufficient access to
viable stem cell lines.
Researchers are also concerned that stem cell
lines usable today may deteriorate and become unusable for
research in a year or two. Under the President's policy, that
could mean no stem cell lines at all would be available in the
future for federally funded research.
Stem cell research is in its earliest stages. We cannot
know what remarkable breakthroughs will be made in the years to
come. In particular, new ways of establishing cell lines may be
developed that can unlock the medical potential of these cells
far more effectively. Yet under the President's plan, NIH-
funded doctors would have no access to cells established with
breakthrough techniques discovered after August 9th. Who knows
what price we may pay by freezing medical progress as of this
particular date? Imagine if we had imposed a similar
restriction on the use of fetal tissue in 1954--a year before
Jonas Salk announced that he had used fetal tissue in
developing the polio vaccine that saved countless lives.
These are serious questions, and they deserve serious
answers, because the life and health of millions of patients
and their families are at stake. It would be unacceptable to
offer these patients and families the promise of effective stem
cell research, but deny them the reality of it.
Our committee today begins its oversight of stem cell
research as it evaluates the need for legislation to assure
vigorous, ethical stem cell research. I hope our hearing will
contribute to the understanding of these important scientific
and ethical issues, and I look forward to the testimony of our
witnesses and the comments of our colleagues.
Opening Statement of Senator Gregg
Senator Gregg. Thank you, Senator Kennedy. I thank you for
holding this important hearing, which goes to a subject which
has captured the Nation's attention because the potential for
it is so immense. The potential is to address diseases which
have plagued many citizens of our Nation and the world for
years, for generations, and which now have a potential source
of relief.
Clearly, stem cell research creates the opportunity to
provide treatments for thousands of Americans suffering from a
variety of diseases including cancer, diabetes, Alzheimer's,
Parkinson's, leukemia, spinal cord injuries, and various other
areas of care.
The therapeutic advances that have already been made by
nearly 50 years of research on adult stem cells is very
encouraging and deserves our continued and enthusiastic
support, also. These therapies, unlike the unproven and
untested potential application of embryonic stem cell
therapies, which are at a minimum 5 and potentially 10 years
away from clinical application, are actually being used today
and are being used very successfully, and we should not forget
that.
We will hear today from Dr. Chute, who will talk about some
of the applications of adult stem cell research and the
therapies which have derived from adult stem cells. So as we
embark on the road of addressing the issue of embryonic stem
cell use, we certainly should not ignore the use of adult stem
cells and should make that a priority of the focus of this
committee also.
Clearly, embryonic stem cells and the policies which the
President have outlined, however, have become the issue which
we are addressing as the priority in this hearing. I certainly
congratulate the President for bringing forward this issue in
the manner in which he has. He has presented a thoughtful and I
think comprehensive approach to how we should proceed.
The questions before this committee which we are to address
are: 1) how is the President's policy going to work, and how is
it going to be applied; and 2) we need to examine how we as a
Federal Government should support the further research in the
area of stem cells.
Senator Kennedy has noted that the President has identified
64 lines of potential stem cells which have been derived from
embryonic cells. The issue, of course, is whether or not that
is an adequate number and whether that number correctly
addresses the issues which are involved, the questions of
principles of life.
This is not an easy issue for us to address as a political
body, but it is clearly one which we must address. I believe
the President has made a comprehensive and aggressive attempt
to try to bring this issue forward in a way which will allow
science to pursue the opportunities that are there. It is my
belief that we should not act precipitously to expand or to set
out on another course, but rather, see and determine what the
effects of the President's initial proposal are. We do not yet
really know whether the 64 lines which have been identified are
going to be adequate and whether the science which will be
developed from those lines will effectively address the needs
of people who are suffering from diseases which may be relieved
as a result of the use of those stem cells. We do not even know
whether those stem cells are going to be available for
application to humans because of the manner in which they have
been derived and been maintained.
These substantive questions have to be addressed as a
threshold issue, I believe, before we start second-guessing the
issue of whether the 64 lines is the appropriate number.
So there is a lot for us to talk about, a lot for us to
look at. I believe this hearing is really an entry-level
discussion of the topic, but hopefully, the results of the
hearing will be that we will set ourselves on a path of
reaching conclusions which will move forward the great
opportunities that lie with this new area of stem cell therapy.
Thank you, Mr. Chairman.
The Chairman. Thank you very much.
Senator Harkin, we welcome your comments.
Opening Statement of Senator Harkin
Senator Harkin. Thank you, Mr. Chairman, and I thank
Senator Dodd for yielding his time.
I will not take long, and I will ask that my statement be
made a part of the record as well.
The Chairman. It will be made apart of the record. We know
that you and Senator Specter have conducted extensive hearings
in your committee, so we are looking forward to your comments
and to working with you.
Senator Harkin. Thank you, Mr. Chairman.
I applaud you for having this hearing; it is very timely.
It is something that, as both you and Senator Gregg have said,
has captured the public's attention and imagination all over
this country.
I think we have to pay very strong attention to whether we
are opening the door, as you said, far enough to really be able
to move aggressively in this research area. Certainly we are
all concerned about the ethical implications of this new
science, but we have wrestled with this over the last few
years. We had an Ethics Committee at the NIH that came up with
I thought very strong ethical guidelines that are now in
existence at this point in time, but if there are others who
have other views on this, we certainly welcome those also.
I have become more and more convinced that we have to move
forward on this basic research. Every week, new findings come
out. Just yesterday, we saw the potential of using stem cells
to make blood cells at the University of Wisconsin, and the
potential that that alone holds for our country.
So I think the concern that we have is whether or not,
within the strictures of ethical guidelines, we can conduct
good, sound science and use the massive resources of what my
colleague and friend, Senator Specter, has often called the
crown jewel of our Federal Government, that is, the NIH; can we
use the power and the structure of the NIH and all the money we
provide to the NIH to involve researchers all over this country
to move this science forward as rapidly as possible, as I said
again within ethical guidelines.
That is really what I think we are about, and those are the
questions that we have to answer. I think the essential
question is whether there are 64 cell lines, whether they are
viable, and whether they can actually lead to therapies in the
future. I think these are the essential questions that we have
to wrestle with.
I thank you, Mr. Chairman, for holding this hearing, and I
thank Senator Dodd for yielding me the time.
[The prepared statement of Senator Harkin follows:]
Prepared Statement of Senator Harkin
Thank you, Mr. Chairman. As you know, I've spent a great
deal of time over the past two-and-a-half years thinking about
stem cells. On the Labor, HHS, and Education Appropriations
Subcommittee, Senator Specter and I have held nine hearings on
this matter since December 1998, and we'll hold two more later
this month.
We've heard from scientists like Dr. James Thomson, the
first researcher to isolate stem cells from early human
embryos. We've heard from ethicists and religious leaders.
We've heard from several of our Senate colleagues, who have
offered their thoughtful perspectives on both sides of this
issue. And we've heard from victims of the many devastating
diseases that could be cured as a result of stem cell
research--diseases like Parkinson's, diabetes, ALS, and
Alzheimer's.
After each one of those hearings, I've become more and more
convinced that we need to move forward on this research. Every
week, it seems, brings new findings about the potential of stem
cells--just yesterday, in fact, scientists at the University of
Wisconsin reported that they have figured out how to coax these
cells into making blood.
We need to power that research with Federal dollars, and we
need to do it quickly, ethically, and without unnecessary red
tape. That's why I welcomed President Bush's announcement last
month in support of Federal funding for research on existing
stem cell lines as a positive first step.
Since then, scientists have raised a number of concerns
about the President's plan. They question how many of the 64
stem cell lines identified by the NIH will actually prove
useful for research. And there's a possibility that all of
these lines have been mixed with mouse cells--a situation that
could make it dangerous to test them on humans.
I share these concerns, but that's why I'm here today, and
that's why my Appropriations Subcommittee will hold two more
hearings later this month--to get some answers.
Mr. Chairman, you've assembled an excellent panel of
witnesses for us this morning, and I look forward to the
testimony.
The Chairman. Thank you very much, Senator Harkin.
Senator Frist?
Opening Statement of Senator Frist
Senator Frist. Thank you, Mr. Chairman and Senator Gregg,
thank you for jumping right in with this hearing so that we can
more closely and more clearly examine the important issues of
embryonic stem cell research.
On July 18, a little over a month ago, I announced my
strong support for Federal funding of both embryonic and adult
stem cell research. And, as we all know and have heard again
and again through the press and through the various hearings
that have been held, embryonic stem cell research holds great
potential for advancing treatments for a broad range of
diseases, illnesses, injuries, and conditions.
Yet I think we need to be very, very careful at this
juncture, early in our discussions--and yes indeed, it is early
in the evolution of this relatively new science--not to
oversell the promise of this research to the American people.
We must recognize that the field of embryonic stem cell
research is young, it is early, it is pioneering; it is not yet
tested. The benefits of this research, although we all attach
huge hope to this particular field, have not yet been realized,
and they are just possibilities.
I think we also need to recognize that there are millions
of Americans, including myself, who hold very deeply-felt moral
and religious concerns about research using stem cells derived
from embryos.
The whole topic is important for us to address in a
straightforward way. It is important because we as a Government
have not yet come to grips with what is the appropriate ethical
and moral construct, how much oversight, what should the
guidelines be, as we enter into a field, maybe for the first
time--the one exception might be genetics--but for the first
time enter into a field that will so profoundly affect the
course of human life and disease by manipulation by human
beings and altering those basic, fundamental building blocks of
life, what makes life, what is living, and indeed what makes us
human. We just have not had to address that in the past in much
of science or medical science. This is the first time.
We have the possibility of producing powerful treatments--
we talk about cures and treatments--as we address this in a
stepwise fashion. However, there is also the possibility of
unintended outcomes, of outcomes that are unanticipated--and
yes, you could even throw harm or potential harm into those
unintended outcomes.
We are just beginning to understand the capacities of this
science, the potential for this science, and therefore it is
critical that we as public servants respond in a way that
treats this pioneering research with awe and moral respect and
great care.
The one thing we have realized over the last several weeks
is how little we know about the State of the art today.
Although people can be critical of the way public policy is
being developed, we know a lot more today than we did 4 weeks
ago and than we did 8 weeks ago. The press has participated
through the hearings that have been held previously and through
this hearing today.
All of this has made clear to me the lack of knowledge that
we have as we address this issue of stem cells. Therefore, I
think that, for the welfare of mankind, there is a moral
imperative that we proceed with embryonic stem cell research
but that we do so with caution and that we do so with
restraint, remembering that it is untested and untried.
I have argued in the past that we need to proceed within a
fully transparent, carefully regulated framework that respects
the potential of this science but, at the same time, respects
the moral significance of the human embryo. Earlier this year,
I set out 10 principles which I felt establish this larger
ethical framework of oversight and regulation.
I am very excited about the hearing today, the hearings
that are planned among the various committees of the U.S.
Senate and the United States Congress as we address these
issues. I am 100 percent in support of what the President has
put forward. For the first time--for the first time under
President Bush's carefully balanced policy, NIH and NIH-funded
scientists will be able to access embryonic stem cell lines
that do hold the potential that we all understand is there.
These issues are difficult because they involve life, human
life, the intersection of science and religion that we simply
have not had to address to this degree in the past. I believe
the President of the United States has put forward a balanced
approach that will allow stem cell research to begin
immediately, right now, quickly, but to do so in a very careful
way.
We will also talk a little bit in the hearing about the
stem cell registry, which I am delighted that the President has
put forward, as well as the Bioethics Advisory Commission,
which I think is critical as we go forward.
Mr. Chairman and Senator Gregg, thank you for calling this
hearing as we address these important issues.
The Chairman. Thank you very much.
If other members wish to include a statement in the record,
they will be so included.
[The prepared statement of Senator Jeffords follows:]
Prepared Statement of Senator Jeffords
Mr. Chairman, thank you for holding this hearing on an
extremely significant issue that will have a profound impact on
all of us. I want to especially applaud the effort of our
colleagues, Senator Specter and Senator Harkin for their
leadership on this issue. The hearings they have held in the
Appropriations Committee have been instrumental in focusing
attention on the promise and hope of stem cell research.
I also want to thank all of our witnesses and especially
Secretary Thompson who, based on his experience as Governor in
Wisconsin, has been able to provide so much guidance to the
Administration.
President Bush's proposal focuses on allowing Federal
funding for research on a small subset of the stem cells
derived from very early stage embryos. Although it is a good
first step, we need to explore other options. We need to make
sure that his proposal is strong enough to propel the research
and not impede it.
It is crucial that we allow our scientists to move forward
with stem cell research because it holds the promise of
providing answers for a host of diseases from Alzheimer's to
heart disease.
Because the possibilities to eradicate so many diseases and
disabilities are endless, the promise has raised hope for so
many Americans.
Christopher Reeves, has encouraged all of us with his grit
and determination to overcome the disability he suffers because
of neurological damage. He knows all too well what stem cell
research can do for him and thousands of others living with
disabilities.
Another brave American, with whom we are all familiar, is
Michael J. Fox. By publicly facing his Parkinson's disease and
becoming a spokesperson for stem cell research, he too has
pointed to the profound promise of this research. These men and
many other Americans have testified in front of Senator Specter
and Harkin and in numerous other venues about the potential of
stem cell research.
Science has brought us to a fork in the road and I have
confidence that we will take the right fork. We can approach
this road morally and ethically and still continue to walk down
a path that will save lives and minimize suffering.
I want to thank our other witnesses as well. These are
profound issues that require the best advice available so I'm
looking forward to their statements. Thank you again for
holding this hearing Mr. Chairman.
The Chairman. We will hear now from two Members of
Congress, first from Senator Specter, who with Senator Harkin
has been conducting very extensive hearings in the
Appropriations Committee on the NIH and has taken a very
serious interest in this issue. We look forward to hearing from
him, and after that, a good friend from my neighboring State of
Rhode Island, Congressman Langevin, someone who has thought
about this over a very considerable period of time and is one
of the new very bright lights in the Congress of the United
States who can help us all understand this issue better as
well. We look forward to hearing from him after Senator
Specter.
Before we begin, I have a statement from Senator Collins.
Prepared Statement of Senator Collins
Mr. Chairman, thank you for calling this important hearing
on the President's plan for providing federal funding for
embryonic stem cell research. I am a strong supporter of stem
cell research and want to commend President Bush and his
Administration for not only allowing this potentially life-
saving research to move forward, but also for moving so quickly
and aggressively to implement the plan.
Stem cell research holds tremendous potential to treat and
even cure a vast array of devastating diseases and conditions,
ranging from Alzheimer's disease, to Parkinson's disease, to
ALS, spinal cord injury and cancer. This research has
tremendous promise for millions of American families, and I
applaud the President for taking such a positive step forward.
As the founder and co-Chair of the Senate Diabetes Caucus,
I am particularly excited about the promise that stem cell
research holds for a cure for juvenile diabetes, which has had
such a devastating impact on millions of American children and
their families. Early research has shown that stem cells have
the potential to develop into insulin-producing cells to
replace those that have been destroyed in people with Type 1
diabetes. One of the major limitations to success in this
research is the limited number of insulin-producing cells for
transplantation--an obstacle which could be overcome through
embryonic stem cell research.
I do have some question, however, about the restrictions
that the President has imposed in limiting this research to
those stem cell lines that are currently in existence. As we
will hear this morning, some researchers have expressed concern
that there will not be enough stem cells available to support
the hundreds and possibly thousands of research teams that
stand ready to investigate them, which could potentially delay
progress in bringing stem cell therapies into medical practice.
Others have questioned whether the 64 stem cell lines that have
been identified are all viable and of good quality and whether
they would all be available to researchers.
Mr. Chairman, stem cell research offers tremendous hope to
those suffering or dying from devastating illnesses. This
morning's hearing provides us with an excellent opportunity to
examine these questions and issues further, and I look forward
to the upcoming testimony.
The Chairman. Senator Specter?
STATEMENTS OF HON. ARLEN SPECTER, A U.S. SENATOR FROM
PENNSYLVANIA AND HON. JAMES LANGEVIN, A REPRESENTATIVE IN
CONGRESS FROM RHODE ISLAND
Senator Specter. Thank you very much, Mr. Chairman and
members of this distinguished committee, for an opportunity to
present some of the findings which the Appropriations
Subcommittee on Labor, Health, Human Services, and Education
has noted during the course of some nine hearings which began 2
weeks after stem cells were first broached on the American
scene in November of 1998.
The President made a profound statement on August 9 and has
made an important opening of the door; but there is a real
question as to whether the door is open sufficiently, and there
is a real question about the accuracy of the facts which were
presented to the President by the Department of Health and
Human Services.
A key statement by the President related to 60 stem cell
lines now expanded to 64, but in the intervening several weeks,
it has become apparent that many of the lines cited are not
really viable or robust or usable. For example, Gothenburg
University in Sweden was reputed to have 19 lines, and they
have at most 3. In India, the researchers were supposed to have
7 lines; none is ready for research. The San Diego Consortium
was reputed to have 9 lines; again, none is ripe for
utilization.
It is up to the congressional hearings to make a detailed
examination as to the accuracy of the representations by HHS of
robust, viable, and diverse lines.
Then, there are the intricate questions of informed
consent. And then, perhaps most fundamentally is the issue of
therapy. It was not addressed in the President's statement, but
it has come to light in the intervening weeks that all of the
stem cell lines have had nutrients from mice and have had
bovine serum. Under the FDA regulations, there cannot be a
mixing of the species.
Now, it is a complex matter, and there have been some
exceptions, but I think it is going to be up to Congress in
these hearings to make the determination as to what the facts
are.
I think it is very important to focus on the need for an
independent review of all of these facts. The information given
to the President has not been complete. HHS has insisted, for
example, that there are 19 lines from Sweden when Dr. Lars
Hamburger has said that he personally advised the Secretary to
the contrary. So these are facts which we must determine for
ourselves.
I agree totally with what Senator Gregg has said, that we
should not be precipitous. The issue as to the hope has been
documented in a detailed manner by responses to letters which
Senator Harkin and I sent to all the directors of the 25
institutes of the National Institutes of Health, who wrote back
on the enormous potential for stem cell research in so many
lines. And I regret but think it is necessary to inform this
committee that when those letters were transmitted to the
subcommittee, we found that they had been censured, and that
many of the very, very positive statements which had been made
by the directors of the institutes, illustrative of which is
Dr. James Bailey, omitted from the letters we got, that ``it
would be unfortunate if the ban on the NIH support for human
stem cell research results in a missed opportunity to restore
hope and quality.'' Similar statements were made by Dr.
Klausner as to cancer and many of the other institute
directors.
When the issue of research was omitted, that is something
which I believe has to proceed apace at the present time. There
is some conversation about let us do the basic research if the
lines are sufficient. The hearings in our subcommittee
suggested that you need 200 lines. And bear in mind that the
Congress has been enormously generous with NIH. That
appropriations process started again with our subcommittee, and
so far, we have added more than $8 billion, and by the end of
this year, on our anticipated doubling, we will have added $12
billion to NIH funding. So there is ample funding to proceed.
And let there be no mistake--we believe that there should
be funding on adult stem cell lines, on cords, on placenta, on
every line, so that science should have the full range of
opportunity.
Before the President's announcement, there was considerable
sentiment in the Congress and a considerable head of steam that
the existing prohibition on use of NIH funds on stem cells had
to be changed. Some 64 Senators signed a variety of letters,
saying they favored stem cell research, and more than a dozen
others made commitments to stem cell research but did not want
to put it in writing. It is up to the Congress to take a look
at these hard facts, and I suggest with a sense of urgency.
Karl Rove has been quoted as saying, ``The President
equates the enormity, gravity, and magnitude of this decision
to an issue of war and peace and whether to commit American
troops.'' That is obviously a pretty strong statement.
My own statement has been that I believe that this issue of
stem cell research, with its potential to touch virtually every
family in America, all of whom are afflicted with either
Parkinson's or Alzheimer's or heart disease or cancer, that
there is no more important issue facing Congress except the
issue of weapons of mass destruction.
I have a friend and constituent in Pittsburgh named Jim
Kordi who suffers from Parkinson's. Whenever I see Jim Kordi,
he carries an hourglass, to remind me that the sands of time
are passing and that the days of his life are slipping away.
That is a pretty emphatic message from the hourglass.
So it seems to me that this is the kind of sense of urgency
which ought to motivate this very distinguished committee and
what we will be doing on two hearings later this month in our
Appropriations subcommittee.
Thank you, Mr. Chairman.
The Chairman. Thank you very much, Senator Specter.
Congressman Langevin?
Mr. Langevin. Thank you, Mr. Chairman, members of the
committee. I would like to thank you, Chairman Kennedy, and
Senator Gregg, and of course, my senior Senator from Rhode
Island, Senator Reed, and the entire HELP Committee for
convening today's hearing on stem cell research.
I am honored to be joining Senator Specter and Secretary
Thompson, who will be testifying later, as well as several
eminent cellular biologists, shedding light on the
ramifications of President Bush's August 9 decision.
Ladies and gentlemen, the issue that we face today is not
whether to move forward with embryonic stem cell research but
how. How do we ensure that all unnecessary barriers to the
research and development of life-saving cures are removed? How
do we establish parameters that provide ethical oversight of
this most delicate issue? And how do we help as many people as
possible as expediently as possible?
Unfortunately, today these questions are being answered in
the context of a policy that impedes the potential of this
Nation's leading scientists.
As many of you know, on November 7 last year, I became the
first quadriplegic elected to the United States Congress. While
my physical condition does not define me, it does affect me on
a daily basis, providing me with a unique perspective on stem
cell research.
At the age of 16, I spent my summer vacation participating
in the Warwick Police Cadet Explorer Program. I had dreamed of
becoming a police officer or an FBI agent for most of my young
life. But on August 22, 1980, my dream was shattered. I stood
in a locker room with a fellow cadet, watching two members of
the SWAT team examining a new weapon which they thought was
unloaded. That weapon accidentally discharged, launching a
bullet that ricocheted off a metal locker and entered my neck,
severing my spinal cord and leaving me paralyzed for life--
perhaps until now.
While embryonic stem cell research could give me the chance
to walk again, please understand that I am here today not just
for myself or others with spinal cord injuries but also to help
alleviate the pain and suffering of millions of people whose
lives could be saved, lengthened and dramatically improved by
this research.
Nearly half of all Americans could benefit from embryonic
stem cell research, including the one million children with
juvenile diabetes, the 8.2 million people with cancer, the 60
million people who are struggling with heart disease, the 4
million Alzheimer's sufferers, the 10 million people fighting
osteoporosis, the 43 million arthritis suffers, the quarter of
a million people with spinal cord industries, and the 30,000
people suffering with Lou Gehrig's disease.
Every family in America, ladies and gentlemen, has been
touched by these diseases and conditions, and now we have the
opportunity to offer them real hope.
That is why I support using stem cells derived from excess
frozen embryos that would otherwise be discarded, which would
allow us to save, extend, and improve lives. Every year,
hundreds of thousands of couples experience the joy of
childbirth through in vitro fertilization, a process which
unfortunately creates more embryos than can be used. To
relegate these potentially life-saving cells to the trash heap
after the arbitrary deadline of August 9 is simply wrong.
While I applaud the door President Bush has opened with the
new embryonic stem cell policy, I am frustrated with the
discovery of just how little room it leaves for medical
advancement. Despite NIH's recent disclosure of the 64 cell
lines that existed before August 9, we are now learning that
they are not all ``robust'' as once claimed, and some of these
cells are still in development and cannot yet be classified as
lines.
Questions about the safety of using the cells in human
trials are also surfacing because many researchers have mixed
human cells lines with mouse cells, which poses the risk of
infecting people with animal viruses.
Finally, we must recognize that irrespective of the
President's guidelines for the existing embryonic stem cell
lines, the private sector in the United States as well as the
public and private sectors abroad will continue to conduct
research on stem cells that fall outside the parameters
established by the Bush Administration.
I would like to conclude with this. What will we do when an
embryonic stem cell derived from the in vitro fertilization
process after August 9 leads to a cure for heart disease, the
number one cause of death in this country? Will we deny 60
million Americans this life-saving cure? And worse, what if
such a cure is found through the morally offensive procedure of
creating embryos purely for harvesting stem cells?
We must fund research on other cell lines besides the 64
cell lines identified by NIH, and we must provide strong
oversight of this research to ensure that it is conducted by
ethical means that do not force us to wrestle with similar
moral quandaries in the future.
The administration's policy impedes unprecedented life-
saving research and raises critical ethical dilemmas that we
must not ignore. Because embryonic stem cell research cannot
deliver on its promise of therapeutic benefit for millions of
people under this policy, I am forced and compelled to oppose
it.
I understand the struggle very well to balance a pro-life
position with embryonic stem cell research. This is perhaps one
of the most difficult decisions that I have ever had to make.
Having come so close to losing my own life, I am reminded every
day of how precious the gift of life truly is. That is why I am
pro-life.
However, nothing is more life-affirming than using what
otherwise would be disposed of to save, extend, and improve
countless lives. I urge my colleagues to open the door to
research on all excess embryonic stem cells derived in the in
vitro fertilization process and to do so with Government
oversight that ensures ethical research procedures.
Mr. Chairman, I believe that as a determined Nation, we
have an obligation to get behind this research and to see it
move forward and offer the hope of easing so much pain and
suffering for so many million Americans.
Thank you, Mr. Chairman.
The Chairman. Thank you, Congressman, for a very moving
presentation and statement, one that obviously includes a great
deal of thought and examination on this issue from a personal
perspective.
And I thank Senator Specter, who has given important
leadership on this issue.
I think that unless there is a different opinion, I will
excuse our two guests, thank them for their excellent
statements, and ask that any questions to them be submitted in
writing.
Senator Reed. Mr. Chairman, can I simply say how proud we
are in Rhode Island of our Congressman and how distinguished
and effective he is with his principled discussion of these
issues.
The Chairman. You certainly may.
Senator Reed. He was an extraordinary public leader in our
State in terms of his management as the secretary of State
before he came here. We are all very proud of Jim and thank him
for his wonderful statement.
Thank you, Mr. Chairman.
The Chairman. Thank you.
Thank you both very, very much.
It is a pleasure to welcome Secretary Tommy Thompson to
speak to our committee on stem cell research. His sense of
timing is impeccable.
The Secretary has a longstanding interest in this important
issue since much of the groundbreaking research in this area
was done at the University of Wisconsin. We look forward to
hearing from him on how the Department of Health and Human
Services plans to implement the President's proposal for
funding stem cell research.
I can say just personally that the Secretary spent a great
deal of time thinking about this issue. I know that he has
spoken with most of the members of our committee, and I know as
well that he spent a good deal of time during the President's
consideration attending many of the briefings and expressing
his views on this matter.
We know you have given a great deal of thought to this, Mr.
Secretary, as you do to other issues. We welcome you to the
committee and look forward to your testimony.
STATEMENT OF HON. TOMMY G. THOMPSON, SECRETARY, U.S. DEPARTMENT
OF HEALTH AND HUMAN SERVICES, WASHINGTON, DC.
Secretary Thompson. Good morning, Mr. Chairman, Senator
Kennedy, Senator Gregg, and all the other members.
Let me start by thanking you for holding the hearing, and
thank you all for being so interested in a subject that is
very, very important to the American citizens and to the world.
I certainly appreciate this opportunity to appear before this
committee to talk about a subject that I am very personally
deeply involved in and have been ever since Jamie Thomson
discovered the embryonic stem cell procedure at the University
of Madison 3 years ago, in 1998.
I am accompanied by Lana Skirboll, director of the Office
of Science Policy at the NIH; Mark Rohrbaugh, deputy director
of the Office of Technology Transfer; Maria Ferrar, director of
the Office of Technology Transfer--and this happens to be her
last day as a Federal employee, and she is here to support this
procedure; and also, Kathy Zoon, director of the Center for
Biologics Evaluation and Research at the FDA.
Let me begin by thanking all of you for your tremendous
support of the Department of Health and Human Services and more
specifically NIH. I want to thank you, Chairman Kennedy and
Senator Gregg, for your support of research, all of you who
have worked so hard to assist us in getting the dollars
necessary to do the medical research at NIH, and for your
support for the great potential of stem cell research, both
adult and embryonic.
I have submitted my written testimony for the record, Mr.
Chairman, but there are some additional points that I would
like take the opportunity to make this morning.
All of us stand today at the precipice of a new era where
science holds the promise of curing the most devastating
diseases. And most important, President Bush is ushering
America into this new era by opening the door to Federal
funding of human embryonic stem cell research in an ethical and
solemn manner.
The President came to a thoughtful and deliberate decision
that the administration will support policies that preserve and
promote the sanctity of life while allowing important medical
research to proceed.
There is nothing easy about human embryonic stem cell
research. It is a complex issue with great ethical and moral
implications. It is an issue that speaks not only to our
greatest hopes and passions as a society but also to who we are
as a society. The moral considerations cannot and must not be
lost in this debate.
President Bush sent us on a wise and deliberate course with
his decision to allow Federal funding of research on existing
embryonic stem cell lines. His decision balanced our Nation's
deepest respect for life with our highest hopes for alleviating
human suffering.
These existing stem cell lines no longer hold the potential
for life, but they do hold the potential to save life. It is
that potential to save life that we must tap and bring to
fruition, Mr. Chairman and members.
Our challenge now is to move beyond the halls of debate
into the laboratories of science where we can do the basic
research that will 1 day lead us to the therapies and the
treatments for the most horrible maladies that plague humanity.
This is an emotional debate. It is a debate that spawns a great
deal of speculation and feeds many misunderstandings. My hope
is that we can clear up the misunderstandings and recognize
that the only way that we are going to resolve the speculation
is to do the research and to find the answers.
The first thing we must all understand is the underlying
need to conduct the basic research into embryonic stem cells. I
cannot stress this point enough. We need to create a
fundamental base of knowledge about how these cells function
and how they can be manipulated, as well as get the answers to
many other scientific questions.
Some people want to make the grand leap from the onset of
federally-funded research to the cures for Parkinson's,
Alzheimer's, and other diseases. If only it were that easy. It
is easy to make such a leap in the emotion of this debate, but
it is also inaccurate and unfair to do so.
The cures for these diseases are not just around the
corner--I wish they were. Before we can even get to the stage
of credibly talking about therapies for diseases, we must
complete the basic research. This will take years, possibly 3,
5, and maybe even 8. No one knows for sure. And Senator Frist
probably knows more than any of us about the time it took for
transplants in heart surgery and how long it took to perfect.
But we now have the ability to do the basic research with
the stimulus of Federal dollars. The role of the Federal
Government should be and will be to make sure that that basic
research takes place. With the support of Congress, this is
where our investments will be going. And as we are investing in
the basic research of embryonic stem cells, we will continue to
fund research into the other types of stem cells, including
adult, cord blood, and placenta. There is great value in all
the research, and we have so much yet to learn about how much
each can contribute to treating these diseases.
The private sector is going to continue to pursue stem cell
research as well. And the logic of the American free enterprise
system suggests that President Bush's decision is going to
provide the incentive for the private sector to get more
involved. Once the basic research is completed, the private
sector will likely have great incentive to step in and
transform the basic research into therapies for disease.
It is in that context of basic research that we must then
address the underlying question of whether we have enough
embryonic stem cell lines to meet the eligibility criteria. We
believe the answer is yes. Let me explain by first addressing
how we arrived at this number and then why we believe the
number is sufficient.
So far, the National Institutes of Health has identified 64
stem cell derivations that meet the President's eligibility
criteria. The President never spoke about or drew any limits on
these lines based on where they were in their development.
Furthermore, we have consistently said that these lines are at
various stages of development. I have spoken to that fact; the
NIH has spoken to that fact, and the NIH white paper
identifying these derivations makes that fact crystal clear.
But unfortunately, and I believe unfairly, some are
choosing to engage in word games or hear only parts of the
story. What is most unfair is that some are trying to create
conflict between myself, the NIH, and the Swedish scientists
from the University of Gothenburg over the number of lines they
have that qualify for Federal funding. They have 19 lines that
qualify for Federal funding. That is what we said; that is what
they have.
Let me be perfectly clear that there is no misunderstanding
and no conflict between us. We are on the same page. In fact, I
talked to Dr. Hamburger yesterday at 5 o'clock in the
afternoon.
I would like to point out to the committee that this is the
blastocyst, and this is at 5 to 8 days. The intercell mass
takes 3 days, depending upon what procedure you use to take
from the blastocyst to develop the cells. They are there in the
proliferation stage in the petri dish. This is the
characterization, and this is what you have to do to
characterize if you are going to have a viable embryonic stem
cell line.
This is the cell line established. This period takes 6 to 8
months from here, after it is removed, until it is perfected
and before they start freezing the cells and are able to use
them in the vials for further research.
The scientists in Sweden have three cell lines at this
stage that are completed. They have four lines in the
characterization process right now, and they have 12 in the
proliferation stage. That is 19. All 19 of those lines qualify
under the President's remarks for funding. That means that they
have been taken from the blastocyst and are able, before August
9 at 9 p.m., to qualify for Federal funding. That is the 19,
and all 64 meet those criteria. All 64 are at different stages
of development.
The Gothenburg scientists have 19 derivations that meet the
eligibility criteria, and we have always acknowledged that most
of these are in the earliest stages of development. We agree
with their scientists that they only have three fully-developed
lines, but they also agree with us that they have 19 in various
stages that meet the eligibility criteria for funding. In fact,
the scientists from Gothenburg had inquired if 50 other
blastocysts that they own would qualify for Federal funding as
well; they did not, because the embryo had not yet been
destroyed.
I spoke yesterday with Dr. Lars Hamburger to once again
make sure we have the same understanding, and there is
absolutely no disagreement or misunderstanding between us.
Now let me explain why we believe that the stem cell
derivations that we have identified are adequate and ample for
basic research even though some are at various stages of
development.
First, we will not be taking applications for funding until
after October 1. To go through the procedure at NIH will
probably take 8 to 9 months to get the dollars out unless there
is an amendment to an existing grant, which could be sooner.
During that 8 to 9 months, a lot of those 12 lines from
Gothenburg will have been fully characterized and fully
developed, making those available but at different stages.
I began by putting into perspective how much work can be
done with the use of a small number of lines--and keep in mind
that embryonic stem cells reproduce, and to the best of our
knowledge right now, they do so endlessly.
For the pasta two decades, there has been research done on
embryonic stem cells from mice. Ninety percent--20 years of
research on mice--90 percent of that research has taken place
with just five mice lines.
But a more impressive example is the work being done at the
University of Wisconsin in Madison. UW scientist James Thomson
was the first to isolate human embryonic stem cells and I
believe probably has more experience and knowledge working with
them than nay scientist in the world. He has done nearly all of
his research using just two of his five stem cell lines.
The Wisconsin Alumni Research Foundation which owns the
five stem cell lines and licenses them through WiCell says it
has enough to supply every researcher with a Federal grant.
Those are not my words; that is WiCell, that is WARF, and that
is what Jamie Thomson has indicated to them. So that is one
owner with just five lines who indicates that they can feed all
the scientists in the world at the present time wishing to
engage in this research with Federal funds. That is a powerful
statement, yet one that many are choosing to ignore.
What cannot be ignored is the remarkable amount of work
already being done on these few lines. As you know, the
University of Wisconsin Medical School reported Monday that its
researchers under the direction of Jamie Thomson have turned
human embryonic stem cells into blood cells called
hematopoietic blood cells. They did so using the WARF line.
This breakthrough is a profound contribution to the research
and understanding of stem cells.
I asked Jamie last night ``How soon could you put that into
therapy?'' He said, ``If everything broke, it would probably be
4 years, more likely 5 years or 6 years.''
The hematopoietic blood cells means that they have
differentiated after they have been frozen the embryonic stem
cells into a blood cell line, which makes it different then,
after it is differentiated from am embryonic stem cell line.
And even while a handful of lines can supply scores of
researchers, the good news is that we have far more than just
the five lines from the University of Wisconsin. We have
identified dozens of already-developed lines--64 lines that
meet the President's criteria for Federal funding--but dozens
of lines that have already been fully characterized and are
ready to be sent out to researchers.
