[House Hearing, 111 Congress] [From the U.S. Government Publishing Office] ALZHEIMER'S DISEASE: THE ONGOING CHALLENGES ======================================================================= HEARING BEFORE THE SUBCOMMITTEE ON HEALTH OF THE COMMITTEE ON ENERGY AND COMMERCE HOUSE OF REPRESENTATIVES ONE HUNDRED ELEVENTH CONGRESS SECOND SESSION __________ DECEMBER 9, 2010 __________ Serial No. 111-162 Printed for the use of the Committee on Energy and Commerce energycommerce.house.gov U.S. GOVERNMENT PRINTING OFFICE 78-139 WASHINGTON : 2013 ----------------------------------------------------------------------- For sale by the Superintendent of Documents, U.S. Government Printing Office, http://bookstore.gpo.gov. For more information, contact the GPO Customer Contact Center, U.S. Government Printing Office. Phone 202�09512�091800, or 866�09512�091800 (toll-free). E-mail, gpo@custhelp.com. COMMITTEE ON ENERGY AND COMMERCE HENRY A. WAXMAN, California, Chairman JOHN D. DINGELL, Michigan JOE BARTON, Texas Chairman Emeritus Ranking Member EDWARD J. MARKEY, Massachusetts RALPH M. HALL, Texas RICK BOUCHER, Virginia FRED UPTON, Michigan FRANK PALLONE, Jr., New Jersey CLIFF STEARNS, Florida BART GORDON, Tennessee NATHAN DEAL, Georgia BOBBY L. RUSH, Illinois ED WHITFIELD, Kentucky ANNA G. ESHOO, California JOHN SHIMKUS, Illinois BART STUPAK, Michigan JOHN B. SHADEGG, Arizona ELIOT L. ENGEL, New York ROY BLUNT, Missouri GENE GREEN, Texas STEVE BUYER, Indiana DIANA DeGETTE, Colorado GEORGE RADANOVICH, California Vice Chairman JOSEPH R. PITTS, Pennsylvania LOIS CAPPS, California MARY BONO MACK, California MICHAEL F. DOYLE, Pennsylvania GREG WALDEN, Oregon JANE HARMAN, California LEE TERRY, Nebraska TOM ALLEN, Maine MIKE ROGERS, Michigan JANICE D. SCHAKOWSKY, Illinois SUE WILKINS MYRICK, North Carolina CHARLES A. GONZALEZ, Texas JOHN SULLIVAN, Oklahoma JAY INSLEE, Washington TIM MURPHY, Pennsylvania TAMMY BALDWIN, Wisconsin MICHAEL C. BURGESS, Texas MIKE ROSS, Arkansas MARSHA BLACKBURN, Tennessee ANTHONY D. WEINER, New York PHIL GINGREY, Georgia JIM MATHESON, Utah STEVE SCALISE, Louisiana G.K. BUTTERFIELD, North Carolina CHARLIE MELANCON, Louisiana JOHN BARROW, Georgia BARON P. HILL, Indiana DORIS O. MATSUI, California DONNA M. CHRISTENSEN, Virgin Islands KATHY CASTOR, Florida JOHN P. SARBANES, Maryland CHRISTOPHER S. MURPHY, Connecticut ZACHARY T. SPACE, Ohio JERRY McNERNEY, California BETTY SUTTON, Ohio BRUCE L. BRALEY, Iowa PETER WELCH, Vermont Subcommittee on Health FRANK PALLONE, Jr., New Jersey, Chairman JOHN D. DINGELL, Michigan NATHAN DEAL, Georgia, BART GORDON, Tennessee Ranking Member ANNA G. ESHOO, California RALPH M. HALL, Texas ELIOT L. ENGEL, New York BARBARA CUBIN, Wyoming GENE GREEN, Texas JOHN B. SHADEGG, Arizona DIANA DeGETTE, Colorado STEVE BUYER, Indiana LOIS CAPPS, California JOSEPH R. PITTS, Pennsylvania JANICE D. SCHAKOWSKY, Illinois MARY BONO MACK, California TAMMY BALDWIN, Wisconsin MIKE FERGUSON, New Jersey MIKE ROSS, Arkansas MIKE ROGERS, Michigan ANTHONY D. WEINER, New York SUE WILKINS MYRICK, North Carolina JIM MATHESON, Utah JOHN SULLIVAN, Oklahoma JANE HARMAN, California TIM MURPHY, Pennsylvania CHARLES A. GONZALEZ, Texas MICHAEL C. BURGESS, Texas JOHN BARROW, Georgia DONNA M. CHRISTENSEN, Virgin Islands KATHY CASTOR, Florida JOHN P. SARBANES, Maryland CHRISTOPHER S. MURPHY, Connecticut ZACHARY T. SPACE, Ohio BETTY SUTTON, Ohio BRUCE L. BRALEY, Iowa C O N T E N T S ---------- Page Hon. Frank Pallone, Jr., a Representative in Congress from the State of New Jersey, opening statement......................... 1 Hon. Phil Gingrey, a Representative in Congress from the State of Georgia, opening statement..................................... 3 Hon. Lois Capps, a Representative in Congress from the State of California, opening statement.................................. 4 Hon. John Barrow, a Representative in Congress from the State of Georgia, opening statement..................................... 5 Hon. Donna M. Christensen, a Representative in Congress from the Virgin Islands, opening statement.............................. 6 Hon. Zachary T. Space, a Representative in Congress from the State of Ohio, opening statement............................... 7 Hon. Anna G. Eshoo, a Representative in Congress from the State of California, prepared statement.............................. 57 Hon. Joe Barton, a Representative in Congress from the State of Texas, prepared statement...................................... 59 Hon. Joseph R. Pitts, a Representative in Congress from the Commonwealth of Pennsylvania, prepared statement............... 63 Hon. Marsha Blackburn, a Representative in Congress from the State of Tennessee, prepared statement......................... 65 Witnesses Marcelle Morrison-Bogorad, Director, Division of Neuroscience, National Institute on Aging, National Institutes of Health..... 9 Prepared statement........................................... 12 Harry Johns, President and Chief Executive Officer, Alzheimer's Association.................................................... 22 Prepared statement........................................... 25 Eric J. Hall, President and Chief Executive Officer, Alzheimer's Foundation of America.......................................... 31 Prepared statement........................................... 33 Marc Cantillon, M.D., Executive Director, Coalition Against Major Disease, Critical Path Institute............................... 39 Prepared statement........................................... 41 Submitted Material Article entitled, ``Evidence for Neurocognitive Plasticity in At- Risk Older Adults: The Experience Corps Program,'' in Journal of Gerontology: Medical Sciences, 2009, submitted by Mr. Shimkus........................................................ 68 ALZHEIMER'S DISEASE: THE ONGOING CHALLENGES ---------- THURSDAY, DECEMBER 9, 2010 House of Representatives, Subcommittee on Health, Committee on Energy and Commerce, Washington, DC. The subcommittee met, pursuant to call, at 1:47 p.m., in Room 2322, Rayburn House Office Building, Hon. Frank Pallone, Jr., [chairman of the subcommittee] presiding. Present: Representatives Pallone, Engel, Capps, Barrow, Christensen, Space, Gingrey, and Markey. Staff Present: Ruth Katz, Chief Public Health Counsel; Sarah Despres, Counsel; Steve Cha, Professional Staff Member; Allison Corr, Special Assistant; Alvin Banks, Special Assistant; Elizabeth Letter, Special Assistant; Ronald Allen, Staff Assistant; Clay Alspach, Minority Counsel, Health; Sean Hayes, Minority Counsel, Oversight and Investigations; and Ryan Long, Minority Chief Counsel, Health. OPENING STATEMENT OF HON. FRANK PALLONE, JR., A REPRESENTATIVE IN CONGRESS FROM THE STATE OF NEW JERSEY Mr. Pallone. The meeting of the House subcommittee is called to order. And today we have a hearing on Alzheimer's disease and its ongoing challenges, and I will recognize myself for an opening statement. As many of us know, Alzheimer's disease is an irreversible progressive brain disease that slowly destroys memory and thinking skills and, eventually, even the ability to carry out the simplest task. Damage first strikes the areas of the brain that control memory, and problems in memory are the first symptoms to be noticed in early stages of Alzheimer's disease. As deterioration progresses to other areas of the brain, problems with other brain functions develop as well, as severe Alzheimer's disease can affect every part of the brain and rob its victims of their very lives and dignity. Alzheimer's disease is fatal. It is estimated to be the sixth leading cause of death in our country. NIH estimates that as many as 5.1 million Americans may have Alzheimer's today, and this figure is expected to grow to 13.5 million Americans by 2050. The truth of the matter, though, is that these figures give an incomplete snapshot of Alzheimer's disease and related dementias. Alzheimer's disease is, at the heart, a family disease, as the intense caretaking that those afflicted with the disease requires places a heavy financial and emotional burden on the family. The serious medical complications related to Alzheimer's disease mean that caregivers often struggle to maintain work outside their home to earn a living, while balancing a never-ending schedule of monitoring and treatment for family members living with the disease. For many adults, known as the sandwich generation, they are duly responsible for caring for their aging parents while they are also caring for their children. And Alzheimer's has a devastating impact not just on families but on our national economy. It is projected that the national cost associated with caring for those with Alzheimer's exceeds $172 billion each year, with the figure expected to rise to $1 trillion by 2005. And these costs represent the burden on Medicare, Medicaid, private insurance caregiving and out-of-pocket costs for families. Of this figure, $123 billion can be attributed to Medicare and Medicaid alone. Until we cure Alzheimer's, it is imperative that our health care system provides stronger support for families caring for loved ones with the disease. The Affordable Care Act, which we passed earlier this year, establishes the Community Living Assistance Services and Support, or CLASS programs, a new national long-term care insurance option. This legislation also provides Medicare beneficiaries with free annual wellness visits, which may increase the detection of early cognitive impairment, enabling patients and families to better plan for care needs. And, finally, the Affordable Care Act will ensure that Americans living with Alzheimer's disease and other preexisting conditions will not have to worry about having their insurance coverage discontinued or denied. In the future, whether families are subject to the triumph or tragedy of Alzheimer's will be dependent on the innovation and new drugs and therapies being investigated in laboratories across our Nation. And today we are going to hear from the National Institute on Aging at the NIH about the great work their scientists are doing to better understand, diagnose, and treat Alzheimer's disease. NIA's translation objectives have focused on supporting early drug discovery and preclinical drug development of novel compounds for Alzheimer's therapy. We are also going to hear from the Coalition Against Major Diseases within the Critical Path Institute on its efforts to improve applied regulatory science and how this will strengthen our ability to treat diseases like Alzheimer's. And, finally, we will hear from the Alzheimer's Association and Alzheimer's Foundation of America about the key research they are supporting, as well as their initiatives and resources that help support day-to-day caretaking of individuals with Alzheimer's. Now, many of the members of this committee have been touched by Alzheimer's and expressed interest in examining the complex issues related to the disease. I do want to highlight the exemplary leadership and advocacy for Alzheimer's disease by a member of our full committee, Mr. Markey. I think he is going to be able to come today, but he basically has been touched, of course, by