[Senate Hearing 106-373]
[From the U.S. Government Publishing Office]



                                                        S. Hrg. 106-373
 
               PARKINSON'S DISEASE RESEARCH AND TREATMENT

=======================================================================

                                HEARING

                                before a

                          SUBCOMMITTEE OF THE

            COMMITTEE ON APPROPRIATIONS UNITED STATES SENATE

                       ONE HUNDRED SIXTH CONGRESS

                             FIRST SESSION

                               __________

                            SPECIAL HEARING

                               __________

         Printed for the use of the Committee on Appropriations




 Available via the World Wide Web: http://www.access.gpo.gov/congress/
                                 senate

                                 ______

                     U.S. GOVERNMENT PRINTING OFFICE
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                      COMMITTEE ON APPROPRIATIONS

                     TED STEVENS, Alaska, Chairman
THAD COCHRAN, Mississippi            ROBERT C. BYRD, West Virginia
ARLEN SPECTER, Pennsylvania          DANIEL K. INOUYE, Hawaii
PETE V. DOMENICI, New Mexico         ERNEST F. HOLLINGS, South Carolina
CHRISTOPHER S. BOND, Missouri        PATRICK J. LEAHY, Vermont
SLADE GORTON, Washington             FRANK R. LAUTENBERG, New Jersey
MITCH McCONNELL, Kentucky            TOM HARKIN, Iowa
CONRAD BURNS, Montana                BARBARA A. MIKULSKI, Maryland
RICHARD C. SHELBY, Alabama           HARRY REID, Nevada
JUDD GREGG, New Hampshire            HERB KOHL, Wisconsin
ROBERT F. BENNETT, Utah              PATTY MURRAY, Washington
BEN NIGHTHORSE CAMPBELL, Colorado    BYRON L. DORGAN, North Dakota
LARRY CRAIG, Idaho                   DIANNE FEINSTEIN, California
KAY BAILEY HUTCHISON, Texas          RICHARD J. DURBIN, Illinois
JON KYL, Arizona
                   Steven J. Cortese, Staff Director
                 Lisa Sutherland, Deputy Staff Director
               James H. English, Minority Staff Director
                                 ------                                

 Subcommittee on Labor, Health and Human Services, and Education, and 
                            Related Agencies

                 ARLEN SPECTER, Pennsylvania, Chairman
THAD COCHRAN, Mississippi            TOM HARKIN, Iowa
SLADE GORTON, Washington             ERNEST F. HOLLINGS, South Carolina
JUDD GREGG, New Hampshire            DANIEL K. INOUYE, Hawaii
LARRY CRAIG, Idaho                   HARRY REID, Nevada
KAY BAILEY HUTCHISON, Texas          HERB KOHL, Wisconsin
TED STEVENS, Alaska                  PATTY MURRAY, Washington
JON KYL, Arizona                     DIANNE FEINSTEIN, California
                                     ROBERT C. BYRD, West Virginia
                                       (Ex officio)

                           Professional Staff

                            Bettilou Taylor
                             Mary Dietrich
                              Jim Sourwine
                               Aura Dunn
                        Ellen Murray (Minority)

                         Administrative Support

                             Kevin Johnson
                       Carole Geagley (Minority)




                            C O N T E N T S

                              ----------                              
                                                                   Page

Opening statement of Senator Arlen Specter.......................     1
Opening statement of Senator Thad Cochran........................     2
Statement of Gerald D. Fischbach, M.D., Director, National 
  Institute of Neurological Disorders and Stroke, National 
  Institues of Health, Department of Health and Human Services...     3
    Prepared statement...........................................     5
Staying within the caps..........................................     7
Statement of Michael J. Fox, actor...............................    12
    Prepared statement...........................................    14
Statement of Joan I. Samuelson, President, Parkinson's Action 
  Network........................................................    15
    Prepared statement...........................................    17
Statement of James Cordy, President, Greater Pittsburgh Chapter, 
  National Parkinson's Foundation and leader, Parkinson's 
  Alliance.......................................................    19
    Prepared statement...........................................    21
Statement of Dr. J. William Langston, President, Parkinson's 
  Institute......................................................    22
    Prepared statement...........................................    24
Issue in hands of congress.......................................    26
Prepared statement of Senator Patty Murray.......................    29



                PARKINSON'S DISEASE RESEARCH AND TREATMENT

                              ----------                              


                      TUESDAY, SEPTEMBER 28, 1999

                           U.S. Senate,    
    Subcommittee on Labor, Health and Human
     Services, and Education, and Related Agencies,
                               Committee on Appropriations,
                                                    Washington, DC.
    The subcommittee met at 9:40 a.m., in room SH-216, Hart 
Senate Office Building, Hon. Arlen Specter (chairman) 
presiding.
    Present: Senators Specter, Cochran, Gorton, and Murray.
    Also present: Senator Wellstone.


               opening statement of senator arlen specter


    Senator Specter. Good morning, ladies and gentlemen. The 
hearing of the Appropriations Subcommittee on Labor, Health and 
Human Services, and Education will now proceed.
    We have a hearing today which focuses on Parkinson's 
Disease. This is a medical problem of enormous impact. We have 
with us today a very distinguished panel, including Mr. Michael 
J. Fox.
    One of the issues of developing public concern, public 
support, for research occurs in a very natural way, when 
someone of the prominence of Michael J. Fox comes forward and 
talks about his own situation. Senator Cochran, who has been 
the originator of the idea for this specialized hearing, and I, 
were just talking to Mr. Fox, who told us about his own 
personal reaction on going public, so to speak, of how he felt 
good this morning, with a sense of purpose, in coming to this 
hearing. Mr. Fox is one of thousands, tens of thousands, 
hundreds of thousands, of people in America who suffer from 
Parkinson's.
    A very distinguished Pennsylvanian, Mr. Jim Cordy, will be 
with us today as a witness. Whenever I am in Pittsburgh, which 
is often, Mr. Cordy is by my side with an hourglass. He holds 
the hourglass up to demonstrate that time is fleeting. This 
subcommittee has been very active in increasing the funding for 
Parkinson's research, as part of our overall drive for funding 
of the National Institutes of Health.
    Senator Harkin, who is the ranking Democrat, will be here 
shortly. But he and Senator Cochran and I and others have been 
working very hard to increase that funding. Last year, we added 
$2 billion, which was unprecedented, to NIH funding.
    I say frequently that the National Institutes of Health are 
the crown jewel of the Federal Government--perhaps the only 
jewel of the Federal Government.
    We had a subcommittee markup yesterday and a full committee 
mark up today, and we go to the floor tomorrow. I said to 
Michael that this was a very unique time for him to be here, 
because we put $2 billion additional in for NIH funding. The 
testimony which we had heard earlier--we will ask Dr. Fischbach 
about that today--is that we are within 5 years of conquering 
Parkinson's. While that is good, I do not think it is good 
enough, if we can do it faster. Because every day that we 
spend, it takes lives.
    The Parkinson's issue is related to another very 
controversial matter, and that is stem cell research, which had 
a major breakthrough last year, last November, a veritable 
fountain of youth when stem cells can be substituted, posing 
enormous promise for Parkinson's and Alzheimer's and many, many 
other diseases. There is a prohibition on NIH funding being 
used for development of stem cells. In the bill, we have a 
provision to curtail that limitation, to have broader NIH 
funding, which we are going to defer action from this bill 
until February, because we want to pass this bill by Thursday 
night, the end of the fiscal year, September 30th.
    That issue, which is going to require extensive debate, 
would preclude our effort to do that. So I talked to the 
Majority Leader, Senator Lott, who says that if we take it out 
and avoid the debate now, we will be able to have extensive 
hearings and have that debate on a freestanding bill next 
February. I do not like that, but it is the best that can be 
done under these circumstances. So that all of our efforts are 
being trained on this issue.
    I have been asked to announce that the National Institute 
of Neurological Disorders and Stroke plans to support eight new 
Parkinson's Disease Centers of Excellence in fiscal year 1999, 
raising to 11 the number of funded Parkinson's centers, 
averaging about $1.3 million each, and that the NIH has 
committed a total of $73 million to Parkinson's Disease 
research for excellence, authorized by the Morris K. Udall 
Parkinson's Disease Research Act, which we included in our 
appropriations bill in fiscal year 1998.
    Now, I am delighted to defer to my colleague, Senator 
Cochran.


               opening statement of senator thad cochran


    Senator Cochran. Thank you very much, Mr. Chairman.
    Let me thank you, first of all, for conducting the hearing, 
convening the hearing, with this outstanding panel of 
witnesses. We have an opportunity and an obligation. The 
opportunity is to achieve a cure of Parkinson's Disease. And we 
are told by experts that this is the most curable of the 
neurodegenerative diseases.
    We have an obligation to fund this at a level as high as 
possible. The authorized level is our target. The Morris K. 
Udall Act creates that target. It emphasized the commitment of 
the Congress, when that Act was passed, to find a cure to 
improve the quality of life of those who suffer from 
Parkinson's Disease. We intend to carry out that mandate and 
that obligation.
    We also have the example of Morris K. Udall, whom the 
chairman and I knew very well personally, and others from the 
Congress who suffered from this disease. Former Senator, the 
late Millward Simpson, of Wyoming, the father of Alan Simpson, 
our distinguished colleague, who is Assistant Leader of the 
Senate, was a victim of Parkinson's Disease.
    In my State of Mississippi, one of my best friends, Noah 
Swett, a circuit judge, who was known around the country for 
his wit and wisdom, was also a victim of Parkinson's. There are 
many others. Celebrities like Michael J. Fox, members of 
Congress, judges, and many, many people throughout our country, 
whose names are not that well-known but who are just as 
important and should be just as important to this Congress.
    So we are hopeful that this hearing will serve as a 
catalyst to give us information that can move this process 
along more rapidly. We thank you all for being here, and 
particularly for the medical researchers and physicians who are 
working so hard to make this dream come true.
    Senator Specter. Thank you very much, Senator Cochran.
    We now proceed to our first witness. He is the 
distinguished Director of the National Institute of 
Neurological Disorders and Stroke at NIH. Dr. Gerald Fischbach 
has been there since July of 1998. Before that, Dr. Fischbach 
was Chairman of the Neurobiology Departments at Washington 
University, Harvard Medical School and Massachusetts General 
Hospital. He is past President of the Society for Neuroscience 
and a member of the National Academy of Sciences.
STATEMENT OF GERALD D. FISCHBACH, M.D., DIRECTOR, 
            NATIONAL INSTITUTE OF NEUROLOGICAL 
            DISORDERS AND STROKE, NATIONAL INSTITUES OF 
            HEALTH, DEPARTMENT OF HEALTH AND HUMAN 
            SERVICES
    Senator Specter. Welcome, Dr. Fischbach. Thank you for your 
very productive work in this very important field. The floor is 
yours. We are going to set the clock at 5 minutes for each 
witness, which is our custom, to leave us the maximum amount of 
time for dialogue.
    Dr. Fischbach.
    Dr. Fischbach. Thank you, Senator Specter, for having this 
hearing. Thank you, Senator Cochran, for support of the 
hearing, as well. I wanted to thank Senator Harkin, as well, 
for your steadfast and wonderful support of biomedical science 
and the NIH budget.
    I also want to acknowledge the many hundreds, actually, of 
physicians and scientists who have brought us to this point 
where we can speak optimistically about halting a 
neurodegenerative disorder like Parkinson's Disease, and where 
there is hope for many neurodegenerative disorders of all 
sorts--childhood, adult and in the aging population.
    I want to start and give you a 30-second primer on 
Parkinson's Disease. This is a disorder that begins with a 
small group of cells deep in the brain. These cells make a 
neurotransmitter called dopamine. When these cells are lost, 
and dopamine is lost, the powerful action that dopamine exerts 
on brain circuits that control movement is lost. These circuits 
are inhibited, they are locked up, and smooth, coordinated 
movements suffer because of that.
    In the past, Parkinson's Disease has been called a 
progressive disorder. I want to tell you some reasons why I 
personally am optimistic that we can change that. There is a 
chance to make real inroads in halting the steady progress of 
dopamine loss and the symptoms of Parkinson's Disease.
    My reasons can be boiled down to three. One, we know a lot 
about these dopamine neurons. Two, we know how they die. Three, 
we have the wonderful appearance on the scene of stem cell 
biology, which offers a type of promise that is really 
unprecedented in the past 25 years of biomedical research.
    These neurons, we know where they live. We know exactly 
where they are located. We know how they function. We know the 
circuits in which they are a part. This is the result of years 
of intensive study in animal models, subhuman primates and 
lower species.
    We know the circuits well enough so that, using modern 
techniques of imaging and precise microelectrode recording, we 
can ablate parts of the circuit that are uncontrolled, that are 
firing inappropriately, which lead to inhibition of movement. 
Many of you have heard of the success, the partial success--
hopefully the increasing success--of pallidotomy, to remove a 
region of the brain which is no longer controlled by dopamine.
    There has been another really important advance, which is 
to insert electrodes deep in the brain and stimulate parts of 
the circuit that are deficient in Parkinson's Disease. This 
technique of deep brain stimulation may revolutionize the 
therapy of Parkinson's Disease. It says that the circuits are 
still there, even though the dopamine neurons have degenerated. 
They are there to be reawakened and to function once again. So 
I personally have great hope that NINDS, in collaboration with 
the VA and other institutes, is going to make a major effort in 
studies of deep brain stimulation.
    Perhaps the most exciting advent is the appearance of stem 
cells on the scene. These are cells derived from the embryo, 
the fetus or the adult, which can proliferate, renew themselves 
and, on cue, can be made to differentiate into the cell that is 
needed. In the case of Parkinson's Disease, it has already been 
possible in animal models to place stem cells in the region of 
damage and to encourage them to produce dopamine and, 
remarkably, to cure, in these animal models, the movement 
disorder that is triggered by one of several experimental 
procedures.
    We also know how these dopamine cells die. It turns out 
that nerve cells die in very few ways. There is a limited 
number of programs of cell death. It is a type of cell suicide. 
They do not die passively, but cells have to activate a suicide 
program. We know a lot about that program. It is activation of 
a cascade of enzymes, each one of which offers novel 
therapeutic targets.
    Here is where Parkinson's Disease will benefit a number of 
other neurodegenerative disorders and will in turn benefit from 
them. Because it seems that cells die in Alzheimer's Disease 
and in ALS and in Huntington's Disease by exactly the same 
mechanism. Indeed, cells die in disorders that you would not 
ordinarily consider neurodegenerative, such as epilepsy, or 
depression, or any one of a number of childhood disorders. Once 
we understand this cascade better, perhaps, through studies of 
Parkinson's Disease, we will shed light on all of these 
disorders.
    There are an enormous number of needs. I do not want to be 
overly optimistic. We must be able to detect this disease much 
earlier than we do. We must understand the environmental 
factors and the genetic factors that predispose people to the 
disease. We need better animal models. We need better ways of 
delivering drugs to the brain. We need more knowledge of stem 
cell biology.
    Now, much of this will be addressed through the Udall 
Centers that Senator Specter mentioned. I suspect that, 
altogether, we will bring about 200 new investigators into the 
field--counting students, junior faculty and the senior staff 
involved. If you have a chance to look through the list of 
projects in those centers, they touch on all the vital needs in 
Parkinson's Disease.
    This path will be hard. It is going to take great effort by 
cooperation among all of the National Institutes of Health, 
cooperation with other agencies in the government, and 
especially cooperation hopefully through a public/private 
partnership with the very powerful and very wise advocacy 
groups for the patients.