We have identified dozens of developed lines with the
potential for many more to become fully developed and useful.
As I indicated, it takes 6 to 8 months to fully perfect and
develop an embryonic stem cell line from derivation. These
lines come from five different countries--India, Sweden, the
United States, Australia, and Singapore.
Certainly we wish each of the derivations were fully
developed; but we must appreciate and we must not underestimate
the basic research value of those stem cells in developing
stages. We can benefit from research into their development. So
the 12 stem cell lines from Gothenburg University at this
stage, at the proliferation stage or even at the
characterization stage, can offer tremendous research on what
stage they are in and how they may be further developed. So
just because they have not been fully established as a cell
line does not mean the research cannot go forward on different
aspects of embryonic stem cells.
Certainly we wish that each of the derivations were fully
developed; but we must appreciate and we must not underestimate
the basic research value of those stem cells in developing
stages. But we could even benefit from research in their
development.
There are some who still wish and will argue that we do not
have enough. Well, we disagree for the reasons outlined. The
only way that you and I will be able to fully answer that
question correctly is to do the research, get the scientists in
and do the research. We need to move beyond the back-and-forth
over the numbers and get to actual work and doing the basic
research on the science.
The President has singled out the embryonic stem cells that
are most immediately available for research, and he has done so
in an ethically sound manner. We must seize the moment,
Senators, and take advantage of the opportunities for research
that these cells present.
Before I wrap up, let me just quickly touch on a few of the
other hurdles we are clearing at NIH and the Department so that
this research can go forward.
First, the NIH is in the process of developing a stem cell
registry and making it available so that scientists know
exactly what lines are eligible and who and how they can
approach those lines for access. We are working to make the
registry available on NIH's website very soon, and I would be
able to tell you today that we will have that website up
sometime within the next 10 days to 2 weeks.
Second, the FDA is making it clear that the use of mouse
feeder lines or layers in the development of lines is not an
insurmountable impediment to research including clinical
trials.
I have a letter here, Senator Kennedy, and one for you,
Senator Gregg, that outlines for the committee FDA's policy on
xenotransplantation. I would like to point out that the FDA has
assured me that the issue is not unique to embryonic stem
cells. In fact, at the present time, we have 13 INDs dealing
with xenotransplantation--currently--not embryonic stem cells--
13 currently under development, new drugs using
xenotransplantation.
They have several investigations for new drugs beyond that
for xenotransplantation, products currently in clinical trial.
So scientists should not let this issue deter them from
research.
I am submitting this letter from the FDA for the record
that outlines their stance on this issue.
Third, we are aggressively tackling many of the proprietary
issues regarding the stem cell lines and their availability. We
are encouraged that the owners of the lines want to make them
available for basic research and are working very closely with
all of them.
I would like to point out parenthetically that every one of
the entities has been in to see NIH, and I have personally
talked to them, and every one of them wants to cooperate and
wants to contribute and wants this basic research to get
started and continue.
In fact, I am very pleased today, Senator Kennedy, to
announce that we have negotiated as of yesterday afternoon a
memorandum of understanding that includes an MTA, Material
Transfer Agreement, that will accelerate research on stem cells
within the scientific community. The National Institutes of
Health and WARF, the WiCell Research Institute, signed that MOU
last night which will allow for the research use of its five
existing stem cell lines that meet the eligibility criteria.
The NIH scientists will have these lines available to them;
they will not be limited in the amount of research they do and
will not be limited to the publication.
This agreement allows scientists to access these cell lines
for their own research, permits scientists to freely publish
the results of that research, and allows the NIH to retain its
ownership--its ownership--of any intellectual property that
might arise from its research using those lines. Furthermore,
the MOU provides for a simple letter of agreement to govern the
transfer of cell lines with minimal administrative burden.
This is a groundbreaking agreement that we are happy to be
able to report to this committee this morning, that hopefully
will serve as the model for making the other lines available.
But it also gives an indication of how serious the owners of
these lines are about making their products available for basic
research. We will continue to work with all stem cell owners to
address proprietary issues.
Carl Gulbrandsen, of the Wisconsin Alumni Research
Foundation is here today, and I would like to take this
opportunity to publicly thank him, the folks at WiCell and the
dedicated team from the NIH Office of Technology Transfer,
headed up with Maria Ferrar, for the hard work they put into
reaching this agreement so quickly.
This agreement gives us even more momentum and incentive to
get to work.
In closing, thank you again for giving me the time and the
opportunity to outline some important and fundamental issues
regarding the President's decision to allow embryonic stem cell
research to go forward. Yes, I am passionate about this; I am
excited and enthusiastic, as you are, about the President's
decision. We all should be. There is great potential for good
from stem cell research. But there is also much work to be
done.
So let us come together and move forward. The only place
where we are truly going to find the answers to all of our
questions is in the laboratories of America and the world.
President Bush has opened the laboratory door. Now let us get
our best and our brightest scientists into the lab so that they
can do the work.
Thank you, Mr. Chairman.
[The prepared statement and attachment of Secretary
Thompson follow:]
Prepared Statement of Secretary Thompson
Mr. Chairman, Senator Gregg, and Members of the Committee, I am
pleased to appear before you today to testify about the President's
decision to permit Federal funding for research using human embryonic
stem cell lines. I am accompanied by Lana Skirboll, Director of the
Office of Science Policy at the NIH, Mark Rohrbaugh, Deputy Director of
the Office of Technology Transfer at the NIH, and Kathy Zoon, Director
of the Center for Biologics Evaluation and Research at the FDA.
I am pleased to be here today because I believe that President Bush
has made a wise decision, one that we can only guess at the import
right now. This was not an easy decision, and as his words to the
Nation made crystal clear, it was not a decision that was made lightly.
President Bush is guided by a strong set of principles, and he, like
all of us, strongly values human life. Importantly, no Federal funds
will be used to support the destruction or creation of new embryos, and
Federal funds will not be used to support research on stem cell lines
that are derived from newly destroyed embryos. The principle that the
Federal government should not encourage or sanction the destruction of
embryos was a cornerstone of the President's decision.
This President, by exhibiting such strong leadership, has helped to
create a research environment that we all hope will lead to cures for
such diseases as diabetes, Parkinson's disease, spinal cord injury,
stroke, Lou Gehrig's disease, and Alzheimer's disease--just to name a
few. President Bush has given hope to us all; the scientific community,
those suffering from these devastating illnesses, and anyone who has
ever seen a loved one suffer from a debilitating disease.
Very early on in my tenure at the Department, I asked NIH to
prepare a report on what was known about human embryonic stem cells: in
short, to do a review of the science. What investigators found, and
what the scientific community has been saying, is that today very
little is known. The first human embryonic stem cell was isolated only
a little over 3 years ago. Very few people in the world are working
with this type of cell. Yet, in that short time, and combined with
research that has been done on animal embryonic stem cells, we all have
every right to feel hopeful about what scientists may be able to
accomplish.
Human embryonic stem cells are unique. They are capable of
continuous self-renewal and of the ability to give rise to all cell
types that comprise the human body. But at this point, we know almost
nothing about their potential to treat disease. And we don't know how
they compare to adult stem cells.
On August 9, 2001, the President announced that Federal funds may
be awarded for research involving the use of human embryonic stem cell
lines that meet the following criteria:
The derivation process was initiated prior to 9:00 p.m.
EDT on August 9, 2001;
The stem cells were derived from an embryo that was
created for reproductive purposes and was no longer needed for such
purposes; and
An informed consent was obtained for the donation of the
embryo, and that donation did not involve financial inducements.
When NIH first presented its report on the State of the science to
me in June, it had documented at least 30 lines. In follow-up
discussions, it was clear that NIH had heard about the possibility of
others, even though scientists had not yet published their findings
regarding some of these other lines, and many were in the early stages
of development. So I asked NIH to go back, talk to these scientists and
companies, and find out everything it could about the stem cell lines
that currently exist. What investigators at NIH found was that more
stem cell lines existed, in various stages of development, than anybody
had realized.
Some were identified from publications and presentations at
scientific meetings. Others were identified in the course of preparing
the NIH report on stem cells through general discussions with the
scientists who had worked in the field. In some cases, the
organizations called NIH, and in others, information was provided to
NIH during inquiries regarding compliance with the former NIH
Guidelines.
To date, investigators from ten laboratories in the United States,
Australia, India, Israel, and Sweden have reported to the NIH that they
have derived stem cells from 64 individual, genetically diverse early
embryos. These human embryonic stem cell lines are currently viable,
exhibit characteristic stem cell morphology, and have undergone at
least several population doublings. The majority of these cell lines
are reported to express all of the markers known to be associated with
human embryonic stem cells.
All 64 of these human embryonic stem cell lines, which are in
various stages of development, meet the President's criteria and are
therefore qualified for use in federally funded human embryonic stem
cell research. The NIH has met or spoken extensively with each of the
investigators responsible for the derivation of these cells. These
scientists have expressed interest in working with the NIH and the
research community to establish a research infrastructure that ensures
the successful handling and use of these embryonic stem cells in the
laboratory. How researchers will be able to use these stem cell lines,
some of which are in the earliest stages of development, for laboratory
research is a valid and important research question, and I encourage
scientists to begin looking for an answer as soon as possible.
Likewise, the question of how to determine the quality or usefulness of
any cell line is an open question that also requires research, and we
encourage investigators to apply for NIH grants to answer it.
By allowing, for the first time, the use of Federal funds for
research on human embryonic stem cells, the President has opened the
door to a promising field of scientific opportunity for some of the
best and the brightest investigators who conduct their work with the
financial support of the Federal government.
I am working with NIH to ensure that the scientific community will
be able to use Federal funds to tap the extraordinary research
potential of human embryonic stem cells. We are hoping to begin funding
this research using a variety of mechanisms:. grants, contracts,
cooperative agreements, and supplements to existing grants. Today, our
goal is to ensure that cells are available and ready for distribution
and to help and encourage researchers who are working with these human
embryonic stem lines, which are in various stages of development, to
conduct the additional research that will make these lines available
for research use. There are challenges ahead.
First, in order to facilitate the much-needed basic research using
human embryonic stem cells, the NIH is creating a Human Embryonic Stem
Cell Registry that will list the human embryonic stem cells that meet
the eligibility criteria and provide basic information about the cells.
And there is more to do. We need to encourage the further
characterization of those lines that are in the earliest stages of
development. We need to provide technical assistance and funds for the
large-scale expansion of stem cell lines so that they are available to
as many researchers as wish to use them. We need to minimize the
administrative burden, with regard to requests for the distribution of
cells, both for scientists who have derived these cells and researchers
who wish to use them. And because these cells are challenging to work
with, we need to determine in what manner we will provide training to
researchers on how to maintain, grow and handle these cells in their
own labs. NIH may hold workshops and conferences to encourage broad
scientific dialogue about research ideas, and the identification and
resolution of technical problems inherent to any new arena of research.
Finally, and most importantly, NIH needs to do what it does best: fund
the most promising ideas, gain new knowledge from research, and use
that knowledge to improve human health.
As with other types of research, recipients of NIH funding will be
responsible for arranging access to particular cells that they
determine are necessary for their research. The NIH is interested both
in accessing cells for use in its intramural research program, as well
as in facilitating access for the broader research community. The goal
is to encourage the transfer of cells from qualified providers under
acceptable conditions and with as minimal an administrative burden as
possible.
Recently, the NIH has met with investigators who have derived these
cells to discuss these topics. At these meetings, the NIH has, on
behalf of its intramural investigators, initiated negotiations with
organizations that have derived human embryonic stem cells. Although
the NIH does not have the authority to negotiate agreements on behalf
of grantee institutions or third parties, it has been the NIH's
experience in other cases that the agreement into which it enters may
serve as a model for subsequent agreements negotiated by NIH-funded
investigators, should their institutions choose to adopt it.
Some scientists have asked about the effect that patents filed or
issued over the past few years will have on human embryonic stem cell
research. The issuance of patents on new discoveries need not adversely
affect continuing research, provided that the patent owners devise a
licensing and sharing strategy to allow basic research to proceed.
Experience has shown that conditions imposed by patent owners can be
crafted both to ensure research uses and to provide appropriate
incentives for commercial development. Although the specific terms and
conditions of availability must be determined between providers of the
cells and the recipients, we are pleased by the willingness of the
researchers who have derived cells to make them available for use by
federally funded researchers.
Although scientists will soon have the opportunity to explore the
promise of human embryonic stem cells, I wish to make it very clear
that this research must proceed responsibly and ethically. We have much
to learn about these cells--much basic research that needs to be
conducted. Clinical applications, which could possibly emerge only
after considerable basic research, are years away. What is important
now is that we begin the process of gaining a thorough and
scientifically based understanding of the promise and potential of
embryonic stem cell research.
The NIH is now implementing the President's policy. It is our hope
that federally funded investigators will take full advantage of this
new opportunity to conduct research on existing human embryonic stem
cells and explore the enormous promise of these unique cells, including
their potential to produce breakthrough therapies and cures. At the
same time, NIH will continue vigorously to support research on animal
stem cells and human adult stem cells, including those found in
umbilical cord blood, so that in the not too distant future we will be
in position to understand the relative benefits and limitations of all
types of stem cells. With the help of the scientific community, this
research will mark the beginning of a new era in modem medicine.
Mr. Chairman, it is time for federally funded scientists to begin
the fundamental research that is needed to determine the true potential
of stem cells. We will provide scientists with ample opportunities to
fully investigate this potential. We urge the research community to
begin these explorations with the profound hope that we stand at the
threshold of a true breakthrough in our ability to treat disease and
disability.
attachment
Department of Health & Human Services,
Rockville, MD 20857,
September 5, 2001.
Hon. Judd A. Gregg,
U.S. Senate,
Washington, DC. 20515-2101.
Dear Senator Gregg: During the past few weeks, concerns have been
raised about human embryonic pluripotent stem cells (HEPSC) and their
eventual use in human clinical trials for a variety of therapies. The
Food and Drug Administration would like to take this opportunity to
clarify these issues. A similar letter is being sent to Senator Edward
M. Kennedy.
While many questions are being raised, the science has not advanced
far enough to answer them yet. HEPSCs were first isolated in 1998, and
the scientific research about them is in its infancy. The FDA stands
ready to work with the scientific community as they near the stage of
human clinical trials. We encourage investigators to work closely with
us to ensure that testing occurs in the most expedient and safest
manner.
Most of the concerns raised to date are not unique to human
embryonic stem cells. The use of irradiated mouse feeder layers in
deriving HEPSC raises concerns that also occur for other
xenotransplantation products. FDA regulations do not prohibit using
mouse feeder layers to make HEPSC products for human clinical trials.
Of course, appropriate testing and precautions are necessary. FDA has a
number of active Investigations for New Drugs (INDs) for
xenotransplantation products currently in clinical trials.
Xenotransplantation products raise issues that several Public
Health Service agencies address, including the Food and Drug
Administration (FDA), Centers for Disease Control and Prevention (CDC),
National Institutes of Health (NIH) and Health Resource Services
Administration (HRSA). In order to provide guidance to maximize the
safe conduct of xenotransplantation trials, these agencies together
with staff from the Office of the Assistant Secretary for Planning and
Evaluation (OASPE) developed the PHS Guideline on Infectious Disease
Issues in Xenotransplantation (1/18/01). The PHS Guideline on
Infectious Disease Issues in Xenotransplantation and several FDA
Guidance Documents (see below) \1\ have defined xenotransplantation as
``any procedure that involves the transplantation, implantation, or
infusion into a human recipient of either (a) live cells, tissues, or
organs from a nonhuman animal source, or (b) human body fluids, cells,
tissues or organs that have had ex vivo contact with nonhuman animal
cells, tissues or organs.''
---------------------------------------------------------------------------
\1\ Guidance documents defining xenotransplantation:
PHS Guideline on Infectious Disease Issues in
Xenotransplantation, January 2001
Draft Guidance for Industry: Source Animal, Product,
Preclinical, and Clinical Issues Concerning Use of Xenotransplantation
Products in Humans, February 2001
Draft Guidance for Industry: Precautionary Measures to
Reduce the Possible Risk of Transmission of Zoonoses by
Xenotransplantation Product Recipients and Their Close Contacts,
Through Whole Blood, Blood Components, Source Plasma, and Source
Leukocytes, December 1999
Guidance for Industry: Public Health Issues Posed by the
Use of Nonhuman Primate Xenografts in Humans, April 6, 1999 (The actual
wording of the definition of xenotransplantation in this document ``the
use of live cells, tissues, or organs from a nonhuman animal source
transplanted or implanted into a human, or used for ex vivo contact
with human body fluids, cells, tissues or organs that are subsequently
given to a human recipient.'')
---------------------------------------------------------------------------
FDA regulates xenotransplantation products using the regulatory
framework established for other biologics. Xenotransplantation products
are, by their nature, also cellular therapies, and some are also gene
therapies, and as biologics are subject to the provisions of the Food,
Drug and Cosmetics Act and the Public Health Service Act and the
appropriate regulations in 21 CFR. A number of clinical trials of FDA-
regulated xenotransplantation protocols, including several in which the
contact with the animal cells is ex vivo, are currently enrolling
patients, after adequately addressing FDA's concerns. Addressing FDA's
concerns have included use of precautions such as: (1) testing the
xenotransplantation product for infectious agents by appropriate
methods including co-culture assays to detect potentially unknown
viruses, (2) educating patients regarding the potential infectious
disease risks and monitoring and testing them for xenogeneic infections
following treatment, (3) educating xenotransplantation product
recipients to not donate blood or tissues for use in humans, (4)
educating patient contacts, and in some cases monitoring them, (5)
maintaining appropriate records of treatment with xenotransplantation
products, (6) maintaining archived biologic samples from the patient,
animal source, and product in the event that a public health
investigation is needed, and (7) maintaining animal sources of
xenotransplantation products in a manner appropriate to reducing the
infectious disease risks of the product when used in humans. This
latter precaution might not be necessary if a long-term established,
well-characterized cell line with adequate documentation is used in
xenotransplantation.
Thus, as intended and practiced, the FDA regulation of
xenotransplantation products, while aimed first and foremost at
safeguarding the public health, should not impose a substantial
impediment to xenotransplantation product development, including HEPSC
that are produced by culture in vitro with mouse cells. HEPSC, as with
any other product, will be reviewed on a case-by-case basis to evaluate
safety when an application for investigational use is submitted to FDA.
In the meantime, sponsors planning the clinical use of HEPSC that have
been developed by exposure to animal cells should visit the FDA
Xenotransplantation Action Plan website () where links to all the published material and transcripts can
be found. They are also encouraged to phone the agency (301 827-2000)
with questions and concerns. Potential sponsors of any
xenotransplantation products are often counseled directly by FDA
personnel on how best to comply with FDA requirements on
xenotransplantation.
Sincerely yours,
Bernard A. Schwetz, D.V.M., Ph.D.,
Acting Principal Deputy Commissioner.
______
The Chairman. Thank you very much, Mr. Secretary, for your
presentation and for the obvious thought and commitment that
you have reflected in the presentation and your knowledge about
this issue. That will be of great help to all of us and to the
country in terms of developing policy.
We will try to have 7-minute rounds; I think it will take
at least that amount of time to ask a few questions.
First--and I want go through very quickly--you talked in
the presentation this morning about 64 derivations on the stem
cells. The President talks about as a result of the private
research, more than 60 genetically diverse stem cells already
in existence. Maybe it is semantics--I do not think it is--but
there was a difference in terms of those two concepts.
Let me get to my first question. If the current stock of
stem cells proves inadequate for effective research, or if
there are no new breakthroughs in ways to extract stem cells,
or if the existing cell colonies are unavailable due to patent
restrictions, would you then consider revising the
administration's policy to ensure that doctors will have access
to the stem cells they need to develop the cures of the future?
Secretary Thompson. Senator, we feel very strongly that the
cells are available. We feel that we have already knocked down
the proprietary concerns with the first MOU, and we think there
are many available.
There is also another procedure called ``subclonal'' that
allows for the subcloning of these existing stem cell lines for
further research being done. So we think it is adequate, and we
do not believe that we would advise the President to change his
policy.
The Chairman. Well, as you know, there are many scientists
who disagree with you about the adequacy and the availability
of the current stem cell lines, including some of the
scientists who developed the lines in the first place.
As you pointed out, we only discovered human stem cells
approximately 2\1/2\ to 3 years ago, and no one can know what
new advances are just around the corner. We have seen repeated
examples of the progress that scientists can make in a few
short years--for example, it took 5 years after the start of
the Human Genome Project just to determine the DNA sequence.
Only 5 years after that, they sequenced the entire genetic
code. Wouldn't it be a tragic mistake to freeze this field and
its progress to August 9, when we do not know what improvements
are going to come down the pike?
Secretary Thompson. Senator, the only real answer to that
is that we have to do the research; we have to do the basic
research. There has not even been the comparative research done
between embryonic, adult, placenta, and cord blood stem cell
lines. This research has got to be done.
I do not know if you or I or any scientist will be able to
say at this point in time that the breakthroughs are going to
be that quick or whether these stem cell lines are going to be
enough. I think that they are. I really believe that. And I
think there is adequacy to do it. But the only way that we can
get these answers is to get our researchers and our scientists
into the laboratory to start doing that basic research.
The Chairman. I agree with you, and I think we all want
them in the laboratory doing as well as they can, but as you
well know, the stem cells receive their nutrients from mice
tissue at the present time.
Secretary Thompson. Mice layers; right.
The Chairman. Yes. I will come back to this. There may be
the development of a technology that could remove those once
the determination is made about what those nutrients are. We
would be denied under the August 9 restrictions, or the
researchers would be, from taking advantage of breakthroughs in
terms of new technologies in terms of the derivation of the
stem cells, would we not?
Secretary Thompson. Some scientists are working on that
particular question as we speak, Senator.
The Chairman. But they would be precluded from taking
advantage of that new technology under the August 9
restriction, would they not?
Secretary Thompson. We do not believe so. We think that
the----
The Chairman. Since all the current cells now derive their
nutrition from mice cells--all of them now----
Secretary Thompson. That is correct.
The Chairman [continuing]. And I will get to the question
about that issue.
Secretary Thompson. I am not sure----
The Chairman. Just let me finish.
Secretary Thompson. OK.
The Chairman. There may very well be the ability to derive
those products as stem cells without using the mice; under the
August 9th restriction, that would not be possible as I
understand it.
Secretary Thompson. All I can say in answer to that is that
one of the scientists did tell us that they think they have
developed a system using pre-August 9 embryo derivations
without mouse line. I am not at liberty and I do not know for
sure. That was something that was said in passing in one of the
discussions.
The Chairman. Well, it was an example of what might happen.
Now, here in this book are the CDC guidelines for
transplanting animal tissues into human beings. ``All cells,''
it says in these guidelines--and this was published August 24--
``All cells that come in contact with human cells designed to
be transplanted should come from animals that are from a colony
that is continually monitored for infectious diseases or from a
colony that was not allowed to interbreed with animals outside
the colony or routinely tested for infectious diseases or
quarantined for 3 weeks prior to procurement of the cells.''
Now, virtually all of the stem cells approved under the
President's plan were grown in the presence of mouse cells.
these mouse cells were almost certainly not procured from
animals treated consistent with the guidelines described above.
Secretary Thompson. All I can tell you, Senator, is that
these are questions that I raised with FDA, and they have
indicated to me that they have many current clinical trials and
INDs going on using xenotransplantation, and they have
indicated to me--they are the experts--that they do not see
this as really an impediment. They will be questioning it, they
will be supervising it and doing the investigation, but as this
letter points out that I am submitting for the record, Senator,
it indicates that they do not believe this is a real
impediment.
The Chairman. This is again an area where research takes
strong exception with the nature of the research that is being
done.
Let me come to another area, and this is the final area
that I will have a chance to mention given the time. Under your
policy, there are 10 organizations that are allowed to provide
the stem cells to federally-funded researchers. People complain
about OPEC being a monopoly, but even they have 11 members. I
know that you are working hard to get adequate agreements, and
I commend you for the agreements that you have announced here.
You ought to be commented on that. The concern will be how many
other agreements will be able to be signed, and that is
basically what we want to do, because without signed agreements
from the suppliers, it is hard for me to share the confidence
that the problems will be satisfactorily resolved.
You have 10 organizations; you have been able to sign up
one or two--one--whatever it is. You have these organizations
that basically have control on these items. How can you give us
the assurance given that kind of restriction that there will be
availability and accessibility for researchers to do the job
that is out there to be done?
Secretary Thompson. We have talked to all the entities, all
10 of them, and all have indicated that they would like to see
the basic research go on. All of them have cooperated. WiCell
is the one that has the patent in the United States at the
present time. They are the one that has signed the MOU plus the
Material Transfer Agreement letter as of yesterday, and they
have also indicated, Senator, that they have five cell lines
available right now, and they have also indicated that there
will be no limitation on the scientists who want to apply to
use those lines and that those liens will then be able to be
used in research, and it will not be limited to the 10
entities. Any scientist--and they have already reached an
agreement with the scientists at NIH to make them available for
the NIH scientists as of today.
The Chairman. Senator Gregg?
Senator Gregg. Mr. Secretary, just as a threshold issue
here, I want to clarify the situation of adult stem cell
funding. Is it your intention and can you assure us that funds
will not be diverted from adult stem cell funding research into
fetal activity, fetal and embryo stem cell?
Secretary Thompson. Absolutely not. That is the beauty of
the President's policy, Senator Gregg, that the basic research
on the comparison of adult stem cells, embryonic stem cells,
placenta flat and cord blood has never been done. The best
thing we can do is to do that basic research and get it started
in the laboratories. And no, there will be no reduction on
adult stem cells. We used $250 million last year on stem cell
research, and I can assure you there is going to be more
available for all forms of stem cells including and most
important, adult stem cells and embryonic stem cells.
Senator Gregg. I think that that is very important. We have
doubled, as was mentioned, the funding for NIH as a result of
the efforts of Senator Harkin, Senator Specter, and a lot of it
was really started by Senator Mack when he was serving in the
Senate. There is certainly a lot of money at NIH these days,
and it seems to me that with this exciting new area of therapy,
we should be able to fully fund especially the adult area which
is already producing results. We actually have physical
therapies that benefit from adult stem cells right now today.
Secretary Thompson. We will not limit that at all. In fact,
I talked with the NIH scientists as of last night and
yesterday, and they have indicated they have enough money
available for all good research projects. Also, because of this
hearing and because of the President's decision and all of the
publicity around embryonic stem cells, there is a tremendous
amount of interest from scientists, researchers, and
investigators around the country and the world who want to
apply.
Senator Gregg. Let me go through a couple of elementary
questions, because I think there are some initial policy issues
that we have to get clear before we go into the substance of
some of the issues that Senator Kennedy was talking about.
As I understand it, without Federal dollars, research on
stem cells can proceed carte blanche; correct?
Secretary Thompson. That is correct.
Senator Gregg. So explain to us if you would why it is
important to have Federal funds involved in this research.
Secretary Thompson. Because the private dollars will want
to go as quickly as possible to a therapy. It is more important
to do the basic research at the beginning, the kind of research
that Jamie Thomson and other scientists who are the forerunners
in this research field are doing. That comparison that I talked
about in response to a previous question that you asked,
Senator, needs to be done, and that kind of basic research
comparison adult, embryonic and all the other stem cell lines
will not be done just by the private sector. The Federal
dollars allow that. That is point number one.
The second point is that Federal research dollars when they
do this research encourages other scientists to get involved.
And once the basic research is done, the privates come in and
do more of the therapy research. So it is sort of a building
block. That is why it is so important to get those Federal
research dollars out quickly and into the laboratories as soon
as possible.
Senator Gregg. That is an important point to make.
Second, what is the role of patents in this exercise? This
company, Geron, which I am not familiar with, has the patents
from the Wisconsin projects to the extent there is
commercialization. What do you see as the role of patents in
this exercise?
Secretary Thompson. First of all, Geron does not have a
patent, Senator Gregg. WiCell is a subsidiary----
Senator Gregg. Or a license.
Secretary Thompson. They have the license. Geron has the
license of three of the five lines, and they have already
reached an agreement with WiCell to make those lines available.
That is part of the agreement, part of the MOU. Really, WARF
and WiCell are directing this. They made the arrangements with
NIH yesterday by signing the MOU and the Material Transfer
Agreement letter to make these available to any scientist in
the world who wants to apply through NIH.
Senator Gregg. I am talking more philosophically. What is
the role of patents in this?
Secretary Thompson. Patents will come into play much more
importantly when the therapies are developed and they go into
commercial products; hopefully, as soon as possible.
Senator Gregg. So in the basic research area, you do not
see that as being an issue.
Secretary Thompson. I do not see that as much of a
restriction, no--based upon the agreement signed yesterday.
Senator Gregg. Can you explain why you picked the date
August 9 at 9 p.m.?
Secretary Thompson. Because that is the time the President
made his speech. And it was not my decision; it was the
President's decision.
Senator Gregg. And what is the implication of that date, in
your opinion?
Secretary Thompson. The implication is that there are
embryonic stem cell lines available for basic research, there
is money available for that basic research, and it allows us
now to open the door to the laboratories, to get the money out,
and to get the comparative and basic research done which is so
important in this whole embryonic field of stem cells.
Senator Gregg. I believe the key to that decision was that
you believe the embryonic stem cell lines that are out there
can be used in a manner which, because they replicate
themselves limitlessly, I think was the term you used----
Secretary Thompson. Endlessly.
Senator Gregg [continuing]. Endlessly--that because of
that, there is no need to go past that date in order to find at
this time adequate stem cells for the research that is going
on. Is that correct?
Secretary Thompson. That is correct. They are pluripotent,
which means they replicate, and as long as they do not
differentiate, those cell lines are able to be used for basic
research. And Jamie Thompson--we have five cell lines, and he
is sort of the creator, the father, of the embryonic stem cell
lines, and he personally uses about two of his stem cell lines
for his research. As far as embryonic stem cell research, it
has been done for 20 years on mice, and 90 percent of that
research has been done on five cell lines.
Senator Gregg. And it is also, as I understand, the
position of FDA as presented to us in this letter--and I have
not read it yet, but you are characterizing it--that the
xenotransplantation issues are not a problem when you move from
basic research to therapy, and they are not going to be a
problem for the FDA as to----
Secretary Thompson. FDA will certainly be looking at every
facet of the procedures as we go along, but they also have
currently 13 INDs going on with different aspects of
xenotransplantation, and they have said that that should not
act as the impediment or reluctance to proceed on the research.
But they will continue to supervise, they will continue to make
sure that it meets all of the safety rules and regulations that
CDC and FDA have put out.
Senator Gregg. Let me ask you another question which I
think raises the issues that get to the issue of when--well, my
time is up, so I am not going to get into that.
The Chairman. Senator Dodd?
Senator Dodd. Thank you very much, Mr. Chairman.
Mr. Secretary, welcome.
Secretary Thompson. Thank you, Senator Dodd.
Senator Dodd. It is good to have you with us today, and I
appreciate your deep interest in this and your obvious
knowledge of it. That is obviously helpful, and I am pleased as
well that you have some wonderful folks from NIH with you here.
This is a tremendously important issue as you and others
have pointed out. I have just a couple observations. I have
asked unanimous consent that my opening statement be included
in the record. Obviously, we are not going to resolve this
issue specifically with a piece of legislation or even a speech
on August 9. This is going to be an ongoing effort, and my hope
would be that whatever policies are developed, and whatever
finally comes out of this debate in the short term, that a
decision will be made to revisit this issue periodically,
almost sunset decisions, so that we will have the ability to
come back and review decisions that have been made to determine
whether in fact, since we are wandering or heading into
uncharted areas, to put it mildly; I would hope that would be
part of the consideration.
It causes me to comment and ask you to further comment
yourself on Senator Kennedy's questions regarding these mouse
feeder cells--and I hear you, and I know you are confident and
hopeful that the research will be there to be able to extract
these mouse feeder cells so as not to contaminate these stem
cells in any way that would jeopardize human life.
Secretary Thompson. That is correct.
Senator Dodd. But there is the possibility that that will
not happen. Just as you hope that it will, we all understand
that it may not work. So my question to you to conclude this
line of questioning would be if in fact that is not the case,
will the administration go back and revisit the August 9
deadline to determine whether we can develop some new lines.
Secretary Thompson. It is going to take such a long period
of time to go from the laboratory to therapy to human clinical
trials, Senator Dodd, that--everybody, including myself, has
great anticipation and hope that we are going to be able to
move directly to a cure for Parkinson's and so on and so forth,
but it is going to take 5 to 8 years.
Yesterday, the hematopoietic blood cells were developed,
and it is probably going to take 5 years----
Senator Dodd. I understand that.
Secretary Thompson [continuing]. And during that period of
time, I am sure that what I say today and the questions that
you ask will be sort of arcane and will probably be ludicrous
by the time we look at it 5 years from now, Senator.
Senator Dodd. It is always dangerous when Congress--we all
get worried about mad scientists--nothing is more frightening
to me than Congress trying to be a scientist. So I appreciate
what you are saying. The point I am trying to get at is sort of
a mindset from the administration standpoint as to whether or
not there is the flexibility to come back and revisit issues. I
realize we cannot answer these questions, and it will take
time, but I am trying to elicit from you as the spokesman for
the administration your policy questions so that if in fact we
are unable to extract the mouse feeder cells, thereby making
these lines precarious at best, will the administration
reconsider its August 9 deadline?
That is really my question. I understand all the scientific
issues that we cannot answer today.
Secretary Thompson. The administration will not reconsider
its deadline as far as the destruction of embryos.
Senator Dodd. OK.
Secretary Thompson. It will reconsider all the policies and
all the procedures and research going into embryonic stem cells
on an ongoing basis as we find new things----
Senator Dodd. I understand.
Secretary Thompson [continuing]. But the destruction of
embryos for new embryos--no, it will not.
Senator Dodd. OK. Let me if I can--I do not know if you
heard Senator Specter or had a chance to read his statement,
but he made some comments in there regarding the viability of
the various lines that exist and raised questions about the
Gothenburg University 19 lines, which you have addressed to
some degree in your conversations apparently as late as
yesterday with one of the scientists there. But I read a
statement today that says that out of those 19--and I am
quoting here from Hamburger, who I guess is the same fellow you
talked to----
Secretary Thompson. Dr. Hamburger, right.
Senator Dodd [continuing]. He says--and I am quoting him--
``If we get three good lines out of them, we will be
satisfied''--out of the 19.
How many lines do we need in order to--what is the minimum
viable lines at the end of this process that we would need in
order for there to be a viable level of research to be
conducted?
Secretary Thompson. We believe that the five lines that
currently exist will be able to get us a long way down the road
doing the basic research, but there are many more. We feel
right now that roughly, there are 25, 24 or 25, full cell lines
established, and there are 64 in the various phases from the
proliferation to the characterization to the cell line
established, and out of those remaining ones, we think there
will be a lot more that will be established that will be able
to use the vials at the end where the cells are frozen and be
able to send out to researchers around the world.
Senator Dodd. In fact, you may want to--and I am just
trying to understand what minimums are, and I agree with you, I
am hopeful that is the case as well--but I want to get some
sense of what is the bottom line that we are looking at here.
If in fact we are only getting three, four, or five, would that
be adequate, and your statement is that you believe it is. AT
the end of all of this, at the end of all the 64 that are out
there, the potential that are out there, and you go through the
steps which you have very clearly outlined here, if at the end,
in that last step, there are five viable lines, your answer to
my question is that that is more than adequate?
Secretary Thompson. We have 25, 24 or 25, adequate right
now, so I am confident that that is enough, but I am also
confident that there are going to be many more of the 64 that
will be available.