his late mother's own struggle with the disease, and because of that and for a lot of reasons he has been an energetic leader in Congress and also chairs the Congressional Caucus on Alzheimer's disease. So he may be joining us later, but I did want to mention him today. And I will now recognize for an opening statement our ranking member, Mr. Gingrey. OPENING STATEMENT OF HON. PHIL GINGREY, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF GEORGIA Mr. Gingrey. Mr. Chairman, thank you. And I certainly want to thank our witnesses for their patience with us today. The witnesses will testify, as we hear from them, Alzheimer's disease presents a looming threat to the health of our Nation, and today I think the number is as many as 5 million Americans in the United States have this dreaded disease. And the scientific community is in agreement that these numbers will only increase significantly in the coming years as our society ages. According to the United States Census Bureau, the number of seniors age 65 and older will actually double to about 72 million in the next 20 years as these baby boomers all reach retirement age. However, as Dr. Morrison-Bogorad will testify, the generation behind them, I think this is what is referred to as Generation X, and these are the folks born between 1965 and 1980, it is a significantly smaller generation than the baby boomers. So why is this comparison significant for the purposes of the hearing today? As Alzheimer's is a degenerative and, as of yet, incurable disease that requires constant monitoring, the role of the main caregiver often falls to our family members, as we all know. According to a study conducted by the Alzheimer's Association, an estimated 11 million Americans provide 12.5 billion hours of unpaid care annually for people, usually their loved ones, with Alzheimer's and other dementias. The disease also does require large amounts of medical care in addition to in-home and community-based services. This high use of medical services results in substantial cost to the Federal Government, States, employers, and indeed families. If the number of Alzheimer's patients does indeed double over the next 20 years as many predict it will, we could be facing a health care crisis if the number of family caregivers declines, and of course the costly care for these patients ultimately shifts to paid health care professionals. Alzheimer's disease is also a personal issue for me. My wife's mother, Laura Neill Ayers, was a very healthy and vibrant 88-year-old woman. She didn't hear very well. I think she loved that. But she was active. She loved to watch sports, and she spent time in her room every day watching baseball. Her husband, Bill Ayers, actually played baseball for the New York Giants in the 1940s, and Laura picked up her love of the game from her husband. Watching baseball became one of her favorite pastimes and thoughts of spring training I think kept her warm all winter long. Seemingly overnight, all of that changed. She was diagnosed with dementia and, we suspect, Alzheimer's halfway through her 89th year, and the woman that we know and love was changed forever. Gone was the active woman, 88. In her place was a woman that seemingly was trapped inside her own body, unable to enjoy the comforts of these golden years. I cannot begin to describe how emotionally destructive this disease was, as many of you I am sure know, not only for my mother-in-law but for my entire family. There are many stories like this one that help underscore the importance of finding a cure. Today there are no known treatments to prevent, cure, or even delay the onset of Alzheimer's. The five drugs currently approved by the FDA have been shown to be successful in reducing the symptoms of the disease, but much more needs to be done to ultimately find a cure. And so with that thought in mind, I would like to welcome all of our witnesses here today. Specifically, I am interested in hearing from Dr. Marc Cantillon, the executive director of the Coalition Against Major Diseases for the Critical Path Institute. As Dr. Cantillon will touch on in his testimony, the goal of his organization is to promote a forum for scientists from the FDA, academia, and industry to evaluate innovative testing methods for the use in drug development. As members of this subcommittee have heard during past hearings, pharmaceutical drug development in the United States can benefit from greater collaboration between industry and the FDA. To the FDA's credit, they have recognized this fact in many areas, including antibiotic drug development, something for which I and a bipartisan group of members on this subcommittee will be advancing pieces of legislation in the 112th Congress. As a representative of the citizens of the 111th of Georgia, I believe the government works best when its processes are constantly scrutinized and reformed, when necessary, to ensure they work as efficiently as possible. That theory applies not only to Congress and the White House, but government agencies as well. Therefore, I hope to learn more about this phrase, ``applied regulatory science,'' during the question-and-answer portion of the hearing and how it might improve patient access to quality care. Mr. Chairman, thank you for scheduling this hearing today. And with that, I see my time has expired and I yield back. Mr. Pallone. Thank you, Mr. Gingrey. Mr. Gingrey. Mr. Chairman, I did want to ask for unanimous consent to submit for the record our actual subcommittee ranking member, Mr. Shimkus, a study that comes out of John Hopkins, ``Evidence for Neurocognitive Plasticity in At-Risk Older Adults,'' and the experience of that study. Mr. Pallone. Without objection, so ordered. [The information appears at the conclusion of the hearing.] Mr. Pallone. Next for an opening statement, the gentlewoman from California, our vice chair, Mrs. Capps. OPENING STATEMENT OF HON. LOIS CAPPS, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF CALIFORNIA Mrs. Capps. Thank you, Chairman Pallone, for holding this, the final Health Subcommittee of the 111th Congress. And I want to thank you for your wonderful service as chairman over the last 4 years. Alzheimer's disease is a very topical subject for this hearing, because the challenges faced by patients and their loved ones are so reflective of the challenges we face throughout our health care system. Long term, these are some of the challenges which fit with Alzheimer's so well: long-term care availability and its costs; respite care; an adequate workforce; and research for better treatments and a cure. Now, that is just touching the surface of the many challenges that we face with this and so many other chronic conditions. And thankfully, the new health care law provides much-needed relief for many of these concerns. One of these is the new long-term care insurance option. There is also Medicare prescription cost relief. And there is opportunities for training the next generation of health professionals; in addition, the Cures Acceleration Network, along with what our chairman mentioned as the CLASS Act. And, of course, we have a lot more that we should undertake now within this framework of the new legislation. Some of these other steps we can take, such as passage of legislation that I am very proud to cosponsor in the National Alzheimer's Act I know you are going to be addressing today in your testimony, including the National Alzheimer's project, with the goal being that we can provide and develop a comprehensive strategy for how to address Alzheimer's disease, how to continue, as many of you have been doing over these past many years. So I do look forward to hearing from our witnesses today about the latest in Alzheimer's research. I understand there are some very critical breakthroughs that have been working their way through the clinical trials and so forth, and also what you see for us in guidance in the next Congress as pressing needs that patients and so many of them, increasing numbers of their family members and caregivers, are going to experience. As has also been mentioned, there is probably not one person in this room who hasn't been touched personally by this devastating disease. And we need to know it is a crisis proportion, really, in our society now, how to best equip ourselves and our communities to cope with it. So I yield back and look forward to today's hearing. Mr. Pallone. Thank you. And I also wanted to thank you also as the vice chair. You have been really tremendously helpful, and so often giving us insight on issues because of your practical experience also as a nurse and your background. So thank you. Our next member for an opening statement, the gentleman from Georgia, Mr. Barrow. OPENING STATEMENT OF HON. JOHN BARROW, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF GEORGIA Mr. Barrow. Thank you, Mr. Chairman. I want to thank you also for your leadership throughout the 111th Congress on this committee. The poet said that comparisons are odious, and at the personal level no one wants their personal trauma or tragedy compared to someone else's. But at the level of public health and social impact, I think some comparisons are useful. The scourge of cancer has many faces. The scourge of Alzheimer's has a very definite profile, many different impacts on folks, but a very different impact in terms of the family. I think it is useful to point out that we didn't really begin to marshal the Nation's resources in the war on cancer until folks could say, everybody knows somebody who has been touched by cancer. I think it is fair to say that everybody now knows somebody who has been touched by Alzheimer's. Nobody knows the particular impact it has on family and on the communities of interest around families that is the hallmark of this dreaded disease. So I think we can take inspiration from the fact that so many folks are impacted by this. I think it is important to point out also in ways that supplement what others have said, the cost of this disease. The uncompensated care and the out-of-pocket expense to our system is staggering and growing exponentially in ways that I think compare very unfavorably to some of the other diseases that we deal with at a social level. Right now, we are spending something like $170-plus billion a year in compensated and uncompensated care, taking into account the fair market value of the services that folks are being forced to render and not getting any kind of compensation for at all. That is coming out of our gross social product. It is projected to cost our society something on the order of $1 trillion a year at current growth levels by the time we reach 2050. So it is staggering in its potential for exponential growth and impact on folks. The point is, this is not only the right thing to do in terms of the quality of the life of folks affected by this. It has never been truer to say that this is not only the right thing to do, but it is the smart thing for us to do, to marshal our resources in more effectively and economically managing and treating and preventing this dreaded disease. So, with that, I look forward to hearing the testimony of our