                           prepared statement


    We have to be targeted and we have to be broad. We have to 
allow for serendipity. Not everything is known. We must fund 
research that may have bearing on Parkinson's Disease in a very 
immediate and direct way. I agree with Louis Pasteur, who said 
that chance favors the prepared mind. Our job at the NIH is to 
prepare us constantly and as well as we possibly can.
    Thanks.
    Senator Specter. Thank you very much, Dr. Fischbach.
    [The statement follows:]
            Prepared Statement of Gerald D. Fischbach, M.D.
    Mr. Chairman and members of the committee, I am pleased to tell you 
what NIH is doing to reduce the burden of Parkinson's disease. I want 
to convey my enthusiasm and optimism. I also want to emphasize that the 
task before us, conquering Parkinson's disease, will not be easy. The 
problems ahead will challenge the insight and ingenuity of scientists 
and physicians throughout the country and require coordinated effort by 
several NIH Institutes working closely with private Parkinson's groups. 
Finding a cure for Parkinson's is not like sending a man to the moon or 
making the atom bomb, where a resolute effort to apply what is known 
produced success. We still need to learn a great deal before we can 
stop this disease, but I am encouraged that the pace of discovery is 
increasing each year, and that we are on the right track.
    Parkinson's disease is a devastating, complex disease. Starkly put, 
Parkinson's destroys the ability to control movement. It begins with 
tremor and difficulty in initiating voluntary movements, and it 
progresses relentlessly, with a broad spectrum of symptoms, including 
depression and dementia in some patients. Nevertheless, there are 
several reasons for hope.
  --At first, the degeneration of nerve cells is confined to one region 
        of the brain and one type of nerve cell. These are nerve cells 
        that normally transmit messages to other cells by releasing a 
        chemical called dopamine. We are rapidly learning, down to the 
        level of single molecules, how cells make dopamine and respond 
        to it. Therefore the target early in disease is clear.
  --A second reason for optimism is the discovery that nerve cells 
        often follow a ``final common path'' to degeneration in 
        Parkinson's disease and in many other disorders. Apoptosis, 
        this death program, is often called ``cell suicide'' because 
        cells participate in their own destruction by activating a 
        cascade of enzymes that disrupt the integrity of genes and 
        normal cell metabolism. Each step in the cascade offers new 
        therapeutic targets to halt the progression.
  --We have new insights about what damages nerve cells provoking the 
        cell death pathway. Mechanisms such as free radical damage, 
        malfunction of mitochondria (the cells' energy factories), 
        ``excitotoxicity'' from excessive release of neurotransmitters, 
        abnormal protein aggregates, and sudden elevations of calcium 
        inside cells have been implicated. Again, each event offers 
        opportunities to slow the damage caused by disease.
  --Levodopa, when first introduced, seemed to be a miracle drug 
        liberating Parkinson's patients from immobility. This drug 
        helps replenish the brain's diminishing supply of dopamine. 
        Unfortunately the effects of levodopa are not sufficiently 
        lasting, side effects can be serious, and, most importantly, 
        levodopa cannot halt the underlying death of nerve cells. It is 
        encouraging that as we learn more about dopamine and other 
        neurotransmitters in the brain, we are learning how to prolong 
        and enhance the effects of levodopa and develop new drugs.
  --Neurotrophic factors, an entirely new class of therapeutic drugs, 
        were identified as natural brain chemicals that promote the 
        growth and survival of nerve cells in the development of the 
        nervous system. We are now learning how neurotrophic factors 
        can be used to protect against neurodegeneration in adult 
        brains, with promising results in animal models of Parkinson's 
        disease.
  --Years of analysis of the brain circuits that control movement are 
        leading to dramatic advances in surgical repair of Parkinson's 
        disease. Pallidotomy is a surgical procedure designed to 
        rebalance the normal interplay of brain circuits that initiate 
        and restrain voluntary movement. The procedure is now carried 
        out with exquisite precision guided by advanced brain imaging 
        and microelectrode recordings from single brain cells. An 
        astounding new technology, chronic brain stimulation, involve 
        electrodes implanted deep in the brain. Beyond relief of 
        symptoms, chronic brain stimulation may even slow the 
        progression of the disease. We must pursue this possibility and 
        determine the long term consequences of these surgical 
        procedures.
  --Stem cells offer an entirely new therapeutic approach. Cell 
        implantation offers hope for actually replacing nerve cells 
        lost in Parkinson's and many other diseases. Clinical trials of 
        fetal tissue transplantation, still underway, have developed 
        methods for implanting cells into the brain, and demonstrated 
        the viability of the concept and promising results for at least 
        some patients. Now, neural stem cells, cells that have the 
        capacity to renew themselves indefinitely and to specialize to 
        form all cell types of the brain, offer a potentially unlimited 
        supply of dopamine cells. Stem cell therapy has already 
        produced dramatic success in animal models of Parkinson's and 
        other neurological diseases.
    Beyond the impact on Parkinson's disease itself, Parkinson's 
research will certainly lead to insights about many other diseases in 
which nerve cells die. Neurodegeneration--the death of nerve cells--is 
a ubiquitous problem. Most notable are the classic chronic 
neurodegenerative diseases such as Alzheimer's, Huntington's, and ALS. 
Many devastating neurodegenerative disorders also attack the brain of 
infants and children. Nerve cell death is critical in stroke, brain and 
spinal cord injury, and in epilepsy. Alchohol and drug abuse can cause 
neurodegeneration. Even severe depression, long thought to be related 
to a chemical imbalance in the brain, is associated with degeneration 
of nerve cells. The same destructive processes come into play and 
provoke the same cell death programs. Advances in Parkinson's disease 
will shed light on all of these disorders, and research on these other 
disorders may also advance understanding of Parkinson's disease.
    Let me now focus on a a few critical issues that must be resolved 
as we move forward.
  --Early detection of Parkinson's disease is absolutely crucial. More 
        that 75 percent of the dopamine cells have already died before 
        the first symptoms are detected. Preventing cells from dying in 
        the first place is the best hope for effective medical therapy. 
        Extensive efforts to develop early detection of 
        neurodegenerative diseases, though brain imaging and other 
        approaches, are a major thrust of programs at the NINDS, the 
        National Institute of Aging, and other components of NIH.
  --Thorough epidemiological and environmental studies are essential to 
        identify factors that set off the disease process. The National 
        Institute of Environmental Health Sciences is leading a major 
        NIH initiative to detect risk factors in the environment that 
        may influence the onset or progression of neurodegeneration in 
        Parkinson's disease.
  --We must also follow the genetic trail. Though most people do not 
        inherit Parkinson's disease, we can learn a great deal by 
        studying the rare families that carry a Parkinson's disease 
        gene. The first gene defect that causes Parkinson's disease, a 
        mutation in the protein synculein, was identified just three 
        years ago, and two more Parkinson's genes have since been 
        discovered. We already have clues that synuclein plays a role 
        not only in familial Parkinson's disease but also in the more 
        common non-inherited form. Synuclein may also play an important 
        role in the development of Alzheimer's disease, again 
        demonstrating the close ties among brain diseases.
  --The advent of new surgical therapies, like deep brain stimulation, 
        reinforces the importance of better understanding the brain 
        circuits that control movement. If we understand the circuits 
        perhaps we can reactivate them. Likewise, the more we are 
        learning about dopamine and other neurotransmitters the greater 
        the options to restore motor control to Parkinson's patients.
  --We are expanding our efforts in experimental therapeutics to keep 
        the pipeline full of potential new treatments. Finding better 
        animal models that truly mimic the slow neurodegeneration of 
        human Parkinson's disease is critical to expediently move 
        candidate therapies to human testing. This is one area where 
        genetic technology may be essential. Other technologies, like 
        high-throughput drug screening and gene arrays, promise to 
        greatly expedite the search for cures and must be made 
        accessible to any researcher with a good idea.
  --We need to develop methods to deliver drugs to the brain. Many 
        potentially therapeutic substances, such as neurotrophic 
        factors, do not cross the blood-brain barrier which excludes 
        substances from the general circulation.
  --For no area of medicine is the promise of stem cells greater than 
        for treating diseases of the human brain. We must learn how to 
        control the survival, proliferation, and specialization of 
        neural stem cells so we can repair the damage wrought by 
        Parkinson's disease. The recent startling demonstration that 
        even 60 year old human brains harbor stem cells presents the 
        possibility that we may someday learn how to empower the 
        Parkinson's ravaged brain to repair itself, if only we can 
        learn the control signals.
    In addition to the National Institute of Neurological Disorders and 
Stroke (NINDS), the National Institute of Aging, the National Institute 
of Mental Health, the National Institute of Envronmental Health 
Sciences, the National Human Genome Research Institute, the National 
Institute on Drug Abuse, the National Institute of Diabetes and 
Digestive and Kidney Diseases, and the National Center for Research 
Resources all support research on Parkinson's disease. Led by NINDS, 
the Parkinson's Disease Coordinating Committee has undertaken several 
initiatives, including a major workshop in 1995 that identified new 
directions for Parkinson's disease research and a cooperative program 
announcement on ``Mechanisms of Cell Death and Injury in 
Neurodegenerative Disorders.''
    Finally, as you have just heard, the NINDS has now funded 11 Morris 
K. Udall Parkinson's Disease Research Centers of Excellence. These 
centers will play a key role in coordinating and carrying out research 
efforts in Parkinson's disease. The centers will explore many aspects 
of Parkinson's disease, from basic science to clinical applications. 
They will play a particularly important role in bringing scientists and 
clinicians together to move research advance to therapy that can 
benefit patients.
    We believe that current extensive efforts by the NIH in Parkinson's 
research are justified by the extraordinary opportunities that 
neuroscience research now presents for fighting this disease and the 
implications for other diseases. Because we know so much about 
Parkinson's, this disease can lead the way in confronting the broader 
problem of neurodegeneration. What we learn about the broader problem 
of neurodegeneration will also help in the fight against Parkinson's 
disease. We have an extraordinary opportunity and a great challenge. 
Neuroscience has arrived at a state when we can contemplate translating 
fundamental discoveries into a cure for seemingly inexorable 
neurodegenerative disorders. Thank you Mr. Chairman. I would be happy 
to answer any questions.