Senator Dodd. In the minute remaining, if I can--and again,
for those of us up here who are trying to learn, this is pretty
complicated stuff, and Bill Frist and others whom many of us
listen to very carefully when they speak on these issues--and I
have been fascinated from a personal standpoint with cord blood
research and have been doing a lot of work in the last few
days, and NIH has been very, very, helpful, and I have talked
with them at length about the subject matter, and there is some
great potential in cord blood research--maybe that is a subject
for another discussion later on--but the position the
administration has taken, that as of August 9, only those
embryos that have already been moved in this process are the
ones to be considered--I think all of us here would tell you
that if there were any issue raised about whether somehow we
were going to create a ``hatchery,'' producing embryos
specifically for developing stem cells, I think we would
vehemently oppose it, every one of us. I do not know of anyone
who would take a different point of view. On the issue,
however, of embryos being developed as a result of the
wonderful advances in the science of in vitro fertilization,
there are existing embryos out there, many of which will be
destroyed. Does the administration take the position that we
ought to pass legislation or that they are going to offer
legislation that would ban the destruction of these embryos
that are sitting there today that will be discarded?
Secretary Thompson. I do not think the administration has
taken a position on that, Senator Dodd, but I would like to
point out that those embryos right now--in order to establish a
good line, it takes a lot of scientific ability to do that.
Senator Dodd. I understand.
Secretary Thompson. And I have discussed that with Jamie
Thomson, and he says a good line takes, with optimum
conditions, 6 months, but it is usually closer to 8 months.
So that right now, we have these 64 in different stages, 24
that are almost fully developed. It would take another 8 months
to be able under the best of circumstances to establish a
brand, new line.
All I am saying is that we should get the research dollars
out there for these basic lines. And it is going to take us
some time, because the procedure at NIH to put out grant
dollars as they tell me will be 8 to 9 months from now. So
during this 8 to 9 months, several of those remaining from 24
to 64 are going to be fully developed, so there will be many
more lines available for research and for development.
Senator Dodd. I thank you, Mr. Secretary. My time is up,
but just a brief, quick comment. My concern--and others will
express their views--is with your answer to the question of
whether you would revisit at all the August 9 deadline if in
fact it turns out that we are not able to remove the mouse
feeder cells. The rigidity of that concerns me, and also the
decision--if we are going to draw ethical lines here, if we are
going to discard cells, there will be no effort to bad the
discarding of those unused embryos, then we are blurring the
lines further, it seems to me, and creating some great
potential conflict here. So again, I appreciate your
involvement, your determination, and your passion about the
issue and look forward to an ongoing dialogue.
Secretary Thompson. Thank you, Senator Dodd.
The Chairman. Senator Frist?
Senator Frist. Thank you, Mr. Chairman.
I briefly want to explore, since you have had so many
conversations, the relationship between public and private
funding. Right now, of the 64 cell lines, or the cell lines
that are out there, all been developed with private funding, as
of today--the research.
Secretary Thompson. No, I cannot answer that in the
affirmative, because some of the universities in Sweden and
some of the lines in India and Australia I am sure could have
had public funding.
Senator Frist. But in the United States, we have not been
funding----
Secretary Thompson. In the United States, yes, that is
correct.
Senator Frist [continuing]. The creation of these cell
lines.
Secretary Thompson. I do not know if they have or they have
not internationally, Senator Frist. I cannot answer that.
Senator Frist. As we look ahead, we know that this science
is moving fast.
Secretary Thompson. Yes, it is.
Senator Frist. It is hard even to project how long all
these lines are going to be available, what the research is
going to be like, what the breakthroughs are going to be, what
the roadblocks are going to be. There is just no way to know,
and we can debate it.
We are going to hear the argument again and again that we
ought to have sort of a freestanding, let us use all the cells,
and we need unlimited cell lines.
I think that what we have learned a lot recently is that
you do not need unlimited cell lines, that there is a limited
number, and now, unlike 4 weeks ago, we are saying is it 64 or
20. At Senator Specter and Senator Harkin's hearing about a
month and a half ago, we initiated a lot of this discussion
about how many cell lines do you need. It is different than
transplantation, where you take the heart out of one person,
you transplant it into another, and it helps them. These cells
are self-perpetuating. So we have come a long way in the
debate.
What is interesting as I listened broadly is that we have
Federal funding, which is important; it legitimizes the field
and gives you some control in terms of ethical oversight and
prioritization. But at the same time, we have the private
sector which, if this promise is real, is going to be heading
off full-steam.
Secretary Thompson. Absolutely.
Senator Frist. And by having some public funding, in fact
we are going to accelerate that because it legitimizes the
field.
What I want to make sure of, and what I have argued in the
last several months, is that we have the ethical oversight.
That will come through the hearing process here, but we need
not just the ethical but the moral, ethical, and scientific
oversight of what is going on broadly, because the drive for
science is powerful. It is to create, it is to discover, and
that is good. On the other hand, as I said earlier in my
opening statement, the potential for unintended consequences is
there. And we are dealing with the creation of life, the
manipulation of the basic building blocks of life, and what I
am concerned about--and we have not talked about, and we do not
have time to go into it in much detail now--is that larger
ethical oversight.
The registry is a step in the right direction because that
will give you at least a handle on the State of the art,
private and public. The Bioethics Commission--at some point, I
would like to hear more about that in terms of what the plans
actually are in terms of oversight, what the focus will be, and
the composition--you do not have to go into that right now, but
again, I would think that they are going to look at the public
sector as well as the private sector as we go forward.
Secretary Thompson. Yes.
Senator Frist. And third, I am interested in scientific
oversight and how we as a Nation--should we legislate it; do
you do it through HHS--provide the appropriate scientific
oversight. Right now, we are arguing--is 64 too many, or where
is it in terms of the 64; is it 19, is it 5, is it 4--it is
good because we are asking the questions, but we need ongoing
oversight so that 3 months from now, when we find out that
there are 200 cell lines, or there are 5, appropriate decisions
can be made, or appropriate input is received.
And I guess what I would like to introduce as a question is
what are we doing not just as far as Federal funding for the 60
cell lines, but in terms of getting the larger ethical, moral,
and scientific construct both to oversee--not regulate, but
oversee--and monitor the issues of both public and private
sectors?
Secretary Thompson. That is a very good question, and let
me respond this way, Senator. That is the reason why the
President set up the Bioethics Committee, to look at those
questions and to be able to make the recommendations to the
administration on those particular questions that you have
raised.
In regard to the Department of Health and Human Services
and NIH, our responsibility is to implement the policy. We are
doing several things to implement that policy. We are setting
up the registry. We are talking to the scientists about whether
or not we should have a repository of these cells at NIH. Some
scientists would like that, other scientists are fearful of
that. We have not been able to do that.
The third thing is to negotiate MOUs and Material Transfer
Agreements, which we have already started.
So we are already a long way down the road in dealing with
this implementation question. The ethical questions certainly
will be considered as we go along, through the Department of
Health and Human Services, but the overall review and direction
and new policies on these will come out of this new commission
set up by the President, headed by Dr. Kass.
Senator Frist. Thank you.
Thank you, Mr. Chairman.
The Chairman. Senator Harkin?
Senator Harkin. Thank you, Mr. Chairman.
Again, Secretary Thompson, I appreciated the conversation
we had on the night the President made his decision. I have
given my support to the President's decision. I think that he
has moved us ahead in this area. Not being a scientist but
having been involved with NIH for many, many years, I have been
watching very carefully the development of this issue over the
last couple of weeks.
There is a lot out there that we do not know and do not
understand; a lot of controversy about these cell lines and how
many Sweden has or India has. We do not really have all the
answers yet, but I think it is incumbent on us to continue to
delve into this to see if we can shed more light on it, to
expand our knowledge of exactly what we are doing and what we
are talking about.
Again, I am not saying that I do not doubt for a minute
that the President did not give a lot of thought and a lot of
time and consideration to this; it is obvious that he did. But
a lot of us have been giving thought and time and consideration
to this for years, and we still do not have all the answers.
So I am not going to take the position that what the
President said on August 9 is the final end-all and be-all of
everything, because even if he did give some consideration and
time and thought to it, he could not have given--I mean,
because of new things that are opening up and new things that
are happening, we have to be able to adapt to this new science.
So my position is going to be one of basically just
continuing to ask questions to try to find out whether or not
under the ethical guidelines that we have established under the
Bioethics Commission--by the way, most of which the President
followed; the only difference was the timing, the 9 p.m. on
August 9--but the other guidelines are all the same--informed
consent, no monetary consideration, and only using the embryos
for----
Secretary Thompson. The excess.
Senator Harkin [continuing]. And not using any therapeutic
type of embryo development. So, basically, it is all the same
except for that time line; it is basically the same. And we may
want to think about that time line, too, at some other point.
But I guess my concern is along the lines of what you said
about the university in Sweden--and I have talked extensively
with my colleague Senator Specter on this. Yes, there are three
developed lines. I did understand that before I came to the
hearing today. But the real open question is whether or not
early on in the process, those cells that were extracted will
lead to viable cell lines, and we do not know that yet.
Secretary Thompson. We do not know that.
Senator Harkin. We just do not know that. I am glad to hear
you--you agree with that, that we do not know that yet?
Secretary Thompson. We do not know that for sure.
Senator Harkin. We can hope they do, obviously, but if they
do not, and if we find out that in other cases in India and
some of these other places, that the lines are not fully
developed, then we might wind up with something quite less than
60 or 64; we might wind up with something quite a bit less than
that. Is that correct?
Secretary Thompson. That is possible, Senator Harkin, but
at the same--if I could answer in a little bit more detail,
there are dozens already in existence, and the time line--it
takes 6 to 8 months to develop a good cell line. There are four
in the characterization stage from Sweden and 12 over here that
have just been derived. The four look very promising, and the
12--there are certainly some, I am sure, that will fail and
some that will succeed. I cannot give you that number. But
there are dozens that are available right now.
Even at this stage, there is some great research that can
be done on those 12, and those are questions for the scientists
to do, and we have not seen that kind of research done yet.
The President has allowed for the Federal dollars now to
flow toward research in all of these areas, and hopefully soon
to get to some therapies.
Senator Harkin. And that is the second thing--it has to do
with the therapies--and we will be delving into this more in
our committee hearings in the future also. But you responded to
I think it was either Senator Kennedy or Senator Gregg when
they asked about the fact that the nutrient supply used for the
development of these cells was evidently mouse cells. I did not
know that before, but I guess this is true in almost--in every
case.
The Chairman. All.
Senator Harkin. All of them.
Secretary Thompson. Yes.
Senator Harkin. So there is a contamination problem that I
was not aware of before. I fully admit that I was not aware of
this contamination problem.
And if we are looking at just what happened yesterday at
the University of Wisconsin and the fact that they are able to
at least initially take some of these embryonic stem cells and
move them into blood lines, that is pretty astounding.
Secretary Thompson. It is exciting.
Senator Harkin. That is astounding. In this short a period
of time, we have done this. Mind you, they only derived these
cells 3 years ago.
Secretary Thompson. Three years ago.
Senator Harkin. And now we are making red and white blood
platelets out of it.
Secretary Thompson. Platelets, right.
Senator Harkin. That is pretty amazing.
But if there is a contamination problem, we could go down
this line for, as you say, the next 5 years or so, but be
working with things that, while they might prove out to be
beneficial therapeutically, would not be able to be used
because prior on, they were grown with a nutrient out of mouse
cells.
It would seem, then, that we would have to go back and
almost start all over again. Why waste all that time? My big
concern is that I did not know and was not aware that all of
these cell lines were grown in mouse cell nutrients as a basis,
and that disturbs me. We need to ask the scientists more about
what that means in terms of the therapies and how soon we can
derive those therapies if in fact they are contaminated.
Secretary Thompson. Can I just answer in two ways? First,
when we started on the 64 lines, nobody knew--you did not know,
I did not know, nobody knew--how many lines were available. I
asked NIH to make a complete inventory of the lines--not at
what place they were but whether or not the embryos had been
derived as of August 9.
NIH did it once; they did it the second time after the
President's speech; and the third time is coming in and talking
to them. We found that there are 64, but they are in different
stages; but there are dozens that have already been through the
cell line established. That is number one.
At the bottom of the petri dish is where the mouse layers
are placed. And it is sort of a traumatic experience for these
cells as I understand it when they are taken away from the
blastocyst and removed and put into the culture; they have to
have something in order to grow and be able to replicate. That
is probably the most difficult portion, and according to Dr.
Thomson, that is where a lot of scientists failed. They need to
be able to do that.
Then, FDA has established policies, and they have looked at
this procedure. Kathy Zoon is here. She is in charge of this,
and she has indicated that right now, we have 13 INDs,
Initiatives for New Drugs, that have already been started using
xenotransplantation. And she does not believe that this alone
is going to prevent moving from here to therapy, and neither
does Dr. Jamie Thomson.
Senator Harkin. I appreciate that, but we need to delve
into this further.
Secretary Thompson. Yes. And the blood cells, the
hematopoietic blood cells--I talked to Dr. Thomson yesterday,
and he said that it is going to take probably 3, 5, 6, 7 years.
Senator Harkin. But those are still contaminated lines.
Secretary Thompson. I do not classify them as contaminated.
Senator Harkin. Well----
Secretary Thompson. They have the cell----
Senator Harkin. There is a cloud over them. There is a very
dark cloud there.
Secretary Thompson. I do not want to argue with you because
I am not a scientist, and I cannot know----
Senator Harkin. Well, I am not, either, I am not either,
but----
Secretary Thompson [continuing]. But I do not think they
are contaminated, because I think they are going to be very
useful and usable. I cannot say for sure. All I can tell you is
what I talk to people and they tell me, Senator Harkin.
Senator Harkin. We have got to follow up with the
scientists on this. I do not know.
Secretary Thompson. Yes. The scientists have got to tell us
that. That is why it is so important to get the scientists in
the laboratory to start answering these questions, Senator.
The Chairman. As the Senator is winding up, as I understand
it, mad cow resulted from a completely unanticipated transfer
of infectious agent from beef to humans. No one anticipated
that, no one knew about it. That is why these guidelines that
you have issued in the CDC are so important, to make sure that
we do not have unanticipated transfer and why those guidelines
are so important. These stem cells that you have outlined here
and the President, I do not believe meet those kinds of
requirements, and looking through the letter that you sent
here, I do not think that should give us much satisfaction. But
we will have another chance to come back to it.
Secretary Thompson. Senator, could I respond?
The Chairman. Yes, please.
Secretary Thompson. The truth of the matter is that the
embryonic stem cells probably would not survive--would probably
not survive--the removal from the blastocyst into the culture
dish without the mouse layers that the cells rest upon and get
the nutrients from. In order for us to do this research, we
have to have this, and it is going to take 3 to 5 years to find
out all of these answers.
The truth of the matter is we have got to get this basic
research done, because the questions you ask are good ones,
Senator Kennedy, and I cannot answer them, but I do know that
the embryonic stem cells would not grow and replicate without
the mouse layers, because they provide the security and the
proteins.
The Chairman. But Mr. Secretary, they may very well have to
use the mouse cells, but not these mouse cells--mouse cells
that meet the kinds of requirements that CDC has outlined that
might take 6 or 8 months. But the ones that are now being
identified in these 64 lines do not meet the requirements of
the protections that have been announced by your own agency and
CDC; they just do not do that. We will spend more time on this,
but the August 9 date arbitrarily says that we are only going
to be able to use those that are already out there when, as you
have said here, we could develop these in between 6 to 8
months, and we could do it with mice that meet the requirements
of the CDC. But we are not going to be able to do that because
we have the August 9 deadline, nor will we be able to even
consider research that might be able to detect the kinds of
nutrients that are so important in the mouse cells--that is
what it is; it is these nutrients--and many of the researchers
that I have talked to say we will be able to find out what
those nutrients are, and we might not even need those mouse
cells down the road, but we are going to be prohibited from
using those cells in the future because of the August 9
deadline as well. These are some of the concerns.
Secretary Thompson. But Senator, the basic research can be
done with the Federal dollars. On the private research dollars,
there is no prohibition or inhibition whatsoever on private
research coming in and using a new procedure and getting it
ready for therapy, getting it ready for the development of a
pill or a device.
The Chairman. Senator Warner?
Senator Warner. Thank you, Mr. Chairman. I wish to commend
you and our distinguished ranking member for initiating this
hearing.
Those of us who have been privileged to attend this hearing
I think will remember it in years to come, because this is an
historic threshold for the Congress. This is the first full
committee hearing on a subject that will place challenges to
our Republic unlike any I have seen, literally, in the 23 years
I have been privileged to serve in the Senate.
We are fortunate to have you at the helm at this particular
time. You bring to your position vast experience in diverse
subjects, and most importantly as Governor, caring for people.
That is what the bottom line is in this.
And I commend the President, even though I and others
stepped out somewhat ahead of the President in a letter--which
I would ask unanimous consent be included as part of the record
today----
The Chairman. It will be so included.
Senator Warner [continuing]. Where 11 Republican Senators
took a stance on this early on.
[Letter follows:]
U.S. Senate,
Washington, DC 20510.
The President,
The White House,
Washington, DC 20510.
Dear Mr. President: We strongly urge you to continue the last
Administration's policy of using Federal funds for research on human
stem cells after these cells have been derived from embryos. In
addition, we strongly urge you to support legislation which would
remove the existing ban on the use of Federal funds to derive stem
cells from embryos.
On the issue of stem cell research, we think our colleague, Senator
Gordon Smith, went to the heart of the matter when he pointed out the
difference between an embryo in a petri dish, which would not produce
human life, as opposed to an embryo in the womb of a woman? where
further development would produce life.
The essential consideration is that there are many excess embryos
created for the purpose of vitro fertilization.The only issue is
whether these embryos will be discarded or used for stem cell research
to save lives. Stem cell research has demonstrated a remarkable
capacity of these cells to transform into any type of cell in the human
body. Stem cells could be transplanted to any part of the body to
replace tissue that has been damaged by disease, injury or aging. If
scientists are correct, stem cells could be used to treat and cure a
multitude of maladies such as Parkinson's, Alzheimer's, diabetes, ALS,
heart disease, spinal-cord injury, all types of cancers, burns, stroke,
macular degeneration, multiple sclerosis, muscular dystrophy,
autoimmune diseases, hepatitis and arthritis.
Current law prohibits Federal funding to create human embryos for
research purposed through cloning, or through any other means. We do
not object to these important prohibitions. However, creating embryos
for research purposes is entirely different from using spare embryos
left-over from infertility treatments. These spare embryos are now
destined to be thrown away. Rather than discarding them, we support
using these embryos in medical research to treat and cure disease.
Sincerely,
Arlen Specter.
Strom Thurmond.
John Chafee.
Olympia Snowe.
Ben Nighthorse Campbell.
Gordon Smith.
Susan Collins.
Ted Stevens.
Kay Bailey Hutchison.
Orin Hatch.
Dick Lugar.
John McCain.
John Warner.
______
Senator Warner. So I look upon this as, not unlike our
great pastime, a football field--at one goalpost, those
standards of ethics, morality, and religion which have made
this Nation the strong Nation and the envy of the world that we
are today--and we have got to protect those in the future. At
the other end of the field is the goalpost of alleviating human
suffering and the need for this Nation to move ahead on the
forefront of science.
My particular area in this Senate is national defense. Our
country is a superpower, not because we desire to be, but
because the rest of the world has ceded to us that
responsibility. We are working tonight on a military budget for
the United States which is greater than cumulatively adding up
all the other budgets of the world. It is astonishing. But we
have taken that responsibility on.
The question before this President and successive
Presidents, the question before this Congress and successive
Congress is are we going to be a superpower in this exciting
science, or are we going to put in a lot of arbitrary
standards? If that is the case, what will happen is two things.
If our Federal system--the President setting down the
leadership, the Congress enacting the laws to support our
President, and the courts trying to fairly arbitrate the
differences of views--all three of our entities of Government
will work together on this--but if we fail to convey to the
American people and the rest of the world that we are fair and
have objectivity and a realistic approach to this, the Federal
Government will be left in a cloud of dust as the private
sector marches off on this. We will also no longer be a nation
of importers of brain power and scientists; our own scientists
I am fearful will go abroad to where they can work on this.
Then, we will lose as a nation that degree of oversight and
control over our standards of ethics and morality if they all
depart. Then, that science will come back into the United
States, either legally or illegally, because you are just not
going to stop the advancement. So this is a whale of a
challenge.
Do you basically agree with that sort of philosophical
approach that I have, and if so, what can we do to keep the
private sector in a fair set of rules to do those things that
perhaps our Federal Government will not do for the moment and
to keep our scientists from leaving this country and going
abroad?
Secretary Thompson. I think your comments were very
thoughtful and very passionate, and I could not disagree with
anything you said, Senator, and applaud you for saying it.
I would like to only clarify that prior to August 9, there
were no Federal research dollars available. As of the
President's speech there is money available. We made an
inventory, and we have found that 64 embryonic stem cell lines
exist that meet the President's criteria--that means for
Federal funds.
They are in different degrees of development. Some are in
the proliferation stage, some are in the characterization, some
are in the cell line established. But they are all available
for research.
The mouse research on embryonic stem cells has been going
on for 20 years, and 90 percent of that work has been done on
five lines. Dr. Jamie Thomson, who discovered the embryonic
stem cells, has five lines, but he uses for his research
purposes two lines, and he has lines available.
And WiCell, who has the patent, has indicated they have
enough cell lines available for any researcher in America who
wants to do the research.
I think it is a tremendous opportunity. This whole area of
embryonic stem cells opens the door. Everybody who has some
sort of malady or has a loved one or a dear friend who is
suffering from dementia or cancer or so on is looking for this
as the panacea. We have to be careful not to indicate that a
cure is right around the corner. It is going to take several
years. We need now to get the Federal research dollars and the
basic research done, Senator, so that private dollars can come
in and develop the therapies once they are developed.
I believe that there is more interest because of this
hearing, more interest because of the President's speech, more
interest because of all the discussions around the country
about embryonic stem cells, and that people know more about
human biology and physiology than they ever have before, which
is in and of itself a positive. And I think that scientists now
calling NIH are excited about the potential.
I cannot answer all the questions, and none of the
individuals up there can at this point in time. The important
thing is to get the research started and make the comparisons
so we know some of the answers, so we can come back here 2
years or 3 years from now and talk about this issue.
Senator Warner. You are the President's principal advisor,
and as I said, we are fortunate to have you. And I commend the
President even though I personally feel somewhat that we should
move a step further.
But the point is let us take one adjunct question, and that
is human cloning. I am totally opposed. The President is
totally opposed.
Secretary Thompson. So is the President, and so am I.
Senator Warner. Should we effect here in the Congress rigid
standards of law and indeed attach criminal penalties to that
one area?
Secretary Thompson. I think we should prohibit human
cloning, Senator.
Senator Warner. And indeed go so far as to attach criminal
penalties?
Secretary Thompson. That is a decision for the Senators.
Senator Warner. I thank the chair. I thank the witness.
The Chairman. Senator Mikulski?
Senator Mikulski. Thank you very much, Mr. Chairman.
Mr. Chairman, I would like to ask unanimous consent that my
opening statement be included in the record.
The Chairman. It will be so included.
[The prepared statement of Senator Mikulski follows:]
Prepared Statement of Senator Mikulski
Thank you, Mr. Chairman, for holding this important hearing
on stem cell research today. I am happy to have the opportunity
to hear from Senator Specter, Congressman Langevin, Secretary
Thompson, and from experts on this issue. The President had a
difficult decision before him, and I appreciate the
deliberative, responsible way he has approached it. I know that
stem cell research is an extraordinarily complex scientific and
ethical issue, and I am pleased that the President took such
great care in making his decision.
One of the reasons I became a U.S. Senator was to save
lives. Scientists tell us that stem cells may help lead to
cures for diseases ranging from Alzheimer's to diabetes. Stem
cell research could save lives. We owe it to every American
suffering from these diseases to explore the scientific
possibilities in front of us.
First, I share the President's opposition to human cloning.
I also agree that we should use our resources to investigate
the research possibilities of stem cells derived from adults
and from umbilical stem cells.
Yet I disagree with the President's decision to allow
Federal funding for embryonic stem cell research only in the
most narrow way. I'm concerned that he has created an enormous
loophole for researchers in private, profit making firms.
Embryonic stem cell research will go forward with or without
Federal funds. The President's decision leaves the majority of
embryonic stem cell research to private firms in an unregulated
atmosphere. My position is that there should be Federal support
for embryonic stem cell research. With Federal funds comes
Federal scrutiny, regulation, and transparency.
Stem cell research offers us a cornucopia of opportunity
that may lead to treatments or even cures for diseases like
Alzheimer's, diabetes, Parkinson's and more. Public funding--
and the public oversight that goes with it--ensures that this
research is conducted within a rigorous ethical framework. We
can have both sound science and sound ethics.
Again, I thank the Chairman for calling this hearing on
stem cell research. I look forward to working with my
colleagues and the Administration on this important issue, and
I look forward to hearing from our witnesses. Thank you.
Senator Mikulski. Mr. Secretary, I just want to really
welcome you today and thank you, in your 6 months in office,
for all the courtesy and collegiality and how responsive you
have been to many issues that I have raised with you. I want to
thank you for that.
I also want to thank you for inviting the women in the
Senate to meet with you to talk about a women's health agenda.
I must say that I am impressed with the mastery that you
have of the technical issues here. For a while there, I thought
you were defending a Ph.D. in biology.
Secretary Thompson. I think I could almost write one right
now, Senator.
Senator Mikulski. Yes, really. You can see that you have
really put a lot of work into this personally and are very
hands-on about it.
Let me go right to my question, which follows along the
line of Dr. Frist. First, in terms of President Bush's
position, we thank him for his deliberativeness. I too oppose
human cloning, support the adult umbilical stem cells, and am
glad that he took the next step in embryonic.
Let me tell you what my concern is. My concern is that the
Bush framework creates a loophole, and the loophole is that
embryonic research from discarded embryos will go on anyway,
but that it will go on within the private sector environment,
unscrutinized and unregulated, therefore lacking any type of
transparency.
The fears that I have about that are twofold--number one,
that big breakthroughs could come from those who hold the reins
of profitmaking rather than being in a large public domain and
therefore could not only profit but even profiteer by it.
No. 2, in an unregulated and nontransparent atmosphere, I
am also worried about some of those grim things that President
Bush is worried about. The biological revolution is stunning in
terms of its opportunities, but it is also stunning in the way
it can present to us a ghoulish, Huxley-like environment.
I wonder what you think about this loophole and the
concerns that I have about this, and where do you see us
heading in that, really, so much of the research could go on in
this unregulated, untransparent, very profitmaking environment?
Secretary Thompson. Senator Mikulski, first off, thank you
for your kind words. I appreciate it very much.
The President set up this Bioethics Commission headed by
Dr. Kass to do just exactly what you are talking about--to
develop the theories, encourage the adoption of laws, to make
sure that the profiteering and other problems that you have
indicated will not exist. Saying all that, before we get there,
I do not believe that the privates are going to do the basic
research; they never really have. The basic research is not
what it is going to get them the money. The basic research is
what NIH does; it is what Johns Hopkins does, it is what
University of Massachusetts and University of Wisconsin and all
the others do. They are the ones who are going to have to do
the basic research. Once that basic research is developed, I
believe that then, the private dollars are going to come in and
try to take that basic research--but we will have intellectual
property rights to that. Geron, WARF, NIH--the agreement we
signed yesterday, the MOU that we signed with WARF, allows us
to maintain our intellectual property rights on our discoveries
at NIH, which is exciting, and the publications that are going
to be going on in this area.
It is so important for us to get this research done. I
cannot scientifically tell you that there will not be
profiteering or a problem existing as you point out. I cannot
say unequivocally that that is impossible. I can tell you that
we are trying to contain any of those problems, and we are sure
that they are in the future and not right now. What we need to
do is get our best scientists to get the Federal dollars to do
that basic research that is just crying for discovery, I
believe.
Senator Mikulski. Thank you very much, Mr. Secretary. As I
understand it, the thrust of your presentation is that
essentially, this is a work in progress, the stem cell
research, both from an administrative perspective as well as
setting our bioethical standards. We have a benchmark.
Actually, we have broad agreement on a certain bioethical
framework. Much is made of the prickly distinctions, but by and
large, I think we are all agreed that we do not want grim and
ghoulish things to go on.
But as I understand it, while NIH begins its role, this
Bioetehics Commission will also commence its role and be
looking at the issues of the greater in-depth of a bioethical
framework as well as evaluating these breakthroughs. Am I
correct in that?
Secretary Thompson. You are absolutely correct.
Senator Mikulski. And in other words, we are going to be
continually reviewing it.
Secretary Thompson. Plus you cannot forget that Congress is
also very much involved in holding hearings on this subjects as
well, and making laws.
Senator Mikulski. But my point is that Congress will
continue to hold hearings based on now what NIH is doing and
also the work of this commission.
Secretary Thompson. Right.
Senator Mikulski. But essentially, this is the framework
for embarking on this biological revolution.
Secretary Thompson. That is correct.
Senator Mikulski. Now a technical question--and I think
that is good; I think we are going to have to hold periodic
hearings on this--these stem lines that we have talked about,
do they have a shelf life, and if they do, this could present
great dilemmas for the Bush position.
I take the position of being allowed to do research on
discarded embryos where there has been informed consent to do
so and oppose creating embryos for therapeutic reasons. But
what do we know about the shelf life, or should we save it for
the ``bio gurus'' here?
Secretary Thompson. We know that for 3 years, the first
embryonic stem cell line is still replicating. Dr. Thomson says
that he believes they can continue to replicate indefinitely,
and other scientists have said the same thing. I am not a
scientist, and I have to take what they tell me as the gospel--
but I believe that they will.
Now, can I say conclusively that there is no shelf life? I
cannot say that. I can tell you that they are usable as long as
they are not differentiated. When the cell starts
differentiating into a particular part of the body, then, like
the hematopoietic blood cells--what has taken place at the end,
after the embryonic stem cell line has been frozen, they have
taken the cells out, and they have then differentiated those
cell lines into blood cells, which is really exciting, creating
blood, white corpuscles, red, and platelets. That to me is
truly exciting, and it is a tremendous breakthrough, but
whether or not it is going to be able to be used in therapy
within 3 years, 5 years or 6 years, I do not think anybody can
say at this point in time, Senator Mikulski.
Senator Mikulski. Yes. There are scientists, many of whom
are involved in cellular research, at Hopkins who apprise me
that they do believe that these cells do have a shelf life. But
I think that you are saying that the science is so new, we are
going to have to see this, and should it have a shelf life,
confront those issues, because both the Commission and NIH will
have had a chance to do their work.
By the way, how are we doing on getting new heads of NIH
and FDA? You have great acting directors.
Secretary Thompson. I have made my views known to Senator
Kennedy and to the White House, and I will hopefully hear good
news from both of those people.
The Chairman. I hope to have good news, too.
Senator Mikulski. Well, like E.F. Hutton, I cannot wait
until I hear the news since both of these flagship agencies are
located in my State.
Secretary Thompson. I know. I am as anxious as you are,
Senator Mikulski.
Senator Mikulski. Thank you, and let me know if I can help
move the process along--again.
Secretary Thompson. I appreciate that.
Senator Mikulski. Ted, can we talk later?
The Chairman. We will talk. [Laughter.]
Senator Murray?
Senator Murray. Thank you very much, Mr. Chairman, and
thank you for having this hearing.
Clearly, this is a very complex issue, and I appreciate all
the knowledge that you have developed on this and the
considerable amount of effort you have taken on a very
difficult subject. Certainly the public is very, very
interested in what we are doing and how we are moving forward
from many different perspectives--obviously, from moral and
religious, but also from a perspective that they do not want us
limiting what we can do in the future in terms of life-saving
processes that can be developed from what we set out here
today.
I share the concern of Senator Mikulski in terms of private
research. You have said very clearly that the administration
has said that there will be no further destruction of embryos
after the August 9 deadline, and I am worried about what kind
of impact this will have on our research. I understand the
commission that you have set out and that you see the Federal
Government and NIH doing sort of basic research and then
allowing private interests to move further. But you yourself
know that private industry has a different motive than the
Federal Government, which is why we have NIH and why we do
research and why we put money into it. We can do the basic
research with the best of intentions that the development will
occur for diseases like multiple sclerosis or Parkinson's or
other diseases, but private companies may well see that
marketing and profit comes from male baldness and may set aside
what we see as a public interest.
I am very concerned that a commission set up to look at
bioethical standards is not going to--they cannot determine
what profit means, but that is what private industry will do.
How do you reconcile this August 9 deadline with what
private interests are going to want to do once we have done the
basic research?
Secretary Thompson. I clearly can reconcile it because the
private sector would not do the basic research that I think is
so important. I do not think the private sector would spend its
time or money making the comparison of embryonic stem cells
versus adult stem cells versus placenta versus cord blood. I
think that that kind of research needs to be done for you and
for me to make the proper decisions, and I do not think the
private sector will do that.
That is why, prior to August 9, the Federal research was
not being done. The President----
Senator Murray. But you are limiting us to that basic
research rather to what we can develop maybe in the future, 2,
5, 10 years from now, on specific diseases.
Secretary Thompson. No, I am not, Senator Murray, because
the MOU that we signed yesterday with WiCell allows us to have
the five cell lines available right now for research and that
the scientists at NIH will be able to keep their intellectual
property rights and will be able to publish on what they
discover. That may be a therapy on baldness, as you have
indicated--I do not think that that is what we are that
concerned with right now; we are more concerned with----
Senator Murray. But it may be the best marketing and the
best profit for somebody.
Secretary Thompson. But that is for the private sector.
Senator Murray. Right, but we have to protect the public
interest, and my concern is that having an August 9 deadline
set, with no intention of ever going back and looking at it
again, may very well preclude some of the important research we
talk about at every hearing we have on this, that we tell our
constituents that it may be available in the future for
diseases that touch many, many families, that it will never be
developed because we had a hands-off approach once the basic
research is completed.
Secretary Thompson. I cannot answer that question except to
say that we think there is ample supply of embryonic stem cell
lines to do the basic research for the areas that you are
concerned about for your citizens and that I am concerned about
for all Americans and that the President is concerned with. He
has just drawn a moral line, an ethical line, saying that there
will be no further destruction of embryos that will get Federal
funding of dollars. And that is probably something that you and
I would never agree on.
Senator Murray. And I guess I am just stating a concern
here.
Well, how do you see this affecting university research,
which is often a collaboration of both private and Federal
dollars?
Secretary Thompson. I think that that is going to have to
be worked on, but I am confident that----
Senator Murray. How will it be worked on? Do we have a
process in place to look at that?
Secretary Thompson. We do not as of yet, but we are working
on it, Senator Murray. As you know, 75 to 80 percent of NIH
dollars go back to universities for research dollars.
Senator Murray. Right, and it is often used in
collaboration with private money. If private money uses stem
cell lines that were developed after August 9, will we be
precluded from doing collaborative research at universities on
that?
Secretary Thompson. That question has not been raised,
Senator, and a decision has not been made.
Senator Murray. I think it is an important one.
Secretary Thompson. I cannot answer that.
Senator Murray. Thank you, Mr. Secretary.
Thank you, Mr. Chairman.
The Chairman. Thank you very much, Senator Murray.
Senator Clinton?
Senator Clinton. Thank you, Mr. Chairman, and thank you,
Mr. Secretary.
I think, as probably all of us feel after these couple of
hours together, there are a lot of unanswered questions which
we will look forward to struggling through with you and those
with whom you consult because clearly, as Senator Warner said,
this is an issue that has such great importance and resonance
not only throughout our country but throughout the world.
I am still at the level of acquiring information and trying
to get it straight, because there are just a lot of
contradictory attitudes that are being expressed about what we
have and what we do not have. I think it is important that we
have a factual base.
You ere not here, Mr. Secretary, for Senator Specter's
testimony, but I am sure that your staff will fill you in and
give you a copy of it. He certainly takes the position that is
I think somewhat different from the one that you have expressed
about what the viability and accessibility of the stem cell
lines are.
We need to come to some basic agreement about what the
evidence shows, and I hope that you will work with us on that,
because certainly, Senator Specter and Senator Harkin have held
a number of hearings on this issue going back now nearly 2
years, and Senator Specter has a different basis of information
available to him than what you have presented. So I hope that
we can work through that so we all know what we are talking
about.