witnesses and thank them for coming today. And I thank you, Mr. Chairman, for making this, the last hearing of this committee and this Congress, a very large issue that we are going to be dealing with for a very long time. With that, I yield back. Mr. Pallone. Thank you, Mr. Barrow. Next, the gentlewoman from the Virgin Islands, Mrs. Christensen. OPENING STATEMENT OF HON. DONNA M. CHRISTENSEN, A REPRESENTATIVE IN CONGRESS FROM THE VIRGIN ISLANDS Mrs. Christensen. Here thank you, Mr. Chairman. And thank you for holding this hearing. And thank you, welcome to the folks who are here to testify. And thank you not only for taking the time to be here but for the work that you all are doing. I really appreciate the approach that is being taken to this devastating disease, a disease that is devastating to not only individuals but families and communities, and potentially to our country, because it is a wholistic approach, looking at prevention, treatment, and research, but also looking at the caregivers who are often forgotten. I wanted to call attention to several areas. First, the cost of care. Due to the cost of care, this disease has the potential to bankrupt our health care system unless we invest in all of these aspects today. In fact, by 2050, it is estimated that 13.5 million Americans will be suffering from Alzheimer's, and the cost might be as much as $1 trillion a year. Second, there is a grave discrepancy between the funding for research for this sixth leading cause of death compared to other leading causes of death, where billions are being spent compared to just over $400 million on Alzheimer's. Third, I want to call attention to the fact that racial and ethnic minorities are disproportionately impacted with Alzheimer's, with African Americans being twice more likely and Hispanic Americans 1.5 more likely to suffer from Alzheimer's and other dementias, despite the fact that they are underdiagnosed compared to their white counterparts. And I wanted to mention, too, other pieces of legislation just to show that the Congressional Black Caucus has been aware and engaged in this issue for several years. One is H.R. 4123, the Alzheimer's Treatment and Caregivers Support Act, introduced by Representative Maxine Waters, and H.R. 1192, the Alzheimer's Family Assistance Act, introduced by Eddie Bernice Johnson. Both of these bills, like the others that are being mentioned in the testimonies, will help to take the fight against Alzheimer's several steps forward and be a part of this solution to this devastating problem. So, again, thank you, Mr. Chairman. And thanks, all of you, for being here. Mr. Pallone. I thank the gentlewoman. And next is the gentleman from Ohio, Mr. Space. OPENING STATEMENT OF HON. ZACHARY T. SPACE, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF OHIO Mr. Space. Thank you, Mr. Chairman. Today is my final hearing in committee, and I wanted to first express my gratitude to Mr. Chairman and to Chairman Waxman, as well as members on both sides of the aisle, for their work in some of the most profound issues of the day and even a generation: issues like energy and health care and the transformational effect that the whole broadband era has brought to this country and to our culture. And while we didn't accomplish everything that I think we had set out to accomplish, we got a lot done. And there is a lot to be done. My district back in Ohio is a very rural district. It is Appalachian Ohio, where the people are good and they work hard, a lot of good things going for us back there. But there are a lot of challenges that we have, and we share those challenges with the rest of rural America. And one of the things that is unique about this committee, I think, is the effect that it can have on bridging the divide that exists between urban and suburban America and rural America. And certainly the divide does exist when it comes to accessing education, certainly with regards to accessing health care. And I encourage the committee to be every bit as ambitious in tackling some of those challenges in the upcoming session of Congress. With respect to the issue of today, Alzheimer's, I sympathize with the remarks of my colleague and friend from Georgia, Mr. Barrow; $172 billion a year, those are war-like numbers. In other words, those are the kind of numbers you spend when you go to war. If you couple the amount of money that we are spending as a society on a disease like Alzheimer's with what we are spending on diseases like diabetes, cancer, and the list goes on and on, it soon reaches the trillion- dollar level. In fact, Alzheimer's alone, by the year 2050, will reach that level. And we cannot sustain that as an economy. When John Barrow says it is not only the humane thing to do, it is the right thing to do, what he is saying is if it is not important enough to deal with these issues simply because of the mitigation of human suffering that occurs as a result of these diseases, then certainly we should be able to justify it because of the cost. And like Alzheimer's and diabetes and cancer, the answer, at least from the congressional perspective, is in medical research. We cannot simply rely upon the private sector to tackle these diseases, not simply because it is not the right thing to do but we can't afford it. For every dime we invest today in research, we will receive a return of dollars in the future. Many dollars. So I urge this committee in future efforts, including its efforts with regards to Alzheimer's, to bear in mind the obligation that we have as an institution, as a government, and the duty we have to our people to conquer these diseases through early diagnosis, advanced diagnosis, advanced treatment, and above all, cure. Let's not forget cure. With that, Mr. Chairman, again thank you for your leadership, and it has been a pleasure serving on your committee. Mr. Gingrey. Mr. Chairman, could I have a brief point of personal privilege, a friendly point of personal privilege? Mr. Pallone. Absolutely. Mr. Gingrey. And let me first of all, to my friend from Ohio, Mr. Space--and he truly is my friend and he and I know that--indeed we will miss him. He is a great member with a great heart, and I think the words that he just expressed to this committee indicate the type of person that he is. And, Zack, we will miss you and I really enjoyed serving with you. And I hope we will keep in touch, my friend. Also, I wanted to say to you, Mr. Chairman, and I say this on behalf of my colleagues who I guess are on airplanes about now, trying to get home to their families, and not the least of which, of course, would be ranking member John Shimkus. We have really enjoyed serving with you, under you, on this Health Subcommittee of Energy and Commerce, not always agreeing on every vote, in fact disagreeing a lot of times. But no one could be more agreeable when he disagrees. Mr. Chairman, We respect you and hold you in high regard and look forward to serving with you in the 112th. Mr. Pallone. Well, thank you. I really appreciate that. And I want to say the same about you. Your input not only as a physician, but just in general, has been fantastic. And I kind of wish that Mr. Shimkus was here today, too, because I wanted to say how easy it was to work with him in the last--I guess not 2 years, a little over a year or so. And it is true, I think, that even though we often disagree on a lot of issues, that we have been able to work together on many issues. And it kind of bothers me sometimes when the media, I guess, pays attention to the differences and doesn't highlight how many bills we have actually passed out and worked on together and got signed into law that were very important for the American people. So thank you. And let me say about Mr. Space. Again, I know that I am really going to miss him. He has really contributed a lot and he has been a friend on so many issues. So thank you. Thank you very much. So we will get to our panel. Let me welcome you, first of all. We only have one panel today, so I will introduce the members. Starting on my left is Dr. Marcelle Morrison-Bogorad, who is director of the Division of Neuroscience at the National Institute on Aging, with the National Institutes of Health. And next is Mr. Harry Johns, who is president and chief executive officer of the Alzheimer's Association. And then we have Mr. Eric J. Hall, who is president and chief executive officer of the Alzheimer's Foundation of America. And, finally, Dr. Marc Cantillon, who is executive director of the Coalition Against Major Disease, from the Critical Path Institute, and also happens to be a constituent, so I should know how to pronounce his name, from one of my towns, Kingsbury. So thank you in particular for being here today. We have 5-minute opening statements that become part of the record, and each of you can also submit additional statements in writing for inclusion if you would like. STATEMENTS OF MARCELLE MORRISON-BOGORAD, DIRECTOR, DIVISION OF NEUROSCIENCE, NATIONAL INSTITUTE ON AGING, NATIONAL INSTITUTES OF HEALTH; HARRY JOHNS, PRESIDENT AND CHIEF EXECUTIVE OFFICER, ALZHEIMER'S ASSOCIATION; ERIC J. HALL, PRESIDENT AND CHIEF EXECUTIVE OFFICER, ALZHEIMER'S FOUNDATION OF AMERICA; AND MARC CANTILLON, M.D., EXECUTIVE DIRECTOR, COALITION AGAINST MAJOR DISEASE, CRITICAL PATH INSTITUTE Mr. Pallone. And I will now recognize Dr. Morrison-Bogorad. STATEMENT OF MARCELLE MORRISON-BOGORAD Ms. Morrison-Bogorad. Thank you very much, Chairman. And I really thank you for inviting me to appear before you today to discuss the pressing issue of Alzheimer's disease. The National Institute on Aging is the lead institute for Alzheimer's disease of the NIH. So in this regard I want to tell you that you can now download copies of our 2009 congressionally mandated ``NIH Progress Report on Alzheimer's Research'' from the NIH Web site. I am retiring at the end of this year and am truly sad that a cure for Alzheimer's has not been found on my watch. But the momentum is there, and I believe that my successor will have this joy. Federal researchers, other scientific agencies, the private sector, and not-for-profit are collaborating as never before to try to solve the mysteries of this disease. For example, we have regular meetings with each of the entities that will testify at this hearing. We have partnered with the Alzheimer's Association on many occasions, and ongoing is a joint venture on updating the definition of AD. The Alzheimer's Foundation of America and other organizations held House and Senate briefings a couple of weeks ago where Richard Hodes, director of NIA, was asked to testify. And we work with the Coalition Against Major Diseases for the Critical Path Institute on matters related to innovations and marker use on clinical trials. The NIA plans and manages an extensive program to better treat and ultimately to prevent Alzheimer's. How do we do this? Well, from our Alzheimer's Summit in 2006, the 2010 State of the Science Conference, from numerous specialized workshops, from program review by our NIA Council every 4 years, we get input from all these sources, and from these decide on the best ways to advance Alzheimer's research