                        Staying within the caps

    Senator Specter. We are facing a controversial 
appropriations bill because of the Balanced Budget Act. We are 
determined to stay within the caps. We have projected a budget 
which does that, but has substantial forward funding, which is 
a practice that the Congress has engaged in for many, many 
years.
    If you would turn the green light on, we are going to take 
5-minute rounds for everyone, including me.
    The budget for our subcommittee is at $91.7 billion, which 
is $4 billion over last year. That is largely accounted for by 
the $2 billion increase in NIH funding and the increases in 
education funding, again, where there is a consensus. We are 
going to have a problem on the floor of the Senate. We are 
going to have a bigger problem in conference with the House. We 
have to run through the raindrops in a hurricane to find 
something that is acceptable to the Congress and to the 
President to get this bill signed.
    I think it is very important for America that we get a bill 
signed on appropriations on the two big priorities, health care 
and education. I think the American people are really sick and 
tired of the kind of partisan bickering that has come out of 
Washington for so long.
    But a big help in persuading the Congress to accept this $2 
billion increase is to be as tangible as we can. Last year, we 
increased the funding by almost $120 million, coming up to $920 
million. This year, the projection will make an increase again 
of that magnitude--$120 million.
    Now, what tangibly can you say will be accomplished on 
Parkinson's--or let me articulate the question a little 
differently--what tangibly was accomplished last year by this 
appropriation of $920 million, almost a billion dollars, and 
the increase of $120 million last year?
    Dr. Fischbach. Well, in the area of Parkinson's Disease, we 
were able to fully fund the 11 centers. This all happened 
within a year--two review cycles. That was an extraordinary 
effort and a wonderful accomplishment.
    Senator Specter. I do not want to cut you short, but the 
time is limited. Fine, for last year. Now, how about next year? 
If you get this $120 million more, if we push you up to a 
billion, 20 million dollars, what will that increase in funding 
enable you to do?
    Dr. Fischbach. I think it will enable us to pursue these 
efforts at the level we funded them. It will allow us to reach 
out to all neurodegenerative disorders and use what we learn 
there to focus on Parkinson's Disease. It will allow us to 
undertake expensive clinical trials, which we could not do now. 
It will allow us, I believe, to begin to contemplate a national 
effort, an epidemiologic effort, in cognitive health, which 
will identify early risk factors for the disease.
    Senator Specter. The subcommittee has heard testimony 
earlier that I referred to briefly in my opening statement, 
that it is realistic to conquer Parkinson's within 5 years. Now 
that was about a year ago, so I guess it is 4 years now. A two-
part question. Would you concur that we are that close to 
solving Parkinson's? Second, what could we do to even make it a 
shorter time interval to conquer Parkinson's?
    Dr. Fischbach. I concur that we are close to solving--and I 
mean the word ``solving''--Parkinson's Disease. I hesitate to 
put an actual year number on it. I think, with all the 
intensive effort, with a little bit of skill and luck, 5 to 10 
years is not unrealistic. We will do everything possible to 
reduce that below 5 years. I would not rule that out.
    Senator Specter. Well, will more money enable you to do it 
in less than 5 years?
    Dr. Fischbach. I believe that we are doing a great deal 
now, in terms of clinical trials. We have to be concerned with 
the sanction and the ability of the community to undertake 
these efforts.
    The advent of stem cells, the possibility of applying them 
aggressively in a variety of disorders will only be limited by 
the resources around the country.
    Senator Specter. Will the elimination of the restriction on 
stem cells be a significant factor in expediting solving 
Parkinson's?
    Dr. Fischbach. Yes, it certainly will. It absolutely will. 
If NIH-funded investigators can use stem cells, understand how 
to make them form dopamine neurons, ensure their survival in 
the brain, enhance the placement of those cells, it will 
certainly lead to a more rapid solution of those problems.
    Senator Specter. Thank you very much, Dr. Fischbach.
    Senator Cochran.
    Senator Cochran. Thank you, Mr. Chairman.
    Doctor, let me, first of all, thank you for your hard work 
in the effort to develop information, a full range of 
information, that will equip medical doctors and scientists to 
be more successful in the future in coming up with a cure and 
improving the quality of life for people who have Parkinson's.
    Dr. Harold Varmus, who is not here today, the Director of 
NIH, is out of the country. We want the record to show that we 
appreciate his attention to this subject and his efforts to 
emphasize and improve the response that NIH is making to this 
challenge.
    For some time, we were a little concerned, and I want to 
ask you about this, about how the score-keeping works at NIH. 
We appropriate money here and we identify areas of priority and 
concern, where we think emphasis ought to be placed by NIH, and 
then we are given a report that so much money has been spent in 
Parkinson's research or cancer research or some other disease 
research.
    Some worry that there is a lot of overlapping, and that 
while we are trying to target funds for Parkinson's, we are 
seeing funds that are described as being used for Parkinson's, 
but may not be as specific to the disease as some in Congress 
would like. What is the response that you could give to those 
who worry about whether or not the score-keeping is accurate?
    Dr. Fischbach. My response is that grading grants, rating 
them as to whether they are directly or indirectly relevant to 
a particular disorder is not an exact science. I am anxious 
personally to make this as precise as possible, and would like 
to work with all experts who have opinions about it. There are 
different opinions about it.
    There was a concern raised 2 years ago about the relevance 
of the funds to Parkinson's. I personally, with senior members 
of the staff, reviewed our grants and tried to categorize them 
better than a slightly, admittedly, outdated system at the NIH. 
So I think we are on the right track. I look forward to working 
with all classes of opinion about the relevance of the grants. 
I think we can come as close as possible.
    I am concerned about closing down the window of opportunity 
too narrowly. I think there are unknowns in Parkinson's Disease 
research. They may well be found in studies that are not 
directly related to Parkinson's Disease. This is all a matter 
of judgment that I think the community has to come to some 
consensus about and inform Congress about.
    If you trace the history of discoveries, really fundamental 
discoveries in Parkinson's, not many of them were because the 
research was directly and immediately focused on Parkinson's 
Disease. I think we have arrived at the time in our history 
when we can focus money directly on this disease, given the 
advances we have in hand. But I think we need enough money to 
do both, to do the direct and do the relevant research, as 
well.
    Senator Cochran. When Senator Hatfield was winding up his 
service here in the Senate, and particularly as chairman of the 
full committee on appropriations, he convened a series of 
hearings looking into how we could better use appropriated 
dollars to support the work that is done by the medical 
community and the research community in coming up with cures 
for illnesses generally. One of the things that we found out in 
those hearings was that fewer and fewer medical doctors and 
research scientists were going into the field.
    What can we do here to encourage those who are the best and 
the brightest and have the capability of really finding the 
answers we need to solving these problems to devote a career to 
medical research, so that we will have the kind of talent and 
resource pool we need to carry out the work that you and others 
like you are doing?
    Dr. Fischbach. I think that is something our Institute and 
our National Council struggle with every day. It is alarming 
that the number of talented young people going into biomedical 
research is declining. There is some hope in the last year that 
it may be on the upswing. But among the things we have thought 
about is to shorten the training period. We need to get people 
into the scientific work force before their late thirties, to 
increase stipends, to reduce medical school debt, and to make 
this type of career attractive by providing funds for them to 
continue their career.
    Some would say it is just not acceptable for someone in 
their forties or fifties to have only a 25 percent chance of 
renewing their grant when they are doing good work. I think all 
the arrows are pointed in the right direction, and that adds to 
my optimism.
    Senator Cochran. Thank you.
    Senator Specter. Thank you very much, Senator Cochran.
    One final question, Dr. Fischbach, before moving on to the 
next panel. That is that the Parkinson's Action Network has 
been concerned as to the utilization of funds. Senator Cochran 
touched on this. But let me put into the record their specific 
concerns, so that that will be before the public, and your 
specific response.
    The assessment by the Parkinson's Action Network 
researchers found that 54 percent of the grant portfolio, they 
say, was not Parkinson's focused, and that 26 percent of the 
funding was spent on ``research completely unrelated to 
Parkinson's.'' I think it is important for the record that you 
respond to that.
    Dr. Fischbach. We have studied their report. We actually 
were shown the figures of the panel of 15 judges. We would be 
eager to work with that panel to try and rectify the 
disparities.
    I would note that a significant fraction of that panel--I 
think it was 6 or 7 out of the 15--essentially agreed with our 
scoring. So there were two populations of judges on that panel, 
those who agreed were within 5 to 10 percent--some within 2 
percent--of our figures, but a significant percentage, the 
remaining eight or nine judges, did not feel that our research 
was focused on or relevant to Parkinson's Disease.
    My only response is that we are trying to reach some common 
ground. We offer and would welcome discussion, with no holds 
barred and with no animosity, with those judges to try and 
reach a more rational agreement about what is and is not 
meritorious as directed to Parkinson's Disease.
    Senator Specter. Well, I thank you for that response, and I 
commend you for your willingness to sit down and work with them 
to try to come to common ground. One of the difficulties that 
this subcommittee has and that I do personally is the 
tremendous number of requests from every organization--and 
there are many--in a variety of fields, wanting a bigger share, 
and many very unusual ailments.
    So that people are understandably desperate to find a cure 
to their problem. That is one of the motivating factors that I 
find in trying to give you extra funds, so that you can tackle 
a broader range of problems. The allocation of funding is 
extremely difficult. But that is essentially a professional 
matter which the Congress leaves to the experts at the National 
Institutes of Health, as you see where the money can be most 
productively used, considering a wide variety of factors.
    But I think it is very important, when people come to this 
subcommittee or to you, that we listen to them and try to 
accommodate their interests to the extent we can. If there is a 
challenge as to how the funds are being used, to try to analyze 
it and try to come to common ground.
    Dr. Fischbach. We will.
    Senator Specter. OK, thank you very much, Dr. Fischbach.
    We turn now to our second panel, Mr. Michael J. Fox, Mr. 
James Cordy, Dr. J. William Langston, and Ms. Joan Samuelson.
    If you, lady and gentlemen, would step forward, we will 
proceed with your testimony.
    We welcome you all here. Ms. Samuelson is president of the 
Parkinson's Action Network and has been very active in 
promoting funding. Dr. Langston is the president of the 
Parkinson's Institute and a renowned expert in the field. Mr. 
James Cordy--where is your hourglass, Jim? OK--has been an 
extraordinarily effective advocate in this field.
    As I noted earlier, we have with us today Mr. Michael J. 
Fox, a successful actor for many years. First, as Alex P. 
Keaton, on the television series ``Family Ties.'' You always 
work with a middle initial, do not you Mr. Fox? Later in many 
movies, including ``Back to the Future,'' and, most recently, 
on television again in the highly acclaimed ``Spin City.'' 
Michael was diagnosed with Parkinson's in 1991, at the age of 
30.
    He has become very, very active in Parkinson's advocacy. 
One of the facts of life is that when someone like Michael J. 
Fox steps forward, it very heavily personalizes the problem, 
focuses a lot of public attention on it, and has the public 
understanding of the need for doing whatever we can as a 
country to conquer this disease and many, many others. So we 
thank you for being here, Michael J. Fox, and look forward to 
your testimony.
    Again, we will put the lights on, for 5 minutes, on 
testimony.
    Mr. Fox, we are going to start with you.
STATEMENT OF MICHAEL J. FOX, ACTOR
    Mr. Fox. Mr. Chairman and members of the subcommittee, 
thank you for inviting me to testify today about the need for 
greater Federal investment in Parkinson's research.
    Some, or perhaps all, of you, most of you, are familiar 
with me from my work in film and television. What I wish to 
speak to you about today has little or nothing to do with 
celebrity save for this brief reference. When I first spoke 
publicly about my 8 years of experience as a person with 
Parkinson's, many were surprised, in part, because of my age. 
Although 30 percent of all Parkinson's patients are under 50, 
and 20 percent are under 40, and that number is growing.
    I had hidden my symptoms and struggles very well, through 
increasing amounts of medication, through surgery, and by 
employing the hundreds of little tricks and techniques a person 
with Parkinson's learns to mask his or her condition for as 
long as possible. While the changes in my life were profound 
and progress, I kept them to myself for a number of reasons--
fear, denial for sure, but I also felt that it was important 
for me to quietly just soldier on.
    When I did share my story, the response was overwhelming 
and deeply inspiring. I heard from thousands of Americans 
affected by Parkinson's, writing and calling to offer 
encouragement and to tell me of their experience. They spoke of 
pain, frustration, fear, and hope. Always hope.
    What I understood very clearly is that the time for quietly 
soldiering on is through. The war against Parkinson's is a 
winnable war, and I have resolved to play a role in that 
victory. What celebrity has given me is the opportunity to 
raise the visibility of Parkinson's Disease and focus attention 
on the desperate need for more research dollars. While I am 
able, for the time being, to continue doing what I love best, 
others are not so fortunate.
    These are doctors, teachers, policemen, nurses, and, as you 
had indicated earlier, legislators, and parents who are no 
longer able to work to provide for their families or to live 
out their dreams. The 1 million Americans living with 
Parkinson's want to beat this disease. So do the millions more 
Americans who have family members suffering from Parkinson's. 
But it will not happen until Congress adequately funds 
Parkinson's research.
    For many people with Parkinson's, managing their disease is 
a full-time job. It is a constant balancing act. Too little 
medicine causes tremors and stiffness. Too much medicine 
produces uncontrollable movement and slurring. And far too 
often, Parkinson's patients wait and wait--as I am right now--
for the medicines to kick in.
    New investigational therapies have helped some people like 
me control symptoms but, in the end, we all face the same 
reality--the medicine stops working. For people living with 
Parkinson's, the status quo is not good enough. As I began to 
understand what research might promise for the future, I became 
hopeful that I would not face the terrible suffering so many 
with Parkinson's endure. But I was shocked and frustrated to 
learn the amount of funding for Parkinson's research is so 
meager.
    Compared to the amount of Federal funding going to other 
diseases, research funding for Parkinson's lags far behind. In 
a country with a $15 billion investment in medical research, we 
can and must do better.
    At present, Parkinson's is inadequately funded, no matter 
how one cares to spend it. Meager funding means a continued 
lack of effective treatments, slower progress in understanding 
the cause of the disease, and little chance that a cure will 
come in time.
    I applaud the steps you are taking to fulfill the promise 
of the Udall Parkinson's Research Act. But, we must be clear, 
we are not there yet.
    If, however, an adequate investment is made, there is much 
to be hopeful for. We have a tremendous opportunity to close 
the gap for Parkinson's. We are learning more and more about 
this disease. The scientific community believes that with a 
significant investment into Parkinson's research, new 
discoveries and improved treatment strategies are close at 
hand. Many have called Parkinson's the most curable 
neurological disorder and the one expected to produce a 
breakthrough first.
    Scientists tell me that a cure is possible--some say even 
by the end of the next decade--if the research dollars match 
the research opportunity.
    Mr. Chairman, you and the members of the subcommittee have 
done so much to increase the investment in medical research in 
this country. I thank you for your vision. Most people do not 
know just how important this research is until they or someone 
in their family faces a serious illness. I know I did not.
    The Parkinson's community strongly supports your efforts to 
double medical research funding. At the same time, I implore 
you to do more for people with Parkinson's. Take up Parkinson's 
as if your life depended on it. Increase funding for 
Parkinson's research by $75 million over the current levels for 
the coming fiscal year. Make this a down payment for a fully 
funded Parkinson's research agenda. It will make Parkinson's 
nothing more than a footnote in medical textbooks.
    I would like to close on a personal note. Today you will 
hear from, or have already heard from, more than a few experts 
in the fields of science, bookkeeping and other areas. I am an 
expert on only one--what it is like to be a young man, husband 
and father, with Parkinson's Disease.
    With the help of daily medications and selective exertion, 
I can still perform my job, in my case, in a very public arena. 
I can still help out with the daily tasks and rituals involved 
in home life. But I do not kid myself--that will change. 
Physical and mental exhaustion will become more and more of a 
factor, as will increased rigidity, tremor and dyskinesia.