Second, the serious question that has been raised about the
potential impact, if not contamination, from the mouse cells--
and also, I believe blood serum from cattle is also used to
nourish these cells in some of the settings--is one that we all
have grave issues about. We are concerned that our Government's
own standards about how to treat the introduction of animal
elements into human material have not necessarily been followed
in the various locations where we have identified these stem
cells. So that clearly, we have a lot more work to do on that,
because as you point out, the research is going to take a
number of years, and I think many of us would be very
disappointed if, at the end of all those years, the work could
not be useful to the alleviation of human suffering because of
the contamination that affected these cells.
One other question that I need some guidance about is in
the President's speech, I believe he said he supported the
House bill. As I understand the House bill, it not only bans
reproductive and therapeutic cloning in the United States and
certainly any Federal involvement in such efforts, but it goes
a step further and says that if, in England or Germany or
Japan, any therapy or treatment is created from therapeutic
cloning, that treatment could not be imported into the United
States.
Am I understanding that correctly in both the House bill
and the President's position?
Secretary Thompson. Senator Clinton, I have not done any
research on that subject, I am sorry to say, so I do not
particular want to answer that question at this point in time.
I would be more than happy to do the research and call you or
write you with an answer on that.
But I would like to hopefully make a correction, and not to
be confrontational. On the contamination, I do not believe that
there is contamination. Just because these cells rest on a
mouse layer of cells does not mean that they are contaminated.
I have talked to FDA, I have talked to NIH, and it is such
a traumatic experience for these cells to be taken out of the
embryo that they have to have this cushion----
Senator Clinton. Mr. Secretary, I understand that, but the
point that was made this morning--and I am just trying to get a
base of information so that I can make the best possible
decisions--but as I understood the chairman's point this
morning, our Government has issued guidelines about how mouse
nutrients are to be injected into any kind of human material.
So that clearly, we know what happens--the xenotransplantation
efforts that you referred to are ongoing. I read the letter
that was distributed. Frankly, it still is a little concerning
to me, because the long list of steps to be taken to guard
against any kind of untoward consequences of
xenotransplantation is a little bit daunting to me. But putting
xenotransplantation to one side, as I understand what the
chairman said, our Government has issued very strict guidelines
about the introduction of mouse nutrients into the human
material, and we have no guarantee that these stem cells in
India or in Sweden or anywhere else have followed the American
Government's guidelines, do we?
Secretary Thompson. We do not.
Senator Clinton. We do not. So I do not think any of us
wants to be confrontational. We are trying to understand what
it is we are doing.
Secretary Thompson. I just----
Senator Clinton. So if our Government has rules about how
to avoid contamination that they have issued to researchers
about how to use mouse nutrients, I think it is a fair
conclusion to draw that in the absence of following those
rules, some might conclude that contamination has or could
occur. That is my only point.
So my only point is that as we go forward to try to
understand what it is we are doing here, we need to be open. We
cannot close our minds. This is not an inquisition. This is not
trying to determine who is right and who is wrong. We are
trying to figure out what we are doing. And there have been
many questions raised since the President's speech that deserve
an answer. The scientific community deserves an answer, the
Congress deserves an answer, and certainly people who are out
there wondering about this deserve an answer.
So on those several points, I would appreciate additional
information.
Also, as we look at this, the question that was raised by
Senator Mikulski, which I think is a very important one, is
that we have chosen to adopt a different approach than Great
Britain, for example. As I understand it, they have an
Embryonic Research Advisory Board that attempts to govern both
public and private sector investments in this research. We have
adopted a different approach. We have adopted these
restrictions on Federal funding and very few on private
funding. And I take seriously your point and happen to agree
with it that the basic research is likely to be done with the
public sector dollars. That, though, raises some of the
concerns that scientists have suggested to me, that is, that it
is very difficult to do the kind of basic research without
those public dollars. That really is the core of whatever
therapeutic use can come from this research.
So that we are concerned, I think, about the potential
shelf life of these stem cell lines, the specialization that
can occur, rendering them useless for the research, the
numerous references to the mouse lines that have been
developed--we do not know how many mouse lines it took to get
those that are now replicable and usable. We are really out
there in the dark, trying to figure it out. If we are going to
make an analogy to the mouse lines, we ought to have as much
information as we can to know if it was 1,000 mouse lines that
eventually produced five viable mouse lines, or was it 64 mouse
lines that produced five viable mouse lines. I do not have any
idea.
So I think that every one of us is grateful for the hard
work that you and the NIH and the professional staff have done,
and we are grateful that the President had a deliberative
process that led at least to the door being opened. But as we
learn more about these 64 stem lines, as we try to figure out
how to reconcile Senator Specter's very strong statement--it
was a passionate statement this morning about what he believes
to be the facts about these stem lines--with your very
optimistic statement about the stem lines and the fact that if
we go with the optimistic view, which is that this is all we
need, and this is all we are going to get, how many years do we
lose, what do we give up--the questions that Senator Warner
raised.
So I think, Secretary Thompson, that many of us are still
in the asking questions phase, and if I may, just one final
question. You have said several times, and you said in response
to Senator Murray that you envisioned private researchers
taking up where the publicly-funded basic research leaves off.
What thought has been given to how these private researchers
will be able to overcome the proprietary rights issues as they
engage in commercial development? With the kind of memorandum
of understanding that you have reached between the Government
and the research institutions--how will the private researchers
be able to do that?
Secretary Thompson. Senator, first off, I did not mean to
imply anything. I just did not want to leave this hearing
saying that all of these lines are contaminated, and I do not
think they are.
Second, you raised a lot of points. I do not know the
answers. I can tell you that I have been involved in this thing
since a month after Jamie Thomson discovered--first as a
Governor; I have been an advocate and a passionate believer
that this shows great promise, and I still believe that.
We have to do the research, and we have to do the basic
research as you have indicated, to find out how can these cells
be used. Adult stem cells may be more usable, more placid, than
embryonic stem cells. That comparison has not been made; that
has to be done.
In regard to the patent rights, I have talked to the people
at NIH, and they tell me that this is no different, that the
MOU and the statement of material transfer allow for this
research to continue. The intellectual rights, the research
writing of the scientists, will be able to continue and will be
saved for NIH; but once they develop a product, like they do
any other product, that does not use embryonic stem cell lines,
the private sector and the patent laws and the commercial laws
of America take over, and they will have to negotiate with
WiCell or get a license to use that. But that is no different
than any other product that has been developed by NIH or by the
private sector when there is an already-existing patent out
there.
Senator Clinton. Thank you.
Thank you, Mr. Chairman.
The Chairman. Thank you very much.
Senator Edwards?
Senator Edwards. Good morning, Mr. Secretary.
Secretary Thompson. Good morning, Senator Edwards. How are
you?
Senator Edwards. I am fine. We appreciate your passion
about this issue and the work you have done on it in the past.
There were a number of us who had concerns when the President
made his speech about the rigid guidelines that he established,
understanding that there are serious scientific and ethical
questions that have to be resolved, and how those guidelines
would be applied in the context of a very new science. I think
all of us recognize that this is a very new and rapidly
developing science.
Secretary Thompson. And very exciting.
Senator Edwards. And very exciting, potentially very
exciting.
I think our concern is that we want to make sure that that
potential in fact is realized. At the time the President spoke
and shortly thereafter when you talked about the subject, you
talked about 60 some-odd stem cell lines being available and
the nature of the research that you thought could be done based
on those stem cell lines. Let me ask you first are you aware--
have you actually been on site where these stem cell lines are
located--and we have a list of where they are across the
country and across the world. Have you actually been on site
and examined the stem cell lines?
Secretary Thompson. No, I have not. I have been at the
University of Wisconsin lab many times, but I have not been
anyplace else, if that is your question.
Senator Edwards. The American Society for Cell Biology has
established four criteria that they think should apply in this
context in order to satisfy the research that needs to be done.
The first is that the cell line be available to publicly-funded
scientists both at the NIH and at universities around the
country.
The second is that the owners of the stem cell lines not
impose restrictions that would limit that research.
Third is that they have growth and handling characteristics
that are compatible with quality research.
And fourth is that they retain the capacity to generate
every adult cell type.
They are pretty simple, actually. One is to make sure it is
available to the scientists who need to do the research;
second, that there are not legal and other restrictions that
would limit the ability to do the research; third, that the
stem cell lines themselves are usable for the research that
needs to be done; and fourth, that they have the capacity to
generate the adult cell types that need to be generated.
Do you know as you sit here today how many of the stem cell
lines that meet your test also meet those four simple tests?
Secretary Thompson. I know that 64 meet the President's
guidelines in regard to Federal funding. There are 64 that we
have inquired about and re-inquired about and have met with
personally.
Senator Edwards. Can I interrupt you--do you think that
those are reasonable tests, the tests that they lay out, those
four simple tests?
Secretary Thompson. I think that those four are adequate,
and I think that they will support----
Senator Edwards. You think they are reasonable.
Secretary Thompson. They are reasonable.
Senator Edwards. OK. Can you tell me as you sit here
today--and you and the President have already established the
limitations--can you tell me as you sit here today how many
stem cell lines meet those four tests?
Secretary Thompson. Dozens.
Senator Edwards. What does that mean, ``dozens''--25, 30?
How many?
Secretary Thompson. I would say--dozens--I would say 20 to
25 at this point in time. But--you were not here--there are
three stages that these embryonic stem cell lines go through.
The first one is the proliferation, and there are several of
those 64 lines that are in the proliferation line; there are
several in the characterization line, which is over here; and
several are in the ``cell line established.'' It takes 6 to 8
months to develop a good embryonic stem cell line, and there
are some of these that have not met the full cell line
establishment criteria yet--they will be by the time they apply
for the money, and the money goes out at NIH. That is going to
take somewhere between 8 or 9 months to get the money out.
To give you an example, in Sweden, Gothenburg University
has 19 lines, Senator Edwards. Three of them are in the ``cell
line established,'' which means they are ready right now for
research and that further research could be done. Four of them
are in characterization, and 12 are in proliferation.
Over the course of the next 6 to 8 months, several will
move from proliferation to characterization to cell line
established, and that is during the process that they will be
applying for Federal research dollars.
To tell you exactly today how many, I cannot tell you. If I
were pressed, I would say there would be 24 to 25 that would
meet the ``cell line established'' criteria, Senator Edwards.
Senator Edwards. That leads me to my next question. When I
originally asked you, you said ``dozens,'' and then, when I
asked you a follow-up question about it, you said ``24 or 25,''
something in the----
Secretary Thompson. That is what----
Senator Edwards. How do you know that those 24 or 25 meet
this reasonable, what you described as reasonable criteria that
the cell biologists have established?
Secretary Thompson. Basically, because the MOUs that we
have discussed with the 10 entities that have them have
indicated they want to share with the scientists; two, that
they can be used; three, that they replicate or are
pluripotent, which is the scientific word for that. And I do
not know what the fourth one was, but as I understand it, they
meet those four qualifications.
Senator Edwards. Let me ask a follow-up question to that.
How many genetically diverse stem cell lines, again as we sit
here today--not the potential, but that we actually know
about--have the ability to regenerate themselves indefinitely?
How many do you know about today?
Secretary Thompson. We think all 64.
Senator Edwards. OK. So it is some of the other tests that
you are not certain whether they are----
Secretary Thompson. Yes. The other characterizations, the
other things like the freeze-thaw testing, the cell marker
assays, which are SSEA-3's and 4's and PTA-61's and 80's, and
the chromosome analysis, and the testing for pluripotency--we
are not sure about all of those things. But we do know that in
order for this to be proliferating, they have already started
proliferating right after they have been removed from the
blastocyst and are in the cells in the culture dish. They are
proliferating right then. So all 64 lines have proliferated; we
do know that for a fact.
Senator Edwards. But you, HHS, NIH, have not physically
been to the sites to actually examine the cells yourselves?
Secretary Thompson. No.
Senator Edwards. Do you plan to do that?
Secretary Thompson. I probably will not, but I know the
scientists will be doing an exhaustive study, which they
already are. We are in the process of setting up the registry.
We have signed--you were not here--but we did sign an MOU and a
Material Transfer Agreement yesterday with Wisconsin Alumni
Research Foundation that allows for their five embryonic stem
cell lines to be made available to the NIH scientists right
now.
Senator Edwards. Are those five lines available to the
nonNIH scientists around the country, publicly-funded
scientists?
Secretary Thompson. Yes. Carl Gulbrandsen is here, and you
can ask him that question, but he has indicated that, yes, any
scientists who want those lines----
Senator Edwards. And what do we know about the availability
of all the other stem cell lines?
Secretary Thompson. All we know--all I can tell you,
Senator Edwards, is that the entities have been in to meet with
Dr. Lana Skirboll and her staff, and I have personally met with
them. I have not negotiated with them, but I have met with
them, and they have all indicated to Dr. Lana Skirboll and to
the staff at NIH that they want to make their cell lines
available to scientists and for NIH to get the basic research
done. They are excited.
Senator Edwards. But at this point, who controls that
decision? Do they control it?
Secretary Thompson. The entities, the entities.
Senator Edwards. We as a nation have left to them control
over what kind of research can be done on these cell lines?
Secretary Thompson. No, no. They own it; I mean, they have
developed them, so they have control over them. And now what we
are doing is----
Senator Edwards. So we have created through this decision
by you and the President a monopoly in those people who have
existing cell lines. If I understand you correctly, what you
are saying is that they now have control over what research, if
any, can be done by--it is all well and good that they are
well-intentioned about it--I appreciate that--but suppose they
decide that no, in fact, they are not going to permit the
research to take place, or they are only going to allow the NIH
to do it, they are not going to allow the other scientists to
do it?
Secretary Thompson. That is not what I have said, Senator.
What I have said is that they have all indicated that they are
in agreement and want to cooperate not only with NIH but with
other scientists to do the basic research on their stem cell
lines.
WARF has gone a step further. They have negotiated now with
NIH an actual agreement which was signed yesterday with NIH to
make their five cell lines that Jamie Thomson, the father of
embryonic stem cells, has developed. He has licensed those to
WARF; WARF in turn has licensed them or has given the
permission to be used by NIH and other scientists through an
MOU and a Material Transfer Agreement for any type of research
that scientists want to do on those.
The second thing that we are doing is developing a registry
at NIH, making all of these 64 lines hopefully available for
scientists around the world to be able to pick the line that
they want to use for their research projects when they apply
for Federal research dollars.
Senator Edwards. The critical word there was ``hopefully,''
and I know my time is up, but my obvious concern is--and I
think it is a critical issue in this ongoing process--that by
establishing the criteria that the President established, he
limited the number of lines that are even potentially available
and left in the hands of the people who presently have the
proprietary control of those lines the decisionmaking about
whether they are going to be publicly available. We cannot
force them to make them available; they have a legal right to
do whatever the choose to do. And I appreciate that they are
well-intentioned, but it certainly has not been an unusual
thing in the past to have people act out of their own personal
financial best interest, and it would not be a shocking thing
to see that happen in this case.
So I think the restrictions that have been imposed
obviously create ongoing problems. I am hopeful with you that
we will be able to get these folks to make these stem cell
lines available, but I think it is a serious question as we sit
here today.
I thank you, Mr. Secretary, for your answers.
Secretary Thompson. Thank you. All I can tell you in
answer, Senator Edwards, is that we have five--the first one we
have negotiated with, which is the one that has the patent in
America, is the one we have reached an agreement with, and we
feel very good about that, and we think the other ones will
follow through.
I cannot promise you today that we are definitely going to
have them all signed up next week, but we are working on them,
and we are working as fast as we possibly can to reach an
agreement with them.
Senator Edwards. Thank you, Mr. Secretary.
Thank you, Mr. Chairman.
The Chairman. Thank you.
Mr. Secretary, I will just make a very brief comments, and
if others wish to ask a final question, obviously, they are
welcome to.
No one could sit through the course of this hearing since
9:30 and not know of your own very strong commitment in terms
of research in this area and your belief that this offers some
enormous opportunities and hope for families across this
country in regard to these dreaded diseases that afflict every
family in America--every family in America. That is very
commendable. The President is fortunate to have the kind of
advocate and advocacy that you have expressed here.
There are those who view this situation somewhat
differently, and we are here trying to listen as we have
carefully to your presentation, made in a very convincing way.
Will there be the availability of stem cells for the research
and the opportunities in an area that was really unthought of,
or maybe thought of but not in reality until 2\1/2\ or 3 years
ago, and with the best of the research that is done at NIH.
Will there be sufficient product out there to permit
opportunities for breakthroughs?
You make a very convincing and compelling case that there
is, and I think there have been some legitimate issues and
questions raised that cause us some concern. I think one of
those issues that I raised very briefly, as others have, is the
question of the conditions of safety and the issue of the use
of nutrients from mice and the fact that in these various stem
cell lines that you talk about here, you would be hardpressed
to think that any of those followed the very strict guidelines
that were issued on August 24th of this year on mice, from a
colony that is continuously monitored for infectious disease.
We know about mad cow disease; no one expected that that kind
of infectious disease would be able to move from animals to
human beings from a colony that was not allowed to interbreed
with animals outside the colony and was routinely tested for
infectious diseases and quarantined for 3 weeks prior to the
procurement of cells.
Now we are told that we have to stay with these cells; even
if we are able to take mice and create new stem cell lines
further after August 9 that would meet all the requirements
that the administration and CDC have put out, you are saying,
``No, no, you cannot do it. August 9th is it.'' And even if we
are able to discover after 2\1/2\ years that we may be able to
do it without nutrients from mice--maybe other kinds of
nutrients would save us that kind of risk--we are told that we
cannot do it because it is after August 9.
We commend you for the arrangements that you have made in
being able to get one of the ten suppliers to make a strong
commitment to make available their stem cell lines, and we are
impressed by your own work in the past and saying that you have
been in touch with all the others, and you believe that they
will make them available. I think most of us would say that no
one underestimates your ability to talk to someone and get an
understanding and form an impression about whether that is for
real or not, and I have a lot of respect for that. I think many
would say we ought to get it in writing, because what we have
seen as I understand it from similar situation is that there
has not been the kind of readily available access when similar
situations have been out there in terms of research at the NIH.
I think most of us have been around here long enough to see
what OPEC was able to do to our economy and the impact that it
has had on our society when they get together, 11 of them. And
we are talking not about 11 countries here, we are talking
about some countries, but basically 10 entities, and that
raises concerns.
So these are all important issues. We had comments about
the patents, and you have commented about that, and I know you
have worked on it. So these issues do raise some serious
questions, but as far as I am concerned, I want to take what
you have said here today and hear from our next panel their
reactions, and then to work with you.
I do find at the end of the day that an August 9 deadline,
sort of a drop-dead date, and the unwillingness, in spite of
what follows in terms of breakthroughs in the areas of
research, would serve as a barrier to important progress in
terms of cancer or Alzheimer's, very troublesome.
But we have learned a good deal this morning, and I have
been enormously impressed by your presentation. We will keep in
close touch with you on this issue. We want no surprises--there
could not be in any event. We want to work with the
administration. We again thank the President for addressing it.
It is just a desire to make sure that we are going to be able
to get done what he has said he wanted done and what I think
offers the greatest hope for the American people.
Senator Dodd?
Secretary Thompson. Can I just say thank you, Senator
Kennedy, for your tremendous interest and passion on this
subject. We may not totally agree on whether or not the August
9 deadline is correct or not, but I think we both agree that we
need to get the research done and the questions answered----
The Chairman. Yes, yes.
Secretary Thompson [continuing]. And I thank you for your
passion and your advocacy on this issue, and I want to publicly
State that I support that and want to work with you as much as
I possible can to accomplish that.
The Chairman. Thank you very much.
Senator Dodd. Mr. Secretary, just to pick up on some of
Senator Kennedy's comments--and Senator Clinton used the right
word--this is not an ``inquisition'' at all. Obviously, there
is deep interest in the subject matter not only here, obviously
in the scientific community, and as you have heard, painfully,
many cases from people at-large around the country who do not
understand all of this. The news media may be in a sense hyping
a bit what the potential is, but understandably, when you have
a family member or a loved one who is suffering, and you hear
about the potential to be able to deal with Alzheimer's or
diabetes and other such illnesses, it is very, very exciting.
I for one want to thank you. You are in the box here, and
we are raising the questions to you, but I think you deserve a
great deal of credit for bringing this as far along as you
have. There are some of us here who would have liked to see the
administration go a bit further with this, in my view--not that
I wanted to see it all; I strenuously oppose the idea of
somehow creating what someone once used the word
``hatcheries''; I would vehemently oppose that--but I also
understand that with the ability to provide people with
families through in vitro fertilization, and that there are
excess embryos here--and I raised with issue you earlier as to
whether or not there is then going to be a position taken on
banning the destruction of those excess embryos; and if there
is, I suppose that would be consistent with the view that they
cannot be used. If it is not, it raises the quandary--if you
are not opposed to their destruction, how could you be opposed
to using them, so that if in fact these other lines that
hopefully are going to do exactly what you have described do
not pan out, we have another source there to deal with.
But I think you have done a tremendous job, and you are
right--we now have a position taken by this administration to
publicly finance research, having drawn lines--and I have
difficulty with the arbitrariness of August 9 and 9 p.m.--but
nonetheless, this is breakthrough, this is good, and we ought
not allow this hearing to end on a note that somehow, while
there are still some disagreements over whether or not after
August 9, there are some additional embryos to be used--this is
very positive.
So I did not want the hearing to end without expressing my
gratitude to you. I suspect you had an awful lot to do with
this, the fact that there was that speech on August 9, and I
suspect you had a lot to do with the fact that there was some
breakthrough here, moving the line a bit further than I suspect
some inside the White House wanted it to be moved. You do not
have to answer that; I just have my own suspicions about it,
and I am grateful to you, and a lot of other people are.
And I know that you have a first-rate team. I cannot tell
you how excited I am about the work that is being done in adult
stem cell research. I have had some wonderful conversations
with my friend from Tennessee who has been enlightening me on
cord blood stem cells and what that can mean. And at NIH, I
have had wonderful conversations with the acting director out
there about how realistic cord blood stem cells might be.
Nonetheless the work that is being done is very exciting.
So I thank you for being here today and thank you for the
work that you have done.
Obviously, some of the questions that have been raised,
Senator Clinton's questions and Senator Kennedy's, we need to
follow up on pretty quickly, because if it turns out that you
are wrong, and we cannot extract these things, we will have to
revisit this issue. I know you do not want to admit that this
morning, but the only thing you said that worried me is that we
will never reconsider the August 9th date, and I am not going
to ask you to repeat that because I like to believe you might
want to rethink it. So if we have to come back to that, we will
have to come back to it, and we will talk to you when that
occurs.
Thank you.
Secretary Thompson. Senator Dodd, thank you, and I thank
all the members of the committee.
I will tell you this has been a great discussion. The
beauty of it is that this holds so much promise for everybody
out there who has a loved one suffering from breast cancer or
dementia or anything else and is waiting for the possibility.
We just cannot get the information out there that the cure is
just around the corner; we have to get this basic research
done.
Senator Dodd. I agree.
Secretary Thompson. The President has allowed that to
continue. I am excited about it, and I know that you are,
Senator Dodd, and I thank you very much for all of your
comments and all of your questions today.
The Chairman. Senator Frist?
Senator Frist. Mr. Chairman, if I could have just 2
minutes. I again want to thank Secretary Thompson and the
President for a lot of thought, a lot of consideration, a lot
of individual meetings--and this issue deserves it because it
is, again, as we look at science one of the few issues that we
have had to face, that humanity has had to face, that has the
opportunity for both promise, altering the basic building
blocks of life, but also that could have unintended
consequences. I want to thank you for that.
I do believe that we should expeditiously implement the
policy put forth by the President, which is carefully crafted.
As this moves forward, it will be important, again because
science changes so rapidly, that we continuously reevaluate
both the progress and the needs of this research.
If there is one thing that has come out from your comments
and the comments in the last several weeks, it is that this is
an uncharted area of scientific inquiry. This is clear whether
we are talking about the number of necessary cell lines or
about the mouse cells and xenotransplantation issue, which we
have addressed in one context of transplanting whole organs but
not quite as much in cells. It demands an ongoing public
discussion among the policymakers, like those here today, the
scientific community, whom we will hear from shortly,
ethicists, the religious leaders, and the American people. It
is absolutely critical to have that discussion on an ongoing
basis--not just today, but on an ongoing basis--as science does
change and progress.
We are going to have to wait several years before we know
whether embryonic stem cell research is going to yield the
promise that we all hope it will. In the meantime, I believe we
should move forward expeditiously in implementing the
President's policy and continue to examine the progress closely
over the coming months and years.
Secretary Thompson. Thank you.
The Chairman. Thank you very much.
Senator Clinton?
Senator Clinton. Secretary Thompson, I want to thank you as
well. I would not want you to leave thinking that because I and
others have raised some very tough issues, we do not totally
respect what you are doing and have done.
I also want to thank our colleague, Senator Frist, because
he also has had some things to say and do that have led us to
this point, and we are very grateful for that.
But we need this kind of open, honest dialogue. This is a
decision which, because of our living in a global media age, we
are bringing in millions of people to be part of. It is not
going to be made by scientists in a closed lab or by Senators
behind closed doors. This is a society decision that has to be
addressed in that way, and I know, given your background and
what you have done with this particular issue and in general,
that you understand that and will be working to lead public
opinion as well.
Thank you very much.
Secretary Thompson. Thank you.
The Chairman. Thank you, Mr. Secretary.
Secretary Thompson. Thank you.
The Chairman. The hour is late, but we will try to get
started with our panel, if we could. We will invite Dr. Douglas
Melton, who is chairman of the Department of Molecular and
Cellular Biology at Harvard University and a leading stem cell
researcher and has made numerous important discoveries in
diabetes research. Dr. Melton was honored for his outstanding
accomplishments in medical research by being named to the
National Academy of Sciences in 1995.
Ms. Karen Hersey is senior counsel for intellectual
property at the Massachusetts Institute of Technology. She can
speak from extensive professional experience on the
complications that can arise in seeking access to essential
medical research materials.
Dr. James Childress is the Edwin Kyle Professor of
Religious Ethics at the University of Virginia. Dr. Childress
served with distinction on the National Bioethics Advisory
Commission whose report on stem cells is a thoughtful guide to
the complex ethical issues raised by this research.
It is a special pleasure to welcome Dr. Childress, who was
formerly the Joseph P. Kennedy Senior Professor of Christian
Ethics at Georgetown University and has been a great leader in
all areas of bioethics and is someone whom I have respected
over a long period of time.
Dr. Kevin FitzGerald is the Doctor Lauler Professor for
Catholic Health Care Ethics at Georgetown University and an
associate professor of oncology. He has written extensively
about the moral-ethical issues raised by new advances in
medicine.
And Dr. John Chute conducts research on adult stem cells at
the Bethesda Naval Medical Center. I believe that research on
adult stem cells should proceed in parallel with a vigorous
research program on embryonic stem cells. We welcome his
testimony on this important topic.
It is a pleasure to have all of you, and we look forward to
moving ahead.
I will ask Dr. Melton if he would be good enough to start.
STATEMENTS OF DOUGLAS MELTON, THOMAS DUDLEY CABOT PROFESSOR IN
THE NATURAL SCIENCES, AND CHAIR, DEPARTMENT OF MOLECULAR AND
CELLULAR BIOLOGY, HARVARD UNIVERSITY; KAREN HERSEY, SENIOR
COUNSEL FOR INTELLECTUAL PROPERTY, MASSACHUSETTS INSTITUTE OF
TECHNOLOGY; JAMES F. CHILDRESS, EDWIN B. KYLE PROFESSOR OF
RELIGIOUS ETHICS, UNIVERSITY OF VIRGINIA; FATHER KEVIN
FITZGERALD, DOCTOR LAULER PROFESSOR FOR CATHOLIC HEALTH CARE
ETHICS AND ASSOCIATE PROFESSOR, DEPARTMENT OF ONCOLOGY,
GEORGETOWN UNIVERSITY; AND DR. JOHN P. CHUTE, HEAD,
HEMATOPOIETIC STEM CELL STUDIES SECTION, NIDDK, NAVY
TRANSPLANTATION AND AUTOIMMUNITY BRANCH, NAVAL MEDICAL RESEARCH
CENTER
Mr. Melton. Good afternoon, Chairman Kennedy and Senator
Frist. Thank you for inviting me to speak here today about
human embryonic stem cells.
In the last 3 years, the potential of these cells has been
widely debated in the public, and rightly so. The subject
forces us all to revisit the question of when life begins, and
we have to scrutinize the crossroads between scientific
inquiry, our efforts to improve the human condition, and our
moral and ethical responsibilities to preserve human dignity.
I am not here to testify on the moral, religious or
political aspects of this research. I appear before you as a
scientist and as the father of a young boy with Type 1 or
juvenile diabetes. I will furthermore not speak to you about
the human burden of a diabetes and will simply say that I work
on human embryonic stem cells to try to treat or cure diabetes.
My remarks today will therefore be confined to the scientific
potential of these cells and the implementation of the
President's plan about which we have already heard.
In my written testimony which I would like submitted for
the record, I summarize the properties of embryonic stem cells
and put that research in a larger context of recent biological
activities and studies at the NIH.
Let me just say now that the ability of these cells to make
any part of the body is what holds their promise for therapies.
We have already heard about the numerous diseases that can be
potentially treated--Parkinson's, Alzheimer's, osteoporosis,
and the disease that has my full attention, juvenile diabetes.
And I would simply like to say at this point that while adult
stem cells have some similar properties, based on what we know
today, adult stem cells do not have all the properties of
embryonic stem cells.
To give an example, there is no credible evidence for the
isolation or growth of an adult pancreatic stem cell, and that
alone justifies, in my view, the work on embryonic stem cells.
I do not need to remind you all that the President made an
important speech on August 9, and I would simply like to
comment on that date as I feel it has important implications
for the implementation of his plan.
That date was not chosen for scientific reasons, and its
arbitrary selection will unquestionably have an effect on the
progress of research. For example, as we have already heard, it
will not be possible for federally-funded researchers to
explore new ways to derive human embryonic stem cells, stem
cells that have a broader genetic diversity, or perhaps were
grown in the absence of mouse tissue, for example, grown with
human as opposed to mouse tissue.
Nevertheless, as Secretary Thompson has rightly pointed
out, the door has been opened, and some research can now be
done. I would like to address two issues and make a proposal
for the implementation of the President's plan.
One of the things I feel the committee has struggled with
is the lack of information about these 60-plus cell lines, and
legitimate questions were asked about them. Let me simply say
that scientists are by their nature inquisitive and skeptical,
and we hold dear the practice of publishing results following
an independent review by qualified experts.
Moreover, by publishing results, scientists generally agree
that the reagents reported therein, including cells, are to be
made available and shared with the research community. In this
way, the results can be independently verified, and new
directions and discoveries can be explored.
The problem with the present case is that only a handful of
these 60-plus embryonic cell lines have been published, so it
is not yet possible to give evaluation or comment on the
quality of the lines. Nonetheless, legitimate questions can be
asked about their growth, differentiation potential, age, and
purity. These issues have already been raised this morning.
What I can say is that decades of experience with mouse
embryonic stem cells show that they lose their differentiation
potential, become contaminated, accumulate mutations, and tend
toward spontaneous differentiation or uncontrolled
differentiation after a certain period. This is related to the
question that Senator Mikulski raised about shelf life.
Stated otherwise, there is incontrovertible evidence that
old mouse embryonic stem cells do not have the same potential
that young ones do. If I were to give an analogy with a human,
it is true that these lines can grow forever and are in that
sense immortal, but they lose their potential--a 150-year-old
may still be alive but does not have the same potential as a
20-year-old.
I hasten to add that I am not criticizing the NIH nor the
scientists who have reported the isolation of these 60 stem
lines. Indeed, the scientists have not published their work,
and they may well wish to further characterize the cells before
doing so. It is therefore, in my opinion, too early to tell how
many of the 60 lines are truly useful. Preliminary indications
nevertheless suggest that the final number will be
significantly less than 60. If the available lines have been
extensively grown and have a high passage number, that will
further reduce their value.
Let me now turn to the question of availability. A separate
issue concerns whether these lines will be made available, and
we have already heard that the entities that have derived the
lines have proprietary and commercial interests. Experience
shows that the negotiation of transfer from those who own the
reagent to federally-funded scientists can be slow, expensive,
and sometimes accompanied by onerous restrictions. It is
obvious that the legitimate interests of companies may not
coincide with scientists' research plans and our Nation's
public health policy.
I was delighted to hear that Secretary Thompson has made
progress with WARF in establishing one such relationship, but
it is yet unclear whether he will be successful in doing so
with the other entities.
To get to my final point, I would like to make a suggestion
which I have made before to Secretary Thompson and the NIH,
which is a plan to move forward that I think will be most
effective given the limitations presented to the scientific
community.
Specifically, I suggest that the NIH create a repository,
not a registry, for the 60 embryonic cell lines. The NIH could
collect the lines, determine their quality, and certify them
for distribution to qualified researchers. Equally important,
this plan would have the NIH negotiate favorable terms with all
of the suppliers that could be set out in the Material Transfer
Agreement.
At the moment, it is very difficult for scientists to
individually negotiate such arrangements, and the Federal
Government and the NIH are in an immeasurably stronger position
than are individual investigators to obtain the human embryonic
stem cell lines from suppliers. In that way, as I have said,
they could verify their quality and arrange for their
distribution.
I would like to know whether the NIH would be willing to
consider doing that given that their resources far exceed those
of individual investigators.
In conclusion, Mr. Chairman, I think the President and
Secretary Thompson have proposed a plan that will allow
federally-supported researchers to begin to explore work on
human embryonic stem cells and work toward a cure. It is an
important step. If my remarks today seem cautious, the reason
is the uncertainty I have about the quality, availability, and
longevity of the cell lines. Assuming that some of the 60 lines
are made available, federally-supported scientists can work to
understand how these cells can be directed to differentiate.
This will lead to new insights into human biology and disease.
However, it seems to me perfectly clear that as these
studies progress to the point where clinical applications can
begin, I expect the plan will have to be revisited, principally
because the viability or utility of the 60 cell lines will have
been exhausted by that point.
In closing, I thank you and the committee once again for
the privilege of speaking to you about this important area of
biology.
The Chairman. Thank you very much.
[The prepared statement of Mr. Melton follows:]
Prepared Statement of Douglas Melton
Good afternoon Chairman Kennedy, Senator Gregg and other
distinguished members of the committee. It is my pleasure to appear
before you today to speak about human embryonic stem cells.
Mr. Chairman, before I begin my remarks on stem cells, I want to
take this opportunity to thank you and the other members of this
subcommittee for your leading role in supporting the NIH. I thank you
on behalf of the Nation's scientists who work to understand the basic
principles of life and to cure human disease.
In the last 3 years, and increasingly so in the past few months,
the potential of human embryonic stem cells has been widely debated in
the public and rightly so. This subject forces us all to revisit the
question of when life begins. We have to scrutinize the crossroads
between scientific inquiry, our efforts to improve the human condition,
and our moral and ethical responsibilities to preserve human dignity.
Not surprisingly, a subject that combines the science of life's
beginnings with politics and religion has captured the Nation's
attention. Indeed, this topic was recently the subject of President
Bush's excellent speech on 9 August.
I am not here to testify on the moral, religious or political
aspects of human embryonic stem cell research. I appear before you as a
scientist and as the father of a young boy with Type I or juvenile
diabetes. I will furthermore not speak to you about the human burden of
this disease, but simply say that I work on human embryonic stem cells
with the aim of providing a cure for diabetes. My remarks today will be
confined to the scientific potential of human embryonic stem cells and
the implementation of the President's plan.