commensurate with our funding. For example, at the 2006 Summit, it was recommended that we develop a project focusing on early onset AD families. We have since funded the International Dominantly Inherited Alzheimer's Network to study preclinical disease in these families. Earlier this year, an NIH State of the Science Conference reported that there was, so far, insufficient evidence that any behavioral interventions for AD or age-related cognitive decline were effective, and that we ought to devote more resources to these questions. Even before the report was finalized, we had stepped up our funding of clinical trials to get definitive evidence whether or not various exercise and cognitive interventions might impact age-related cognitive decline, mild cognitive impairment, and AD. Now we are funding around 20 such trials. The Alzheimer's Disease Neuroimaging Initiative is another example of our leadership. ADNI is a very successful public- private partnership to identify biological and imaging markers for better ways of monitoring AD clinical trials and also identifying persons at risk for the disease preclinically. Here we initiated the process through a series of meetings where we brought together all interested parties to discuss what initiative would be most useful for them for development in a precompetitive setting, and these discussions led to funding of ADNI in 2004, with substantial financial support from industry and from not-for-profits such as the Alzheimer's Association, coordinated by the Foundation for NIH. Another aspect of our planning process is to ask our director for funds to specifically target new areas that need to be developed. An example is our Translational Initiative. Partly through these targeted funds, this important and innovative portfolio has grown to over 60 projects, each aimed squarely at bringing a new drug to the stage where it can get FDA approval for performing clinical trials. This is a particularly important area for us to develop, as pharmaceutical companies are often unwilling to put monies into the beginnings of the drug discovery process and translational research. One reason that drug trials have not worked so far may be that the drugs are given too late in the disease to have any effect, but prevention trials to test those possibilities take a lot of money and time under current protocols. We are developing new methodologies, and in the meantime we have been able to fund a number of prevention trials by the simple way of adding cognitive measures on to trials started for other clinical conditions often by other institutes. So it is a cheap way of funding these trials. But other possible drug therapies for Alzheimer's are directed against unique aspects of the disease, and so for these more specialized interventions NNIA must continue to develop AD-specific trials. We make difficult decisions all the time about where to put our resources. We do not have a crystal ball to tell us what approach will eventually pay off in relieving suffering from this frightening disease. We are committed to trying every promising avenue, and we will succeed. Thank you. Mr. Pallone. Thank you very much. [The prepared statement of Ms. Morrison-Bogorad follows:] [GRAPHIC] [TIFF OMITTED] T8139A.001 [GRAPHIC] [TIFF OMITTED] T8139A.002 [GRAPHIC] [TIFF OMITTED] T8139A.003 [GRAPHIC] [TIFF OMITTED] T8139A.004 [GRAPHIC] [TIFF OMITTED] T8139A.005 [GRAPHIC] [TIFF OMITTED] T8139A.006 [GRAPHIC] [TIFF OMITTED] T8139A.007 [GRAPHIC] [TIFF OMITTED] T8139A.008 [GRAPHIC] [TIFF OMITTED] T8139A.009 [GRAPHIC] [TIFF OMITTED] T8139A.010 Mr. Pallone. Next is Mr. Harry Johns. STATEMENT OF HARRY JOHNS Mr. Johns. Thank you very much, Chairman Pallone, distinguished members of the committee. I want to also thank Ranking Member Shimkus and all of you for holding this hearing this afternoon. My name is Harry Johns. I am the president and CEO of the Alzheimer's Association. The Alzheimer's Association was created in 1980 and is the leading voluntary health organization in both the provision of Alzheimer's care and support, and in the funding of Alzheimer's research, as the largest nonprofit funder of Alzheimer's research in the world. To do our job well, we spend a lot of time listening, listening to people with Alzheimer's, their caregivers, their families, to researchers, our many collaborators, and to hundreds of thousands of our advocates. We hear their stories and experiences, and they inspire us to go further, faster, to provide better care, and ultimately the cure that we all seek. We listen to those families in your districts and we listen to our own families. My mother had Alzheimer's disease herself. Any of us who have seen the disease up close don't want to see it again, for anyone to have the disease or to be a caregiver. Regrettably, we know it is going to happen much more ahead of us. You know, the effects of Alzheimer's, as it has been stated previously, are truly devastating at the human level. In this country alone, as previously referenced, we estimate actually there are about 5.3 people who have the disease, about 200,000 of them younger onset, younger than 65. And by the middle of the century it could be as high as 13.5, or even as high as 16 million individuals in the United States alone. Today, there are 35 million people worldwide who have the disease. Today, if you do develop Alzheimer's, we can say with certainty, absolute certainty, that you will either die with the disease or of it. And of the 10 leading causes of death, as previously mentioned, Alzheimer's is now sixth, Alzheimer's is growing by far the most rapidly, a 50 percent increase between 2000 and 2007, the last year that statistics are available. And it is the only one of the top 10 causes of death that has nothing to do to prevent, stop, or even slow it. For perspective, even though Alzheimer's is likely seriously underreported, already today it is killing more people than diabetes and more people than breast cancer and prostate cancer combined. The 11 million caregivers in the United States, Alzheimer's can literally take everything they have to give: their time, their money, their jobs, and their own good health. And it happens every day, oftentimes never to be recovered. One study at least indicates that people who are caring for a spouse with Alzheimer's can actually predecease the individual with the disease. The economic impact of Alzheimer's is also devastating, truly staggering numbers. You have already mentioned the $172 billion in costs today for Alzheimer's going as high as $1 trillion by the middle of the century, which was reported in the Alzheimer's Association's trajectory report earlier this year. And those are in today's dollars. Those are not inflated dollars. Those are dollars rated in today's terms. A total of $20 trillion over the next 40 years just to pay for the care costs, not an additional cent for the research we so badly need. And our country is simply not ready for this onslaught of Alzheimer's that is already upon us. Let's take the case of research funding. You know, we have made significant progress in other diseases, in fact, in no small part because of the significant investments we have made in those diseases. Research spending at the Federal level for cancer is about $6 billion today; for a cardiovascular disease about $4 billion; for HIV/AIDS it is about $3 billion. Now, all of those are good investments. They have paid off in lives saved, and they are going to continue to pay in that way for our country. But in Alzheimer's, we are only spending $469 million a year, despite those other huge impacts we have already discussed. We know that more money invested in research save lives, but we also know that too little money invested in research actually costs lives and ultimately will drive those very huge care costs into the trillions. Today, right now, we spend $250 in America on care cost for Alzheimer's and dementia compared to $1 invested in research; 250 to 1. So to address the underinvestment in Alzheimer's research, the Alzheimer's Association strongly supports the Breakthrough Act. It is a bill that authorizes $2 billion in Alzheimer's research. But the Alzheimer's Association will not ask others to do what we won't do ourselves. We play an unparalleled role in the research community in Alzheimer's, globally as well as in the United States, and that certainly includes direct investment in Alzheimer's and effectively investing in science. Our peer-reviewed research program since its inception has funded $279 million worth of research to 1,900 investigators, making us the largest funder in the nonprofit world. And through partnerships and our own funding, we have played some kind of a part in every major advance over the past 30 years as a result. But as I discuss these necessary investments in Alzheimer's research, and more broadly, I certainly recognize that our country is currently engaged in a very appropriate and very necessary conversation about our fiscal situation, our fiscal situation as a country, and we have to address that. But Alzheimer's, unaddressed, is one of our most devastating issues, both human and financial, as we have all discussed. So we must aim at what is the highest return potential we have for investments, Alzheimer's one of them. So if we can't fix Alzheimer's, I don't think we can fix Medicare. Medicare costs three times more for each individual in the system who has Alzheimer's than it does for a normal individual. Mr. Pallone. Mr. Johns, I have been trying not to cut anybody off because we only have the one panel. Mr. Johns. Let me wrap really quickly then, Mr. Chairman. Certainly I want to mention the National Alzheimer's Project Act, and I want to thank this committee for its leadership. I certainly again want to recognize Congressman Markey who you mentioned for his leadership as the author; certainly recognize Dr. Burgess who has also provided leadership on this. And we know, of course, the Senate passed the bill yesterday. We look forward to the real possibility of the House passing it. We urge you to pass it. And we look forward to working with this committee and the Congress to realize the ambition of the Alzheimer's Association, its vision, a world without Alzheimer's disease. Mr. Pallone. Thank you. [The prepared statement of Mr. Johns follows:] [GRAPHIC] [TIFF OMITTED] T8139A.011 [GRAPHIC] [TIFF OMITTED] T8139A.012 [GRAPHIC] [TIFF OMITTED] T8139A.013 [GRAPHIC] [TIFF OMITTED] T8139A.014 [GRAPHIC] [TIFF OMITTED] T8139A.015 [GRAPHIC] [TIFF OMITTED] T8139A.016 Mr. Pallone. Mr. Hall. STATEMENT OF ERIC J. HALL Mr. Hall. Chairman Pallone, members of the committee, thank you so much for convening this hearing and for inviting the Alzheimer's Foundation of America to testify. I am Eric J. Hall. I am the AFA's founding president and chief executive officer, and I am truly honored to be here to testify on behalf of our member organizations and families that we care for across the country. AFA was formed in February of 2002 to provide optimal care and services to individuals confronting dementia, and to their caregivers and families, through member organizations