                           Prepared statement

    I can expect, in my forties, to face challenges most will 
not expect until their seventies or eighties, if ever. But with 
your help, and if we all do everything we can to eradicate this 
disease, in my fifties, I will be dancing at my children's 
weddings, and mine will be one of millions of happy stories.
    Thank you for your time and attention.
    Senator Specter. Thank you very much, Mr. Fox, for those 
very profound and moving words.
    [The statement follows:]
                  Prepared Statement of Michael J. Fox
    Mr. Chairman, Senator Harkin, and members of the Subcommittee--
thank you for inviting me to testify today about the need for a greater 
federal investment in Parkinson's research. I would like to thank you, 
in particular, for your tremendous leadership in the fight to double 
funding for the National Institutes of Health.
    Some, or perhaps most of you are familiar with me from 20 years of 
work in film and television. What I wish to speak to you about today 
has little or nothing to do with celebrity--save for this brief 
reference.
    When I first spoke publicly about my 8 years of experience as a 
person with Parkinson's, many were surprised, in part because of my age 
(although 30 percent of all Parkinson's patients are under 50, and 20 
percent are under 40, and that number is growing). I had hidden my 
symptoms and struggles very well, through increasing amounts of 
medication, through surgery, and by employing the hundreds of little 
tricks and techniques a person with Parkinson's learns to mask his or 
her condition for as long as possible.
    While the changes in my life were profound and progressive, I kept 
them to myself for a number of reasons: fear, denial for sure, but I 
also felt that it was important for me to just quietly ``soldier on.''
    When I did share my story, the response was overwhelming, humbling, 
and deeply inspiring. I heard from thousands of Americans affected by 
Parkinson's, writing and calling to offer encouragement and to tell me 
of their experience. They spoke of pain, frustration, fear and hope. 
Always hope.
    What I understood very clearly is that the time for quietly 
``soldiering on'' is through. The war against Parkinson's is a winnable 
war, and I am resolved to play a role in that victory.
    What celebrity has given me is the opportunity to raise the 
visibility of Parkinson's disease and focus more attention on the 
desperate need for more research dollars. While I am able, for the time 
being, to continue to do what I love best, others are not so fortunate. 
There are doctors, teachers, policemen, nurses and parents who are no 
longer able to work, to provide for their families, and live out their 
dreams.
    The one million Americans living with Parkinson's want to beat this 
disease. So do the millions more Americans who have family members 
suffering from Parkinson's. But it won't happen until Congress 
adequately funds Parkinson's research.
    For many people with Parkinson's, managing their disease is a full-
time job. It is a constant balancing act. Too little medicine causes 
tremors and stiffness. Too much medicine produces uncontrollable 
movement and slurring. And far too often, Parkinson's patients wait and 
wait for the medicines to ``kick-in.'' New investigational therapies 
have helped some people like me control my symptoms, but in the end, we 
all face the same reality: the medicines stop working.
    For people living with Parkinson's, the status quo isn't good 
enough.
    As I began to understand what research might promise for the 
future, I became hopeful I would not face the terrible suffering so 
many with Parkinson's endure. But I was shocked and frustrated to learn 
that the amount of funding for Parkinson's research is so meager. 
Compared with the amount of federal funding going to other diseases, 
research funding for Parkinson's lags far behind.
    In a country with a $15 billion investment in medical research we 
can and we must do better.
    At present, Parkinson's is inadequately funded, no matter how one 
cares to spin it. Meager funding means a continued lack of effective 
treatments, slow progress in understanding the cause of the disease, 
and little chance that a cure will come in time. I applaud the steps we 
are taking to fulfill the promise of the Udall Parkinson's Research 
Act, but we must be clear--we aren't there yet.
    If, however, an adequate investment is made, there is much to be 
hopeful for. We have a tremendous opportunity to close the gap for 
Parkinson's. We are learning more and more about this disease. The 
scientific community believes that with a significant investment in 
Parkinson's research, new discoveries and improved treatments 
strategies are close-at-hand. Many have called Parkinson's the most 
curable neurological disorder and the one expected to produce a 
breakthrough first. Scientists tell me that a cure is possible, some 
say even by the end of the next decade--if the research dollars match 
the research opportunity.
    Mr. Chairman, you and the members of the Subcommittee have done so 
much to increase the investment in medical research in this country. I 
thank you for your vision. Most people don't know just how important 
this research is until they or someone in their family faces a serious 
illness. I know I didn't.
    The Parkinson's community strongly supports your efforts to double 
medical research funding. At the same time, I implore you to do more 
for people with Parkinson's. Take up Parkinson's as if your life 
depended on it. Increase funding for Parkinson's research by $75 
million over current levels for the coming fiscal year. Make this a 
down payment for a fully funded Parkinson's research agenda that will 
make Parkinson's nothing more than a footnote in medical textbooks.
    I would like to close on a personal note. Today you will hear from, 
or have already heard from, more than a few experts, in the fields of 
science, book-keeping and other areas. I am an expert in only one--what 
it is like to be a young man, husband, and father with Parkinson's 
disease. With the help of daily medication and selective exertion, I 
can still perform my job, in my case in a very public arena. I can 
still help out with the daily tasks and rituals involved in home life. 
But I don't kid myself .  .  . that will change. Physical and mental 
exhaustion will become more and more of a factor, as will increased 
rigidity, tremor and dyskinesia. I can expect in my 40s to face 
challenges most wouldn't expect until their 70s or 80s--if ever. But 
with your help, if we all do everything we can to eradicate this 
disease, in my 50s I'll be dancing at my children's weddings. And mine 
will be just one of millions of happy stories.
    Thank you again for your time and attention.
STATEMENT OF JOAN I. SAMUELSON, PRESIDENT, PARKINSON'S 
            ACTION NETWORK
    Senator Specter. We turn now to Ms. Joan Samuelson, 
President of Parkinson's Action Network, an organization 
founded to support and encourage research and funding to 
produce an effective treatment and cure for the disease. She 
earned her degree at UCLA, an undergraduate and a law degree 
from the University of California at Berkeley, the founder of 
the Parkinson's Action Network, she has been President since 
1991.
    Thank you for your good work, Ms. Samuelson, and the floor 
is yours.
    Ms. Samuelson. Thank you very much, Chairman Specter. 
Thanks so much to you and to Senator Cochran for your 
leadership on this issue. Thank you for your determination to 
add the additional $2 billion to the NIH budget, to enable us 
to have adequate funding without robbing Peter to pay Paul. 
Thank you so much for this hearing, for this opportunity to be 
here today.
    Senator Cochran, thank you so much for your leadership on 
that. We just deeply appreciate it.
    When I was thinking this morning about how to use 5 minutes 
to try to talk about how desperately we need adequate funding 
for Parkinson's research, I realized that what I should do is 
try to have you, as best you can, sit in our shoes for those 5 
minutes, because it is so confoundedly hard to describe what 
our life is like. So that is what I am going to try to do. It 
is about waiting for a rescue, basically.
    I am 13 years post my Parkinson's diagnosis--a day I will 
never forget. At this point, the drug we all take, l-dopa 
Cinamet, just does not work as well as it did at the beginning. 
Because my cells have deteriorated to the point where they 
cannot work well enough, and there is not enough there to work 
with.
    I am sure Dr. Fischbach talked about that a bit, and Dr. 
Langston will talk about that problem and all the things that 
they have available to try to solve it. But my reality is that 
this morning when I woke up, I reached over and popped that 
pill. It took an hour for me to be able to move enough to get 
out of bed. That is the frozen body that is the reality that I 
live with part of the time now. That is one of those moments 
when all I am doing really is waiting for a rescue. I am 
waiting for that medication to kick in.
    At first, the medication does provide that rescue. Boy, it 
is the most amazing miracle when it does. Because I would go 
from being in that frozen body to being able to come here and 
talk to you today and power myself on my own two feet and 
function in the world and be an independent citizen, with 
dignity. It is just that pill that did it. It is a miracle. But 
then it stops working.
    In 1991, Anne Udall, one of Mo Udall's kids, took me to 
visit him at the Veterans Long-Term Care Facility. He had 
recently retired from the Congress because of his advanced 
Parkinson's and a fall that he had had as a consequence of 
Parkinson's, which is a frequent occurrence. Anne decided that 
we should go meet him. She blurted out something in the cab 
that I am sure she still regrets, which is that she said, you 
know, I guess I am taking you to see your future. Indeed she 
was.
    He had entered the next stage that I have not entered yet 
and that I pray I will never reach, which is the departure from 
active society. At that point, he was still able to sit up in a 
wheelchair, and I could understand a few words that he was able 
to say, although with great difficulty, but he had departed 
from the society that I now still get to function in with 
dignity.
    Two years later, she took me back to see him again. At that 
point, he was lying in his bed, unable to move, unable to 
speak. That was what I see as the living death which is the 
next stage, which is then followed by death itself. So those 
are three stages that I look forward to with great fear and 
desperation and want to have delayed--to have a rescue from as 
soon as I can.
    What the scientists tell us, and Dr. Langston will talk 
about this more, is that they are ready to deliver that rescue. 
Attached to my testimony is a copy of a research agenda that we 
are collaborating with a wide variety of scientists around the 
country to show the Congress the clarity of their vision. Dr. 
Fischbach talked about needing a targeted and broad research 
agenda. That is in fact what this is. It talks about prevention 
and it talks about brain repair, which is the thrilling array 
of therapies, including stem cells, that the scientists are 
very close to being able to provide.
    It is really astonishing to me their willingness to be so 
precise about these timetables, to talk about a cure within 5 
to 10 years, to talk about effective therapies in fact with 5 
and even sooner. But what they tell us every time they talk 
about it is how little money they have to do it. We are 
thrilled to have these additional centers, pursuant to the 
Udall Act. But what it really is is $8 million to $10 million. 
That is a tiny little step toward what they have identified 
conservatively now as $240 million.
    So, to simply fully fund the Udall Act, which is not done 
yet, is really just a first step. It is a very important step. 
That is what we are asking for this year, for the $75 million, 
$50 million of which would could go to the Neurology Institute 
and $25 million to the Environmental Health Sciences Institute, 
of which Dr. Ken Olden, who is here, is the Director.
    They want to get started. They want to work on this. But it 
is really in the hands of the Congress to provide them with the 
weaponry to make this happen. It is really not the fault of the 
NIH that they are not able. Because we do not want them to rob 
Peter to pay Paul. We want them to be able to focus on this 
aggressively without taking money from anything else.
    But it is really in the hands of the Congress to make that 
decision. So we have tried to get the rescue from the 
medication, and then that stops working. We want the scientists 
to deliver it. But, honestly, we really feel that the rescue is 
in the hands of the Congress now. Because the money has to get 
to the scientists so that they can actually deliver it to us.
    Michael talked about his vision. Every one of us has our 
own. This hearing room is full of people, whether they have 
Parkinson's themselves or they have a loved one that lives with 
it as they do every day. Every one of us has our own personal 
vision of how we are going to get back these precious freedoms 
of movement and speech and dignity that we all so desperately 
want to have our entire lives.
    My personal vision centers on my family. I am lucky to have 
my four nieces here today, who are sitting with me. I have to 
say, without bias, they are among the most adorable and 
wonderful people on the face of the Earth. I thank them for 
being here. I thank my brother and sister for bringing them.