While I'm certain the committee is well aware of the potential uses
for human embryonic stem cells, allow me to briefly put this research
in context. In the last century, biologists showed that genes are the
units of development and heredity, discovered that genes are made of
DNA, and recently completed sequencing the DNA that comprises the human
genome, that is, sequencing the DNA of all human genes. This monumental
achievement will stand as one of the most important scientific triumphs
from the last century. Knowing the sequence of DNA allows scientists to
uncover the basis for development, heredity and disease and challenges
us to understand how the code of life is read or interpreted. At the
same time, this enduring achievement should not cause us to forget that
the unit of life is not DNA nor the gene, but rather the unit of life
is the cell. Cells are alive and reproduce and among cells, stem cells
are unique. Embryonic stem cells are special because they can reproduce
to make more of themselves and they have the remarkable capacity to
make any kind of cell in the body. One might think of them as the fire
hydrant of all cells, having the capacity to renew or replenish lost
cells and tissues. Understanding how these cells can duplicate
themselves and how they specialize to make all types of cells will
undoubtedly reveal important insights into human biology and disease.
The ability of stem cells to specialize or differentiate into any
kind of cell is what holds their enormous therapeutic promise. Many of
the diseases that currently plague our society are diseases of cellular
deficiency, diseases in which one particular cell type is missing or
defective. These diseases include Parkinson's, Alzheimer's,
osteoporosis, some cancers and the one that has my full attention,
juvenile diabetes. It has been estimated that as many as one hundred
million Americans are affected by these diseases. Stem cells have the
potential to replace the missing or deficient cells and it follows that
the Nation's scientists and those suffering from diseases are anxious
to aggressively pursue this research. I should like to note that while
embryonic stem cells have a much broader potential for growth and
differentiation than do adult stem cells, research on both adult and
embryonic stem cells is warranted; it's too early to know which type of
stem cell will be most useful. Whereas the last century of biology can
be said to have focused on the gene and the sequence of DNA, I believe
this century will see biologists come to understand and harness the
unit of life, the cell, specifically stem cells.
president bush's plan for sixty embryonic cell lines
President Bush has made clear his commitment to support research on
human embryonic stem cells, highlighting the importance of this
research. The President's plan provides the opportunity to advance
embryonic stem cell research in the US, at least for a few years, and
as such his plan marks an important commitment. The Honorable Tommy
Thompson has worked diligently for this research and his continued
leadership will be critical in moving forward with the President's
plan.
For this field the date of the President's speech, 9 August 2001,
is important because only stem cell lines in existence at that time,
estimated to be about sixty, are eligible for Federal support. This
date was not chosen for scientific reasons and its arbitrary selection
will have an effect on the progress of research. For example, it will
not be possible for federally funded researchers to explore new ways to
derive human embryonic stem cells nor work with cells that have been
isolated without possible contamination from mouse or other supporting
cells. Nevertheless, it is now possible for the Nation's researchers to
initiate studies on how embryonic stem (ES) cells differentiate and we
can begin to explore their therapeutic potential.
Looking ahead to how the plan will work, I turn to two issues: the
quality of the sixty cell lines and their access or availability.
quality of the human embryonic stem cell lines
Scientists are, by their nature, inquisitive and skeptical and we
hold dear the practice of publishing results following an independent
review by qualified experts. Moreover, by publishing results,
scientists generally agree that the reagents reported, including cells,
are available to be shared with the research community. In this way
results can be independently verified and new directions and
discoveries can be explored. In the present case, only a handful of the
sixty+ embryonic cell lines have been published so it is not yet
possible to evaluate or comment on the quality of cells. Nonetheless,
legitimate scientific questions about the growth, differentiation
potential, age, and purity of the lines must be considered. Decades of
experience with mouse embryonic stem cells have shown that ES cells can
lose their differentiation potential, become contaminated, accumulate
mutations, and tend toward spontaneous or uncontrolled differentiation.
The fact that mouse ES cells lose their full potential with increasing
age or passage number is only one reason to believe that the sixty+
cell lines will not be sufficient for the years of research required to
investigate therapies with these cells. Looking ahead to clinical
applications, including transplantation and the problem of
immunological rejection, there will certainly be a need for broader
genetic diversity of cell lines. There may also be a need for cell
lines that have been isolated without the use of mouse feeder layers.
I hasten to add that I am not criticizing the NIH nor the
scientists who have reported the isolation of the sixty human embryonic
stem cell lines. Indeed, the scientists have not published their work
and they may well wish to further characterize the cells before doing
so. It is therefore too early to tell how many of the sixty+ lines are
truly useful embryonic stem cell lines. Preliminary indications from
press reports do suggest that the final number will be significantly
less than sixty. If the available lines have been grown extensively and
have a high passage number that will further reduce their value.
availability
A separate issue concerns whether the cell lines will be made
available to federally funded researchers in a timely manner and
without restrictions on their use for research. It is noteworthy that
most of the entities that have isolated the sixty+ human embryonic stem
cell lines are companies with proprietary and commercial interests. In
addition, there are relevant patents on some of the cells that may
further restrict their distribution and use. Experience shows that the
negotiation of transfer from those who own a reagent to federally
funded scientists can be slow, expensive, and sometimes accompanied by
onerous restrictions on use. It is obvious that the legitimate
interests of companies may not coincide with scientist's research plans
or our Nation's public health policy.
I believe this problem of access is likely to be quite serious. The
NIH plan to create a registry of cells will leave it to individual
investigators to negotiate for transfer of the cells. This places a
heavy burden on researchers and one can anticipate, at a minimum,
significant delays. In some cases the terms of the transfer may be too
restrictive to allow scientists access to the material. Finally, I note
that some of the potential suppliers have already indicated that they
lack the resources and incentive to prepare their cells for
distribution.
create a federal repository for human stem cells
I would like to suggest a plan that addresses both of these issues.
Specifically, I suggest that the NIH create a repository, not a
registry, for the sixty+ embryonic cell lines. The NIH could collect
the cell lines, determine their quality, and certify them for
distribution to qualified researchers. Equally important, this plan
would have the NIH negotiate favorable terms with the suppliers, set
out in a Material Transfer Agreement, so that scientists could use the
cells for research purposes. The Federal government and the NIH are in
an immeasurably stronger position than are individual investigators to
obtain the human embryonic stem cell lines from suppliers, verify their
quality, and arrange for their distribution.
summary
In conclusion, Mr. Chairman, I think that President Bush and
Secretary Thompson have proposed a plan that will allow federally
supported scientists to begin to explore the potential of human
embryonic stem cells and work toward a cure for numerous diseases. This
is a very important step forward. If my remarks today seem cautious,
the reason is the uncertainty about the quality, availability and
longevity of the cells. Assuming that some of the sixty cell lines are
made available, federally supported scientists can work to understand
how these cells can be directed to differentiate. As the studies
progress to the point where clinical applications can begin, I expect
the plan will have to be revisited because the viability or utility of
the sixty+ cell lines will have been exhausted.
In closing, I thank you and the Committee once again for the
privilege of speaking to you about this important area of biology.
The Chairman. Ms. Hersey?
Ms. Hersey. Good afternoon, Mr. Chairman and distinguished
members of the committee.
My name is Karen Hersey. I am senior counsel for
intellectual property at the Massachusetts Institute of
Technology. It is my understanding that I have been invited
here today for the purpose of providing you with an
introduction and an overview of the subject of Material
Transfer Agreements, known as MTAs, about which you have
already heard this morning, and the role that they
traditionally play in the exchange of materials for scientific
research.
As we already know from our colleagues at the University of
Wisconsin, there is every expectation in the academic community
that access to stem cells for research will involve the
execution of a Material Transfer Agreement between the stem
cell provider and the organization requesting them.
MIT does not have a medical school, and in terms of the
numbers of materials that we request access to, it is small
compared with those institutions that have larger faculties,
larger student bodies, and medical schools. However, MIT's
faculty, students, and staff are engaged in a substantial
volume of research in biology, biotechnology, bioengineering,
and so forth, where biological materials are used every day to
investigate and teach areas of advanced science.
Like every research unit, whether academic or industrial,
MIT investigators depend upon the availability of and the
access to new materials developed by others to move their
research forward. The materials are often proprietary to their
owners and not commercially available, or they may be
commercially available at a very steep price that the
university generally does not have to pay only if it accepts
materials under restrictive conditions. And in some cases, the
materials will be made available to the academic and industrial
communities under a common set of terms that we in academic
find ourselves trying to work with in a contractual framework
that is not suited to our environment.
Twenty years ago when I first joined MIT as a licensing
attorney, the term ``Material Transfer Agreement'' was
virtually unknown, or at least unknown to those of us in the
administration. We can reconstruct from the lament we often
hear recalling the ``good old days'' that materials were
transferred from scientist to scientist and from organization
to organization through a phone call or a verbal handshake.
From time to time, one-page documents or releases arrived,
generally containing warnings that the materials might be
toxic, that they were not to be used for human subject
research, and requiring the receiving party to release the
sending party from all responsibility for use of the materials.
These one-page notifications were almost always signed by
the investigator receiving the materials. A check of our
database shows that MIT institutionally signed a total of eight
MTAs in 1989.
That was yesterday. Today the story is far different. My
office at MIT has overall responsibility for incoming material
transfer activity at MIT. We have one attorney and one
paralegal negotiating more than 75 Material Transfer Agreements
a year. We have a database where all MTAs, their status as
active or inactive, the lab where the materials are placed, and
the responsible investigator are logged, and where the
restrictions on use of the materials are also logged. There are
over 600 entries covering materials in active use, and we are a
relatively small university. The larger State universities and
medical schools are looking at exponential increases in these
numbers.
So what has happened to change the simple handshake between
scientists into a fullblown negotiation between organizations,
often taking months? No doubt there will be any number of
theories out there that might be supported, but as I consider
events over the last decade and a half, it leads me to
attribute the change to three factors.
The first is the explosion of the biotechnology industry
and the recognition by both industry and the academic community
during the 1980's and 1990's that certain combinations of the
materials, certain methods of producing them or methods of
using them, could in fact be patented.
From then on, the materials, especially those that were
unique, took on an added value, and the transfer of them under
terms that would protect intellectual property rights of the
owner became singularly important.
The second was the companion recognition in the late 1980's
that the sharing of materials might just translate into an
advantageous business opportunity for the owner. If materials
could be cast among academic scientists, industry bench
scientists, and Government researchers, might there not be
interesting knowhow, improvements, and new discovers that could
be reeled back in if the materials owner implanted hooks into
its agreements?
Third, it is clear from the negotiations we conduct now
that fear also played a role in the demise of the traditional
transfer by handshake. That is, fear of deep-pockets liability
if materials were misused and also fear that a potential
business opportunity for the materials owner might be thrown
away if materials were sent out with no hooks applied.
That background should provide a clearer understanding of
why, in today's typical Material Transfer Agreement received by
an academic institution, it is now common that some combination
of limitations, restrictions, give-backs or reach-throughs will
be found. Before the materials can be introduced into
scientific research programs, the terms under which the owner
is willing to make them available will need to be reconciled
with the proposed scientific research program, the sources of
funding for it, and the institutional policies having to do
with freedom to publish, the importance of sharing research
findings with colleagues, and technology transfer.
If I may, I would like to just take a very short time to
take you through some of the very common but problematic terms
that we are apt to see in the agreements that must be signed
before the transfer of materials can be accomplished.
I would like to say that except as between nonprofits, it
is totally unusual for us to see a one-page Material Transfer
Agreement unless the type size is minute. Most often these
agreements will run well beyond the two-page quick transaction
limit. They will commonly define or identify the materials they
cover and routinely expand that definition of materials to
subsume any progeny that might be split off or replicated.
While that is clearly understandable, unfortunately, all too
often the definition of ``the materials'' is expanded by the
owner to encompass ``modifications and derivatives'' of them.
This is where the problems for the receiving scientists are
likely to begin, for if the owner owns the materials under this
expanded definition, the owner also ends up with ownership of
the modifications and the derivatives made by the receiving
scientist. The ownership problem is exacerbated if the
materials provider also wants ownership rights to all
improvements, inventions or discoveries developed as a result
of using the materials.
If there is not an ownership issue, there is most likely a
licensing issue. Materials owners may require an exclusive
license, royalty-free, to ``all inventions and research
results'' made through use of the materials; or they may
require open-ended options or first refusal rights to license
inventions relating to use of the materials.
The Chairman. We can give you another minute or two to wrap
up.
Ms. Hersey. Thank you, Senator.
At a minimum, if the materials provider is a commercial
company, a university may expect to grant that company a
royalty-free, nonexclusive license.
I would just like to end by giving you a couple of
observations that might help as you look at or contemplate the
kinds of agreements that may come in with the materials that
are going to be used by NIH research.
We do have at MIT a slightly more difficult time with
material transfer agreements coming in from foreign
organizations. There is a problem with control over use of the
materials, intellectual property rights, and in fact, some of
the materials are not owned by the foreign institutions at all
but by the faculty, the scientists who develop them.
Finally, how do material transfer agreements really affect
the scientific work of a university? They tend to be
comprehensive legal agreements presenting unique and complex
issues for a university. That means they are time-consuming to
negotiate, as you have already heard, and hold up research
efforts. They often contain ownership, licensing and reach-
through terms and conditions that are inconsistent with Federal
requirements attached to federally-funded research. They may
prohibit or restrict publication of research in a way that is
unacceptable for an academic institution. They may discourage
innovation because in fact the materials provider will control
commercialization rights.
Thank you very much.
The Chairman. Thank you very much, Ms. Hersey.
[The prepared statement of Ms. Hersey follows:]
Prepared Statement of Karen Hersey
Mr. Chairman and distinguished members of the committee. My name is
Karen Hersey. I am Senior Counsel for Intellectual Property at the
Massachusetts Institute of Technology. I am pleased to have the
opportunity today to provide you with an introduction to the subject of
material transfer agreements and the role they traditionally play in
the exchange of materials for scientific research. As we already know
from our colleagues at the University of Wisconsin, there is every
expectation in the academic community that access to stem cells for
research will involve the execution of a material transfer agreement
between the stem cell provider and the organization requesting them.
MIT is a small institution compared with most other U.S. research
universities and we do not have a medical school. However, MIT's
faculty, students and staff are engaged in a substantial volume of
research in biology, biotechnology, bioengineering, biochemistry and
related disciplines where biological materials, including cell lines,
plasmids, vectors, sequences, monoclonal antibodies and others are used
every day to investigate and teach areas of advanced science. Like
every research unit, whether academic or industrial, MIT investigators
often depend upon the availability of, and access to, new materials
developed by others to move their research forward. The materials are
often proprietary to their owners and not commercially available; or
they may be commercially available at a steep price that the university
generally does not have to pay if it accepts the materials under
restrictive conditions; and, in some cases, the materials will made
available to the academic and industrial communities under a common set
of terms and we, in academia, find ourselves trying to work within a
contractual framework not suited to the academic environment.
background
Twenty years ago, when I first joined MIT as a licensing attorney,
the term ``material transfer agreement'' was virtually unknown, at
least unknown to the administration. We can reconstruct, from the
lament we often hear recalling the ``good old days'' that materials
were transferred from scientist to scientist and from organization to
organization through a phone call and a verbal handshake. From time to
time, one page documents or releases arrived that generally contained
warnings that the materials might be toxic, were not to be used on
human subjects, and required the receiving party to release the
materials owner of all responsibility for any use of the materials.
These one-page notifications were almost always signed by the
investigator receiving the materials. A check of our database shows
that N41T institutionally signed a total of 8 MTAs in 1989.
That was yesterday. Today, the story is far different. My office at
MIT has overall responsibility for the in-coming material transfer
activity for MIT. We have one attorney and one paralegal negotiating
more than 75 material transfer agreements each year. We have a database
where all MTAs, their status as active or inactive, the lab where the
materials are placed, and the responsible investigator are logged.
There are over 600 entries covering materials in active use--and we are
a very small university. The larger State universities and medical
schools are looking at exponential increases in these numbers.
What has happened to change a simple handshake between scientists
into a full-blown negotiation between organizations, often taking
months? No doubt there will be any number of theories that might be
supported, but as I consider events over the last decade and half, my
experience leads me to attribute the change to three main factors.
The first is the explosion of the biotechnology industry and the
recognition by both industry and the academic community during the
1980s and early 1990s that certain combinations of these materials,
certain methods of producing them or methods of using them, in fact,
could be patented. From then on, the materials, especially those that
were unique, took on an added value and the transfer of them under
terms that would protect intellectual property rights of the owner
became singularly important. The second was the companion recognition
by the late 1980s that the sharing of materials might just translate
into an advantageous business opportunity. If materials could be cast
among academic scientists, industry bench scientists, and government
researchers might there not be interesting know how, improvements, and
new discoveries that could be reeled back in if the materials owner
implanted hooks into its agreements. Third, it is clear from the
negotiations we conduct now that fear also played a role in the demise
of the traditional ``transfer by handshake''. That is, fear of ``deep
pockets'' liability if materials were misused and also fear that a
potential business opportunity for the materials owner might be thrown
away if materials were sent out with no hooks applied.
This background should provide a clearer understanding of why, in
today's typical material transfer agreement received by an academic
institution, it is now common that some combination of limitations,
restrictions, give-backs or reach-throughs will be found. Before the
materials can be introduced into scientific research programs, the
terms under which the owner is willing to make them available will need
to be reconciled with the proposed scientific research program, the
source of funding for it, and institutional policies having to do with
freedom to publish, the importance of sharing of research findings with
colleagues, and technology transfer.
common mta terms, today
First, let me say that, except as between nonprofits as I will
explain later, it is very unusual for us to see a one-page material
transfer agreement today, unless the type size is minute. Most often
these agreements run well beyond the two-page quick transaction limit.
They will commonly define or identify the materials they cover, and
routinely expand the definition of the materials to subsume any
``progeny'' that might be replicated. Unfortunately, all too often the
definition of what are ``the materials'' is expanded by the owner to
encompass ``modifications and derivatives'' of them. This is where the
problems for the receiving scientist are likely to begin; for, if the
owner owns the materials, the owner also ends up with ownership of the
modifications and derivatives made by the receiving scientist. The
ownership problem is exacerbated if the materials provider also wants
ownership rights to all improvements, inventions or discoveries
developed as a result of using the materials.
If there is not an ownership issue, there most likely is a
licensing issue. Materials owners may require an exclusive license,
royalty-free, to ``all inventions and research results'' made through
use of the materials. Or they may require open-ended options or first
refusal rights to license inventions relating to use of the materials.
At a minimum, if the materials provider is a commercial company, a
university may expect to grant the company a royalty-free, nonexclusive
license to inventions made as a result of using the materials. As a
result, the promise of an MTA-encumbered invention as a viable business
opportunity for the inventor and his or her institution is
significantly diminished.
Where ownership and licensing issues can be successfully maneuvered
around, other limitations, restrictions or encumbrances often result in
additional obstacles. For instance, the materials provider may require
the researcher to hold all results learned from using the materials in
confidence, may require a right of review and approval for any
publication dealing with the materials, may require a right of approval
over any transfer to a third party of the materials, their progeny,
derivatives, improvements and so forth, may tag on ``reach-through''
rights to second and third generation uses of the materials or
derivatives of them in terms of royalty-sharing or first commercial
rights to exploit. It should not be a surprise that the list of
encumbrances generally grows with the perceived value of the materials
as a business opportunity.
general observations
I will finish up this short discussion of the role material
transfer agreements play in current-day research efforts with a very
few general comments.
The transfer of materials from university to university or
nonprofit to nonprofit is most often accomplished under a one-page
document called a Uniform Biological Material Transfer Agreement, or
UBMTA. This is a simplified method of transfer agreed upon among the
U.S. research universities during the early 1990s with the help of NIH.
The providing university does not attach strings to the use of the
materials, but, in some cases does have an interest in where they may
be transferred by the receiving university. An attempt was made at the
same time to convince industry to join in a simplified transfer
mechanism, but it was not successful.
At MIT we have a slightly more difficult time with
material transfer agreements from foreign organizations or companies.
They seem to be concerned with maintaining control of their properties
either by claiming ownership of derivatives and modifications or by
prohibiting the receiving scientist from obtaining any independent
intellectual property rights through uses of the materials. And,
significantly, we find restrictions placed on uses, ownership and
further transfer because foreign universities or institutes often do
not own materials, or do not have the right to control the distribution
of them. Unlike the situation with U.S. universities, in Europe and
elsewhere materials are often the property of the scientist or
professor who developed them.
Where materials are patented there is an extra layer of
complications that arises. The university community will very often be
successful in getting a noncommercial research right or license to use
a patent covering the materials, but this is as far as it goes. Any
future commercial use requires a license from the patent holder or no
license rights at all may be available, hence any commercial use that
would infringe the patent is cut off all together.
Finally, how do the material transfer agreements really
effect the scientific work of a university? They tend to be
comprehensive legal agreements often presenting unique and complex
issues for a university. That means they are time consuming to
negotiate and hold up research efforts. They often contain ownership,
licensing or reach-through terms and conditions that are inconsistent
with Federal requirements attached to federally-funded research. They
may prohibit or restrict publication of research in a way that is
unacceptable for an academic institution. They may discourage
innovation because the control of the research output rests with the
materials provider or, at a minimum, establishes a preference for the
provider to exploit new discoveries made using the materials. Last, and
a subject we've not considered at all today, they may contain
indemnification obligations that amount to strict liability and are
either not within the statutory authority of a State institution to
accept or present an unacceptable risk to the endowment of a private
institution.
This concludes my brief remarks on a complex subject. I am most
appreciative of the Committee's indulgence and will be pleased to
answer any questions you may have.
The Chairman. Dr. Childress?
Mr. Childress. Good afternoon, Mr. Chairman and members of
the committee.
I am James Childress. I teach religious ethics and
biomedical ethics at the University of Virginia and also serve
on the National Bioethics Advisory Commission.
I have been asked to present my own views on the ethical
issues in human stem cell research. In doing so, I will
sometimes draw on NBAC's report on this topic along with its
recommendations, which I as a commissioner helped prepare and
also endorsed.
I will briefly summarize several points from my longer
written testimony and would respectfully request that that
testimony be entered into the record.
The Chairman. All of the testimony will be included.
Mr. Childress. I thank you very much.
I appreciate the thought and consideration that went into
President Bush's policy, but other more flexible policies are
also ethically acceptable and even preferable.
President Bush's policy suggests that it is ethically
acceptable to use Federal funds for research on stem cell lines
that were derived prior to his announcement on August 9 if the
derivation also met certain ethical requirements--specifically,
that donors or embryos that were created solely for
reproductive purposes must have given informed consent without
any financial inducements.
If this policy is ethically acceptable and satisfies basic
ethical standards--and I believe it does--it should also be
ethically acceptable to do the same thing prospectively--that
is, to provide Federal funds for research on stem cell lines
derived in the future, after August 9 as well as before, within
the same ethical guidelines. The prospective policy would offer
greater and needed flexibility, especially in view of the
scientific uncertainty about the value of the approved cell
lines. And it would be ethically preferable because it would
increase the possibilities for important research without
violating ethical standards.
This prospective policy can be undertaken without
sanctioning or encouraging further destruction of human
embryos. Those were legitimate, major concerns in President
Bush's statement. We can establish effective ethical safeguards
to ensure that a couple's voluntary decision to destroy their
embryos is voluntary and informed, or that their decision to
donate them for research is voluntary, informed, and
uncompensated. The research, then, would only determine how the
destruction occurs, not whether it will occur.
In making these points, I want to stress that no consensus
exists among religious and secular moral traditions in our
society about the moral status of the unimplanted human embryo.
Public policy in our pluralistic society has to respect diverse
fundamental beliefs, and yet it must not be held hostage to any
single view of embryonic life.
Whichever policies are finally adopted to enable stem cell
research to go forward within ethical limits, we will need a
very strong public body to review protocols for deriving stem
cells from embryos and to monitor these research. Perhaps the
Council on Bioethics which President Bush has announced can
fulfill these functions. If not, some other public body will be
needed. For example, the UK has established a statute, the
Human Fertilization and Embryology Authority, which would use
all embryo research as well as licensing of reproductive
technology and fertility clinics. It reviews all embryo
research in public and private arenas. Congress might consider
that model for our society as well.
In a recent editorial in Science, ethicist LeRoy Walters
stressed that ``Government's and their advisors should be
humble and flexible, but also decisive and courageous.'' We
must carefully scrutinize claims of scientific promise, but we
must not unduly constrain research that may help alleviate
human suffering and reduce the number of premature deaths.
Indeed, we have a collective moral duty to try to alleviate
human suffering and reduce premature deaths just as we have a
collective moral duty to respect important ethical limits in
dealing with developing human life.
We must also provide clear and stringent ethical safeguards
in stem cell research, along with strong review and oversight.
And I think in the final analysis, we must avoid unduly rigid
rules that appear to be arbitrary and inconsistent.
Thank you very much for your attention. I will be glad to
answer any questions.
The Chairman. Thank you, Dr. Childress.
[The prepared statement of Mr. Childress follows:]
Prepared Statement of James F. Childress
Good morning, Mr. Chairman and members of the committee. I am James
Childress. I teach in the Department of Religious Studies, the Medical
School, and the Institute for Practical Ethics at the University of
Virginia. I am also a member of the National Bioethics Advisory
Commission (NBAC). I have been asked to present my own views on the
ethical issues in the debate about public policies toward human stem
cell research, especially embryonic stem cell research. Even though I
am not testifying on NBAC's behalf, I will sometimes draw on NBAC's
report and recommendations, which as a commissioner I helped to prepare
and also endorsed. \1\
---------------------------------------------------------------------------
\1\ See National Bioethics Advisory Commission, Ethical Issues in
Human Stem Cell Research, Vol. 1: Report and Recommendations
(Rockville, MD, September 1999). In November 1999, I presented NBAC's
views at the hearing on ``Embryonic Stem Cell Research'' before the
Subcommittee on Labor, Health and Education of the Senate
Appropriations Committee. I request that the Executive Summary of
NBAC's report be entered into the public record of today's hearing.
---------------------------------------------------------------------------
an ethically acceptable policy
I very much appreciate the thought and consideration that went into
President Bush's announced policy on the use of Federal funds in human
embryonic stem cell research, but I would argue that other, more
flexible policies are also ethically acceptable, and even preferable.
Consider three possible options:
(1) Provide Federal funds for research on cell lines derived (using
non-Federal funds) from embryos prior to August 9, 2001 within certain
ethical guidelines (President Bush's announced policy).
Provide Federal funds for research on cell lines derived (using
non-Federal funds) from embryos, earlier or in the future, within
certain ethical guidelines (NIH's earlier proposed policy).
Provide Federal funds for both the derivation of, and research on,
cell lines derived from embryos within certain ethical guidelines
(NBAC's recommendation).
President Bush's announced policy (#1) suggests that it is
ethically acceptable to use Federal funds for research on stem cell
lines that were derived, using non-Federal funds, prior to his
announcement on August 9, if the derivation also met certain ethical
requirements, including the informed consent of donors of embryos
created solely for reproductive purposes and the absence of financial
inducements. \2\ If policy #1 is ethically acceptable--as I believe it
is--then it should also be ethically acceptable to do the same thing
prospectively (policy option #2). That is, it should be ethically
acceptable to provide Federal funds for research on stem cell lines
derived in the future, after August 9 as well as before, with non-
Federal funds and within the same ethical guidelines. This prospective
policy would offer greater--and needed--flexibility for the short-term
and long-term future. And it would be ethically preferable because it
would increase the possibilities for important research, without
violating relevant ethical standards. \3\
---------------------------------------------------------------------------
\2\ See President George W. Bush, ``Stem Cell Science and the
Preservation of Life,'' New York Times August 12, 2001.
\3\ This is similar to the policy NIH initially proposed in
December 1999 and further refined in 2000.
---------------------------------------------------------------------------
President Bush's statement noted that the first policy (#1), which
includes about sixty stem cell lines (about which there is some
scientific uncertainty and controversy), \4\ ``allows us to explore the
promise and potential of stem cell research without crossing a
fundamental moral line by providing taxpayer funding that would
sanction or encourage further destruction of human embryos that have at
least the potential for life.'' However, I believe that ethically we
can provide Federal tax funds for research on stem cells derived after
as well as before August 9, using non-Federal funds, and that this can
be accomplished without sanctioning or encouraging further destruction
of human embryos. To do so, we must establish effective ethical
safeguards. Those safeguards should ensure to the greatest extent
possible the couple's voluntary and informed decision to destroy their
embryos--rather than use them or donate them to another couple--and
their voluntary and informed decision to donate them for research. The
research would then determine how the destruction occurs, not whether
it will occur; as matters stand in most jurisdictions, couples may
determine how to dispose of their embryos.
---------------------------------------------------------------------------
\4\ See ``President Bush's Stem Cell Policy,'' A Statement of the
American Association for the Advancement of Science, August 17, 2001.
---------------------------------------------------------------------------
It is possible to go further than either of these first two
policies and recommend, as NBAC did, a third option--the provision of
Federal funds for both the derivation of stem cells from embryos and
research on those cell lines, again in accord with ethical
requirements. One argument for this option is that a strict separation
between derivation and use would adversely affect the development of
scientific knowledge. For instance, the methods for deriving embryonic
stem cells may affect their properties, and scientists may increase
their understanding of the nature of such cells in the process of
deriving them.
In short, I see no ethical reason for limiting Federal funding to
research with cell lines derived by some arbitrary date, as long as
future derivation, with non-Federal funds (option #2) or Federal funds
(option #3), also respects the same moral limits and we establish
effective ethical safeguards. Indeed, our collective moral duty to
alleviate human suffering and reduce the number of premature deaths
provides a strong ethical reason to support this research, within moral
limits.
respect for the embryo
There is widespread agreement, as NBAC observed, that ``human
embryos deserve respect as a form of human life,'' but, at the same
time, sharp disagreements exist ``regarding both what form such respect
should take and what level of protection is required at different
stages of embryonic development.'' \5\ At the very least this
``respect'' implies
---------------------------------------------------------------------------
\5\ See NBAC, Ethical Issues in Human Stem Cell Research.
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that early embryos should not be used unless they are
necessary for research,
that embryos remaining after in vitro fertilization (IVF),
as well as cadaveric fetal tissue, should not be bought or sold, and
that alternative sources of stem cells should
simultaneously be explored.
Indeed, given the promise of this research, and the uncertainty
about which stem cells might be adequate and which might be superior
for various purposes, research on stem cells derived from different
sources should be eligible for Federal funding. The goal of realizing
the therapeutic promise of stem cell research is ethically significant.
It is also ethically important to treat the different sources of stem
cells with appropriate respect.
One interpretation of appropriate respect for early embryos would
rule out their deliberate creation in order to use them in research. I
supported NBAC's recommendation that, at this time, Federal agencies
should not fund research involving the derivation or use of embryonic
stem cells from embryos made solely for research purposes, whether they
were made by IVF or by somatic cell nuclear transfer into oocytes.
However, in this area, it is ethically dangerous to say ``never.'' For
now, it appears to be possible to develop enough cell lines without
creating more embryos, and there appears to be no need for nuclear
transfer unless and until therapy is possible, at which time matched
tissue may be needed. As the science develops, it may be necessary to
revisit the question about so-called ``therapeutic cloning.'' \6\
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\6\ NBAC's report on human stem cell research anticipated that
privately-funded research would use deliberately created embryos,
whether created through IVF or cloning, and that careful monitoring of
this private sector research would enable the Federal government to
determine when, if ever, the time has come to fund the creation of
embryos for research.
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diversity of religious and secular positions
Views about appropriate respect for the embryo hinge on convictions
about the embryo's moral status. As a scholar in religious ethics, I
have been fascinated by the diverse religious views on human embryonic
stem cell research, both across traditions and within traditions. On
May 7, 1999 NBAC convened a meeting at Georgetown University to hear
presentations on religious perspectives relating to human stem cell
research. Altogether eleven scholars in Roman Catholic, Jewish, Eastern
Orthodox, Islamic, and Protestant traditions presented formal testimony
that day, and two others made statements in the public comment period.
Their statements, as well as later statements of other traditions
(e.g., the Mormon tradition), reveal significantly different
perspectives on the ethical acceptability of research on unimplanted
human embryos. Even when opposed to abortion, different religious
positions may reach divergent moral conclusions about human embryonic
stem cell research. Their different conclusions follow, in part, from
different premises about the moral status of the early embryo existing
outside a woman's womb.
No consensus exists among religious traditions or secular moral
traditions--about the moral status of the extracorporeal embryo. This
diversity sets the context for an ethical assessment of public policy
toward human embryonic stem cell research. An ethical public policy in
our pluralistic society has to respect diverse fundamental beliefs. And
yet it must not be held hostage to any single view of embryonic life.
stem cell lines derived from aborted fetal tissue
Another possible source of stem cells--human embryonic germ cells
from aborted fetuses--has received scant attention recently. \7\
However, precedent exists in U.S. policies for providing Federal funds
to support research on cell lines derived from aborted fetuses. This
precedent appears in the framework developed for the use of cadaveric
fetal tissue in transplantation research. \8\ This framework seeks to
separate as much as possible a pregnant woman's decision to abort from
her decision to donate fetal tissue for research. The rationale for
this separation is to avoid any possibility, however slight, that the
opportunity to donate fetal tissue in federally funded research could
provide an additional motivation for a woman to have an abortion.
---------------------------------------------------------------------------
\7\ See Alta Charo, ``Bush's Stem Cell Decision May Have Unexpected
and Unintended--Consequences,'' The Chronicle of Higher Education,
Tuesday, August 14, 2001.
\8\ For the debate about human fetal tissue transplantation
research, see the discussion in James F. Childress, Practical Reasoning
in Bioethics (Bloomington, IN: Indiana University Press, 1997).
---------------------------------------------------------------------------
Several ``ethical safeguards'' were erected in order to prevent the
use of fetal tissue in federally funded transplantation research from
encouraging abortions. For example, these safeguards separate the
consent process for abortion from the consent process for the donation
of fetal tissue for research, and prohibit the donor of fetal tissue
from designating the recipient of the transplant. These ethical
guidelines, which appear to have been effective in human fetal tissue
transplantation research, should now be extended to stem cell research
as well, as NBAC has recommended and NIH has proposed. \9\
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\9\ See NBAC, Ethical Issues in Human Stem Cell Research, Vol. 1,
pp. 68-69, Recommendation #1.
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another approach to public policy--the u.k. experience
The United Kingdom has responded quite differently than the U.S. to
human embryonic stem cell research, including ``therapeutic cloning.''
Following the 1984 Warnock committee report, the British government
implemented most of that committee's recommendations in the 1990 Human
Fertilisation and Embryology Act, which, among other things,
established the Human Fertilisation and Embryology Authority (HFEA).
Over the last decade, the HFEA, currently chaired by Ruth Deech, has
had authority over in vitro fertilization, in policy and in practice.
The Authority also licenses and monitors all human embryo research in
the U.K., whatever the source of funding. In addition, it approves, in
limited circumstances, the creation of embryos for research purposes.
Over 53,000 embryos have been used in research, while 118 have been
created specifically for research. In January, 2001, following vigorous
public debate, the British Parliament approved regulations to enlarge
the range of acceptable goals for human embryo research and also to
permit the creation of embryos for research by nuclear transfer
(``therapeutic cloning'').
In the U.K., then, years prior to the recent debate about stem cell
research, several substantive and procedural standards were established
for embryo research, including the creation of embryos for research.
Furthermore, the public appears to have considerable confidence in that
framework, based on a decade's experience. As a result, the acceptance
of ``therapeutic cloning'' required only an extension of the existing
framework, rather than the invention of a new one.
The U.K.'s strict regulation of reproductive technologies and
authorization, but also tight control over, embryo research appears to
have created a context for a positive response to the possibilities of
human stem cell research. \10\ By contrast, in the U.S., regulation of
reproductive technologies and fertility clinics, which is under the
control of the states, is, at best, limited and uneven, and the Federal
government has not allowed the use of Federal funds for embryo research
(though, of course, privately funded research proceeds). As a result,
the task of formulating public policy toward human embryonic stem cell
research is much more challenging in the U.S.
---------------------------------------------------------------------------
\10\ For helpful discussions of policy in the U.K., see LeRoy
Walters, ``Human Embryo Research: Lessons from History,'' Science 293
(August 24, 2001): 1401, and Nicholas Wade, ``Clearer Guidelines Help
Britain to Advance Stem Cell Work,'' New York Times, August 14, 2001.