dedicated to improving quality of life. Today our membership consists of more than 1,400 organizations, including grassroots not-for- profit organizations, government agencies, public safety departments, and long-term care communities. Our services include a toll-free hotline staffed by licensed social workers, educational materials; Care Advantage, which is a free quarterly family caregiver magazine that right now reaches about 1 million readers; professional training programs; AFA Teens, which is a Web-based support and scholarship program; and our National Memory Screening Date. As a foundation, our money is generated and disbursed by grants to service organizations as well as respite grants to families who are in need. Recognizing the severe fiscal challenges facing our Nation, it is more important than ever to leverage available private- sector resources in a cost-effective manner to support public- sector initiatives. AFA makes substantial investments in care and services to tackle the enormous challenges associated with Alzheimer's disease and related dementias for both individuals and their family caregivers. But the needs of the population are going to overwhelm our resources in the years to come. The National Institute on Aging reports that as many as 5.1 million Americans over 65 are, today, dying of Alzheimer's disease, and those numbers are projected to increase dramatically in the coming years. The rapidly rising costs associated with this disease will put an enormously heavy burden on families, businesses, and government economically. It is our opinion that increased investment in preventing, treating, and/or curing chronic diseases of the aging, such as Alzheimer's disease, is perhaps the single most effective strategy in reducing national spending on health care. Chronic diseases associated with aging account for more than 75 percent of Medicare and other Federal health expenditures. Unprecedented increases in these diseases as the population ages are one reason why the Congressional Budget Office projects that total spending on health care will rise to 25 percent of the U.S. GDP by 2025. Simply put, our Nation does not have the luxury of time to wait to address the health research needs of this population. Standard & Poors' recent report titled ``Global Aging 2010: An Irreversible Truth,'' stated that no other force, no other force is likely to shape the future of national economic health, public finances, and/or policymaking at the irreversible rate at which the world's population is aging. Standard & Poors believes that the cost of caring for people will profoundly affect growth prospects and dominate public finance policy debates worldwide. As we have learned from the experience that we have all had with polio, heart disease, HIV/AIDS, cardiovascular, and other diseases, medical research and breakthroughs can have a profound impact on reducing health care costs. As the extension of life expectancy from age 47 in 1900 to almost 80 in 2000 demonstrates, medical advances enormously increase national productivity and prosperity. Yet those benefits can only come about if NIH makes the needed investments and research aimed at preventing, treating, or curing age-related diseases and extending healthy life. AFA, again, recognizes the serious fiscal challenges facing our Nation, which will require Congress to carefully scrutinize future funding priorities. We believe it is critical to leverage critical resources within the private sector, including not-for-profit organizations such as our own, to support proven, cost effective initiatives, and that is why we will all need Congress to be our partner. This subcommittee and the full Energy and Commerce Committee have played a critical role in overseeing and supporting the mission of the NIH, and we respectfully urge your support for continued commitment to NIA's important research. AFA is seeking $1.4 billion, an increase of $300 million in fiscal year 2012 National Institute on Health budget specifically for the National Institute of Aging. This funding is the minimum essential to sustain the research needed to make progress in attacking the chronic diseases that are driving mass increases in our national health care costs. That level of funding would make the NIA's baseline consistent with comparable research initiatives conducted elsewhere under the auspices of NIH. If NIA funding is not significantly increased, we stand to lose a generation of more young and emerging investigators in aging and Alzheimer's disease. This would be an enormous waste, since the NIA is poised to accelerate the scientific discoveries that can be translated quickly into effective prevention and efficient health care to reduce the burden of this silver tsunami of age-associated chronic diseases. Breakthroughs from NIA research can lead to treatments and public health interventions that can delay the onset or slow the progression of costly connections such as heart disease, stroke, diabetes, bone fractures, age-related blindness, Parkinson's, and indeed Alzheimer's disease. From a budgetary perspective alone, such advances could save trillions of dollars by the middle of this current century. At the Alzheimer's Foundation of America, our incredible strength and our quick success has come from collaboration. AFA looks forward to working with members of the subcommittee to address the important issues raised in today's hearing and, in the long term, to end the devastation caused by Alzheimer's disease. Mr. Pallone. Thank you. Thank you, Mr. Hall. [The prepared statement of Mr. Hall follows:] [GRAPHIC] [TIFF OMITTED] T8139A.017 [GRAPHIC] [TIFF OMITTED] T8139A.018 [GRAPHIC] [TIFF OMITTED] T8139A.019 [GRAPHIC] [TIFF OMITTED] T8139A.020 [GRAPHIC] [TIFF OMITTED] T8139A.021 [GRAPHIC] [TIFF OMITTED] T8139A.022 Mr. Pallone. Dr. Cantillon. STATEMENT OF MARC CANTILLON, MD Dr. Cantillon. Mr. Chairman, members and staff, thank you for the opportunity to present testimony on this very important topic. I am Dr. Marc Cantillon, the executive director of the Coalition Against Major Disease of the Critical Path Institute, also known as C-Path. I am a practicing physician and a neuroscientist, with 15 years' experience in research and drug development at the NIH, academia, nursing homes, and within the pharmaceutical industry. C-Path is a nonprofit organization founded in 2005 by the FDA and the Arizona community in order to build collaborations that identify more reliable and efficient methods to test new medicines' applied regulatory science. As you have heard, in spite of the exciting laboratory discoveries in Alzheimer's research, we lack full translation, we lack new medicines that could significantly alter the course of the disease. And, indeed, we have seen huge Alzheimer's disease drug trials fail. Nevertheless, there is reason for renewed hope. Across the hall into science is a proverb written up that I would like to quote. ``Where there is no vision, the people perish.'' Proverbs. Thanks to the work of this subcommittee, the FDA Amendments Act of 2007 included a provision for the FDA to create the Critical Path public-private partnerships. We are extremely grateful to Congresswoman Marsha Blackburn of Tennessee, Congressman Elliot Engel of New York, and Congresswoman Gabrielle Giffords of Arizona for their leadership on this legislation. The Coalition Against Major Diseases, or CAMD, was one of the first of these partnerships launched by the FDA, and it is already creating and identifying new tools that will speed the safe development of new medicines for Alzheimer's and other neurodegenerative diseases. CAMD seeks to recreate the sense of urgency and open collaboration that made the unprecedented rapid progress against AIDS possible. Sharing of knowledge was the hallmark and is generally accepted as the reason that the rapid and enduring success was secured against that epidemic. Created by C-Path and the Engelberg Center of the Brookings Institute, CAMD is a consortium that currently includes scientists from 12 major pharmaceutical companies, NIH scientists, as well as experts from patient organizations such as my colleagues here today, the Alzheimer's Association and Alzheimer's Foundation. The FDA, along with the European Medicines Agency, the EMA, and indeed the Japanese PMDA, provide advisers to our over 250 scientists who participate in CAMD by sharing what they know about Alzheimer's disease and how they can better test new therapies. CAMD's accomplishments have already changed the way we attack this devastating disease. Firstly, CAMD researchers compare the way that they and other researchers score dementia, scored dementia in a clinical trial, and subsequently corrected over a dozen inconsistencies. Now it is possible for them and for EMA and FDA to compare results directly from study to study. This is a great example of applied regulatory science because it improves the quality, the accuracy, and efficiency of decisions made by both the regulators and the regulated industry of the pharmaceutical industry. A first ever, CAMD was able to pool the data from 11 clinical trials conducted by several different pharmaceutical companies. This has created the largest publicly available Alzheimer's disease database in the world, and it describes the natural course of the disease in over 4,000 patients. Over 200 teams of scientists around the world are already using this. For example, modeling. In the past, former scientists had to design trials based on their clinical experience or data within the company or what they read in the medical literature. This database allows the individual patient-level data to show progression over time in this number of patients. This is far more precise than their clinical experience or what they can glean from the medical literature. This database also allows them to more accurately predict the outcome for a particular trial, or how long the trial must be, or how many patients must be included; indeed, how genetic subsets of the population might respond differently, et cetera. CAMD is now working with NIH and with academic centers to pool their data in the same standardized database to further enrich this as a leading-edge tool. CAMD is also helping define the FDA's new qualification process described in the new guidance. In this work, CAMD submits data and requests that the FDA accept certain brain- imaging tools or cerebral spinal fluid tests as qualified for identifying patients much earlier in their disease when there is still brain function to be saved. Yes, there are many reasons for hope. The Critical Path public-private partnership is improving the applied regulatory science for Alzheimer's at the FDA. However, we do need your help. Understaffing is a serious problem throughout the FDA and is especially serious for CAMD. The FDA needs your support to be able to dedicate the required number of scientists and staff to participate in CAMD and other Critical Path partnerships. This is the kind of applied critical science that is changing the way drugs are tested and evaluated today, so that Alzheimer's can be prevented, not just slowed. I thank you