                           Prepared statement

    My personal vision is that I live a normal life and that I 
am able, as they grow up, to continue to be their buddy and 
their role model, as I feel I am today. I do not want that 
taken from me. I need that rescue. We need the help of the 
Congress to deliver it.
    Thank you.
    [The statement follows:]
                Prepared Statement of Joan I. Samuelson
    Thank you for this opportunity to testify before you on Parkinson's 
research funding. We are most grateful to Chairman Specter and the 
other members of the Committee, in particular Senator Cochran, for 
making this day possible. The Parkinson's Action Network was created in 
1991 to give voice to a community that has been largely invisible, and 
to increase funding for Parkinson's research in an effort to speed 
research, deliver breakthroughs and cure this dreadful disease.
    I am one of a million Americans who suffer with Parkinson's. 
Parkinson's is a devastating progressive neurological disorder that 
makes it difficult to walk, causes uncontrollable tremors, and in its 
final states robs individuals of the ability to speak or move. It is 
caused by the degeneration of brain cells that produce dopamine, a 
neurochemical controlling motor function.
    After 13 years with a Parkinson's diagnosis, I am at a crossroads--
physically and medically. Despite all my efforts and the best medicine 
available, I have moments every day when I live in a frozen body, 
waiting for my remaining dopamine cells to receive the drugs and let me 
move. I watch, with frustration and fear, my ability to speak and 
swallow begin to slip. I have already been forced to give up so much 
that I love: my law practice, running, hiking--and some of my dreams. 
The hardest thing is being unable to do all the automatic unappreciated 
routines like getting out of bed in the morning, turning on the light, 
dressing. Every day these activities get more and more difficult--and 
some days they are almost impossible.
    Without a medical rescue, I know what is coming: the retreat from 
active, independent life; then the living death of the so-called ``end 
stage.''
    Eight years ago in 1991, Anne Udall, whom I met in my first years 
as an advocate, took me to meet her father, Congressman Mo Udall. At 
that time he was 15 years post diagnosis, retired from Congress because 
of his advanced Parkinson's, and living at the Veterans' Long Term Care 
Facility. On the way there, Anne said almost without thinking, ``I 
guess I'm taking you to see your future.'' And she was.
    At that time, Congressman Udall was still able to sit up in a 
wheelchair and could speak somewhat, although it was very difficult to 
understand. When I returned just two years later he was completely 
bedridden and frozen, but in all likelihood his mind was entirely 
intact. We'll never know. I'm so much closer to that fate than when I 
first started advocating for a greater investment in Parkinson's 
research.
    Losing independence and freedom is what scares me and those in the 
Parkinson's community the most. Perhaps you can understand my 
increasing frustration; and why I am not content to wait patiently for 
a cure--not when I know more can be done.
    I have been meeting with Parkinson's scientists from across the 
country--dozens of eminent researchers--working to shape a research 
agenda and budget that would match the promise in finding more 
effective treatments and getting us closer to a cure. Wherever I go and 
to whomever I speak, I have found an almost unanimous consensus among 
the experts that we are close to unraveling this disorder. But they all 
say they could be working much harder.
    The real problem is not the science--it's the meager (and 
unacceptably small) size of the federal commitment to eradicate 
Parkinson's.
    The attached research agenda is the first attempt to summarize the 
serveral areas showing great promise for a rapid return on a research 
investment. The estimated annual cost for this focused research 
campaign is conservatively estimated by the neuroscientists at $185 
million--almost double the initial Udall Act authorization.
    Passing the Udall Parkinson's Research Act in 1997 was a great 
achievement, but the promise of that Act has yet to be realized. The 
law authorizes the National Institutes of Health to spend at least $100 
million for focused Parkinson's research. Small increases to 
Parkinson's research have been made, and several additional Parkinson's 
research centers are promised. We're glad to see that. But the new 
spending is a tiny effort in contrast to what the scientists could be 
doing.
    Over the last eight years, we have tried with little success to 
significantly increase funding to Parkinson's research. As the attached 
chart shows, when we started, the number for Parkinson's funding was 
pitifully low [stuck for years at about $26 million]--and it has never 
grown much greater. The NIH has increased its reported number 
significantly but primarily by including increasing amounts of 
``related'' funding, not funds for focused or direct research.
    In fact, the gap between the funding and the potential research has 
become a chasm. The small increase in Parkinson's spending has produced 
only a skirmish when what we need is a serious war.
    It has never been altogether clear how much is being allocated for 
Parkinson's-focused research--the requirement in the Udall Act. As a 
result, beginning in fiscal year 1997, Representative Fred Upton, the 
Udall Act's House sponsor, asked the NIH to document its reporting by 
providing information on the grants it included as ``Parkinson's 
research.'' Then, we asked scientists who are experts in Parkinson's 
research to evaluate the NIH research portfolio on Parkinson's. In both 
years, the results confirmed what we were hearing from the nationwide 
research community: despite the passage of the Udall Act, funding for 
research that actually would benefit Parkinson's patients remains 
unacceptably low.
    This past year, the Parkinson's Action Network asked a group of 15 
key Parkinson's researchers from many of the nation's top academic or 
independent research centers to review abstracts of the grants the NIH 
identified as spent for Parkinson's research in fiscal year 1998 at the 
National Institute of Neurological Disorders and Stroke (NINDS). Many 
are the chairs of their departments; the majority receive and/or have 
received NIH research funding and currently serve and/or have served on 
NIH study sections. (We had waited for several months for the NIH-wide 
list requested by several members of Congress but it was unavailable.)
    Their evaluation found the federal research investment in 
Parkinson's to be far less than that report by NIH to Congress. 
Specifically, the scientists found that 26 percent ($19 million) of the 
grants allegedly spent on Parkinson's research, were spent on research 
that is non-related to Parkinsons. For example, the grants funded 
research focused on Alzheimer's disease, Huntington's disease, drug 
abuse, AIDS, and work at the National Institute of Diabetes and 
Digestive Kidney Diseases, among other things, and had no likely 
benefit for Parkinson's. Furthermore, the evaluation found that of the 
$75 million NINDS claims to spend on Parkinson's, only 44 percent ($33 
million) is spent on research directly related to Parkinson's. Another 
28 percent ($21 million) is spent on research that may indirectly 
benefit Parkinson's, with the remaining 26 percent ($19 million) spent 
on research that will not help Parkinson's patients.
    While we have felt enormously frustrated in our efforts to get a 
clear picture of Parkinson's research funding, we do not want this to 
be a debate about numbers. The real message is this: more funding must 
be devoted to Parkinson's focused research. Without it, the scientific 
community won't have the ammunition to find effective treatments and 
the path to a cure to help me and the million Americans living with 
this disease.
    The solution is with the Congress. We believe NIH and the 
institutes with a particular focus on Parkinson's want to do more--but 
need the resources to do so. They don't want to take funding away from 
other critical research--and neither do we. What the Parkinson's 
community is asking is for the Committee to provide an additional $75 
million more for Parkinson's--over and above what is currently being 
spent. Of this funding we would like to see $50 million for the 
National Institute of Neurological Disorders and Stroke and $25 million 
for National Institute of Environmental Health Sciences--where the 
promise for finding a cure is the greatest.
    The consequences of inaction are very real for the Parkinson's 
community. The costs to society are enormous as well. With annual costs 
now in excess of $25 billion, we are only seeing the tip of the 
iceberg. Very soon the Baby Boom generation will reach the average age 
of onset--57--and the annual costs in medical care, lost wages, 
disability will grow exponentially.
    At the height of the polio epidemic there were 58,000 people 
diagnosed with the disease. And of that 20 percent became what they 
called ``paralytic''--those permanently disabled and crippled by the 
disease. People took enormous precautions in the summer polio 
``season'' when it seemed to strike the most.
    Parkinson's strikes 60,000 people every year and the season for 
Parkinson's is 365 days a year.
    We must rally against Parkinson's as we did so successfully against 
polio. We must bring an end to this disease that disables so many. And 
the only way that is going to happen is through an adequately focused 
research effort that is driven by the desire to save lives.
    Please don't let another year go by without fulfilling the promise 
of the Udall Act. Thank you.