---------------------------------------------------------------------------
conclusion
In conclusion, if President Bush's announced policy is ethically
acceptable, as I believe it is, there is, in my judgment, no cogent
ethical reason for stopping where that policy stops with the use of
stem cell lines that were derived from embryos by August 9, 2001.
Indeed, that temporal restriction is difficult to defend from an
ethical standpoint. It is possible to use non-Federal funds (or even, I
would argue, Federal funds) to derive stem cell lines from embryos
within certain ethical requirements, and to provide Federal funds for
research on those lines without sanctioning or encouraging the
destruction of embryos or the creation of so-called ``extra'' or
``surplus'' embryos in clinical IVF. I would support these other policy
options (that is, derivation with non-Federal funds or with Federal
funds) on the grounds that they will probably enable important research
to proceed more rapidly, and will not breach crucial ethical
boundaries. In addition, it is, in my judgment, ethically justifiable
to provide Federal funds for deriving and conducting research on stem
cell lines developed from aborted fetal tissue, in accord with the
guidelines and regulations already established for human fetal tissue
transplantation research.
Whichever policies are finally adopted to enable important and
promising stem cell research to go forward within ethical limits, we
will need a strong public body to review protocols for deriving stem
cells from embryos (and from fetal tissue) and to monitor this
research.'' \11\ Perhaps the Council on Bioethics, which President Bush
has announced, could fulfill these functions. If not, some other public
body will be needed, as the U.K. experience suggests. In the UK, the
Human Fertilisation and Embryology Authority is statutorily
established, and that might be a model for Congress to consider.
Indeed, Congress might consider whether we now need some oversight of
human embryo research in the private arena.
---------------------------------------------------------------------------
\11\ See NBAC, Ethical Issues in Human Stem Cell Research, Vol. 1,
pp. 74-77, Recommendation #8.
---------------------------------------------------------------------------
It is safe to assume that no policy currently under discussion will
be the final one. We will need to revisit this research again and again
as the science develops and as its ethical implications become clearer,
particularly through a public body's review and oversight. Thus, no
policy will end the national conversation about how to balance, over
time, the relevant ethical considerations. Our public dialogue needs to
continue, with the kind of reflective leadership that Congress, at its
best, can provide. While this dialogue continues, we need a policy with
greater flexibility than the one President Bush announced, but also
with closer review and oversight than some would like.
In a recent editorial in Science, ethicist LeRoy Walters stressed
that ``Governments and their advisors will need to be humble and
flexible, but also decisive and courageous.'' \12\ We must carefully
scrutinize claims of scientific promise, being wary of unfounded
optimism, but we must not neglect research that offers a significant
prospect of major medical breakthroughs that may alleviate human
suffering and reduce the number of premature deaths. As a society, we
must provide clear and strong ethical guidelines, regulations, and
safeguards in stem cell research, while avoiding unreasonably rigid
rules that appear to be arbitrary and inconsistent.
---------------------------------------------------------------------------
\12\ Walters, ``Human Embryo Research: Lessons from History.''
---------------------------------------------------------------------------
Thank you--I will be glad to answer any questions. \13\
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\13\ 1 am grateful to Alta Charo and LeRoy Walters for their
thoughtful and helpful comments on an earlier draft of this testimony;
they are absolved of any responsibility for its content.
The Chairman. Father FitzGerald?
Father FitzGerald. I would like to thank the chair, Senator
Kennedy, and the members of this distinguished community for
this opportunity to come before you today to discuss this
issue.
I would like to bring to bear on this issue my background
in molecular genetics, in bioethics and in religion to present
a somewhat different perspective on this issue than I think we
have heard yet today, and in doing so, I would like to offer a
caveat. There is no way that I can do justice in the brief time
allotted to me to the numerous serious, well-informed and
thoughtful people who are deeply concerned about this research
and the ethical and moral ramifications of pursuing it. These
people, of course, include scientists who have no specific
religious affiliation.
Since I have limited time, let me focus on an issue that
was raised earlier by Senator Frist and then again by Senator
Dodd. That is the issue of what do we do when we do not know.
There is much in this area that we do not know. We do not know
much of the science, and we do not know much of what the
ethical, moral, and social ramifications will be. How are we
going to respond to that lack of knowledge, because in that
lack of knowledge, we truly run the risk of overselling the
promise and underemphasizing the problems associated with human
embryonic stem cell research.
As Senator Clinton stated, it is extremely important that
we receive and create a factual base here on this issue, and
that factual base will, of course, include more than just the
scientific facts.
In addressing this controversial issue, many groups and
committees, such as the National Bioethics Advisory Committee
as well as the proposed committee to be directed by Dr. Leon
Kass, have been gathered together, bringing experts from
various fields of inquiry and interest to propose how our
pluralistic society should respond to this controversial area
of biological research in pursuing progress while protecting
justice.
For instance, as Dr. Childress has mentioned, in trying to
balance the concerns of many people in our Nation, the National
Bioethics Advisory Commission came to the understanding that
human embryos are not just tissue; they do have some moral
status and some value to society. In deciding that, the
National Bioethics Advisory Commission as well as others who
have come to similar conclusions set the bar higher for us to
pursue human embryonic stem cell research than it might be set
for other avenues of research. So in order to clear that bar
and justifiably pursue this research, one would have to meet a
higher standard.
Addressing a comment made earlier by Senator Specter--he
said it is important that science may have the full range of
opportunity--I think it is also important to recognize and
remember that we already limit what science can do, especially
in terms of human research.
The two areas that are most often raised as important to
address in meeting that higher standard for human embryonic
stem cell research are need and the number of people who will
benefit from this research. Let me first address the issue of
need. Do we need human embryonic stem cell research?
Granted, human embryonic stem cell research is
scientifically quite interesting, fascinating, and of great
interest to the people who do that research to pursue. However,
oftentimes we talk about pursuing this in connection with the
number of therapies and cures that will be discovered. In fact,
I believe the phrases of countless numbers of people, and as
Senator Specter said, it will touch virtually every family in
America.
I would argue that we could make the same claims for other
avenues of research, certainly research being pursued in
genetic therapies, research in proteomics, research in drug
development, and of course, research in adult stem cells. All
these other areas of research do not need to set the bar as
high in being pursued. Embryos, human life, is not destroyed.
Yet at the same time, scientists involved in those avenues
of research could make similar claims as to the potential of
their research and the number of diseases they are addressing.
Second, when we talk about the number of people who will be
treated and/or cured by any of these medical areas of research,
we must be careful. We must again be careful not to oversell
the promise, and remember that in fact there are many, even in
our own Nation, who do not have access to these new
technologies and cutting-edge medical products.
Actually, Senator Murray brought up a very interesting
example. She asked the question would we have to keep human
embryonic stem cell products hostage to such things as male
baldness. And the interesting parallel there is that in the
early 1990's, a drug was developed with FDA approval to treat a
disease that afflicts hundreds of thousands of people a year.
It is called sleeping sickness. It happens in Africa. The drug
was the most effective drug known against that disease. That
drug was developed because it might be a cancer research tool
or a cancer therapy. When it proved not to be so, the company
that was producing the drug decided not to produce it anymore,
even though it could cure or at least treat hundreds of
thousands of people. However, the drug is back. It is back
because it has a side effect--it causes hair loss. So they have
turned it into a cream that women can use to treat excessive
facial hair.
When we say that we are going to promise people these
wonderful potential therapies and cures, are we indeed giving
them the accurate assessment of how our systems work? My issue
is not necessarily specifically with the pharmaceutical
industry but in promising the people of this country and
perhaps the world a solution and a promise that we cannot
fulfill.
I very much appreciate the work of this committee, and I
appreciate the reflection that has gone into the many proposals
here and the many presentations, and I certainly hope that we
continue in our reflection on this very serious issue.
Thank you very much.
The Chairman. Father, that drug that you are talking about
is methyltrexate. My son, who had osteosarcoma, took that drug
when he was in an NIH trial, and he survived. Now, there is
about an 85 to 90 percent survival rate in children with that
disease. So you would have a tough time convincing me on that
particular one--I just picked that up.
Father FitzGerald. Actually, the drug is not methyltrexate;
it is F-fluorothene.
The Chairman. OK. I stand to be corrected.
[The prepared statement of Father FitzGerald follows:]
Prepared Statement of Father Kevin FitzGerald, SJ
Advances in medical research are happening at such a rapid rate
that it seems new breakthroughs are announced every week in the media.
These advances present the possibility for treatments and cures unheard
of only a generation ago. However, advances in research can also create
ethical problems seemingly so complex and convoluted that we wonder
whether or not the research is worthwhile. In our society, which claims
to value both progress and justice, we must find a balance between the
Utopian hopes and Frankensteinian fears generated by these advances in
medical research.
One area of recent advance in medical research has raised new hopes
for treating serious illnesses that result from the death or
deterioration of cells and tissues we require for good health. These
illnesses include terrible neurological disorders such as Parkinson's
and Alzheimer's, as well as tragic injuries such as paralysis caused by
damage to a person's brain or nervous tissue. The basis for these new
hopes is a new understanding of human stem cells and their function.
What do these stem cells do? The normal function of stem cells is
to produce new cells and to replace cells we lose through the natural
processes of cellular aging and death. From the time a human being
begins as a fertilized egg, that human being grows and develops by
cells continually dividing to make more cells. Early on in our
development, when we are embryos, the primary function of most of the
cells is to divide and rapidly make more cells. ``Embryonic'' stem
cells have not yet become any particular type of cell, such as muscle
or nerve, and so embryonic stem cells are thought to be capable of
becoming any type of cell in the human body.
As a human being continues to develop as a fetus, infant, child,
and adult, the number of cells in the body increases. During these
later stages, most of the cells stop dividing and take on specific
duties and become brain cells, or liver cells, or skin cells, etc.
However, some cells keep their ability to divide and replace other
cells that have been lost due to damage or normal aging. These stem
cells are generally called ``adult'' stem cells. Until just the past
few years, adult stem cells were considered to be only in certain
tissues, like blood and skin, and to have only the capacity to replace
the cells of the particular tissue within which they were situated.
Recent research has now indicated that adult stem cells are present in
many, if not almost all, of the tissues of the body, including the
brain. In addition, adult stem cells are not limited to replacing cells
from only the type of tissue wherein they are found.
New breakthroughs in stem cell research have involved both
embryonic and adult stem cells. Researchers have discovered ways to
isolate and grow embryonic stem cells such that they can be directed to
become different types of cells that are found in the body. Since
during embryonic development embryonic stem cells do become all the
different types of cells in the body, researchers speculate that it
should be possible, eventually, to direct embryonic stem cells to
produce whichever kind of cell is needed.
Though this research sounds very promising, there are both
scientific and ethical troubles with embryonic stem cell research. One
scientific obstacle is the problem of controlling the development of
the embryonic stem cells into the type of tissue one might need to
treat a disease or injury. Since these cells have such a great ability
to make more cells of any kind, it is important that researchers know
that there are no uncontrolled embryonic stem cells being implanted
into a person. An embryonic stem cell that does not convert into the
type of tissue desired could cause significant harm to a patient by
making the wrong kind of cells in the treated tissue, or by just
growing out of control and creating a tumor. Much research on embryonic
stem cells would still be required in order to insure the safety of
these proposed treatments. This research, in itself, raises a key moral
issue.
The key moral obstacle to embryonic stem cell research is that
currently the only way embryonic stem cells are obtained is by
destroying an embryo. Unlike tissue or organ transplantation, where the
organs are removed after the death of a person, embryonic stem cells
are not harvested after the embryo has died. The procedure for removing
the embryonic stem cells from the embryo destroys the embryo.
Much of our health care tradition is based on the idea that healing
is a benefit to be made available to all. Consequently, it is not
acceptable that for some to be healed others must be sacrificed--no
matter their State in life. In response to this perspective, some argue
that frozen ``spare'' embryos, left over from in vitro fertilization
treatments and not likely ever to be used to produce a pregnancy, might
justifiably be destroyed in order to get embryonic stem cells. However,
using a fundamental principle of health care which states that first of
all one should not unnecessarily harm another, one can counter that no
human life is ``spare.'' Who among us has the right to decide that
another human life is a ``spare'' life, especially when that human life
does not have the chance to contest the decision? We do not consider it
appropriate to take organs from dying patients or prisoners on ``death
row'' before they have died in order to increase someone else's chances
for healing or cure. Neither, then, should we consider any embryos
``spare'' so that we may destroy them for their stem cells.
This defense of human life does not mean that all stem cell
research must be rejected. Research on adult stem cells should be
encouraged, especially in light of the new results that indicate its
amazing promise for treatment and cure. For example, blood stem cells
may be able to replace lost brain cells or liver cells, and vice versa.
In the not-to-distant future, the issue of rejecting transplants may no
longer be a problem because our own stem cells will be used to repair
and replace our damaged cells and tissue.
Even taking the promise of adult stem cells into consideration,
some scientists argue that the most efficient way forward is to do
research on all the various types of stem cells in order to see which
will be best for treating the many different diseases we wish to cure.
This approach of doing all the different types of experiment that can
be done is often the best way for science to proceed. But is it always
the best way for a society to proceed?
We Americans know from our own history with eugenics and research
on minorities or the mentally disabled the tragedies that can occur
when public policies concerning human experimentation are shaped
primarily by the dictates of science. More broadly, the history of
humankind abounds with examples of how biological characteristics such
as race, gender, intelligence, and age, have been used as justification
for discrimination against certain groups within a society so that the
dominant groups in a given society might take advantage of the
vulnerable and those on the margins of the society. In response to
these wrongs, our society has chosen to limit what experiments can be
performed on human beings, even though these limits may slow scientific
progress. Are we not repeating this same tragic pattern by declaring
embryos of significantly lesser value than other human beings because
of their developmental status? And if human embryos do have some
significant value in our society, as the National Bioethics Advisory
Committee concluded, then considering all the basic research that still
can be done using animal models, human tissue culture, and adult stem
cells, why is there a continuing clamor for the destruction of human
embryos to fuel stem cell research?
One reason often put forth as justification for the destruction of
more and more human embryos is the need to bring healing and cures to
the thousands, if not millions, who suffer from illnesses and diseases
that may be amenable to treatment with embryonic stem cell therapies.
Such an argument as this is of great significance for it connects to
another fundamental principle of medicine: treat sickness and heal when
you can. Yet, as the argument is stated, its significance rests in part
on two assumptions: 1) that embryonic stem cells will be necessary, or
superior to all other options, in the treatment of certain diseases,
and 2) that the thousands and millions who need the treatments will
have access to them.
Addressing the first assumption, we need to recognize that the
diseases suggested as likely targets for human embryonic stem cell
research are also the targets of researchers using other approaches,
such as genetic therapies, drug development, and adult stem cells. It
may well be the case that for many patients the treatments for their
illnesses may come more quickly from research avenues other than human
embryonic stem cell research, and that these alternative treatments may
even be better than any treatment derived from human embryonic stem
cell research.
Regarding the second assumption, we need to acknowledge that even
if treatments from human embryonic stem cell research prove to be the
best available and are developed first, many people who need these
treatments will not have access to them. For example, the World Health
Organization statistics show that approximately 900,000 children die
each year of measles. Human embryonic stem cell research is not needed
to prevent measles infections and the cost of the measles vaccine is
dramatically less than any human embryonic stem cell treatments will be
for a very long time, if they are ever produced. Hence, just because
many people in the world tragically share a devastating disease, the
greater tragedy is that only a relative few get to enjoy the benefits
of cutting edge medical technology. This tragic reality should not
prevent our pursuit of medical advances, but it should also not be
forgotten when we decide as a society which medical research is
justified and which is not.
If the justification for proceeding with the destruction of human
embryos for research rests even in part on these two assumptions of
need and the number of those who will benefit, then this justification
is flawed. Human embryos need not be destroyed in order that thousands
or millions might be saved.
Indeed, without the continual destruction of human embryos the
future of medical advance will still be one of great hope. There are
many avenues of medical research that can be pursued with broad ethical
and societal support. As a people who value progress and justice, we
can decide to pursue every avenue of medical research that is
respectful of human life in all its stages, and brings care and healing
to all those in need.
The Chairman. Dr. Chute?
Dr. Chute. First, I want to thank you, Senator Kennedy, and
the committee for inviting me to appear today.
I come to this issue as a clinician first, as a clinically
trained hematologist and oncologist. For the past 5\1/2\ years,
I have also been directing research in adult hematopoietic stem
cell biology.
I also want to say that it has been with great interest
that I have followed the work of Dr. Thomson and those who have
done embryonic stem cell line research. I have been fascinated
that they have been able to propagate these cells in vitro as
long as they have shown they can in peer-reviewed journals and
that they can get these cells to differentiate into neuronal
epithelial muscle and hematopoietic cells.
It is very understandable that patients with very serious
diseases are very excited and hopeful that there may be cures
in the future for such things as diabetes, Parkinson's, and
Alzheimer's derived from further research on these cells.
But as the national discourse on embryonic stem cell
research has progressed, there has been, ironically, a
diminishing appreciation of the exceptional progress that has
been ongoing and occurring in adult stem cell research, and I
appear today to highlight the critical importance of the
ongoing research in adult stem cell biology.I21In the year
2000, there were 1.2 million new cases of cancer in the United
States. Cancer is the second leading cause of death in the
United States, and the incidence of cancer is increasing. Adult
hematopoietic stem cells have been and are currently used
successful in the treatment and cure of patients with leukemia,
lymphoma and other hematologic malignancies.
Also of great interest is that newly developed methods of
transplanting adult hematopoietic stem cells have now been
shown to effect major remissions in solid tumors. There is a
group at NIH led by Dr. Barrett and Dr. Chiles that has
recently shown that kidney cancer can be put into remission
with the donation of normal peripheral blood stem cells, which
is a fascinating new development in the use of these cells to
treat cancer.
Therefore, as this work moves forward, an even greater
number of patients will benefit from adult stem cell
transplantation. As further evidence, I submit to you that the
two most important medications that we as oncologists give to
patients in their cancer treatments are epagen and nupagen,
which are both growth factors, both of which were developed and
isolated through federally-funded adult stem cell research.
These medications promote the recovery of red and white blood
cells following chemotherapy and allow patients' quality of
life to be significantly improved while also preventing life-
threatening infections.
Adult hematopoietic stem cells are the ideal vehicle for
gene therapy to treat such diseases as sickle cell anemia,
hemophilia and immune deficiencies. More than 100,000 children
in America are threatened with these life-threatening genetic
diseases. For the first time in the past 2 years, two recent
publications have shown the successful gene transfer into
patients with disease. Both of these studies used adult
hematopoietic stem cells.
At the end of the year 2000, there were 70,000 Americans
awaiting organ donation for kidney, liver, heart, or lung
disease. Recent animal studies indicate that the co-
administration of hematopoietic stem cells along with organ
transplantation may dramatically lessen the need for
immunosuppression. If this work moves forward, thousands more
patients will be able to be successfully transplanted with
organs with much less morbidity.
In addition to these current applications, even broader
clinical therapies will derive from adult stem cells in the
near future. Published reports in the last 5 years have shown
that transplants of bone marrow stem cells can differentiate in
vivo into functional liver cells, skeletal muscle, brain cells,
and even functioning cardiac myocytes. These data indicate that
a rare subset of hematopoietic stem cells is in fact
pluripotent and possess at least limited plasticity. Adult cord
blood stem cells can be maintained now in culture for up to 12
weeks. Our laboratory has shown that bone marrow stem cells can
be expanded 10-fold in just a week of culture under specialized
conditions. Whether adult stem cells will match embryonic stem
cells in the treatment of diseases like Parkinson's or
Alzheimer's remains unknown, and in my opinion is probably
unlikely. But given the dramatic progress in adult stem cell
research in the last 5 years, I think continued funding is
merited.
To close, I would like to just make two points about
embryonic stem cell research that are scientific concerns.
First, immune barriers for embryonic stem cells to work in
transplantation for treatments like Alzheimer's and Parkinson's
will require immune barriers to be crossed. Scientists who are
experts in this field, specifically, Dr. Thomson at Wisconsin,
have argued that cloning is the way to get beyond this. In
fact, I think the scientific community recognizes that if these
immune barriers cannot be crossed with some method such as
nuclear transfer, the translation into actual therapies for
patients will be extremely difficult.
Second, animal studies indicate that the transplantation of
embryonic stem cells actually has a high incidence of teratoma,
or tumor development, in small animal models. In vitro
differentiation and genetic engineering of embryonic stem cells
prior to transplant have been proposed as methods to circumvent
this problem, but both of these approaches have further
technical concerns. As Dr. Thomson himself has recently cited,
the heterogeneous nature of embryonic stem cell development in
culture has hampered the use of embryonic stem cell derivatives
in transplantation.
I close with the very brief following statement. The
limitations of embryonic stem cells as a source of
transplantable tissue should be openly and honestly presented
to the public, since treatments and cures using these cells are
certainly not imminent.
In contrast, adult hematopoietic stem cells are
successfully used in the treatment of patients with common
diseases every day. Genetic engineering is not required for
these cells to be safely applied. New treatments for diseases
are imminent with adult stem cells and not hypothetical. While
scientists continue to explore the basic biology of embryonic
stem cell lines, the needs of thousands of patients who benefit
from adult stem cell research should not be sacrificed.
Thank you.
[The prepared statement of Dr. Chute follows:]
Prepared Statement of John P. Chute, M.D.
1 would like to first thank Senator Kennedy and the Senate
Committee for inviting me to appear at this important hearing. As a
clinician who does research, it is an honor to be here. My specialty
training is in Internal Medicine, Hematology and Medical Oncology. Upon
completion of my clinical training in 1996, 1 joined the Stem Cell
Biology Laboratory at the Naval Medical Research Institute. Since June
1999, 1 have been the Head of the Hematopoietic Stem Cell Studies
Section within the joint NIDDK/Navy Transplantation and Autoimmunity
Branch. The focus of our lab has been the development of methods to
cultivate and expand adult hematopoietic stem cells for medical
therapies. Therefore, it has been with great interest that I have
followed the remarkable progress which has been made in the development
of human embryonic stem cell lines. I have admired the work of
investigators like Dr. James Thomson at the University of Wisconsin in
demonstrating that embryonic stem cell lines can be propogated in vitro
through hundreds of cell divisions and that these cells can be induced
to differentiate in vitro into neuronal, epithelial, muscle and
hematopoietic cells, to name just a few (1,2). In contrast, the
experience of investigators in attempting to propagate and induce the
in vitro differentiation of adult hematopoietic stem cells into diverse
tissues has been generally unsuccessful (3,4). Understandably,
physicians, legislators, the media, and most importantly, patients,
have become increasingly hopeful that the further development of human
embryonic stem cell research might lead to treatments and even cures
for diabetes mellitus, parkinson's disease, a1zheimers, muscular
dystrophy, and other degenerative diseases. Unfortunately, the
optimistic pronouncements by members of scientific community about the
``imminent'' therapies which would be forthcoming from embryonic stem
cell research has raised the innocent expectations of the most
vulnerable members of our society (patients with debilitating diseases)
to a level which medical researchers may never meet.
Last month, President Bush set forth a policy to provide Federal
funding to support ongoing research on specified human embryonic stem
cell lines. During his address, he noted that the Federal government,
via NIH, currently spends approximately 250 million dollars annually on
``stem cell research''. The overwhelming majority of this money
heretofore has been applied to basic and clinical research on adult-
source stem cells. In order to jump-start the Federal funding of
embryonic stem cell research, it has been discussed that 100 million of
the Federal dollars which the NIH would have appropriated toward adult
stem cell research will be taken to support the fledgling embryonic
stem cell initiative. Sacrificing 40% of the current adult stem cell
research funding for the purpose of initiating further embryonic stem
cell research would be a mistake of historical proportions and would
risk harming hundreds of thousands of patients in the United States who
currently benefit from adult stem cell based therapies.
The American Cancer Society estimates that there were 1.2 million
newly diagnosed patients with cancer in the U.S. in the year 2000 (5).
Cancer is the second leading cause of death in the United States and it
continues to gain on the leader, cardiovascular disease. Adult stem
cells from bone marrow, cord blood, and peripheral blood have been and
are currently used safely throughout the U.S. in the treatment and cure
of tens of thousands of patients with leukemia, lymphoma, and certain
types of solid cancers (6). Current research advanced by NIH
researchers further indicates that, when given in a
``nonmyeloablative'' manner (less toxic), patients with solid tumors
(e.g. kidney cancer) for the first time are achieving complete
remissions simply due to the anti-cancer effect of the infused bone
marrow cells (7). These results indicate that an even greater number of
cancer patients will benefit in the near future from adult stem cell
transplantation. But more research funding will be needed to meet these
goals. The success of adult stem cell research can also be measured by
examining the two most important medications given to all patients who
undergo chemotherapy and radiation: these medications, erythropoietin
(epogen) and neupogen are natural growth factors which act within human
bone marrow to stimulate red blood cell recovery and white blood cell
recovery following highly toxic chemo- and radiation therapy. Without
these 2 medications, thousands of cancer patients would require longer
hospitalizations, suffer significant life-threatening infections, and
patient quality of life would be dramatically worsened. Both of these
medications were discovered via research on adult hematopoietic stem
and progenitor cells.
In our laboratory, we have utilized Federal research funds to
search for the genes and proteins which induce the self-renewal of
adult hematopoietic stem cells (8). We are currently participating in a
cooperative research agreement with Large Scale Biology Corporation
(Vacaville, CA and Rockville, MD) to apply high level protein
identification techniques to achieve this end. We anticipate
identification of specific human stem cell growth factors within the
next 2 years. This research may lead to new therapeutic growth factors
which could be administered to patients with cancer to allow earlier
recovery from the adverse effects of chemotherapy and radiation. Yet,
despite its potent effects in the elimination of disease, bone marrow
transplantation is a morbid procedure for donors, requiring one liter
of marrow to be harvested for each adult transplantation. Umbilical
cord blood is an alternative and attractive source of hematopoietic
stem cells for this purpose, and the ease of collection and banking of
cord blood stem cells could allow for a universal bank of stem cells so
that the majority, not the minority, of Americans might have an
immunologically matched stem cell graft available for them should they
need it. But more Federal funding, not less would be needed to develop
such a national cord blood bank.
Adult stem cells from the bone marrow or peripheral blood are also
the ideal vehicle for gene therapy to treat such common and
debilitating diseases as sickle cell anemia, hemophilia, thallasemia,
and immune deficiencies (9). More than 100,000 children are afflicted
with life-threatening forms of these genetic diseases in the United
States (10). For the first time, 2 recent reports have demonstrated
successful gene transfer and gene expression of functional proteins in
patients transplanted with adult stem cells. As adult stem cell
researchers continue to provide new methods to successfully introduce
normal genes into adult stem cells, major diseases such as sickle cell
anemia and hemophilia will become treatable for the first time. In
addition, with the advent of the human genome database, an increasing
number of treatable genetic diseases will be identified. But more
research funding will be required to reach these goals.
As of the end of the year 2000, there were 70,373 Americans
awaiting organ donation for end stage kidney, liver, heart, or lung
disease (11). Organ transplant recipients face major morbidities and
life-threatening complications due to the immunosuppression which they
require in order to prevent organ rejection. Recent research in animal
models has indicated that concomitant infusion of donor bone marrow
stem cells can induce an ``immune tolerant'' State in the recipient
which may allow long-term acceptance of the donated organ without the
requirement for prolonged immunosuppression (12). The standardized
clinical transplantation of donor bone marrow coupled with organ
transplantation could allow tens of thousands of more patients to be
successfully cured of their end stage liver or kidney disease with much
less morbidity and complications.
In addition to these current clinical applications, ongoing
research indicates that even broader clinical therapies will derive
from adult stem cells in the near future. Investigators have
demonstrated in animal models that transplanted bone marrow stem cells
have the capacity to differentiate in vivo into liver cells, skeletal
muscle cells, brain cells, and even functioning cardiac muscle cells
(13-17). These data indicate that a rare subset of adult hematopoietic
stem cells are pluripotent and possess at least limited plasticity.
Scientists in the U.S. and Italy have recently demonstrated that cord
blood stem cells could be maintained and expanded in number in culture
for up to 12 weeks and our laboratory has recently shown that bone
marrow stem cells can be increased 10-fold in 1 week of culture under
specialized conditions (18-20). The capacity to expand the numbers of
human adult stem cells in vitro is a critical first step toward
harnessing these cells for future medical treatments. Whether adult
stem cells will be useful in the future clinical treatment of diseases
such as diabetes, parkinson's or a1zheimer's remains unknown and
perhaps unlikely. But given the dramatic discoveries of the past 5
years, along with the fact that adult stem cells are a proven safe and
genetically stable source of tissue for transplantation, continued
research on adult stem cells is merited.
Embryonic stem cells hold great promise as a potential source of
tissues for the treatment of many debilitating and degenerative
diseases. However, that potential faces several important scientific
hurdles prior to the realization of patient application. First, immune-
mediated rejection of transplanted embryonic stem cell grafts will
occur in recipients unless the individuals are heavily
immunosuppressed. This immunosuppression would result in major
morbidities and life threatening complications for patients which might
outweigh the potential benefits of the transplant (1). Alternatively, a
patient's DNA could be introduced into an enucleated egg, for the
purposes of generating blastocytsts (embryos) which contain embryonic
stem cells which would be immunologically identical to the patient.
Even if this form of cloning was not controversial, it is highly
inefficient and would likely require large resources of eggs for the
purposes of developing genetically matched tissue grafts. Second,
animal studies indicate that the transplantation of embryonic stem
cells leads to a high incidence of teratoma (tumor) development. This
risk is clearly incompatible with patient transplantation and requires
that these cells would have to be induced to differentiate in vitro
into desired tissue lineage or genetically engineered prior to
transplantation (1). Such transplanted cells would likely have a
limited in vivo life expectancy and might require additional
transplantations over time. These limitations of embryonic stem cells
as a source of tissue for human transplantation should be openly and
honestly presented to the public, since treatments and cures from these
cells are not imminent. Most importantly, the requisite studies in
small animals and primates to prove efficacy and safety are far from
completed. In contrast, adult stem cells are successfully used in the
treatment and cure of many diseases every day and genetic engineering
is not required for these cells to be safely applied. More importantly,
new treatments are imminent, not hypothetical. While scientists
continue to explore the basic biology of embryonic stem cell lines, the
needs of thousands of patients who benefit from adult stem cell
research should not be sacrificed.
References
1. Odorico J, Kaufman D, Thomson J. Multilineage differentiation
from human embryonic stem cell lines. Stem Cells 19:193, 2001.
Thomson J, Itskovitz-Eldor J, Shapiro S et al. Embryonic stem cell
lines derived from human blastocysts. Science 282:1145, 1998.
Traycoff C, Cornetta K, Yoder M et al. Ex vivo expansion of murine
hematopoietic progenitor cells generates classes of expanded cells
possessing different levels of bone marrow repopulating potential. Exp
Hematol 24:299, 1996.
Antoniou M. Embryonic stem cell research: The case against.... Nat
Med 7:397, 2001.
American Cancer Society Report: Facts and Figures 2000. Cancer:
Basic Facts. Chapter One, page 1, 2000.
International Bone Marrow Transplant Registry. Information
Services, Summary 2000.
Childs R, Chernoff A, Contentin N et al. Regression of metastatic
renal cell carcinoma after nonmyeloablative allogeneic peripheral blood
stem cell transplantation. N Engl J Med 343: 750, 2000.
Chute J, Saini A, Kampen R, et al. A comparative study of the cell
cycle status and primitive cell adhesion molecule profile of human
CD34+ cells cultured in stroma-free versus porcine microvascular
endothelial cell cultures. Exp Hematol 27:370, 1999.
Dick J. Gene therapy turns the comer. Nat Med 6:652, 2000.
American Sickle Cell Anemia Association. Frequently Asked
Questions: How common is sickle cell anemia? 2001; National Hemophilia
Foundation. Information Center, page 1, 2001.
UNOS Transplant Patient Datasource. Statistics 2000, National Data.
Hale D, Gottschalk R, Umemura A, et al. Establishment of stable
multilineage hematopoietic chimerism. and donor-specific tolerance
without irradiation. Transplantation 69:7, 2000.
Lagasse E, Connors H, Al Dhalimy M, et al. Purified hematopoietic
stem cells can differentiate into hepatocytes in vivo. Nat Med 6:1229,
2000.
1. Ferrari G, Cusella-De Angelis G, Coletta M et al. Muscle
regeneration by bone marrow derived myogenic progenitors. Science 279:
1528, 1998.
Mezey E, Chandross K, Harta G et al. Turning blood into brain:
cells bearing neuronal antigens generated in vivo from bone marrow.
Science 290:1779, 2000.
Brazelton T, Rossi F, Keshet G et al. From marrow to brain:
expression of neuronal phenotypes in adult mice. Science 290:1775,
2000.
Orlic D, Kajstura J, Chimenti S et al. Bone marrow cells regenerate
infracted myocardium. Nature 410:701, 2001.
Piacibello W, Sanavio F, Severino A, et al. Engraftment in nonobese
diabetic severe combined immunodeficient mice of human CD34+ cord blood
cells after ex vivo expansion: evidence for the amplification and self
renewal of repopulating stem cells. Blood 93:3736, 1999.
Chute J, Saini A, Wells M et al. Preincubation with endothelial
cell monolayers
increases gene transfer efficiency into human bone marrow
CD34+CD38progenitor cells. Hu Gen Tber 11: 2515, 2000.
Chute J, Wells M, Clark W, Park J, Harlan D, Stull M, Civin C.
Quantitative expansion of human bone marrow repopulating cells occurs
through activation of the primitive CD34+CD38- subset. Am Soc Hematol
Submitted 2001.
The Chairman. Thank you very much.
I thank all the members of the panel. We are going to
recess, and I would like to come back with some questions at
2:15. I know this causes some concern, perhaps, for your
schedules, but if you can come back--if you cannot, I can
understand, and we will submit some written questions. I do not
think it will take long; hopefully, we will have you out by 3
o'clock. So if we could do that, we would very much appreciate
it. We have had a very full morning, and the two parties have
caucuses that start at 12:30, where I see most of our members
have gone. I think that by 2:15, they will have ended, and
hopefully, we will have some additional participation.
I am very, very grateful to all of our panelists.
Senator Frist. Mr. Chairman.
The Chairman. Yes, Senator Frist.
Senator Frist. Let me just add that this is fascinating
based on the discussions this morning. If some of the panelists
cannot stay, if we could just submit to them written questions,
because we do not know what their schedules are. I do have
questions, and I do not think we can spend a long time this
afternoon, so if we could be permitted to submit written
questions.
The Chairman. Certainly.
Thank you. We will meet again at 2:15.
The committee stands in recess.
[Whereupon, at 1 p.m., the committee recessed, to reconvene
at 2:25 p.m. this same day.]
AFTERNOON SESSION
The Chairman. The committee will come to order.
I will invite our witnesses to return to the table if they
would, please. Thank you very much for adjusting your
schedules.
I would like to go through a few questions if I might, and
I will start with Dr. Melton. You expressed several concerns
about the restrictions placed on stem cell research under the
President's proposal. Do you believe that the restrictions
could significantly impede scientific progress and slow the
development of new cures using these cells?
Mr. Melton. Yes, I do. I do think they will impede
progress.
The Chairman. Do you want to elaborate on that? We talked
this morning about the issues of accessibility and reliability
and safety, contamination. As a researcher, what are your
concerns?
Mr. Melton. I could give one example. You could imagine
that with the cells that are available, scientists discover a
means by which they can turn human embryonic stem cells into
pancreatic beta cells for the treatment of diabetes. You would
then want to use human embryonic stem cells that have been
freshly derived in the absence of these potentially
contaminating mouse feeders and use those for therapies,
because that would eliminate concerns about known and unknown
viruses. As I understand it, the new cell lines which would be
derived would be ineligible for Federal researchers. That is
one example of the possible problems that will likely arise.
