[Senate Hearing 106-373]
[From the U.S. Government Publishing Office]
S. Hrg. 106-373
PARKINSON'S DISEASE RESEARCH AND TREATMENT
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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
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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
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Subcommittee on Labor, Health and Human Services, and Education, and
Related Agencies
ARLEN SPECTER, Pennsylvania, Chairman
THAD COCHRAN, Mississippi TOM HARKIN, Iowa
SLADE GORTON, Washington ERNEST F. HOLLINGS, South Carolina
JUDD GREGG, New Hampshire DANIEL K. INOUYE, Hawaii
LARRY CRAIG, Idaho HARRY REID, Nevada
KAY BAILEY HUTCHISON, Texas HERB KOHL, Wisconsin
TED STEVENS, Alaska PATTY MURRAY, Washington
JON KYL, Arizona DIANNE FEINSTEIN, California
ROBERT C. BYRD, West Virginia
(Ex officio)
Professional Staff
Bettilou Taylor
Mary Dietrich
Jim Sourwine
Aura Dunn
Ellen Murray (Minority)
Administrative Support
Kevin Johnson
Carole Geagley (Minority)
C O N T E N T S
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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
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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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