    Senator Specter. Thank you very much, Ms. Samuelson.
    Would you ask your daughters to stand, so we can all see 
them.
    Ms. Samuelson. Stand up. From the left they are Anna, 
Rachel, Sarah, and Leah.
    Senator Specter. Your brother is here.
    Ms. Samuelson. My brother is here, Mark Samuelson, and his 
wife Beth. My sister Judy Samuelson. I am so fortunate to have 
such wonderful family.
    Senator Specter. Thank you very much. That certainly does 
personalize it.
STATEMENT OF JAMES CORDY, PRESIDENT, GREATER PITTSBURGH 
            CHAPTER, NATIONAL PARKINSON'S FOUNDATION 
            AND LEADER, PARKINSON'S ALLIANCE
    Senator Specter. Our next witness is Mr. James Cordy, of 
Pittsburgh, Pennsylvania. It says here he is an effective and 
tireless advocate. I can personally attest to that. He has a 
unique perspective, as a Parkinson's patient, and he has an 
ability to articulate the needs of the Parkinson's community. 
He is a founder of the Parkinson's Alliance, the only national 
group comprised of an administered by individuals with 
Parkinson's Disease. He served as President of the Pittsburgh 
Chapter of the National Parkinson's Foundation, and is a member 
of their Board of Directors.
    Thank you for joining us today, Jim, and the floor is 
yours.
    Mr. Cordy. Thank you, Senator Specter, Senator Cochran.
    I contracted Parkinson's 12 years ago, which is a further 
statement that this is not an old person's disease. I was 40 
when that happened. Prior to that, I was in research and 
development for a specialty steel company. Not noted in my 
credentials, I was part of that magnificent grassroots effort 
that saw enacted into law the Morris K. Udall Parkinson's 
Research and Education Act.
    I am here today to give testimony in support of a dramatic 
increase in Parkinson's research that that bill envisioned. I 
brought this hourglass today, as I carry it a lot of places as 
you well know, to serve several functions. Hopefully it will 
keep me within my allotted time period. But, more importantly, 
it is to convey to you that we who have Parkinson's are in a 
race against time. Just at the top chamber is depleted 
relentlessly grain by grain, so is my top chamber, my brain, 
losing brain cells which control movement, day by day.
    I am here today to help give Parkinson's a human face, as 
Joan and Michael did. Parkinson's is a degenerative disease of 
the brain. When my medications are working, I approach some 
form of normalcy. In fact, I sometimes think I do not do our 
cause a service because I look pretty good. But when those 
medications are not working, I struggle, as Joan and Michael 
talked about. I cannot, at times, button my shirt, tie my tie, 
drive my car, shuffle papers. Some things seem pretty small. A 
friend of mine recently was able to put his socks on again. 
That was a big improvement in quality of life.
    I witness this disease slowly but surely erode my physical 
abilities. I lost my facial expression, my sense of smell and I 
have a monotone voice. I would not be here today if that was 
the extent of my problems. Unfortunately, those are just a 
preview of the horrors to come if we do not cure this sinister 
disease.
    With Parkinson's Disease, what terrifies me and all that 
have it is the real possibility we might end up like the 
recently deceased Morris Udall, bedridden, unable to move or 
talk. I have heard the saying that God helps those who help 
themselves. We certainly try to do that. We successfully 
encouraged Congress to pass the Morris K. Udall bill. We 
supported last year's record increase in NIH appropriations.
    But we did not stop there. In an effort to make sure that 
there is a continual pool of high-quality Parkinson's research 
proposals, a group of us, mostly with Parkinson's Disease, have 
formed a group called the Parkinson's Alliance, with the 
concept of providing seed money. This program is intended to 
encourage new approaches to Parkinson's Disease research, 
thinking outside the box as they say.
    Relatively small grants from the private sector will be 
made to new researchers and researchers not previously working 
in the Parkinson's arena. These grants are intended to 
underwrite the costly pilot data that is a virtual necessity to 
get an NIH grant now. Congress and I think NIH, through your 
appropriations committee, needs to be prepared to fund these 
additional proposals if we are going to reach the potential of 
this new and exciting program.
    I could not help but thinking back to when the Udall bill 
was introduced several years ago, when Congressman Upton said 
we can cure Parkinson's for the price of an on-ramp on an 
interstate. That seems like a fairly small amount.
    We are going to cure Parkinson's Disease. The certainty 
with which I make that statement is based on the opinion of a 
majority of neuroscientists that Parkinson's is curable in the 
near term. When Dr. Fischbach and other scientists make that 5 
to 10 years, I say that despite their extremely good 
credentials, medical science is exploding so rapidly that it is 
impossible for us to predict that.
    I just cite the things like the Internet. Who knew what the 
Internet was 3 or 4 years ago? Now it is part of our daily 
lives. So I look for that 5 to 10 to be cut down to 2 to 4. 
Again, stem cells might do that.
    I have been coming to Washington for 4 years. Conditions 
have changed dramatically. Back then, there was a massive 
budget cutting and deficits. Now we have surpluses. Four years 
ago there were relatively few people who knew about 
Parkinson's. Now, thanks to people like Muhammad Ali and 
Michael J. Fox, awareness has increased and it is widespread, 
all of which should promote a more positive climate for 
Parkinson's Disease.