The Chairman. How concerned should we be about
contamination? Have you used products from other countries, and
what is your own sense or knowledge as to how high you think
the risk might be?
Mr. Melton. I am not really qualified to comment on that,
but I would say that the FDA, as I understand it, will treat
this as a xenotransplant, which means that the hurdles and the
requirements for using a therapy will be much more onerous.
Dr. Chute can probably comment more on the difficulties
with using animal products in treating humans.
The Chairman. Dr. Chute?
Dr. Chute. Thank you, Senator Kennedy.
In our laboratory, we worked with a feeder layer that was a
porcine feeder to grow adult hematopoietic stem cells. I have
had iterations with the FDA both on using a porcine and a human
feeder layer, and without question, there is a long series of
safety tests that you have to do on the human cells that are
cultivated with xeno feeder layers such as mouse/porcine. And
even with a human feeder layer, there is still a very, very
long list of safety tests that have to be done.
But I would make the comment that I do not think it is
impossible if you use a mouse feeder layer that the human cells
derived from those cultures could ever be used in the clinic. I
think it just makes it more arduous, without question. It might
add a year or a two potentially to the time before you get to
the clinic.
The Chairman. Well, you are certainly restricted now, under
the August 9 date, so you probably would not get there.
Dr. Chute. Correct.
The Chairman. Dr. Melton, after reviewing the agreement
signed by NIH and the University of Wisconsin, I see that it
expressly forbids use of the cells for therapeutic or
diagnostic purposes. What are the implications of restrictions
of this kind for the work you do in trying to find new
treatments for juvenile diabetes?
Mr. Melton. Well, first, I am sort of surprised by that
news, because I have not seen the agreement. But if it is the
case as you say that the cells could not be used for
treatments, I would think that would be a damning condition.
Clearly, there is no point in doing the research that I am
doing if it were not for the possibility of treating people. So
I would have to see the conditions to comment further.
The Chairman. Ms. Hersey, the President's plan gives a
handful of suppliers control over all the federally-approved
stem cell lines. Based on your experience negotiating
agreements with private companies, wouldn't you agree that this
type of monopoly is likely to make it difficult for NIH-funded
researchers to get prompt access to these cells?
Ms. Hersey. Yes, I think it is going to make it very
difficult, Senator.
The Chairman. Are you familiar with the other types of
situations that you know of from the past that are similar to
this kind of a monopoly?
Ms. Hersey. There have been several. Especially where the
company holds patent right, we have seen a number of them. In
some cases, the NIH has been able to step in as they have this
time and try to make a difference for the university
researchers. But I would say that at least 20 percent of the
time, we cannot get access to the materials we want because of
the encumbrances.
The Chairman. Dr. Childress, I want to underline one point
you made in your testimony. Is it fair to say that you see no
ethical differences between a stem cell derived from a
discarded embryo on August 9 and one derived at a later date?
Mr. Childress. As long as they meet the kinds of ethical
standards that President Bush laid out, and I think those are
important ones, about the embryo being left over following
efforts at reproduction, that the donors give voluntary
informed consent, and that there be no financial inducements.
If we go forward and apply the same ethical standards to the
derivation of stem cells from embryos in the future, I cannot
see an ethical difference. I cannot see that what happened
before August 9 and what happens after is ethically different.
The concern that some have expressed, that this might well
sanction and encourage the destruction of embryos, I think is
also problematic as a concern, because after all, to this
point, people may well have made decisions about destruction of
embryos and the possibility of privately-funded research, since
that research has been going forward.
Furthermore, we really do not have evidence from fertility
clinics that people deciding to discard embryos make this a
major factor in decisionmaking. So I would be inclined to say
that we can build the ethical safeguards to prevent the kind of
scenario that has concerned many.
The Chairman. You have followed the fetal transplantation
issue closely.
Mr. Childress. Yes.
The Chairman. Have you formed any impression--and maybe I
could ask others on the panel as well--in that debate, we had
the agreement for the use of certain fetal tissue, but we also
established guidelines for utilization in the private sector--
have you thought about that issue as well, and can you tell us
what your thinking has been, what the advantages or concerns
would be, or perhaps the disadvantages?
Mr. Childress. First, I think there are significant
parallels between the kinds of ethical safeguards one would try
to set up, which appear to have been effective, in the area of
human fetal tissue transplantation research, and the kinds of
guidelines that would be appropriate in two different settings.
One would be the use of embryos left over after in vitro
fertilization, but also the research that we have not really
talked about today, the derivation of embryonic germ cells from
aborted fetuses. One could draw a parallel there also, and that
area of research has been omitted from much of the recent
discussion, and I am not sure whether it merits further
attention or not. I have not followed the scientific
developments on that side, but one could perhaps make a case
for paying some attention to that as well.
The Chairman. Throughout your distinguished career, you
have shown a deep reverence for life. Do you think that
allowing federally-funded doctors both to use and derive stem
cells from discarded embryos is consistent with those deeply-
held beliefs?
Mr. Childress. I believe so, first of all, if we work with
the notion which the National Bioethics Advisory Commission
also tried to articulate, that it is very important to
recognize that the embryo deserves an appropriate form of
respect, appropriate to that stage of developmental life. Now,
there will be widespread disagreement in society, as I have
mentioned earlier, about the moral status of the embryo and
exactly what kind of respect it deserves, what kind of
protection is appropriate.
On the National Bioethics Advisory Commission, we drew
several implications from the notion of respect for the early
embryo. One certainly was that in this area, we should not be
buying and selling embryos or, if we go in the direction of
fetal tissue, we should not be buying and selling fetal tissue.
One could also argue that we should not use left over
embryos unless they are necessary.
Now, there would be considerable debate. I happen to be on
the side of those who think that, given the promise of this
research for alleviating suffering and reducing the incidence
of premature death, we ought to be exploring all sources of
stem cells at this point. And I certainly agree with my
colleagues who stress that we should not neglect adult stem
cells in this process, and just seeing which ones will be most
important in developing the kinds of treatments and, we hope,
cures that might be possible.
One implication that we drew from this principle of respect
for the early embryo was that at this point, we should not
deliberately create embryos for purposes of use in research,
whether it is through IVF or so-called therapeutic cloning.
We emphasized--and I would agree with this emphasis--that
the point we were making was ``at this time,'' because it may
well be necessary to revisit this question of so-called
therapeutic cloning if the basic research ends up producing
some clinically effective treatments that may well best be
done, or perhaps only be done, with matched tissue. So it may
be necessary to revisit that at some point. But we have heard
from many colleagues, and we still have a long way to go before
that becomes a critical question again and then might raise the
issue of the necessity of going in that direction.
I would be opposed, though, at this point to a ban on
therapeutic cloning, worrying that that would indeed set an
inappropriate limit for future developments.
The Chairman. Thank you.
We will submit the questions of our colleagues.
[Response to questions from committee members were not
available at time of printing.]
The Chairman. I am grateful to the panel, and I thank all
the witnesses for their excellent testimony. It has been an
extremely informative and important hearing. It is clear that
stem cell research offers a virtually unprecedented opportunity
to find cures for a host of dread afflictions from cancer to
heart disease, from diabetes to spinal cord injury to
Parkinson's disease to Alzheimer's disease.
It is also clear that there are serious concerns in the
scientific community about whether the restrictive rules
currently imposed by the Bush Administration will allow this
research to proceed speedily and effectively. These concerns
range from the number, safety and durability of the existing
cell lines to whether they will be truly widely available to
researchers. The memorandum of understanding that Secretary
Thompson announced this morning specifically prohibits the use
of the cell lines in clinical research, the research that is
done to actually test possible treatments for illnesses.
Millions of patients and their families expect that stem
cell research will move forward as rapidly as possible. It will
be unacceptable to offer these patients and families the
promise of effective stem cell research but deny them the
reality of it. We will continue to examine the questions raised
at the hearing, and I am optimistic that Congress will take
whatever steps are necessary to ensure that stem cell research
proceeds effectively and ethically.
The committee stands in recess. Thank you all very much.
[Additional material follows:]
ADDITIONAL MATERIAL
Statement of the Alpha-1 Foundation
Alpha-1 Foundation supports S. 723: Embryonic Stem Cell Research.
The Alpha-1 Foundation is a national not-for-profit organization
dedicated to providing the leadership and resources that will result in
increased research, improved health, worldwide detection and a cure for
Alpha1-Antitrypsin Deficiency (Alpha-1). Alpha-1 is a
genetic disorder that results in devastating and fatal lung and liver
disease. Alpha-1 is a major cause for lung transplantation in adults
and a leading cause for pediatric liver transplants. Diagnosed patients
can engage in preventative health measures. Treatment of the lung
disease associated with Alpha-1 consists of a sole therapy that is
derived from human plasma, infused weekly and is in critically short
supply. Individuals with the pulmonary destruction associated with
Alpha-1 often require supplemental oxygen and suffer the pain known
only to those unable to catch their breath.
The Alpha-1 Foundation has joined the Coalition for the Advancement
of Medical Research whose membership includes voluntary health
organizations, scientific and academic societies, industry,
universities, medical organizations, and others to add our voice in
support of the initiative to reinstate the Federal guidelines for
embryonic stem cell research. The Foundation supports the NIH
guidelines in the belief that they were developed with stringent
oversight and that Federally funded research will allow for this
research to move forward in an ethical manner.
Further the Alpha-1 Foundation Medical and Scientific Advisory
Committee (MASAC) passed a resolution supporting S. 723 because
embryonic stem cell research may hold great promise in the search for a
cure for Alpha-1. The list of Alpha 1 Foundation MASAC members and a
copy of the resolution in support of S. 723 is attached.
alpha-1 foundation medical and scientific advisory committee resolution
regarding stem cell research
Whereas, the Medical and Scientific Advisory Committee of the
Alpha-1 Foundation (MASAC) is aware of the many issues surrounding the
use of stems cells in biomedical research and the debate regarding the
National Institutes of Health Stem Cell Guidelines, and,
Whereas, MASAC appreciates the potential role of stem cell research
in identifying a cure for individuals with the genetic deficiency of
Alpha1-Antitrypsin,
Therefore, MASAC resolves the following:
1. MASAC endorses the current Federal Stem Cell Guidelines as set
forth by the National Institutes of Health.
1. MASAC endorses and recommends passage of US Senate bill S. 723,
sponsored by Senators Specter and Harkin.
Further, MASAC recommends these resolutions be included in a letter
addressed to both Senators Specter and Harkin and recommends that
individuals address letters to their Congressional members requesting
support of S. 723.
medical and scientific advisory committee members
James K. Stoller, M.D., Chair, Cleveland Clinic Foundation
Mark L. Brantly, M.D., University of Florida College of Medicine
Manuel G. Cosio, M.D., Royal Victoria Hospital, McGill University
Frederick deSerres, Ph.D.*, Chapel Hill, NC
Robert J. Fallat, M.D., California Pacific Medical Center
Ann R. Knebel, R.N., D.N.Sc., NIH, National Institute of Nursing
Research
Joe Reidy*, Waldwick, NJ
Caroline Riely, M.D., University of Tennessee, Memphis
Robert M. Senior, M.D., Washington University School of Medicine
Edwin K. Silverman, M.D., Ph.D., University of Maryland School of
Medicine
Gordon L. Snider, M.D., Boston VA Medical Center
Charlie Strange, M.D., Medical University of South Carolina
Bruce C. Trapnell, M.D., Children's Hospital and Medical Center
Gerard M. Turino, M.D., Columbia University College of Physicians &
Surgeons
Catherine A. Valenti*, Meridian, ID
Debbie Waldrop, M.S.N., R.N., C.C.R.C., University of Texas at
Tyler
Thomas B. Witt*, Severn, MD
Bioethicist Consultant, Evan DeRenzo, Ph.D., Center for Ethics/
Washington Hospital Center
Ex-Officio, Robert A. Sandhaus, M.D., PhD, FCCP, Alpha-1 Foundation
John W. Walsh*, President and CEO, Alpha-1 Foundation
*Diagnosed with Alpha1-Antitrypsin Deficiency
Statement of Don C. Reed
Dear Senator Kennedy, honorable Committee Members: Seven years ago,
my son Roman Reed suffered an accident while playing college football.
His neck was broken; he became paralyzed from the shoulders down. Since
then, our family has become involved in the struggle to find a cure for
paralysis. We were fortunate to have a new law passed in California,
the Roman Reed Spinal Cord Injury Research Act, setting aside a small
amount of money each year for paralysis research.
Imagine our joy, therefore, to hear about the amazing possibilities
of embryonic stem cells. If new nerve cells could be therapeutically
cloned, and imprinted with Roman's DNA pattern, his own body could
regrow nerves to heal the damaged spine. Our son might be able to close
the fingers of his hands again, maybe even rise and walk.
Unfortunately, despite President Bush's public commitment to allow
embryonic stem cell research, steps are being taken which will
effectively kill that research.
1. The President supports and has promised to sign House Resolution
2505. Under this terrifying anti-science law, it will be a Federal
crime to make embryonic stem cells: a felony, punishable by a ten-year
jail sentence, and a one million dollar fine. H.R. 2505 treats
therapeutic cloning of cells as if it was the reproductive cloning of
humans. Obviously, to multiply infant copies of ourselves is wrong, and
should be illegal. But therapeutic cloning? That is about cure: making
cells, healing people, saving lives. Comparing therapeutic and
reproductive cloning is like comparing a surgeon's scalpel to a
criminal's switchblade. Their purposes are completely different.
Under H.R. 2505, if a researcher found the answer to cancer,
paralysis, or AIDS--but cloned just one embryo to make stem cells--he
or she would have to receive the Nobel Peace Prize in jail.
2. The President is also shutting off the only other source of
embryonic stem cells; he will not allow the scientific use of embryos
left over from fertility procedures. Under the Bush guidelines, no more
new embryonic stem cell lines can be made. Ever.
3. The Administration's list of 64 viable stem cell lines is
neither sufficient nor even accurate. Sweden, for example, is credited
with seventeen ``robust, vital'' lines. The Swedes made a phone call to
correct this, stating they have three usable lines, not seventeen. Only
ten laboratories in the world even have viable embryonic stem cell
lines. America has four.
4. Even these extremely few stem cell lines can never be used to
help people. As is the case with all new science, experimental
animals--(laboratory mice)--were used to make the lines. Food and Drug
Administration guidelines on inter-species experiments disqualifies
them for human cure. If we could use therapeutic cloning, this would
not be a problem. We could just manufacture some more--but H.R. 2505
makes that illegal.
The President's proposal, then, leaves us with nothing but the
promise of what might have been.
This decision will hurt every American. For those who suffer
crippling and life-threateninq diseases now, and the families who watch
them suffer, our most promising possibility of cure has been denied.
For sheer financial self-interest alone, the quest for cure must be
allowed. Our country faces an increasingly unpayable mountain of
medical debt, public and private. It is overwhelmingly expensive to
provide longterm hospitalization and attendant care.
Example: spinal cord injury, which my son Roman has, costs America
approximately $20 billion a year in medical costs and lost wages.
That's about $170 per taxpayer, for just one medical condition. And the
cost in suffering to 450,000 paralyzed Americans and their families?
That terrible price can never be calculated.
Why would anyone want to deny cure to the injured and critically
ill? The problem, conservatives point out, is that when we dissect an
embryo to obtain stem cells, we are destroying living tissue. That
near-microscopic dot is technically alive.
And there is our choice. Like the battlefield medic who decides
which soldier's life to try and save, because he cannot save them all,
we too must choose; a 5-7 day old collection of cells in a glass petri
dish--or a hundred million suffering people.
Think of folks you know. Like President Ronald Reagan, who has
Ahlzheimer's disease. Or Michael J. Fox, with Parkinson's. Mary Tyler
Moore, juvenile diabetes. Magic Johnson, HIV. Elizabeth Montgomery, who
died of cancer. Vice President Dick Cheney, heart disease. Christopher
Reeve, spinal cord injury. Annette Funicello, multiple sclerosis. And
other folks, out of the public eye, like a soldier terribly burned on
the battlefield and living in continual pain, or my sister Patty, who
died of leukemia at age 24. Perhaps, God forbid, even someone in your
own family.
Whose rights shall we protect--our loved ones, the living people of
the world--or a dot in a dish, a collection of cells which can neither
think nor feel?
Honored committee members, you who will make this momentuous
decision: do not feel rushed. Give us your best. For in your hands are
the hopes and dreams of those imprisoned by infirmity, confined to a
lifetime of wheelchairs and hospital beds, and the endless humiliations
of helplessness.
As my paralyzed son Roman puts it: ``Take a stand with us today, in
favor of research for cure. Take a stand--so 1 day, everybody can.''
Thank you.
Don C.Reed is the father of Roman Reed, and the sponsor of
California's Roman Reed Spinal Cord Injury Research Act.
Statement of Christopher C. Straub
I am the Executive Director of the Culture of Life Foundation, an
educational foundation dedicated to finding and spreading the
scientific truths that confirm the dignity and inviolability of every
human life from the moment of conception until natural death. I am
submitting testimony today in favor of holding to the course set by
President Bush in his August 9th statement. In other words, I urge this
Committee and the Senate to support robust funding for adult stem cells
and stem cells derived from umbilical cord blood and placentas, and to
reject any increase in the number or sources of embryonic stem cell
lines beyond those already authorized by the President.
Our Foundation was deeply disappointed in the portion of the
President's August 9th statement which permitted Federal funds to be
expended on research on stem cell lines which are the fruits of the
prior killing of human embryos. We also recognize that the President
drew clear lines of limitation, both in the number of embryonic stem
cell lines authorized for Federally funded research, and in the
prohibition on Federally funded embryo destruction.
In support of maintaining those lines, I will review for you some
facts not well covered in the news media: the tremendous advances
already made by researchers and clinicians using adult stem cells. But
even if adult stem cells were worthless and embryonic stem cells
guaranteed immortality, I would still urge you to reject the funding of
research on embryonic stem cells.
The embryo is a human being, fully equipped with the 46 chromosomes
of a human being and with the complete genetic data that mark its
unique humanity. All it needs to develop into a person as apparently
independent as the rest of us is to be appropriately protected and
provided with food and oxygen. I will not mention the embryo's size or
weight or appearance--to reject its humanity on those superficial
grounds is not a serious argument in this age of science. The argument
that its humanity is based on its location (e.g., a uterus) rather than
its nature is likewise absurd; consider whether or not an embryo would
be human if it were placed in some artificial womb of the future. To
deny the embryo's humanity based on the location of the embryo is to
determine its humanity based on the intentions of the person who has
power over it. In this view, if the embryo is intended to survive and
grow, then it is human; if it is intended to be killed, then it is not
human. By this reasoning, there are no human persons on the death rows
of our penitentiaries.
The embryo's humanity from fertilization onward is not a matter of
religious faith, but of reason applied to scientific data. ``The
genetic information (DNA), which will determine a person's physical
characteristics and much of his intelligence and personality is present
at fertilization. Fertilization is the process during which a male
gamete or sperm . . . unites with a female gamete or oocyte . . . to
form a single cell called a zygote. This highly specialized, totipotent
cell marks the beginning of each of us as a unique individual''
(William J. Larsen, Essentials in Human Embryology, New York: Churchill
Livingstone, 1998, p. 17). To determine the embryo human at any point
subsequent to fertilization is arbitrary, subjective, unscientific, and
may coincide with the personal interests of those making the
determination. All the talk about ``treating the embryo with dignity''
is nothing more than a nervous tiptoeing around the awesome and simple
truth of its human personhood.
To obtain stem cells from an embryo, the embryo--this person--must
be killed. Killing innocent people is considered evil in Judeo-
Christian morality and in the laws that derive from that morality, the
laws by which we govern ourselves. Throughout human history, it has
been demonstrated repeatedly that we humans cannot obtain a good
outcome from an evil action. This rule operates with mathematical
certitude. That is why embryonic stem cell research is objectionable
and why tax dollars should not be used to pay for it.
The President, of course, did not authorize the killing of any
embryos, and I am grateful for that. He did authorize Federal funding
for research on 64 stem cell lines derived from embryos that had
previously been killed, and that was a mistake on his part. I ask you
to imagine you are a medical researcher in Germany sixty years ago and
the German Army contracts with your laboratory to research a diet and
medications to help soldiers resist extremes of cold. The data you are
given to work with comes from experiments on condemned prisoners who
were forced to participate and who in most cases froze to death in the
course of the experiment. If you know the source of the data, it is
wrong for you to work on this contract. This is the situation with the
stem cell lines upon which Federally funded research is permitted:
although the killing was done elsewhere, by another, it is wrong to
seek the fruits of it. That is the basis of our Foundation's objection.
The work on the 64 stem cell lines is also the seed of more
killing, as scientists State their requirement for more and better
embryonic stem cells than those authorized by the Administration. The
authorized cell lines may not be sufficient. Many of them may be
tainted by contact with feeder cells from mice. More work reportedly
remains to be done on some of the cell lines to be sure of their
viability. There may be fewer than 64 viable lines among those the
Administration has authorized; the August 29 New York Times reported
that a hospital in Sweden, which the Administration asserts has
nineteen lines, by its own account has only three established lines,
four more that are being studied, and twelve that are in early stages.
In addition, the ownership of patents and rights to the process by
which the cells were derived and developed is not completely clear, and
none of the corporate and academic laboratories seem disposed at this
point to imitate Dr. Jonas Salk's generosity with his epochal
discovery.
If more cell lines are demanded, the in vitro fertilization (IVF)
industry reportedly has an overstock of at least 100,000 frozen embryos
that were supposedly a rich source for stem cell research. But that
source may have been a mirage: the August 26 New York Times reported
that most parents of frozen embryos created in the IVF process do not
want to contribute their embryos to science. The only other source may
therefore be embryos specifically created to be destroyed in research.
Many of the lines President Bush has authorized for federally funded
research are probably in this category, and laboratories like the Jones
Institute for Reproductive Medicine are ready to make many more.
Against this possibility I again recall to you the human personhood of
every embryo, even one destined for destruction within days of
creation. No potential good can justify such a killing.
With the destruction of embryos for medical research we see the
division of humankind into two parts: those who are destined to be
killed for the benefit of others, and those who benefit from the death
of another. This is as profound an inequality as has ever existed in
human history.
Meanwhile, research on stem cells from postnatal human tissue and
from umbilical cords and placentas continues to make spectacular
breakthroughs in the treatment of human patients as well as laboratory
animals. With exception of the news this summer that adult stem cells
reside in one of our Nation's most bountiful resources, human fat,
there has been little coverage of the spectacular breakthroughs made
using adult stem cells against some of the very conditions and diseases
which are used to justify the killing of embryos. Our foundation tracks
these discoveries and issues a report each week, and I submit a list of
those discoveries. You should know that adult stem cells have already
been used to create knee cartilage, heart muscle, liver tissue, kidney
cells, and bone mass in real human patients. Adult stem cells have been
used to grow restorative tissue around spinal cord injuries, to restore
vision, and to help people suffering from diabetes to become insulin-
free. Adult stem cells have been used to treat Crohn's disease, to
overcome immune deficiency in children, to restore bone marrow in
people with leukemia, and to fight a wide variety of cancers. Fresh
successes are reported almost daily. For example, the August edition of
Archives of Dermatology reported that doctors at the M.D. Anderson
Cancer Center in Houston, Texas, used adult stem cells to cure a man
with rare but devastating skin disease, scleromyxedema. This disease
had stiffened and thickened the patient's skin to the point he could
not close his eyelids or eat. His doctors collected adult stem cells
from his own bone marrow, then destroyed his immune system with
chemotherapy and transplanted his stem cells back into his body to
permit them to rebuild his immune system. As a result, the
scleromyxedema is now in remission.
There are also spectacular nonstem cell treatments being developed
to fight the diseases and disorders most often cited as a rationale for
embryonic stem cell research. Against Alzheimer's, for example, a
university in Tokyo has discovered a protein with the potential to
prevent or reverse the disease, and researchers in Dublin and at New
York University have separately discovered what may be an Alzheimer's
vaccine. Against cancer, nonstem cell developments include new toxins
to be directed against cancer cells, using red blood cells guided by
ultrasound to deliver anti-cancer drugs directly to tumors, and
developing antibodies to deliver lethal radiation to a tumor.
Adult stem cells have another advantage: when taken from the
patient the possibility of tissue rejection is greatly reduced. For
this reason, many Americans are now banking their infant's umbilical
cords.
The performance and the promise of adult stem cells are so great,
and the news blackout and scant attention paid them by elite opinion is
so marked, that there may be some motive at work besides concern for
health. Whether it is a desire to derive a supposed benefit from the
death of an embryo, the attraction of huge grants and concomitant fame,
or the urge to smash another traditional human taboo, the embryonic
stem cell is supposedly the new hope of mankind and the adult stem
cell, which has already accomplished much more, is ignored. Unless we
wake up to the facts and respond to them by reinforcing the success of
adult stem cells, we will delay the day we conquer some of the cruelest
diseases and conditions that afflict us, while at the same time
becoming a nation that kills out of utilitarian motives.
Statement of the Culture of Life Foundation
diabetes
Adult Stem Cell Research
Adult Stem Cells Cure Type I Diabetes in Mice: Dr. Denise
Faustman of Harvard University Medical School has discovered an
exciting potential new therapy that could possibly cure type I
diabetics. The treatment effectively cured diabetes in mice, and human
trials are currently being set up. Patients with type I diabetes have a
defect in their immune system wherein their white blood cells (WBC's)
inappropriately attack their own ``pancreatic islet cells'' in their
pancreas. These cells are the primary producers of insulin for the
body. Dr. Faustman's experiments entailed killing the defective WBC's
in mice with diabetes by giving them certain drugs. She then planned to
somehow ``reprogram'' the new WBC's that would be produced, so that
they wouldn't attack the pancreatic islet cells again, and then to
transplant new islet cells into the mice. However, she found that there
was no need to do so. After the defective WBC's were gone, adult stem
cells in the mice ``took over,'' and started producing new pancreatic
islet cells within 40 days. If the human clinical trials prove
effective, this treatment would be helpful for not only diabetics, but
also for other diseases that involve similar ``autoimmune'' symptoms,
like lupus, rheumatoid arthritis, multiple sclerosis, and more than 50
other illnesses. All without need of embryonic stem cells.
Source: Cromie, William; ``Adult Stem Cells Effect a Cure: Diabetes
cure may eliminate need for embryo cells''; Harvard University Gazette,
July 19, 2001, at http://www.hno.harvard.edu/gazette/index.html/01-
stemcells.html.
Non-ES/Fetal Cell Research
Formation of Insulin-Producing Pancreatic Islet Cells from
Adult Pancreatic Tissue: A major goal with scientists studying diabetes
is to come up with a good source of pancreatic cells that produce
insulin. One of the primary insulin-producing cells in pancreases are
``islet cells'', which secrete insulin in response to glucose
stimulation. In patients with type I and type 2 diabetes, a transplant
of such cells would be tremendously helpful, since not nearly enough
cadaver pancreas transplants for such patients become available on
time. In July 2000, scientists at Harvard Medical School announced that
they had found that by specially-preparing and nurturing adult
pancreatic ductal tissue (which normally does not produce much
insulin), they were able to coax the ductal cells to form pancreatic
islet cells. These pancreatic islet cells were then further found to
produce insulin, with increased amounts produced when stimulated with
glucose. If this method of pancreatic cell differentiation and growth
can be optimized to produce large amounts of cells, it could mean an
important new type of cell replacement therapy for type 1 and type 2
diabetics.
Source: Bonner-Weir, et al.; ``In vitro cultivation of human islets
from expanded ductal tissue''; Proceedings of the National Academy of
Sciences USA; July 5, 2000; 97 (410); 7999-8004.
Insulin-Producing Pig Cells Are Helping Diabetic Baboons:
Scientists at Duke University have demonstrated that specially-coated,
insulin-producing pancreas cells from pigs are keeping a diabetic
baboon off of insulin. The pig pancreas cells were encapsulated within
a complex carbohydrate known as alginate. This coating had pores that
were large enough to allow insulin to be released, but small enough
that the baboon's immune system antibodies and cells were not triggered
to give a noticeable response to the foreign cells. The encapsulated
pig cells were injected into the baboon's peritoneal cavity. The baboon
has not needed extra insulin for 9 months, since the injection. With
the success of this treatment, it is estimated by the researchers that
human clinical trials could begin within the year. Some organizations
dedicated to the treatment of diabetes are supporters of embryonic stem
(ES) cell research. However, there have been no such successful studies
to date using ES cells to treat diabetes; likewise, no ES cell studies
have shown such promise or possibility to move to human clinical trials
so quickly.
More information can be found at: http://www.dukenews.duke.edu/Med/
baboon.htm. (From 6/15/01 Duke University News Release)
Fat Cell Hormone Controls Diabetes: A hormone naturally
produced by fat cells shows promise for treating diabetes. The hormone,
called adiponectin, was recently found to be able to reduce the
severity of diabetes in mice. It was able to normalize elevated levels
of glucose in mice, and was also able to regulate the production of
glucose by rat liver cells. Both of these abilities could help to treat
diabetes in humans. One biotech company, Genset in Paris, France, is so
encouraged by the findings that they are already planning clinical
trials in humans using the hormone. There is so much potential with
such nonembryonic/nonfetal research, that there is no need to delve
into research on diabetes using human embryonic stem cells.
Sources: Science http://www.sciencemag.org/ (need subscription);
article entitled ``Hormone Keeps Diabetes in Check'' by Josh Chamot in
ScienceNow section of journal); also Berg, Anders et al.; ``The
adipocyte-secreted protein Acrp30 enhances hepatic insulin action'';
Nature Medicine; August 2001; Vol. 7, No. 8; pp. 947-953.
alzheimer's disease/parkinson's disease/brain & spinal cord injury
Adult Stem Cell Research
Neural cells isolated from adult cadaver human brains were
found to be able to grow into different types of neural cells,
including neurons, astrocytes, and oligodendrocytes. (Palmer, et al.;
``Cell culture: Progenitor cells from human brain after death'';
Nature; May 3, 2001 (411); 42-43.)
Human umbilical cord blood treated with growth factors
express markers that indicate that they can give rise to neurons.
(University of South Florida Health Science Center Press Release, May
9, 2001 and February 18, 2001; also presented at May 6, 2001 American
Academy of Neurology Meeting)
Adult Neural Stem Cells Purified From Mouse Brain Can Differentiate
Scientists in Australia report in this week's journal
Nature that they were able to purify adult stem cells from mouse brain.
This is normally somewhat difficult, since it has been estimated that
stem cells number only 1 for every 300 cells in the adult brain. But
they were able to do so via modem cell separation techniques, and have
also been able to stimulate their collected neural stem cells to
differentiate into muscle cells by growing them together with other
muscle cells. If it is found that human neural stem cells can do the
same, such adult stem cells could possibly be used to treat spinal cord
or muscle (including cardiac) injury and many brain diseases (such as
Alzheimer's or Parkinson's), without the use of a single embryonic stem
cell.
Source: Rietze, Rodney, et al.; ``Purification of a pluripotent
neural stem cell from the adult mouse brain''; Nature; Vol. 412, Aug.
16, 2001; pp. 736-739; also Pearson, Helen; ``No stemming the tide'';
Nature; Vol. 412, Aug. 16, 2001.
Adult Skin Stem Cells Can Turn Into Brain Tissue
Researchers at McGill University in Montreal, Canada have
found that they can repeatably isolate adult stem cells from the skin
of mice. Repeatability is a necessary objective for researchers with
stem cells, and so is versatility--the ability to become more than one
type of cell. These skin stem cells, called SKPs (for SKin Precursor
cells) have both going for them. Over and over, the scientists were
able to coax the isolated stem cells to form other types of cells, from
neurons to muscle cells to fat cells. The scientists' studies with
adult human scalp tissue have found that similar stem cells can be
found in human epidermis. This is extremely encouraging, both from an
ethical point of view and from an immunological point of view. If taken
from a patient's own skin, SKP adult stem cells would likely provoke
little or no immunological response, and would not necessitate the use
of strong drugs to suppress the patient's immune system. In contrast,
the use of embryonic or fetal stem cells would both be immoral and
would likely necessitate the use of such drugs. SKP stem cells appear
to have much potential for use in diseases or disorders of the central
nervous system.
Sources: Toma, Jean, et al.; ``Isolation of multipotent adult stem
cells from the dermis of mammalian skin;'' pp. 778-784; Sept. 2001,
Vol. 3, issue 9 (published online on 13 Aug. 2001), also http://
www.washtimes.com/national/20010814-73539816.htm (8/14/01 Washington
Times article by Carolyn Abraham entitled, ``Stem Cells from Skin Grow
Into Brain Tissue''); also, http://www.sciencedaily.com/releases/2001/
08/010814063557.htm (ScienceDaily 8/14/01 report) and http://
www.mcgill.ca/public/releases/2001/august/stemcells/ (McGill University
8/13/01 Media Release)
Other Non-ES/Fetal Cell Research
At Keio University in Tokyo, scientists have discovered a
protein that stops the death of brain cells that occurs in Alzheimer's
patients. The findings were reported in the May 22, 2001 edition of the
Proceedings of the National Academy of Sciences. This protein, called
humanin by its discoverers, may not only prevent further deterioration
from Alzheimer's, but could be the first step in curing the disease.
Alzheimer's is one of the diseases most often mentioned in
justifications of embryonic stem cell research.
NYU Researchers Design Successful Vaccine Against
Alzheimer's Disease: Scientists at New York University have created a
vaccine against Alzheimer's disease which has proven effective in mouse
studies at removing amyloid plaques in the brain. These plaques are
aggregated clumps of the protein amyloid in the brains of Alzheimer's
patients, which are thought to contribute to the characteristic memory
loss, confusion, and mood disturbances in such patients. Like the
recent report of an Alzheimer's vaccine produced by the Elan
Corporation in Dublin (Adult Stem Cell Research Weekly Highlights #3)
this vaccine is also made of a synthetic protein. However, the NYU
group believes that their vaccine has less chance of causing later
potential plaque formation than the vaccine from the Dublin group.
Their experimental results were very promising in mice with plaques: 7
months after injection with the vaccine, a bad form of the amyloid was
reduced in their brains by 89 percent in the cortex (the seat of higher
thought), and by 81 percent in the hippocampus (the brains memory
center), compared to the control, nonvaccinated groups. In addition,
the vaccinated mice had 57 percent less soluble amyloid present. This
is yet another promising discovery in treatment options for Alzheimer's
disease which does not resort to utilizing embryonic or fetal stem
cells.
Source: http://www.eurekalert.org/ (from 8/2/01 report)
Alzheimer's Drug Being Used to Treat Brain Injury: The
University of Florida and 14 other centers will soon participate in
clinical trials testing a drug, donepezil (more commonly known by the
trade name Aricept, and manufactured by Eisai Co. Ltd.), in patients
with brain trauma. The drug is currently used to slow memory decline in
Alzheimer's disease. It is thought that because patients with brain
trauma often have trouble with memory and cognition as with Alzheimer's
disease, an effective treatment for Alzheimer's could be helpful for
treating brain injury. Likewise, people that have received brain trauma
have been found to be more likely to develop Alzheimer's later in life.
Thus, a treatment that alleviates the symptoms of brain trauma could
also have the effect of stemming the onset of Alzheimer's. This is the
first randomized clinical trial ever conducted that treats chronic
traumatic brain injury. It does not necessitate the usage of embryonic
or fetal stem cells.
Source: http://unisci.com/stories/20013/0801012.htm (from 8/l/01
report)
Manipulation of a Single Gene in Neurons Can Lead to
Profuse Cell Regeneration: Could Lead to New Treatments for Brain and
Spinal Cord Patients: A July 1, 2001 News Release from the National
Institutes of Health/National Institute of Neurological Disorders and
Stroke focuses on the recent work of researcher Maureen L. Condic,
Ph.D. (University of Utah School of Medicine in Salt Lake City). She
has shown that by genetically modifying adult rat neurons to produce
more of a protein called integrin, a dramatic increase in nerve fiber
growth could be achieved. The amount of growth was more than 10 times
greater than in any other published study of adult neuron regeneration,
and was indistinguishable from neuron growth in newborn adult animals.