for the opportunity to provide this testimony, and everybody in the CAMD thanks you for your leadership and foresight in authorizing the FDA's Critical Path public-private partnerships that are giving new hopes to patients and families at risk for this devastating disease. I would be happy to answer any questions. [The prepared statement of Dr. Cantillon follows:] [GRAPHIC] [TIFF OMITTED] T8139A.023 [GRAPHIC] [TIFF OMITTED] T8139A.024 [GRAPHIC] [TIFF OMITTED] T8139A.025 [GRAPHIC] [TIFF OMITTED] T8139A.026 [GRAPHIC] [TIFF OMITTED] T8139A.027 Mr. Pallone. Thank you, Doctor. I thank all of you. We will take questions from the members, and I will start with myself, and I will start with Dr. Morrison-Bogorad. Scientists know Alzheimer's attacks the brain long before people exhibit cognitive decline. But the specifics are crucial because, so far, drug after drug has failed to effectively treat Alzheimer's in people who already show symptoms. I know you suggested that that was part of the problem, that perhaps the answer is earlier treatment before you actually have the signs of the disorder. So what I wanted to ask is why are biological markers, whether gene mutations or pathological brain changes, important to the development of effective treatments for Alzheimer's and what research is NIA conducting to better understand these markers? I am sort of going back to that same issue that you mention, is perhaps we should be starting earlier but then we would have to know if people have the disorder. Ms. Morrison-Bogorad. It is probably one of the items that we are pushing most of our effort into these days, because we do think that understanding the earlier stages of Alzheimer's disease is very important and we have thought that ever since we reissued the request for applications for funding our Alzheimer's disease centers across the country. Because about 8 years ago now we said to them, forget about late stages of disease. We want you to really, really concentrate on the earlier stages. So we have thought about this for quite a long time; and, obviously, one of the things that held us up is not being able to identify preclinical stages. That is one of the things that is really being addressed by the Alzheimer's disease in your imaging initiative, especially in people who aren't yet showing symptoms or in people who are developing mild cognitive impairment, which is a precursor to Alzheimer's. Researchers working together with industry and people funded by us are identifying markers in the cerebrospinal fluid, and these markers are lowered beta-amyloid and higher levels of protein called tau that signal that a person is approaching the stage of mild cognitive impairment. The other technology which has been developed by ADNI and by others is actually being able to look at in the brains of individuals cerebral amyloid plaques through positron emission tomography, through imaging. And this is perhaps the most amazing breakthrough over the last several years because that has allowed us to see that in a number of folk, older folk, older than 65 or 70, about 20 percent of these folk who otherwise would have been thought of as normal, who are quite normal cognitively, have levels of amyloid plaques in their brain that in some cases are equivalent to a person with Alzheimer's disease who can't at the moment function for themselves. So the CSF markers and the brain markers are two ways we have of identifying preclinical disease which we didn't have before, and they could be used to identify people who have got markers in the brain, amyloid in the brain for earlier clinical trials than we are able to do right now. So I agree. It is really, really, really important that we develop these markers and that we use them to do more efficient clinical trials in the preclinical stage. Mr. Pallone. I had a second question, but I maybe would prefer if anybody else would want to comment on this issue because I think it is pretty important. Would any of the others like to---- Mr. Johns. I simply add that what Marcelle has said is certainly one of the most exciting areas that is occurring in Alzheimer's research today. She has indicated that we have worked together on this at the Alzheimer's Association International Conference on Alzheimer's Disease over the summer. There were significant findings released on this very front. It is potential to go to the point where we can actually identify Alzheimer's presymptomatically in the future. It is not ready for the clinic. That is for the lab at this point. That is a significant set of advances that are very important to us. What is of course very important is that we have a parallel in treatments. Right now, we are making faster advances on the diagnostic science than we are on treatment side. So what we really need to do is catch that up. And that is one of the most important reasons for the additional funding that is really needed for Alzheimer's disease. Mr. Pallone. Thank you. Mr. Gingrey. Mr. Gingrey. Mr. Chairman, thank you. I think I will shift then to the treatment aspect of it and maybe come back to very interesting things that the two of you have just talked about with regard to early diagnosis. Dr. Cantillon--I hope I did better. I know I botched that up pretty bad the first time--I have a lot of respect that the FDA, the Food and Drug Administration, performs. With that being said, I am interested in exploring how the FDA drug approval process might be improved in the hope it may help spur a greater drug development for diseases such as Alzheimer's. I think I mentioned in my opening remarks that I have introduced legislation along with my colleagues here on the committee, Energy and Commerce Committee, Health Subcommittee, bipartisan, Mr. Green and Ms. DeGette on the Democratic side and myself and Mr. Rogers from Michigan on this side, the GAIN Act. This is in regard to the shortage of antibiotics. So a different disease, a different category of drugs, but equally as important. Can you tell me, Dr. Cantillon, how applying scientific advances such as the use of biomarkers or drug development tools might aid in drug development in this country? Dr. Cantillon. Yes. Thank you for that question. So, actually, as a medical director of Alzheimer's and other programs with Schering Plough over the last couple of years before CAMD, I did sit on an industry advisory group for this ADNI trial that Dr. Bogorad was speaking about. So I was giving some advice into the choice of the instruments and that these markers were being developed, had my hat on in terms of both drug development and facing the FDA with a package for an approval for a treatment. The trouble is when you are coming from a drug developer's point of view, this is all very cutting edge. And as I said in my testimony, the science by itself is truly not enough. It is not enough to have exciting markers that may predict something that is going on in the brain if it can't be harnessed into a path and the development steps to use a new drug and prove that. I will give you an example. So we talked just now about the cerebrospinal fluid. We know there are certain proteins that can indicate both the disease and perhaps even the type of progression predictive. We would take that and we are taking that and in a collaborative way with the whole field. So we are just one collaboration that I had mentioned. Look at the evidence for that in a critical way, in a scientific manner. The regulatory science part is having our regulatory scientific colleagues internationally review that in a context of use. Does that allow you to choose a population for a trial who are not yet demented? So to call somebody demented, you don't need to be a doctor. It is very clear that the person does not have their brain functions in the same way for memory and so forth. To find somebody that is very early in the disease or even hasn't fully shown clinical symptoms, you need these markers. Can we ask for FDA regulatory approval that these are standardized in such a way that they can become a tool, a standard tool publicly available to any company in this country or anywhere else? Can take off the shelf and put into a program such that, by using this tool, they don't have to defend that tool when they go in front of the FDA? That process has already been done in this qualification. And instead they can focus on their own particular drug that then fits into this pattern and can use that patient population, for example, to show progression over time. Do those people who have low tau, high tau, low a-beta or a particular brain picture with amyloid, do they progress faster than others and can you show a difference in the people who were on drug and off drug? That is how the whole development process can make use of this science and translate it into something that the FDA can then approve or not approve. The FDA did put out a guidance document partially from working with us just 2 months ago, and in that the steps are laid out very clearly. Essentially, it is show us the evidence. Very similar---- Mr. Gingrey. My time is rapidly drawing to a close, but let me quickly ask you, do you think it is important that such drug development tools--biomarkers--that they first receive approval by the FDA before being used by industry and the agency to measure the safety and efficacy of the drug? Do you think that the FDA would have to approve this off-the-shelf kind of testing ahead of time? Dr. Cantillon. The FDA has the possibility just to approve for commercial use or other use, but that is not within the context of use that we are talking about. The context of use would help that tool help define a particular population. So, for example, let me give you another--for another consortium. We developed some markers for renal injury, for kidney injury. They were brand new, and they could allow a drug developer or anybody to show if something was happening very early on before the kidney actually was destroyed. These markers went through this qualification process and are being very widely used by all companies, including my former one, to make decisions about drug development. When they go to the FDA, they don't need to defend those markers for kidney. They have already been approved. So it takes a lot of that work away and you can focus on the drug. Mr. Gingrey. Doctor, thank you. That does answer my question. And I will yield back, Mr. Chairman. Mr. Pallone. Thank you. The gentlewoman from the Virgin Islands. Mrs. Christensen. Thank you. Thank you, Mr. Chairman. Dr. Morrison-Bogorad, given the disproportionate impact of Alzheimer's on minorities, is the National Institute of Minority Health and Health Authority Research, which was formally the center among the NIH institutions that you are collaborating with? And, also, how diverse are the participants in the clinical trials? Ms. Morrison-Bogorad. It is again something that we paid particular attention to, especially in our flagship clinical trials, the Alzheimer's disease clinical consortium, and there we have made a rule that a certain number of people in each clinical trial that we run there are minorities. And that has been