                           prepared statement

    The reasons for passing the Udall were compelling. But we 
have not realized to date the necessary resolve to get the job 
done. It was suggested that we have a neurodegenerative 
initiative, with Parkinson's leading the way. This could result 
in a possible domino effect in neurology and neurological 
diseases. It would rid this world not only of Parkinson's, but 
ALS, Huntington's, and Alzheimer's. To have this domino effect, 
the first piece must fall. We need the sense of commitment and 
sense of urgency to realize the potential of the Udall bill to 
cure Parkinson's in years rather than decades.
    Again, I just want to thank all of you for your support. 
Senator Wellstone, who I see just arrived, thank you.
    Senator Specter. Thank you very much, Mr. Jim Cordy, for 
those very poignant and personal comments. It certainly brings 
the whole issue home.
    [The statement follows:]
                   Prepared Statement of James Cordy
    Mr. Chairman and members of the committee. My name is Jim Cordy. 
I've had Parkinson's disease for 12 years. Formerly I was an engineer 
in R&D at a specialty steel company. Parkinson's forced me onto 
disability 4 years ago. I am president of the Greater Pittsburgh 
chapter of National Parkinson Foundation, on their national board of 
directors, and leader of the Parkinson Alliance. I am also part of that 
magnificent grassroots effort which saw enacted into law the Morris K. 
Udall Parkinson's Research and Education Act. I'm here today to give 
testimony in support of the dramatic increase in Parkinson's Disease 
research envisioned by the Udall bill.
    I brought this hourglass to serve several functions: Hopefully, it 
will help me stay within my allotted time, but most importantly, it is 
intended to convey to you that we who have Parkinson's are in a race 
against time. Just as the top chamber is depleted relentlessly grain 
after grain, so is my top chamber, my brain, losing nerve cells which 
control movement day by day.
    I'm here today to help give Parkinson's a human face. Parkinson's 
disease is a degenerative disease of the brain. When my medications are 
working I approach some form of normalcy. Perhaps as I walk away from 
this table some may think ``he doesn't look so bad to me''. But those 
medications without which I would be unable to function lose their 
effectiveness with time. The beginnings of that loss are just happening 
to me. I'm falling behind in my race against time. As a result my hands 
and legs sometimes shake and my body is stiff. I have witnessed this 
disease slowly but surely erode my physical abilities. I can no longer 
tie my tie, wash my hair or tuck my shirt in. I can't shuffle papers or 
drive my car. I have lost my facial expression, sense of smell and I 
now have a monotone voice. But I wouldn't be here today if that was the 
extent of my problems. Unfortunately those are just previews of the 
horrors to come if we don't cure this sinister disease. What terrifies 
me and all who have Parkinson's disease is the real possibility that I 
might end up as the recently deceased Mo Udall bedridden unable to move 
or talk.
    I sometimes think I do not serve the Parkinson's research cause 
well when I come to Washington.
    The image I want to leave you with is the horror of Parkinson's 
disease. A woman from California wrote to me describing the final 
ordeal her mother suffered. The body of this former Olympic athlete had 
shriveled to 60 lbs and she had assumed a fetal position for her final 
three years. Three years. This is the image of Parkinsons I want to 
leave you with this and the promise of hope.
    I've heard the saying that God helps those who help themselves. We 
have certainly tried to do that. We successfully encouraged Congress to 
pass the Udall bill. We supported last year's record increase in NIH 
appropriations. But we didn't stop there. In an effort to make sure 
there is a continual pool of high quality Parkinson's research 
proposals a group of people, many with Parkinson's disease, the 
Parkinson Alliance, began promoting the seed money concept. This is a 
program is intended to encourage new approaches in Parkinson's disease 
research. Relatively small grants from the private sector are made to 
new researchers or researchers previously not working in the 
Parkinson's field. These small grants are intended to underwrite the 
cost of developing pilot data for the purpose of submitting an 
application to NIH for a much larger research grant. Congress and NIH 
will have to be ready to fund the additional applications that soon 
will sprout from the seeds.
    We are going to cure Parkinson's disease. The certainty with which 
I make that statement is based on the opinion of a majority of 
neuroscientists that Parkinson's is curable in the near term. The 
question is when? I've been coming to Washington for 4 years. 
Conditions have changed dramatically. Back then there was massive 
budget cutting and huge deficits. Now we have surpluses. Four years ago 
relatively few knew what Parkinson's was. Now in part because of our 
efforts, but more because of well known people such as Muhammad All and 
Michael J. Fox, the awareness has increased dramatically. We have 
widespread bipartisan support, we have done everything that we can 
think of to do. All of which should promote a more positive climate for 
Parkinson disease research.
    Senator Specter, committee members, Dr. Fischbach thank you for 
your support. We have made real progress. The reasons for passage of 
the Udall bill were compelling but we have not realized to date the 
resolve necessary to get the job done. It was suggested that we have a 
Neurodegenerative Disease Initiative with parkinsons leading the way. 
This could result in a possible domino effect that would rid the world 
of not only parkinsons but ALS, Huntingtons and Alzheimers. To do this 
the first piece must fall. We need the commitment and sense of urgency 
necessary to realize the potential of the Udall Bill and cure Parkinson 
disease years rather than decades.
STATEMENT OF DR. J. WILLIAM LANGSTON, PRESIDENT, 
            PARKINSON'S INSTITUTE
    Senator Specter. Our final witness is Dr. J. William 
Langston, President of the Parkinson's Institute. He is a 
graduate of the University of Missouri Medical School. He 
served as Chief of the Valley Medical Center. He is a member of 
the faculty at Stanford University and a Senior Scientists with 
the California Institute of Medical Research.
    Thank you for joining us, Dr. Langston, and we look forward 
to your testimony.
    Dr. Langston. Thank you very much. I would like to start by 
thanking you, Senator Specter and Senator Cochran, for having 
us here and having this hearing.
    I am a neurologist. I do research in Parkinson's Disease. I 
have dedicated my entire career to trying to find the cause and 
cure for this disease. I think, after listening to Michael Fox 
and Jim Cordy and Joan Samuelson, you can probably understand 
why.
    I have a very singular purpose in testifying, as a 
researcher, someone out there embattled in the field, trying to 
solve this disease. That is to try to give you the perspective 
of the research community as to why there is optimism in the 
field.
    Senator Specter, you said something that really heartened 
me in your opening remarks. That is that there have been 
estimates that we could possibly make major progress, perhaps 
solve the disease, find the cause, in 5 years, but that was not 
fast enough. Well, we feel the same way. I want to tell you, 
there is a whole cadre of researchers out there, lined up, 
ready to go if you and NIH can give us the support to get 
there.
    A second comment that was made by Dr. Fischbach that I 
think is extremely important and that I would like to emphasize 
is that while science is full of serendipity and unexpected 
surprises in research, sometimes you hit a point where it is 
time to focus. I truly believe that we now are at a point where 
there is enough knowledge--and Dr. Fischbach superbly outlined 
all of the research that is going on in this field--that it is 
time to focus.
    With a focused effort, the pieces are in front of us, the 
science is there, I think we can make major progress towards 
this disease. I laud NIH's efforts. It is a wonderful first 
step. We have a long way to go, and I think everybody would 
agree on that.
    There is a real sense of excitement, promise and urgency in 
the research community. I think most of us feel that this 
disease can be solved. It may be the first of the diseases to 
be solved. But we must pursue every lead relentlessly if we are 
going to get there.
    I would like to mention just several major research areas 
where I think there has been progress. Again, Senator Specter, 
you asked what has been done with NIH funds. Earlier this year, 
in the Journal of the American Medical Association, a twin 
study was published, the largest twin study ever done in 
Parkinson's Disease. It involved every twin, living twin, that 
served in World War II. The results of that twin study were 
very important.
    They showed that the vast majority of patients, 
particularly older patients with this disease is probably due 
to something in the environment or triggered by something in 
the environment. That means that we need to invest in 
epidemiology. Epidemiology is expensive. It is time consuming. 
Without knowing if this was the right direction to go, we would 
not want to put that kind of money into this science. Now we 
know that is the way to go.
    If we can find the triggers, or causes, in the environment, 
we could have primary prevention of this disease, and eradicate 
it. So that is the future and the past in that area.
    There are also genetic forms of Parkinsonism. Two years 
ago, researchers at NIH cloned the first gene that causes a 
form of Parkinsonism. While these families are very rare, it 
has yielded tremendous research dividends already. We now know 
of proteins that are abnormal in the brains in Parkinson's. 
This is a lead that could help us solve and perhaps cure this 
disease.
    In terms of mechanisms of degeneration, there is a huge 
amount of research going on. If we can find out why those cells 
die, we can intervene and block that process. Parkinson's is 
usually mild when it is first diagnosed. If we could stop the 
disease there, we could basically have something that was close 
to a cure.
    There has been a huge amount of progress in surgery. Dr. 
Fischbach has already talked about those.
    Stem cell technology looms as a very exciting area. For 
those of us who lived through the fetal transplant era and 
Federal bans, I think it is like Yogi Berra once said, it's 
deja vu again. We are having trouble because of bans on 
research. That needs to be changed. This is one of our great 
hopes, I think, for a cure for this disease.
    Once cells die in the brain, they are gone forever. To 
repair the brain, we are going to have to find ways to get new 
sources of cells, put those into the brain, so they can take 
over the job of the missing cells.
    I would like to close, since I see my red light is up, with 
one final statement. I really believe what I am about to say. I 
think today it can truly be historical for Parkinson's 
research. I hope that we have convinced you, and ultimately can 
convince Congress, that a major investment in Parkinson's 
research is not only critically needed, but justified many 
times over by the opportunities in front of us.
    Such an investment could yield huge dividends, not only for 
Parkinson's, but other neurodegenerative diseases, as well. 
Possibly, just possibly, we may be able to end this terrible 
disease once and for all.
    Thank you.
    Senator Specter. Thank you very much, Dr. Langston.
    [The statement follows:]
               Prepared Statement of J. William Langston
    Good morning. It is a pleasure and honor to be here. I would like 
to begin by briefly describing my own background. I am a neurologist, 
and have dedicated my entire professional career to research and 
patient care in Parkinson's disease. I have published 250 papers in the 
area and I see patients with this disease every day and. I am also 
founder and President of the Parkinson's Institute in Sunnyvale Ca, 
located in the heart of Silicone Valley.
    My goal today is to impart the sense of excitement, promise, and 
urgency that currently pervades the Parkinson's disease research 
community. I believe with the adequate resources and manpower, we can 
solve the complex riddle of Parkinson's disease. Research opportunities 
abound--never before have we had so many new leads. But we must pursue 
these leads as vigorously as possible if we are to conquer this 
terrible disorder.
    I would like to begin with research on the cause. As a result of a 
study published earlier this year in the Journal of the American 
Medical Associate, we now have a much clearer picture of how to invest 
our resources to achieve this. This NIH funded study involved 
interviewing all of the living twins who served in World War II. Nearly 
20,000 twins were interviewed, the largest twin study ever done for 
Parkinson's disease. After examining all of the identical and fraternal 
twins with suspected disease, the results showed that typical 
Parkinson's disease, when beginning over the age of 50 is not due to 
genetic causes, but rather must be caused or triggered by something in 
the environment.
    For the research community, this is a huge branch point. It means 
that we can and should focus on environmental influences by studying 
populations of individuals, including the WW II twins. Such studies 
require major investments in time and money, but with this new data we 
now know that such an investment is worth it. Studies to date have 
pointed to pesticides, herbicides, rural living, certain heavy metals, 
and of course there is the inverse relationship to cigarette smoking. 
Let me stress that, if causative agents can be identified in the 
environment, ways to avoid and/or minimize effects of exposure could 
lead to primary prevention of the disease. This is our ultimate dream.
    Does this mean there is no role for genetics? Not at all. 
Unexpectedly, the same study in twins showed that when parkinsonism 
begins earlier in life there is a strong genetic component. I think I 
can safely state that there is a near unanimous consensus in the 
research community that unraveling the genetic parkinsonisms, while 
solving a very small percentage of the cases, will provide invaluable 
new clues on the cause of typical Parkinson's disease. Finding new 
mutations that cause parkinsonism will lead to identification of new 
genes. This will lead to the identification of new proteins that may be 
key players in the process of cell death.
    Let me give an example. In 1997, investigators at the human genome 
project identified a mutation in a form of familial parkinsonism. The 
mutant gene produces a protein know as -synuclein. It turns 
out that only a few families on earth have this mutation, but this same 
protein has been found to aggregate in nerve cells in virtually all 
cases of typical sporadic Parkinson's disease, in structures know as 
Lewy bodies, This has opened up an entirely new avenue of research, and 
raised the possibility for the first time that Parkinson's disease may 
be a protein aggregation disorder, something that has been suspected 
for years in Alzheimer's disease. A second and entirely different 
mutation has already been identified in another form of familial 
parkinsonism, and I suspect there will be many more. The affected 
proteins can be used to model Parkinson's disease in transgenic mice, 
and can be used to study mechanisms of cell death. An all out approach 
to identify new genetic forms of parkinsonism could have scientific 
yield, and we are just in the beginning stages of this research.
    And this is only one of the many areas of laboratory investigation 
that are currently underway to better understand Parkinson's disease. 
Areas currently under investigation include studies on free radicals, 
excitotoxicity, nitric oxide, the process of programmed cell death, and 
even inflammation as possible causes of cell death in Parkinson's 
disease. Each represents an exciting and important area of basic 
research, which, if positive could have enormous therapeutic 
repercussions. If we can identify the mechanisms by which these cells 
are dying in the brain, even if we don't know what kicks the process 
off, we may be able to intervene by blocking the process, and slowing 
or halting disease progression. This could lead to secondary prevention 
if we could identify the disease in its preclinical state, something I 
will return to later.
    Now I would like to turn to patients who have already been affected 
and disabled to a greater or lesser degree by Parkinson's disease. 
Primary and secondary prevention are exciting goals, but what can do 
for those who have already been damaged by the disease? We must find 
ways to repair or restore the damaged areas of the brain. It sounds 
impossible, but in fact new strategies are emerging constantly.
    To explain how this works, I need to give you a brief primer. In 
Parkinson's disease, the brain cells that make a substance called 
dopamine begin to die. Without dopamine, the motor system shuts down, 
leaving patients frozen and unable to move. Because the brain is 
incapable of making new cells, one of the few hopes for a cure is what 
we call cell replacement therapy. Progress in neural transplantation 
has been substantial over the last 15 years. We now know that this 
technique is feasible and safe. Furthermore, it is known that 
transplanted cells survive after transplantation into the brain and are 
capable of exerting therapeutic benefit, although technological 
barriers remain (for example, only approximately 10 percent of cells 
survive). However, the recognition that the use of human fetal tissue 
is likely to be limited in the foreseeable future, an intensive effort 
is under way to find alternatives. Promising lines of research in the 
use of xenografts, bioengineered cell lines, and the used of progenitor 
or pluripotent cells. The latter are in the earliest stage of 
development, but may be the most exciting in the long term. Any success 
in this area could lay the groundwork for serious attempts to cure this 
disease. To quote my colleague, Dr. Rusty Gage of the Salk Institute, a 
preeminent researcher in this area, ``This is an ambitious agenda 
which, while focusing on Parkinson's disease, if funded in excellent 
laboratories, will yield broadly relevant results''.
    How do we best get there, the most quickly? To quote Dr. Gage again 
``One should consider establishing regional testing centers, where 
reliable models in rat, mouse and monkey are routinely established; 
where basic investigators can apply to try out their latest ideas 
without having to set up the models in their own labs and learn by 
making all the mistakes that have already been made. These centers 
could also be places where better models are being designed all the 
time. These centers could eventually form an alliance with clinical 
trials to make sure that the trial reflects what is really known from 
the pre-clinical work, and if a clinical trial is conducted, it would 
be done in such a way that no matter how it turned out, the pre-
clinical centers could take the results and build on them''.
    There is an alternative strategy that should be vigorously pursued. 
This involves reviving or restoring cells that are still in the brain, 
but are non-functional. Even though most of dopamine is gone, only 
about 60 percent of cells are lost, well below the threshold that leads 
to symptoms. This means that there are many cells still present that 
are not functioning. If we can turn on even half of these remaining 
brain cells, we might be able to reverse the Parkinsonism entirely, and 
there are substances that may do this. Growth factors are being 
actively investigated, but do not get to the brain. A new family of 
trophic factors has been discovered in the last few years call 
neuroimmunophilins. These compounds can cross the blood-brain barrier, 
and if effective, could accomplish everything we hope to achieve with 
surgery, without the surgery.
    This brings me to currently available surgical techniques. The last 
decade has lead to a true renaissance surgical approaches for 
Parkinson's disease. This was the direct result of the powerful model 
for Parkinson's disease, which has allowed us to learn a great deal 
about the circuitry of the basal ganglia. For the first time we know 
where to intervene to balance out the abnormal brain circuits in 
Parkinson's disease. A particularly exciting innovation is the use of 
deep brain stimulation. Electrodes are placed deep in the brain, and 
stimulated using a device that resembles a cardiac pacemaker. This 
technique is as effective as older ablative procedures, but not 
permanent and therefore much safer. It can be done on both sides and in 
areas of brain that we could not otherwise approach. One deep 
stimulation area in particular has been found to be very effective, the 
subthalamic nucleus or STN. Indications are that between 10 to 30 
percent of patients may be able to go entirely off medications. But to 
continue this work, a great deal of work needs to be done, both 
experimentally and in practice. We still don't understand how it works 
and we may not have found the best area to stimulate yet. Few centers 
in the country are trained or experienced to do this type of surgery, 
and because of expense, large scale trials have yet to be done. A great 
deal of work lies ahead of this to bring this exciting new technique to 
fruition.
    Finally, I want to draw your attention to a critical research area 
where there is a huge gap, and that is the need for a biomarker. Simply 
put, this is a biologic test that can be used to determine presence or 
absence of a specific disease. At the moment there is no biomarker for 
Parkinson's disease. We desperately need one because clinical 
examination is accurate only about 75 percent of the time. This means 
we are wrong 1 out of every 4 times. This not only affects patient 
care, it can severely affect research. For example, when investigating 
the cause, if some of the patients you are studying don't even have the 
disease you think they do, one might easily miss a vital clue as to the 
cause. In carrying out new drug trials, mixing in patients that don't 
have the disease might easily one thinks they have could dilute out an 
otherwise positive result.
    Fortunately we have an exciting start in this area with new imaging 
procedures. Positron imaging technology is a powerful way to look at 
the brain during life, but for cost and technical reasons will likely 
remain a research tool. A newer technology, called SPECT scanning, 
could be widely used, but at the moment less than a handful of centers 
are doing this procedure, and we have a long way to go before this can 
be widely used for both research and practice. The other major gap is 
there is much more to be learned from it. In the long run, we will 
really need a biomarker that can be used to screen the general 
population for preclinical disease. If that can be developed, and we 
learn more about the mechanism by which cells die, we may be able to 
intervene to halt the disease with ``neuroprotective'' before it even 
appears clinically, something that could be the near equivalent of 
cure.
    In summary, I would like to close by saying that I believe that 
this could be a historical day for Parkinson's disease research. I hope 
that, by the end of this hearing, we will have convinced you that a 
major research investment in not only critically needed, but fully 
warranted. I truly believe that we are at a place in the scientific 
history of research on Parkinson's disease where such an investment 
could yield huge scientific dividends. If so, our society and the 
patients we serve will be the real winners.
    Thank you for you kind attention.