Previous studies had primarily focused on somehow changing the
environment around neurons to stimulate them to grow, but this study
shows that a key to neuronal growth may be as simple as changing the
performance of one gene. This could lead to better treatment strategies
(and nonembryonic stem cell research ones) for brain and spinal cord
injury, as well as for Parkinson's and Alzheimer's diseases.
Sources: July 1, 2001 News Release from the National Institutes of
Health/National Institute of Neurological Disorders and Stroke
(original source) and http://www.sciencedaily.com/releases/2001/07/
010702084939.htm
Anti-Alzheimer's Antibody Gives Clues to Why it Decreases
Development of Plagues in Brain: Researchers at Washington University
School of Medicine studying the function of an anti-Alzheimer's
antibody have found that the antibody draws a protein called amyloid-
beta out of the brain in mice and diverts it to the bloodstream.
Amyloid-beta contributes to the formation of ``amyloid plaques'' in the
brains of Alzheimer's disease patients. Scientist David M. Holtzman,
M.D. says, ``Within hours of injecting the antibody into mice, the
concentration of amyloid-beta in the bloodstream rose approximately
1,000 times higher than it had been before the injection.'' In
addition, animals injected with the antibody over a period of months
developed less amyloid plaques than control animals did. The difference
between the groups was statistically significant. Utilization of such
an anti-Alzheimer's antibody could be a useful and nonmorally
problematic therapy for Alzheimer's patients.
Sources: News Release from Washington University School of Medicine
(original source) and http://www.sciencedaily.com/releases/2001/07/
010704093453.htm (7/3/01)
New Hope in Spinal Cord Treatment Utilizing Patients' Own
White Blood Cells: Doctors at Proneuron Biotechnologies in Tel Aviv,
Israel, are using a promising new technique to treat spinal cord
injuries. They inject a patient's own white blood cells directly into
their spinal cord, after activating the cells by incubating them with
skin cells from the patient's body. So far, results are encouraging. A
19-year old girl who was paralyzed from her chest down by a car
accident was treated with the procedure, and a year later, has regained
pain sensation in one of her legs, can contract some leg muscles, and
can curl her toes. It's not clear right now whether the activated white
blood cells are promoting regrowth of the neurons or are simply keeping
more neurons from dying. The treatment tries to utilize the knowledge
that white blood cells are known to usually play a big role in the
healing of injuries. Normally, however, white blood cells do not heal
injuries to the spinal cord, because of the ``blood-brain barrier.''
This is a term for the tight cell junctions in the blood vessels in the
brain and spinal cord areas, which isolate the brain and spinal cord
from the blood cells and potential toxins in the blood. With the new
procedure, the doctors get past this barrier by injecting the cells
directly into the spinal column. Three other people have been treated
by Proneuron with this and other new techniques for paralyzed patients,
including a 19-year-old man from Colorado who was injured while using a
snowboard. The procedure described here can only be performed within 14
days of the spinal cord injury, and Proneuron is seeking more patients
from around the world that are interested in trying the procedure. They
are willing to pick up transportation and living expenses, and the cost
of the treatment itself for the patient. This type of treatment is
especially significant in light of the fact that people who advocate
embryonic stem cell research believe that spinal cord injuries can be
helped by such research. This white blood cell procedure (also known as
``autologous macrophage procedure'') is already showing promise in
humans, and does not face the ethical or immunological problems that
would come from human embryonic stem cell or fetal cell research.
Source:http://www.cnn.com/2001/HEALTH/conditions/07/20/
spinal.cord.injuries.ap/index.html (from 7/20/01 report); also
www.proneuron.com
Alzheimer's Vaccine Holds Promise and Appears Safe in
Human Tests: Scientists at the Elan Corporation, a pharmaceutical
company headquartered in Dublin, have found that a vaccine they have
been testing to treat Alzheimer's disease has shown promise in mice and
appears safe in humans. The vaccine, called AN-1792, is made of a
synthetic protein which is a form of the protein beta amyloid that is
found in the characteristic ``plaques'' (protein aggregates) in the
brains of Alzheimer's patients. These plaques are thought to be what
causes the memory loss and behavior changes which are symptoms of
Alzheimer's disease. In mouse experiments, the synthetic protein
vaccine was injected into mice that had plaques (i.e., they were a
mouse ``model'' of the human disease). Two groups of mice were studied
after such immunization: older mice that had already formed brain
plaques and younger mice that had not yet formed plaques. With the
older mice, it was found that there had been a significant curbing of
plaque formation. With the younger mice, it was found that the
immunization had kept virtually all of the mice from even developing
plaques. It is thought that the vaccine somehow clears plaques out of
the brain. In more recent testing of the vaccine in humans to determine
its safety, it was found that the vaccine appeared to have no
significant adverse effects. In addition, the patients had developed an
immune response to it, which is what should happen if the protein is
having any effect in their bodies. Although the patients did not
exhibit any cognitive or memory improvements, the scientists hope that
the next phase of upcoming human trials, which test for vaccine
efficacy, will show positive results as did the mice experiments. This
research appears to be much safer than the research reported in the
March 8, 2001 New England Journal of Medicine involving fetal and
possibly embryonic cells injected into the brains of Parkinson's
patients (another neural disease). That treatment had disastrous
results, such as wild, aberrant movements in the patients. In addition,
the research involving this vaccine is an example of one of the moral
research routes that can be taken as an alternative to research
involving the destruction of embryonic human beings.
Sources: http://www.cnn.com/2000/HEALTH/aging/07/11/
alzheimer.vaccine/ (from 7/11/01 report); also http://www.cnn.com/2001/
HEALTH/07/23/alzheimers.vaccine/index.html (from 7/23/01 report); also
http://www.elan.ie/alzheimer/pressrelease.asp; also http://www.elan.ie/
alzheimer/research.asp
Promising New Treatment for Partial Spinal Cord Injury
Researchers at the Weizmann Institute of Science in Israel
are experimenting with a new therapy intended for patients with partial
spinal cord injury. With partial spinal cord injury, the several days
immediately following the injury are crucial, because during this time,
there is usually further neural damage that occurs in a wave that
spreads from the injury site, and can be even more destructive than the
initial damage. The goal of the researchers is to find a treatment that
will effectively stop this postinjury degeneration. In their recent
studies on rats with partial spinal cord injury, they injected the rats
postinjury with specific proteins which they had derived from the
central nervous system (and which were selected to be able to boost the
rats' immune system). The treated rats showed significant recovery of
movement, and their spinal cords contained nerve fibers that appeared
substantially healthier than those of nontreated rats (which suggests
that the proteins halted further neural degeneration). These results
are very encouraging, and counteract (with hard data) the hype from
pro-embryonic stem (ES) cell research organizations that ES cells are
needed for healing of spinal cord injury. This nonES therapy is only
one of several we've reported on in the last few weeks which treat
neural injury and disease in a completely moral manner.
Sources:http://wis-wander.weizmann.ac.il/weizmann/doa iis.dll/
Serve/item/English/1.200.7.5.html and http://www.sciencedaily.com/
releases/2001/08/010817081453.htm (8/17/01 report)
heart disease and repair
Adult Stem Cell Research
Bone marrow cells were found to regenerate heart cells
after induced heart attack-type damage in mice. (Orlic, et al.; ``Bone
marrow cells regenerate infarcted myocardium''; Nature; April 5, 2001
(410); 701-705.)
The June 7 edition of the NEJM also reported on research
that found that human heart muscle cells regenerate after a heart
attack. These researchers, at the National Institutes of Health and New
York Medical College, now seek to determine why heart muscle cells are
dividing. One hypothesis is that these cells originate from stem cells
present in the heart. Members of the research team had already shown in
mice that adult stem cells from bone can become functioning heart
muscle.
A team at University of California at Los Angeles
announced in May of this year that they had injected adult stem cells
from a heart attack victim's bicep to replace heart muscle tissue
damaged in a heart attack. Separately, also in May, a team at Baylor
University announced their discovery that stem cells from the bone
marrow of an adult mouse had the capability to transform into blood
vessels and cardiac muscle.
Other Non-ES/Fetal Cell Research
Newly-Discovered Heart Cell Protein Could be Used in
Cardiac Treatment or for Conversion of Other Cells to Cardiac Cells: In
the June 29, 2001 issue of Cell, researchers at the UT Southwestern
Medical Center at Dallas report that they have discovered a protein
called myocardin in heart cells which is responsible for turning on
cardiac genes. Dr. Eric Olson, chairman of molecular biology, says,
``The Holy Grail in the heart field is finding the gene or genes that
can convert noncardiac cells into cardiac cells.'' The scientists
studied the function of the protein in frog embryos and found that when
myocardin is absent, the heart does not develop. It is possible that
the gene that controls myocardin is a type of ``master gene'' for the
heart. If so, then it could bode well for all sorts of possible heart
treatments, such as changing nonheart cells into heart cells (an
alternative to embryonic stem cells), or utilization in heart repair
therapies in all ages (babies born with heart defects to adults with
heart defects or heart attacks).
Sources: Wang, Da-Zhi, et al.; ``Activation of Cardiac Gene
Expression by Myocardin, a Transcriptional Cofactor for Serum Response
Factor''; Cell, Vol. 105, 851-862, June 29, 2001; also, http://
unisci.com/stories/20012/0629013.htm
Heart Cells Divide, Enlarge, and Live Longer With Special Enzyme
Scientists at Baylor College of Medicine in Houston have
found a way to increase heart cell division, increase heart cell size,
and make heart cells live longer. This is great news for people prone
to heart attacks or other heart ailments. The researchers genetically
engineered their laboratory mice to produce a specific enzyme called
TERT (Telomerase Reverse Transcriptase) in their heart muscles. This
enzyme is normally only produced in very young mice, and is involved in
the process of chromosome duplication during cell division. The
engineered mice continued to produce the enzyme even after they grew
older. At first, the mice produced many more heart cells than normal
mice. Several weeks later, cell division ceased and the cells grew
larger (hypertrophy). In addition, the cells appeared to be more
resistant to cell death than normal cells. Usually, the condition of
hypertrophy confers a weakness to the heart. With these mice, however,
their hearts did not have impaired functioning. One of the scientists
in the group, Dr. Michael Schneider, said that the scientists involved
believe that adding TERT to an adult heart could offer protection
against the type of cell death experienced during a heart attack. In
addition, TERT could possibly be added to cells that have been grafted
onto an injured heart. This could probably aid in cell growth and
protection from cell death. It is just one more example of the exciting
research being done without using embryonic stem cells.
Source: http://www.sciencedaily.com/releases/2001/08/
010821074944.htm (8/22/01 report) and Oh, et al., ``Telomerase reverse
transcriptase promotes cardiac muscle cell proliferation, hypertrophy,
and survival'', Proceedings of the National Academy of Sciences,
published online August 21, 2001 www.pnas.org/cgi/doi/10.1073/
pnas.191169098 [Need subscription].
kidney disease/renal failure
Adult Stem Cell Research
Bone Marrow Cells Can Become Kidney Cells: Scientists at
the Imperial Cancer Research Fund and Imperial College School of
Medicine in the UK have recently discovered that bone marrow stem cells
(which are adult stem cells) can change into kidney cells in both mice
and humans. For the mice that they studied, they examined the kidneys
of female mice which had been given bone marrow transplants from male
mouse bone marrow. For the humans, they analyzed kidneys of male
patients who had been given transplants of female kidneys. The
researchers utilized a special DNA probe technique on the kidneys,
which can detect if a cell originated from a male or female. For both
groups, it was found that the mouse and the human kidneys contained
male kidney cells as well as female kidney cells. It was determined
that these male cells had come from the mouse's or patient's own bone
marrow, had implanted into the new kidneys, and had literally become
kidney cells. This finding opens up many promising possibilities.
People whose kidneys are damaged or failing because of disease or
injury could be treated with their own bone marrow cells to form new
kidney cells and possibly whole new kidneys (which would help solve the
desperate current shortage of kidney transplants). This is especially
beneficial for the realm of kidney treatment, because as organs, they
are particularly bad at repairing themselves. Also, there would be less
immune rejection, because the repairing cells would be coming from the
patients' own bone marrow. Professor Nick Wright, one of the authors of
the paper in the Journal of Pathology Online which describes the
experiments, says that an additional possibility using this knowledge
could be to genetically engineer bone marrow stem cells to contain
genes ``resistant to cancer or other disease, to protect the kidneys
from further damage.'' Says Dr. Poulsom, lead author on the paper,
``The potential for advances in medicine from using adult stem cells is
enormous. They can give rise to many different types of cells so any
organ may 1 day be repaired. Using adult stem cells also avoids the
ethical dilemmas associated with embryonic stem cell work.''
Sources: Poulsom, et al., Journal of Pathology Online; ``Bone
marrow contributes to renal parenchymal turnover and regeneration''; 25
July 2001; also Pro-Life Infonet 7/25/01 #2486 (contact:
[email protected]); also http://unisci.com/stories/20013/
0725011.htm (from 7/25/01 report)
hemophilia
Non-ES/Fetal Cell Research
Research showing that the genetic modification of cells
could fight hemophilia, announced in the June 7, 2001 edition of the
New England Journal of Medicine (NEJM), used adult cells taken from the
skin of the patients' arms. The cells were then modified to add the
gene for a missing clotting factor, grown in the laboratory, and
injected in to the patient.
cancer
Placental Stem Cell Research
Placental blood was found to be a useful source of stem
cells for bone marrow reconstitution in patients with leukemia and
other diseases. (Rubinstein, et al.; ``Outcomes among 562 recipients of
placental-blood transplants from unrelated donors''; New England
Journal of Medicine; November 26, 1998; 339 (22); 1565-1577.)
Non-ES/Fetal Cell Research
Cancer Treatment Made Better Through ``Sticky''
Antibodies: A team of scientists at the University of California,
Davis, has developed a new method for altering antibodies for targeting
the destruction of tumors. Antibodies are molecules within the body's
immune system which normally hone in on foreign elements in the body,
to fight infections. It has been found that they can also be directed
to fight cancerous tumors. When they are connected with a radioactive
element (which is joined to a small carrier molecule), the special
antibodies not only hone in on the tumor, but also deliver lethal
radiation to the tumor. Radiation is a commonly-used therapy to fight
cancer. When used in this way, the radiation goes straight to the
cancerous cells, rather than to other parts of the body as well (which
is a frequent side-effect of usual radiation therapy). Previous studies
have combined the antibody-radiation complex in a rather temporary
manner, using methods that unfortunately allow the radiation carrier
molecule to unhinge from the antibody. The scientists at UC Davis have
come up with a new method which engineers the binding region of the
antibody to adhere permanently to the radiation carrier molecule. In
effect, the antibody-radiation juncture is ``stickier''. This should
allow for a cancer treatment regimen that is more precise, delivering
the radiation directly to the tumor site without potential radiation
leakage in other parts of the body. This is yet another cancer therapy
which does not utilize embryonic stem cells (which have been touted as
being a possibility for use in cancer research).
Source: Chmura, et al.; ``Antibodies with infinite affinity;''
Proc. Natl. Acad. Sci. USA, Vol. 98, Issue 15, 84808484, July 17, 2001;
also, http://unisci.com/stories/20013/0717015.htm
Drug Capable of Destroying Only Oxygen-Starved Cells
Within Tumors: Researchers at the University of Ulster in the UK have
developed a promising new drug for cancer treatment. It is different
from other current drugs on the market in that it targets hypoxic
cancerous cells, which are cells deep within tumors that are not
receiving much oxygen. These types of cells have proven to be
particularly difficult to treat with the normal range of cancer drugs
and radiation therapy, which are generally only good at killing cancer
cells on the exterior of a tumor (and which do receive oxygen). The
hypoxic cancerous cells within tumors are also known to be especially
malignant, and have been found to be one of the prime instigators of
secondary bouts of cancer, after the external tumor cells have been
killed off. The new drug, called AQ4N, is also different from standard
chemotherapy drugs, in that when administered, it is not toxic to the
body. It only becomes toxic when it reaches the tumor cells deep inside
the tumor, and is only toxic to those particular cells. This would
avoid the common side-effect of the current chemotherapy drugs to kill
other, noncancerous cells in the body (in doing so, they cause hair
loss and extreme nausea to cancer patients). It is thought that perhaps
this new drug can eventually be used in combination with current modes
of drug and radiation therapy to be an effective cancer treatment
regimen. The development of drugs such as this is a moral and effective
alternative to cancer research involving embryonic stem cells.
Source: http://unisci.com/stories/20013/0719012.htm
Making Deadly Acute Myeloid Leukemia (AML) Treatable:
Scientists at Johns Hopkins University have found that drug called
AG1295 can treat acute myeloid leukemia (AML), which is the most common
form of adult leukemia. At least 40% of patients with AML have a
genetic mutation wherein the gene called ``FLT3'' is altered. This gene
is normally involved in the growth and maturation of blood cells, and
when it is altered, uncontrolled division (causing leukemia) can occur.
The researchers discovered that the drug AG1295 specifically interferes
with the action of the altered FLT3 protein, blocking the uncontrolled
growth and causing the leukemia cells to die. Safety and efficacy
trials in animals and humans will need to be done, but it is a big step
that the investigators have been able to pin down the genetic
abnormalities related to this type of leukemia. This is good news, and
another exciting prospect that doesn't involve embryonic stem cell
research.
Sources: http://unisci.com/stories/20013/0727012.htm (from 7/27/01
report) and http://www.sciencedaily.com/releases/2001/07/
010726102007.htm (from 7/26/01 report)
Teaching the Immune System to Fight Cancer and Other
Diseases: Researchers at Oxford University's Department of Clinical
Medicine have developed a modified adenovirus that can teach the body's
immune system how to fight tumors, malaria, and hepatitis. The vaccine-
type method uses two sequential types of immunization. The first,
called the ``prime,'' introduces bits of the tumor or pathogen into the
body. This allows the body to be able to identify and recognize the
antigen. Later, a second immunization, called the ``boost,'' involves
injecting a modified adenovirus. This adenovirus elicits a larger
immune response which includes production of special ``CD8+ killer T
cells'' by the body. Because of the first ``prime'' injection, these
killer T-cells are specifically made to hone in on the tumor or
pathogen. Clinical trials of such vaccines are currently being started
by Oxford's spinout company, Oxxon Pharmaccines. Such research is
encouraging and is yet another type of cancer research that doesn't
involve human embryonic stem cells or fetal cells.
Source: http://unisci.com/stories/20013/0724011.htm (from 7/24/01
report)
Red Blood Cells Can Deliver Anti-Cancer Drugs With Great
Accuracy: A scientist at the University of Ulster, Professor Tony
McHale, has developed an ingenious way to deliver drugs to target
tumors with precision. He uses normal red blood cells (RBC's). RBC's,
which routinely carry oxygen throughout the body, have been used in
past studies in an attempt to deliver drugs throughout the body before,
without much success. This is because it is difficult to get the cells
to deliver the drugs to the exact point in the body that one is
interested in targeting. Professor McHale, however, has discovered a
way to get around this. He found a way to sensitize red blood cells so
that they will explode when they come in contact with an ultrasound
beam. After sensitizing the RBC's and loading them up with the desired
drug, doctors focus an ultrasound beam on the targeted tumor. Once the
RBC's reach this area, they explode, delivering the drugs in a precise
manner. In this way, surrounding tissue is left unaffected, which is
also a key benefit to his procedure, since most current cancer drugs
have a way of affecting other, noncancerous cells in the body (which
can cause hair loss and nausea in cancer patients). This is yet another
great cancer research discovery that does not use a single embryonic
stem cell.
Source: http://unisci.com/stories/20013/0724012.htm (from 7/24/01
report)
Inactivating Cancer Cells: Scientists at the Lautenberg
Center for General and Tumor Immunology of the Hebrew University--
Hadassah Medical School say that they have a new and safer way to
battle cancer. They say that doctors should induce cancer to remain in
a dormant State, rather than treat it by massive chemotherapy treatment
(which normally kills both cancerous and normal cells). The researchers
have developed a technique whereby they link a toxic molecule with an
antibody directed against specific receptors on cancer cells. After
injecting this complex into the blood stream, the cancer cells can be
induced to internalize the complex. The toxin portion of the complex
then inactivates the cancer cell, and prevents it from replicating. In
mouse experiments, the scientists caused a human tumor to grow on an
immunologically-deficient strain of mice. When treated with the human
antibody-toxin complex, the cancer cells in the mice became dormant.
The mice stayed healthy for as long as they were being treated. If this
treatment proves effective in human clinical trials, it could be used
as a therapy for many different kinds of cancer. Importantly, it does
not necessitate the use of human embryonic stem cells.
Source: http://www.eurekalert.org/ (8/7/01 report entitled ``Hebrew
University researchers seek to `inactivate' cancer cells'')
crohn's disease
Adult Stem Cell Research
Adult Stem Cells Used to Treat Crohn's Disease: Crohn's
disease, which affects more than 50,000 Americans, is an autoimmune
disorder wherein a patient's white blood cells attack their own
stomach. It can cause extreme pain, chronic diarrhea, and an inability
to digest food. Ten weeks ago, Northwestern Memorial Hospital in
Chicago, Illinois performed the first stem-cell transplant to treat
Crohn's disease, with successful results. The outcome was so
encouraging that they performed a second transplant on a 16-year-old
boy on Monday (8/6/01). The first transplant involved a 22-year-old
woman who had suffered from Crohn's disease since childhood. The
doctors first collected some of her own blood stem cells. They then
used powerful chemotherapeutic drugs to destroy her faulty immune
system, and afterwards injected the blood stem cells back into her
circulatory system. The stem cells traveled to her bone marrow and
immediately began producing new immune system cells. After previously
failing all other treatments, the patient now has no diarrhea or
abdominal pain. Similar procedures have been used successfully to treat
lupus (another autoimmune disease) and with promising results to treat
multiple sclerosis. All with the use of a patient's own stem cells, not
embryonic stem cells.
Source: http://www.suntimes.com/output/news/cst-nws-stem07.html
(Golab, Art; Chicago-Sun Times; 8/7/01; ``Stem Cells Used to Treat
Crohn's'')
skin disorder
Adult Stem Cells Successful in Treating Rare Skin Disorder
A team of doctors at the University of Texas M.D. Anderson
Cancer Center in Houston recently performed the first known adult stem
cell transplant to treat a rare skin disorder called scleromyxedema.
The disorder is characterized by waxy, stiff, thickened skin. In the
Houston patient, it had progressed so far that the skin on his face had
a ``cobblestone appearance,'' and he could not close his eyelids
completely or eat. The doctors first collected stem cells from the
patient's own bone marrow, and then purposely destroyed his immune
system with chemotherapy. They then transplanted his stem cells back
into his system in order to allow the cells to reconstruct his immune
system. Three months after the transplant, the patient's face does not
have the cobblestone appearance anymore, and he can now close his eyes
and open his mouth. This is another example of highly encouraging
results from adult stem cells in clinical work on human patients.
Sources: Prolife Infonet 8/20/01, #2509; Reuters Health, Aug. 17,
2001; also Feasel, et al., ``Complete Remission of Scleromyxedema
Following Autologous Stem Cell Transplantation'', Archives of
Dermatology; August 2001, Vol. 137, No. 8, pp. 1071-1072.
thalassemia
Umbilical Cord Cell Transplant Successfully Used To Treat Thalassemia
Doctors from Singapore's National University Hospital and
Singapore General Hospital have succeeded in the first known case of an
umbilical cord cell transplant into a patient with Thalassemia Major.
Thalassemia is a rare genetic blood disorder wherein the patient's red
blood cells are unusually small and contain defective hemoglobin, and
can lead to anemia. If left untreated, it is fatal for children.
Thalassemia Major is one of the most severe forms of the disease.
Usually, thalassemia is treated with bone marrow transplants. If a
matching bone marrow donor cannot be found, the patient can be given
numerous blood transfusions (which carry with them the risk of
infections or development of diabetes or liver cirrhosis due to
overdose of iron). In this recent case, the patient was given the
umbilical cord blood of an unrelated donor. 28 days after the
transplant, the patient was producing normal red blood cells, and was
released 8 days after that. Umbilical cord cell transplants offer
patients an opportunity for an alternative source to bone marrow
transplants; it offers more versatility as well, since the umbilical
cord donor does not appear to need to even be related.
Source: Agence France Press; August 14, 2001; International News
Section; ``Singapore scores medical first in treatment of
thalassaemia.''
applicable to various diseases
Non-ES/Fetal Cell Research
Blocking Programmed Cell Death and Engulfment of Cells
Could Have Potential for Treating Various Diseases: Researchers at Cold
Spring Harbor Laboratory have found methods for decreasing cell death
in the developing nervous system of the nematode worm known as C.
elegans. They have been studying the cell survival and cell death
processes in the worm, since it has a transparent body and such cell
activities can be monitored with a microscope. Like in other animals,
the normal process of cell death involves a signal inside of cells that
are about to die, which causes a programmed death response in the
cells. Soon afterwards, neighboring cells ``engulf'' the dying cell to
complete the process. The scientists have found that when they were
able to weaken the ``death signal'' within the cell slated to die, they
were able to save a percentage of the cells. In addition, when they
were able to weaken the ``engulfment machinery'' in the engulfing
neighbor cells at the same time, an even greater number of cells were
saved from death. The researchers suggest that similarly modifying the
engulfment machinery in some cells in humans could prove to be an
effective therapy for patients with neurodegenerative disease, stroke,
and cancer.
Sources: Daniel J. Hoeppner, et al.; ``Engulfment genes cooperate
with ced-3 to promote cell death in Caenorhabditis elegans''; Vol. 412;
July 12, 2001; pp. 202-206; also News Release from Cold Spring Harbor
Laboratory (original source) and http://www.sciencedaily.com/releases/
2001/07/010712080935.htm
Some Mouse Tissue Capable Of Regeneration: A special
strain of mouse may contain secrets for rapid regeneration of injured
bodily tissue. These laboratory mice, called ``MRL mice,'' have been
noted to have a special ability to heal themselves with minimal
scarring. Recent studies by researchers at the Wister Institute in
Philadelphia have shown that even after serious heart injury, these
mice show a fascinating ability to heal their own heart tissue without
the use of drugs, injections of stem cells, or other intervention.
Their experiments showed that in control mice, only 1-3% of the heart
cells in the region of cardiac injury were capable of dividing. In the
MRL mice, however, up to 20% of cells in the damaged heart area were
capable of dividing after the injury. Further studies on these mice
will possibly be aimed at identifying specific genetic and molecular
markers which are the cause of such rapid regeneration ability. In
doing so, scientists can potentially design drugs and treatments that
can lead to improved healing in many types of injuries and illnesses.
All without necessitating the use of embryonic stem cells.
Source: http://unisci.com/stories/20013/0807014.htm (8/7/01 report)
Platelets Have Ability to Produce Proteins In Response To
Signals: Scientists at the University of Utah have found out some new
things about platelets--they are much more versatile than once thought.
Platelets are one of the components of blood (besides red blood cells
and plasma), and are known to be involved in wound healing, by sticking
together to form plugs at wound sites to help close the wounds up. They
have been long thought to have a relatively limited ability to produce
proteins in response to cellular signals as normal cells do, because
they lack a cell nucleus (which is usually the interpreter of cellular
signals). However, the Utah scientists have found that platelets really
do have the capacity for significant production of proteins for wound
repair, in response to cellular signals. They found that platelets
contain RNA transcripts inside them. These portions of cellular
machinery are involved in the production of proteins. Specifically, it
was discovered that with these RNA transcripts, platelets can produce
``interleukin -1 -beta'', which is a protein that signals the cells in
damaged blood vessels to display receptors for white blood cells (so
that the white blood cells can hone in on the injury). These
discoveries offer new insights into ways that blood vessels are
repaired, and could lead to the development of drugs and treatments
that can be more specifically-targeted. Platelets are involved in many
illnesses (heart attacks, circulation problems, strokes, gangrene).
This new knowledge could be utilized to treat all of them, with no need
for embryonic stem cells.
Source: http://unisci.com/stories/20013/0806011.htm (8/6/01 report)
More Umbilical Cord Cell Possibilities
ViaCell Inc., an Umbilical Cord Blood Research and Banking
Company, Comments on Bush Decision to Fund Alternatives. ViaCell, Inc.,
commenting on a portion of the recent Bush Administration's decision
regarding stem cells, expressed approval at the Administration's
decision to increase funding for alternatives to human embryonic stem
cells such as adult and umbilical cord cells. ViaCell has developed a
new technology which they call ``Selective
Amplification'', which allows the expansion of stem
cell populations from umbilical cords. In addition, they are
``preparing and Investigational New Drug (IND) application to initiate
human clinical trials designed to demonstrate the safety, efficacy, and
clinical benefits of selectively amplified stem cells from umbilical
cord blood.'' They mention that some key advantages of umbilical cord
stem cells include their use in the therapy of over 45 diseases; their
ability to not cause as many immunological problems as other types of
cells when transplanted into donors; and the fact that they can
differentiate into various cell types (including hematopoetic,
mesenchymal, and neural cells). Of course, one of the primary
advantages is that they do not involve the ethical and moral concerns
that the use of human embryonic stem cells do.
Source: http://www.eurekalert.org/ (8/10/01 report); Contacts
include Ernie Knewitz (Media Contact for Noonan/Russo Communications;
[email protected] or 212-696-4455 ext. 204) or Marc Beer (Chief
Executive Officer of ViaCell, Inc.; [email protected] or 800-766-
0966 ext. 4650)
adult stem cells' ability to change into many other types of cells
Adult Stem Cell Research
A group of researchers at Johns Hopkins University has
found that mouse bone marrow cells can change in vivo into the cells
which line the lungs, intestines, and skin. Dr. Sharkis, the leader of
the research team, was quoted in the May 4, 2001, issue of Cell as
saying that ``an infusion of stem cells after toxic cancer treatments
may be able to repair damaged tissue throughout the body, and serve as
treatments for a variety of diseases characterized by tissue and organ
damage, such as diabetes and cystic fibrosis.'' (Krause, et al.;
``Multi-Organ, Multi-Lineage Engraftment by a Single Bone Marrow-
Derived Stem Cell''; Cell; May 4, 2001: 105 (3); 369-377.)
Fat cells taken from liposuction were able to change into
cells resembling cartilage, bone, and muscle. (Zuk, et al.;
``Multilineage cells from human adipose tissue: implications for cell-
based therapies''; Tissue Engineering; 97 (410) 2001; 211-228.)
Skin Of Adult Mammals Contains Multipotent Stem Cells (1/
26/01 www.unisci.com article). A French research team published in the
January 26, 2001 issue of Cell that the skin of adult mammals contains
stem cells which can give rise to the epidermis, sebaceous glands, and
hair follicles. They found that the stem cells are mainly located
within the hair follicles. This discovery can aid in research and
treatment in dermatology, oncology, and cosmetology. In dermatology,
these adult stem cells have the potential to help in the areas of wound
healing and in improving the results of skin grafts in burn patients.
In oncology, the adult stem cells show promise in helping to understand
the origins of some skin cancers. In cosmetology, it is thought that
research on the cells could help to treat hair loss or excess body
hair.
Sources: Oshima, et al.; ``Morphogenesis and Renewal of Hair
Follicles from Adult Multipotent Stem Cells;'' Cell; Vol. 104 (2), pp.
233-245, January 26, 2001; also, http://unisci.com/stories/20011/
0126015.htm.
alternatives to using embryonic stem cells
Adult Stem Cells
Numerous recent studies have revealed the potential of
adult stem cells to become several different types of tissue. This
capability defeats the accepted scientific knowledge of decades past;
it was formerly thought that once a stem cell within a developing
embryo had differentiated and committed itself into one cell type, it
couldn't re-differentiate later in life from an adult cell into another
cell type. It has now been found that adult neural murine stem cells
can transform themselves into blood cells, \1\ adult muscle stem cells
can convert into large quantities of blood cells, \2\ adult bone marrow
cells can become liver cells. \3\
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\1\ Bjornson, et al., ``Turning Brain into Blood: A Hematopoietic
Fate Adopted by Adult Neural Stem Cells in Vivo,'' Science [January 22,
1999] 534-7.
\2\ Jackson, Mi and Goodell, Proceedings of the National Academy of
the Sciences [December 7, 1999] 14193-5.
\3\ Thiese, Krause, et al., Hepatology [January, 2000] 235-40.
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This progress on adult stem cells is wonderful for
potential patient recipients, because there would not be the problem of
immune rejection of the cells that one would find with embryonic stem
cells. Adult stem cell lines could be grown from the patients' own
body, thus overcoming any potential graft-versus-host disease and any
need for strong immunosuppressive drug therapy.
Likewise, adult stem cells are much more accessible than
embryonic stem cells. They are located throughout the human body, even
in adipose (fat) tissue.
Umbilical Cord and Placental Cells
Umbilical cord blood banks and placenta banks are in
operation in several states and are being organized in others. These
cells have shown promise in being used to combat leukemia. \4\
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\4\ Rubinstein, et al., ``Outcomes among 562 Recipients of
Placental-Blood Transplants from Unrelated Donors,'' New England
Journal of Medicine, 339 [Nov. 26, 1998) 1565-1577.
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detrimental findings with es and fetal cell research
A study in Science (Humpherys, et al.; ``Epigenetic
Instability in ES Cells and Cloned Mice''; Volume 293 (5527); July 6,
2001; pp. 95-97; or http://www.sciencemag.org/ [subscription
necessary]) shows that embryonic stem cells and cloned cells are
extremely genetically unstable. The researchers examined the gene
expression (the production of proteins from genes) of the cells of mice
obtained by cloning. The clones were obtained by performing a transfer
of an embryonic stem cell into a de-nucleated egg and then implanting
the embryo into a surrogate mother. The original donor embryonic stem
cells were also examined. It was found that gene expression by both
sets of cells was very different when compared from cell to cell. This
implies that ``even apparently normal cloned animals may have subtle
abnormalities in gene expression.'' (p. 95) It also implies that if
embryonic stem cells would 1 day be able to be coaxed adequately into
cells of interest (i.e., cardiac, neuronal, etc.) for transplant
purposes, the cells may exhibit aberrant behavior due to each cell
being wildly different in the proteins they produce.
Injection of fetal cells into the brains of Parkinson's
disease patients was found to cause wild, aberrant movements such as
arm swinging and head-jerking. It is thought that these fetal cells
produced too much dopamine, and were difficult to control. (Freed, et
al.; ``Transplantation of Embryonic Dopamine Neurons for Severe
Parkinson's Disease''; New England Journal of Medicine; March 8, 2001;
Vol. 344 (10); pp. 710-719).
Unstable Gene Expression Found in Both Clones and
Embryonic Stem Cells: Scientists have just published a study in Science
in which they have found that embryonic stem cells and cloned cells are
extremely genetically unstable. The researchers examined the gene
expression (the production of proteins from genes) of the cells of mice
obtained by cloning. The clones were obtained by performing a transfer
of an embryonic stem cell into a de-nucleated egg and then implanting
the embryo into a surrogate mother. The original donor embryonic stem
cells were also examined. It was found that gene expression by both
sets of cells was very different when compared from cell to cell. This
implies that ``even apparently normal cloned animals may have subtle
abnormalities in gene expression.'' (p. 95) It also implies that if
embryonic stem cells would 1 day be able to be coaxed adequately into
cells of interest (i.e., cardiac, neuronal, etc.) for transplant
purposes, the cells may exhibit aberrant behavior due to each cell
being wildly different in the proteins they produce.
This is a further reason for not utilizing human embryonic stem
cells or human clones in research. There are alternative cells which
are morally, and as this new finding shows, scientifically and
medically, more acceptable: adult stem cells, placental cells, and
umbilical cord cells.
Sources: Humpherys, et al.; ``Epigenetic Instability in ES Cells
and Cloned Mice''; Volume 293 (5527); July 6, 2001; pp. 95-97; or
http://www.sciencemag.org/ [subscription necessary]
[Whereupon, at 2:40 p.m., the committee was adjourned.]