extraordinarily helpful because it has meant that I think a fifth of the folk who participate in certain of these studies are minorities. So it is amazing what a little rule will do. And we certainly have got quite a vibrant program in epidemiology looking at Alzheimer's disease in minorities and comparing that with Alzheimer's disease. And I would say at the moment the results are somewhat equivocal because many of the ways in which you define Alzheimer's disease are also very dependent on things like the education of the person who is taking the tests. And many older African Americans, of course, for other reasons haven't had the education that they should have had and so they don't do as well on these tests as they should. It doesn't mean they have Alzheimer's, however. So this is a very, very thorny issue and we have gotten a number of very good researchers working on that to actually try and tease out what part of the minority burden of Alzheimer's disease is real--and I do believe some of it is because some of the possible things that cause Alzheimer's disease are, as you are aware, much more prevalent in minority communities, things like heart disease. But I do believe that some of the numbers are a little bit over, perhaps larger than they should be because of this issue on how to determine Alzheimer's disease in people with different backgrounds. Mrs. Christensen. That is interesting, because my impression is that it is underdiagnosed rather than overdiagnosed in minorities. And I will let anybody comment on that. But I wanted to ask Mr. Johns, if I can get this question straight, because I know this hearing is really about getting funding for Alzheimer's. And, Mr. Johns, I heard your argument very clearly about the need for an increased investment in Alzheimer's to reduce the cost of health care eventually and to perhaps even save Medicare. We have been trying to get CBO to score prevention for a while, actually introduced legislation to have them do that when they are asked by the committee. So, without having scoring in place for prevention, how do you foresee getting the funding needed, especially in a Congress that is committed to cutting spending? And how important do you think scoring prevention is to this particular issue? Mr. Johns. Well, certainly, as you say, the fact is that CBO won't score what I would describe as a game changer. That is a problem I know for all of us when we have interest in what would be effectively R&D for our country. We really have-- beyond the immediacy of Alzheimer's, we have what is a potential brain drain in our country as a result of research being attracted overseas. While it isn't specific to Alzheimer's, it is generic and related to Alzheimer's, as well as other medical research. So we have as a country to face what is, first of all, a significant problem in that larger regard with medical research but very specific in Alzheimer's; and, of course, we have the challenge of facing our economic realities and also then funding something that cannot be scored. We recognize fully the difficulties of this, but we can also see from the projections we have done--and we have gone to outside sources at the Alzheimer's Association to develop what are these data. We have actually used the CMS data on expenditures on Medicare and Medicaid. We have taken those to Dartmouth. We have had the Lewin Group look at all these data and what we have identified is that $20 trillion cost over the next 40 years. One of the problems we have as a country, of course, is actually addressing problems that are longitudinal. Those of you sitting across from me know that better than I in terms of how hard it is to make those things work. But we certainly know it, too, the Alzheimer's Association and any of us here sitting on this side who are trying to change the course of Alzheimer's disease. We have to find the national fortitude to address this already enormous problem. Everyone who has the disease today will die with it or of it. We do not have a treatment that stops or even slows it. The devastation of the disease at a human level for all of us who have experienced it personally and for all of those who haven't, we recognize just how bad that is. I don't have the easy answer to your question, but the scale of the problem, the enormity of the issue begs for us to find a way to answer the question so that we can address this now. We are running out of time. One of the things that Marcelle mentioned is that the science community believes ever more that we need to intervene sooner, that the plaques entangles of the disease are deposited earlier in life, at least 10 years before the symptoms manifest. If, in fact, we don't make these investments relatively soon, the baby boomers, 10 million of whom will have this disease, will be a lost cause. And the devastation at a human level and the economic toll will be solidified if we don't move relatively soon. Mrs. Christensen. Thank you. Thanks. Mr. Pallone. I thank the gentlewoman. Next, we have our Alzheimer's hero here, who actually sponsored both of the bills that you mentioned, Mr. Johns, the one that I guess is now in the House for action hopefully next week, as well as the larger bill. Mr. Markey. Mr. Markey. Thank you, Mr. Chairman, very much; and I thank our witnesses so much for your participation here today. Robert Browning wrote, grow old with me, the best is yet to be. But the truth is that for millions of millions of Americans the golden years are now the worst years because of Alzheimer's and the family caregiver who has to help. So this is now at 4 million or 5 million Americans already an epidemic since, as we know, not only does the Alzheimer's patient have the disease, but one family member has it as well as. So about 10 million Americans right now are living with it on a daily basis in their homes or in some facility. And when it goes up to 12 million times 2, 24 million, 25 million people, the caregiver and the patient, it is going to be an incredible moment in American history. So we have an incredible responsibility here to make sure that we put in place a plan. And, Mr. Chairman, you made reference to it, which is that the National Alzheimer's Project Act, which I introduced on this side along with Christopher Smith, the co-chair of the Alzheimer's Task Force, passed the Senate last night. And Senator Bayh and Senator Collins did an excellent job in framing it up and we will be able to pass that next week on the House floor. And then we will have a plan. We will have something that makes it possible for us to put in place something that is the plan to attack this disease. And it is long overdue, but it is a good beginning. So let me ask you this, and maybe you could reflect upon it, Mr. Johns, if you could. Last year, the Federal Government spent $122 billion on helping people with Alzheimer's, but we only invested $469 million in finding the cure, and we know we are only at the beginning of an explosion of the bills that are going to come in across America for the Federal Government to help families with Alzheimer's. Can you reflect upon that, give us your insight as to how big it is going to become and why it is imperative that we act now? Mr. Johns. Certainly. And let me thank you, Mr. Markey, for your authorship of NAPA and the input and leadership of yours and the committee's in moving that forward. Certainly Alzheimer's is already costing $172 billion in total, the $122 billion you talked about from the Federal Government. Incidentally, at the Medicare level, Alzheimer's is driving 17 percent of the Medicare budget---- Mr. Markey. Say that again. Mr. Johns. Seventeen percent of the Medicare budget is driven by Alzheimer's today. The total cost for Alzheimer's again to the country and other dementia is, by the middle of the century, in excess of $1 trillion per year; and by far the bulk of that will be the Federal Government's cost projected from today's levels with no changes. So that it simply won't be affordable not only on the economic front but again on the human front. We can't accept what will happen to families. We don't have the ability to deal with the end-of-life considerations of the long-term care. Families at some point, dedicated as they are, with 70 percent of the people who have Alzheimer's living at home and cared for at home, at some point that other percentage as a result of the fact that families, no matter how much time they spend, the 24 hours a day that they often spend as caregivers, especially toward the end of life, is no longer enough or they are simply not capable of handling the difficulty of the care at home so that we are not equipped at this point. As you indicated, we don't have a plan for any of these things at this point. NAPA will hopefully address that, but we are just not prepared as a country to handle any of these problems at the scale they are going to rise to. Mr. Markey. This is just something that is not as well understood as it should be. My mother had Alzheimer's, and she was a valedictorian. My father was a milkman. And my father always said it was an honor that my mother married him. And he used to say, as well, if the strength of your brain determined who got Alzheimer's that he would have had it and my mother would have been taking care of him. But we know that this is an equal opportunity disease. At age 80, 82, 84, 86, 88, my father kept her in our living room with the arms of a milkman, arms the size of my legs; and he was able to do it. But for many families it becomes exhausting. You can't do it. There is a point beyond which you need help and that helps comes increasingly from the Federal Government in the form of $122 billion a year right now. But I don't think actually we are going to be able to solve the Federal budget deficit if we don't dramatically increase the Federal investment in research. It will be a trillion dollars a year just for Alzheimer's care in another 15 years, and it is just a number that is going to increase exponentially. And despite the efforts of people like my father and other families all across the country, these people are heroes, but heroes need help and they need hope. And only the National Institutes of Health, really the institutes of hope, really give people the courage to keep on going. So this whole effort is absolutely--I think it should be the number one issue, to be honest with you, just from a budgetary perspective. From a humane perspective, yes. Coupled with the Alzheimer's Breakthrough Act, which I have introduced, and the HOPE Act, the Health Outcomes, Planning and Education Act, which we have to focus on, we have to put in place the kind of ingredients of this plan that make it possible for us to solve this problem. And I commit to you, all of you, that I am going to continue to just work my heart out to make this something that becomes real in people's lives; and I cannot thank you enough all of you for your support. And, Mr. Chairman, I thank you for conducting this hearing. I don't think there is a more important subject for us to be discussing as Americans. Mr. Pallone. Thank you. And thank you for all you do, Mr. Markey, on