                       Issue in hands of congress

    Senator Specter. In listening to your testimony, Ms. 
Samuelson is exactly right, that this issue is in the hands of 
the Congress. There is no doubt about that. We have a total 
budget which is almost $1.8 trillion, a staggering sum of 
money. Nobody can really comprehend that amount of money. If 
you took a large room like this, there would be insufficient 
space to stuff $10,000 bills into it.
    When Jim Cordy talks about the desire of conquering 
Parkinson's in 2 to 4 years, I agree, and less if possible.
    When Michael J. Fox asks for $75 million more, we could do 
it if we increased overall NIH funding by about $1.3 billion.
    If we start the battles among the various institutes and 
ailments, I think it would be very counterproductive. So what 
has to be done is to raise all the boats with the overall 
funding. That is something that many of us would like to see 
happen.
    Just a very brief statement on the practical politics of 
what happens. Two years ago, we had a sense of the Senate 
resolution to double NIH funding over 5 years, 98 to nothing. 
Then, when the issue came up about adding the money, to add 
first a trillion dollars, 3 years ago, it lost, 63 to 37. So 
Senator Harkin and Senator Cochran and I doubled the request. 
Senator Wellstone joined. If at first you do not succeed, 
double the request.
    We asked for $2 billion. Again we lost. We got a few more 
votes. But this subcommittee went to the drawing board with 
some sharpened pencils, and we found the money by rearranging 
priorities.
    Again, this year, we have determined to rearrange the 
priorities and add $2 billion more. So when you had $120 
billion to research, that is very, very substantial. But I do 
not disagree with you, Mr. Fox, about adding $75 million more. 
When you look at our total budget and you look at the wealth of 
this country, there is no reason why every valid research 
application should not be granted. Every one ought to be 
granted.
    Right now, there are about seven closed doors which are 
unopened. Out of every 10, three are opened for research; seven 
are closed. But that requires the will of the Congress to do so 
in the priority-setting. You have available to you the members 
of the Senate and the House who have voted no on increasing NIH 
funding. So it is a fairly direct matter to mobilize America to 
get the increased funding.
    When you come and tell your stories, and understandably 
with tears in your eyes, and Michael J. Fox wants to see his 
children's weddings, it is very understandable.
    When Ms. Samuelson wants to be the buddy to her family 
youngsters, it is understandable.
    When Jim Cordy gets emotional about having a normal life, 
it is understandable. We have to fund Dr. Langston.
    Any concluding comment, Dr. Langston? I will give you each 
one more chance for a concluding comment.
    Dr. Langston. Well, I just want to say, again, for someone 
who is out there working day to day on this disease, seeing 
patients every day with this disease, something like that is 
heartening and inspiring. I just hope we can look back and see 
that this was the beginning of something very, very important 
that helped us solve this disease as we go into the new 
millennium.
    Thank you again for the opportunity to be here.
    Senator Specter. Mr. Fox, you put your finger right on top 
of the core issue--hope, hope, there is good reason for hope.
    Mr. Fox. Right.
    Senator Specter. But we have to translate that hope into 
action now. Concluding comment, Michael?
    Mr. Fox. I would say my comment is--and I did not graduate 
from high school, but I learned enough Latin to be able to say 
this--carpe diem. We are there. If I can do anything, I hope 
that I can bring a little attention to the fact that--you know, 
all kinds of people have hardships and struggles and face 
issues.
    Certainly by highlighting our battle, we are not 
diminishing anyone else's battle or need for help. But someone 
mentioned the word ``prioritizing.'' We are there with this. We 
are really there. If we can just get a focus on it, I really 
think we can get this done. We will be out of your way.
    Senator Specter. Thank you very much, Michael.
    Any concluding comment, Ms. Samuelson?
    Ms. Samuelson. Well, I do think that says it all. This is 
the time. We realized some time ago that what we needed to do 
as a community was partner up with the scientists, to help them 
get what they needed. I think it is a partnership with the 
Congress. It is thrilling to hear that there is interest in 
that, in getting this done and providing the money to do it.
    Senator Specter. Thank you very much.
    Jim Cordy, any final comment?
    Mr. Cordy. You talked about trying to convince Congress to 
do this. The number that Joan came up with, it would cost 
society $25 billion a year, and if we spend $100 million a year 
to cure that. You were talking about people grasping the 
billions of dollars. I broke that down. For every dollar spent, 
we would save $250. That is just a tremendous return on 
investment and one I do not think we can pass up.
    Lastly, one other thing, just so I do not catch a lot of 
hell from my granddaughter, and my niece is standing up, she is 
in attendance, and I would certainly like to dance at her 
wedding.
    Senator Specter. Thank you very much, Jim.
    Senator Cochran.
    Senator Cochran. Mr. Chairman, let me conclude my part of 
this hearing by thanking you for your strong leadership. You 
have really shown the way, and you have gone out front in 
leading us to more dollars for NIH. We now have to continue to 
support you, as we go to the full committee today and the floor 
of the Senate tomorrow, to get support for this additional 
funding, and then make it stick in conference, and get the 
President to sign one of our bills. That will be helpful, too.
    But we have an opportunity, as I tried to mention in my 
opening statement, an opportunity and an obligation. The 
opportunity is to give people a chance to restore normalcy and 
control over their own life, to find a cure for this dread 
disease. We have the opportunity to give renewed hope to 
millions of Americans who are affected by Parkinson's Disease, 
by making clear our commitment to provide the resources 
necessary to cure the disease.
    For those who are involved in the research, like Dr. 
Langston and Dr. Fischbach, we thank you for your very strong, 
imaginative and dedicated efforts to make this dream a reality.
    Thank you.
    Senator Specter. Thank you very much, Senator Cochran. 
Thank you for all of your leadership and help.
    Senator Wellstone.
    Senator Wellstone. Thank you, Mr. Chairman. I am not really 
a member of the committee.
    Senator Specter. Well, in that event, Senator Wellstone, we 
will still let you speak.
    Senator Wellstone. I thank you for your graciousness. I 
actually do not have any prepared remarks. Let me do this in 1 
minute.
    I always agree with Senator Cochran.
    Senator Cochran. Do not hurt me now.
    Senator Wellstone. I thought I was hurting myself.
    I think that you really have done excellent work, Mr. 
Chairman. I think you are right about the need to expand the 
NIH budget. If I could snap my fingers and have it my way, we 
would do even much more. Because otherwise we would get one 
group of people with a disease pitted against another group of 
people, and it just does not make sense.
    I know we do not earmark, but I love this language, you 
know, having worked on this legislation for a long time, that 
will make it clear that we will get the funding that we 
absolutely believe we deserve, that is in the Udall bill. So we 
have got to do the work. Thad is right. The only other thing I 
would say is I would like to thank everyone.
    Jim, when you talked about the courage of Muhammad Ali, or 
Mr. Fox for being here, you are right. It is important for 
people who are so well known nationally to speak out and to 
say, look, you know, with the funding we are providing, we 
could finally cure this disease, and we are going to tell you 
time is not neutral, it is not on our side, and we need for you 
to do this. I also want to thank the people in the Parkinson's 
community, whether it be people with Parkinson's and whether it 
be their loved ones, for their speaking out too.
    It has been a really important, effective citizens' lobby. 
The only reason we are where we are today is because of the 
strength and the courage and the dignity of the people in the 
community. So I agree with Michael J. Fox, that you will be out 
of our way, but only after we get this job done.
    Thank you.
    Senator Specter. Thank you very much, Senator Wellstone.
    Without objection, we will put a statement from Senator 
Murray in the record.
    [The statement follows:]
               Prepared Statement of Senator Patty Murray
    Mr. Chairman: I want to thank you for scheduling this important 
hearing and I want to also thank all of today's witnesses for coming 
before the Subcommittee to present their testimony. I look forward to 
reviewing your written testimony and want to commend all of you for 
your commitment and dedication to increase the awareness of 
Parkinson's, and working to one day find the cure for this devastating 
illness.
    I have heard from many families in Washington state who have been 
touched by Parkinson's. I have heard their stories and know the 
heartache they face. I know first hand how a disease like Parkinson's 
can strike the entire family. Last year I met with a young father who 
told me that he was not able to go camping with his son last summer. He 
told me how he had always enjoyed the camping trips he had with his son 
but he could no longer endure the physical demands of camping. He has 
lost this precious time with his son, and his son has lost as well.
    As a member of the Senate Appropriations Committee and the Senate 
Health, Education, Labor and Pensions Committee, I have worked hard to 
increase our commitment to biomedical research. As a Member of the 
Appropriations Committee, I have worked, along with our Subcommittee 
Chairman, to Increase NIH funding by well over 40 percent since 1993. 
As a member of the HELP Committee, I was part of the Committee's 
efforts to revitalize and modernize the Food and Drug Administration to 
ensure that life saving, experimental drug treatments got to patients 
faster. I consider enactment of the FDA Modernization Act as one of the 
major accomplishments of the 105th Congress. My work was based on my 
belief that we must improve access to treatments and life saving drug 
therapies.
    I have now become more and more concerned about access. We have 47 
million Americans with no health insurance. We have health care 
decisions being made by health insurance bureaucrats instead of doctors 
and patients. We have health insurance companies that are denying 
access to clinical trials and experimental treatments, and health 
insurance policies that discourage or penalize those who need access to 
highly specialized care. What good does it do to double NIH funding or 
modernize the FDA when millions of patients are denied access to new 
drug treatments and therapies?
    Could you briefly touch on the issue of access and how we can 
ensure that all Parkinson's patients can access life saving treatments? 
What impact or role do clinical trials play in expanding access and 
knowledge of Parkinson's disease? How important is it for a Parkinson's 
patient to have access to speciality care and cutting edge biomedical 
advances?

    Senator Specter. We thank all of you for coming. May the 
record show that in this audience there are many people here in 
wheelchairs, with canes and walkers, showing the disability and 
the further need for action and for adequate funding.

                         Conclusion of hearing

    Thank you all very much for being here, that concludes the 
hearing. The subcommittee will stand in recess subject to the 
call of the Chair.
    [Whereupon, at 10:50 a.m., Tuesday, September 28, the 
hearing was concluded, and the subcommittee was recessed, to 
reconvene subject to the call of the Chair.]

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