this and other issues. Mr. Engel. Mr. Engel. Thank you. Thank you very much, Mr. Chairman. Right in the nick of time. First of all, I want to thank everyone on the panel. This is certainly a very, very important and--and something I have had a lot of concern about. I think that--what all my colleagues have said--this is what we ought to be spending money on when we talk about some of the other issues. I think we should all agree on issues like this. Mr. Chairman, I want to ask unanimous consent to insert my opening statement in the record. [The information was unavailable at the time of printing.] Mr. Engel. Let me ask Dr. Cantillon--first of all, thank you for your comments. I was told the comments you made before, and I deeply appreciate your saying that. The Critical Path Initiative is certainly something that is near and dear to my heart. I have strongly supported public-private partnerships, and I am pleased to learn that this program has been very effective in tackling diseases like Alzheimer's. You mentioned in your testimony, Doctor, that one way Congress can be helpful is to provide the resources to increasing staffing levels at FDA; and, as I mentioned before, I couldn't agree more. We need to increase resources for the FDA to help them bring drugs to market. But, given the limited resources we are working with, I was wondering if you could address other ways that we might help break down the barriers to translational research and help fill in the gap that has opened up between biomedical researchers and the patients who need their discoveries, which we unfortunately referred to as the valley of death. Dr. Cantillon. Indeed, thank you and thank you for your support for these partnerships. I think that the partnership, the public-private partnerships, are certainly a major part of the answer in this fiscal environment. So we have all said several times that this is about to bankrupt our country and many other aging countries around the world and there aren't unlimited funds either to put into any one particular disease. And maybe we need to look at an innovative science and an innovative way of answering some of these questions that have come up. So the FDA is going to be faced with a lot of the new science arriving in different ways. Part of what we have been working together with them was to put a process in place to translate this science, not just, let us say, from the test tube to the rat, but all the way through to a new medication at the very end. In other words, to make a process available to be able to gather the evidentiary information. The FDA doesn't have but a few handful of staff that we deal with on a very regular basis and, as I mentioned, are indeed a part of our consortium; and the Europeans, in fact, are in a similar situation. So what I was referring to is basically stretching the dollar and the people that we have. Part of what we are doing and seek to do more of is in fact build an in-kind work, and there is a lot you can do with that. There is a lot of scientists, just like there was a lot of data, who have been siloed, be it within government--I used to be at NIH--or within companies. Once a trial has failed, that data is essentially put on the shelf somewhere. Definitely with the kind of tools we are talking about, those data can be re-examined, at the very least, and perhaps there will be some pearls in there. But they also can be mined for the learnings that are there and shared. That is public and belongs to all of us. So what we put together are these various methods to do that. It is, I would say, an innovative tool to get people to give up their silo thinking, look at a precompetitive space for companies and even for academics, that they actually don't own the data that they have generated, that it belongs to the people. And if we can set it up that way, have it as a database, for example, as I mentioned, but also the other tools, then that is freed for the best minds in the world to attack and that is actually a very efficient way of doing that. Mr. Engel. Thank you for pointing that out. It certainly is a shame if research is put on the shelf and no one else can get to it or look at it. This has to be a collaborative effort, obviously, and thank you for pointing that out. I would like to ask a question of Mr. Hall and Mr. Johns. As I am sure you are aware, the Patient Protection and Affordable Care Act, which is referred to as health care reform, that we just passed here, we were doing this for as you know almost 2 years. There was a provision in there that improves access to home and community based care for patients. We know that these services greatly improve the quality of life for Alzheimer's patients. Health care reform, as we know, strengthens the long-term care system for chronic and other long-term neurological conditions both by eliminating the arbitrary caps on treatment, which we did--that was one of the crowning glories of this bill--and by expanding coverage to include preexisting conditions, which is the second pillar that is so important in this bill. In an effort to improve upon existing programs that positively impact Alzheimer's patients and help you provide services, can you give us some insight as to how these provisions will improve your ability to provide services for Alzheimer's patients and their families? And can you also address what other Federal programs exist to help you deliver services to more patients? Mr. Johns. Well, I personally think that all of our constituents are in a position, especially, as I mentioned previously, that 70 percent or more of people are cared for at home. All of our constituents who are in a position to be at home need additional assistance. As we see how all this unfolds, we will learn better exactly how it can best serve the individuals who are in those situations. So we certainly always have high hopes for what would help to be a better care at home. Because we know, as I mentioned a little bit earlier, that we do not have the capability to really accommodate all the folks who would ultimately perhaps need to go to long-term care. So additional ways to find ability to handle people at home is critical to the entirety of the Alzheimer's constituency. Mr. Hall. Additionally, I think we all agree that the longer we can prolong institutionalization the better it is for the Federal budget, for sure. But families do need an enormous amount of support. The conversation we have had here around research is pivotal. There is no doubt we need to find a cure as quickly as possible, and any amount of money that we can put towards this disease would be an enormous win at this point. But the reality is that a cure does not seem to be coming anytime soon, and so it does rely on care, and it does rely on those families. So any type of provision that supports individuals with the disease and cares for them and provides for them the greatest quality of life for the longest period of time is great. But also, too, we are recognizing the support that is truly needed for caregivers across the country at this point, at this juncture where we are in relationship to a cure is really the critical step. It is what we need to do. Mr. Engel. Thank you. Thank you, Mr. Chairman. Mr. Gingrey. Mr. Chairman, just very quickly--and I know the afternoon is getting long, and I appreciate our witnesses who have done a wonderful job. I guess I would direct this to Mr. Hall. Can you talk about the benefits of early detection as it relates to both the financial realities that patients face and for making end-of-life decisions? Mr. Hall. Sure. The Alzheimer's Foundation of America hosts and is the initiator of National Memory Screening Day, and the reason that we do that is really just to sort of educate the public as a whole and then to allow individuals to participate in memory screenings across the country. That isn't a diagnosis for Alzheimer's, but rather is looking to see if there is an indication of I guess the most common manifestation of Alzheimer's disease, which is memory problems. That initiative to us is really important because of the fact that it points to a possible early diagnosis of this disease. It is really critical because our experience at the Alzheimer's Foundation of America is that our phone calls, our e-mails in the volume that we receive, every single one of those families is in crisis and chaos. So they are really scrambling now to figure out what does Alzheimer'sdisease care look like? What am I now responsible for as a family? What is required of me legally, financially? What is required of my time? Early diagnosis in our interpretation is really important because, one, I think some of the treatments that are available right now are able too offset the progression of the symptoms of the disease and therefore the person enjoys--the person with the disease enjoys a higher quality of life for a longer period of time. And I have to tell you, traveling the country--and I am sure Harry could say the same thing--We have not met one single family anywhere that hasn't said that all they wanted was one more good day with their loved one who is in the grips of Alzheimer's disease. If we can prolong one more day, I think that is a win. That is what we have right now. But, additionally, planning in this situation is enormous, educating, empowering the family unit so that they understand. It is generally one in four individuals caring for every person with Alzheimer's disease. But also then enabling organizations to surround those families, to hold their hands, to walk with them on the journey and support them, to connect those families at the point of diagnosis with those necessary resources, instilling hope, which is probably the greatest missing link in Alzheimer's disease. At least by surrounding a family with hope you are doing just that. You are giving them a lifeline, and you are letting them know that they are not going to be alone in the process. That takes care of an enormous emotional toil for a family, which is probably the biggest piece of the picture. And then if you are able to bring in other resources of financial planning and legal planning and what all this looks like as far as care in the future, those are enormous wins in alleviating the burden, the stress, depression for caregivers. Mr. Gingrey. Thank you. Mr. Pallone. Thank you. That concludes our questions, but I just want to thank you all of you. It is obvious from listening to the questions and your testimony how important this is both now and in the future. We do plan to move the one bill that passed the Senate on the House floor next week; and, of course, the larger bill will have to wait for another time. But this is--I just want to stress how important really and how we really plan to prioritize this. So thanks a lot. Let me mention that you may get additional questions from the members to answer in writing. The members are supposed to submit those within the next 10 days or so, but you may get those and the clerk will notify you of that. But, without objection, this meeting of the subcommittee is adjourned. 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