[Senate Hearing 106-855]
[From the U.S. Government Publishing Office]
S. Hrg. 106-855
AMYOTROPHIC LATERAL SCLEROSIS
=======================================================================
HEARING
before a
SUBCOMMITTEE OF THE
COMMITTEE ON APPROPRIATIONS UNITED STATES SENATE
ONE HUNDRED SIXTH CONGRESS
SECOND SESSION
__________
SPECIAL HEARING
__________
Printed for the use of the Committee on Appropriations
Available via the World Wide Web: http://www.access.gpo.gov/congress/
senate
__________
U.S. GOVERNMENT PRINTING OFFICE
68-402 WASHINGTON : 2001
______
_______________________________________________________________________
For sale by the U.S. Government Printing Office
Superintendent of Documents, Congressional Sales Office, Washington, DC
20402
COMMITTEE ON APPROPRIATIONS
TED STEVENS, Alaska, Chairman
THAD COCHRAN, Mississippi ROBERT C. BYRD, West Virginia
ARLEN SPECTER, Pennsylvania DANIEL K. INOUYE, Hawaii
PETE V. DOMENICI, New Mexico ERNEST F. HOLLINGS, South Carolina
CHRISTOPHER S. BOND, Missouri PATRICK J. LEAHY, Vermont
SLADE GORTON, Washington FRANK R. LAUTENBERG, New Jersey
MITCH McCONNELL, Kentucky TOM HARKIN, Iowa
CONRAD BURNS, Montana BARBARA A. MIKULSKI, Maryland
RICHARD C. SHELBY, Alabama HARRY REID, Nevada
JUDD GREGG, New Hampshire HERB KOHL, Wisconsin
ROBERT F. BENNETT, Utah PATTY MURRAY, Washington
BEN NIGHTHORSE CAMPBELL, Colorado BYRON L. DORGAN, North Dakota
LARRY CRAIG, Idaho DIANNE FEINSTEIN, California
KAY BAILEY HUTCHISON, Texas RICHARD J. DURBIN, Illinois
JON KYL, Arizona
Steven J. Cortese, Staff Director
Lisa Sutherland, Deputy Staff Director
James H. English, Minority Staff Director
------
Subcommittee on Departments of Labor, Health and Human Services, and
Education, and Related Agencies
ARLEN SPECTER, Pennsylvania, Chairman
THAD COCHRAN, Mississippi TOM HARKIN, Iowa
SLADE GORTON, Washington ERNEST F. HOLLINGS, South Carolina
JUDD GREGG, New Hampshire DANIEL K. INOUYE, Hawaii
LARRY CRAIG, Idaho HARRY REID, Nevada
KAY BAILEY HUTCHISON, Texas HERB KOHL, Wisconsin
TED STEVENS, Alaska PATTY MURRAY, Washington
JON KYL, Arizona DIANNE FEINSTEIN, California
ROBERT C. BYRD, West Virginia
(Ex officio)
Professional Staff
Bettilou Taylor
Mary Dietrich
Jim Sourwine
Ellen Murray (Minority)
Administrative Support
Kevin Johnson
Carole Geagley (Minority)
C O N T E N T S
----------
Page
Opening statement of Senator Arlen Specter....................... 1
Opening statement of Senator Harry Reid.......................... 2
Statement of Representative Lois Capps........................... 4
Statement of Gerald D. Fischbach, M.D., Director, National
Institute of Neurological Disorders and Stroke, National
Institutes of Health, Department of Health and Human Services.. 6
Prepared statement........................................... 8
Statement of Tom Maniatis, Ph.D., chairman, Cure Advisory
Committee, Amyotrophic Lateral Sclerosis Association........... 11
Prepared statement........................................... 13
Statement of Steve Beuerlein, quarterback, Carolina Panthers..... 18
Prepared statement........................................... 20
Statement of Steve Garvey, former first baseman, Los Angeles
Dodgers........................................................ 22
Prepared statement........................................... 23
Statement of Dick Schaap, host, ESPN's The Sports Reporters...... 25
Prepared statement........................................... 26
Statement of Greg Hickock, ALS patient, Jackson, MI.............. 31
Statement of Blair Underwood, actor.............................. 35
Prepared statement........................................... 36
Statement of Shelbie Oppenheimer, ALS patient, New Hope, PA...... 37
Prepared statement........................................... 39
Statement of Steve Rigazio, ALS patient, Las Vegas, NV........... 40
Prepared statement........................................... 41
AMYOTROPHIC LATERAL SCLEROSIS
----------
THURSDAY, MAY 18, 2000
U.S. Senate,
Subcommittee on Labor, Health and Human
Services, and Education, and Related Agencies,
Committee on Appropriations,
Washington, DC.
The subcommittee met at 9:35 a.m., in room SH-216, Hart
Senate Office Building, Hon. Arlen Specter (chairman)
presiding.
Present: Senators Specter and Reid.
Also present: Representative Capps.
OPENING STATEMENT OF SENATOR ARLEN SPECTER
Senator Specter. Good morning, ladies and gentlemen. The
appropriations Subcommittee on Labor, Health and Human
Services, and Education will now proceed with our special
hearing on amyotrophic lateral sclerosis.
We thank you all for coming. We have a very distinguished
witness list, and we have a very distinguished guest list of
all those who are in attendance today to proceed with the
hearing to hear what is being done, what can be done for the
dreaded ailment of amyotrophic lateral sclerosis. This is an
ailment which affects some 15,000 to 20,000 Americans, with
about 5,000 new cases each year. Some 90 percent of amyotrophic
lateral sclerosis patients die within 5 years of being
diagnosed and only 10 percent live longer than 5 years.
At the present time, there is no known cure for amyotrophic
lateral sclerosis. I have inquired of Dr. Fischbach earlier
today specifically on ALS as to whether the new developments on
stem cells might have some potential for treatment or for
prevention. Stem cells, as you may know, are those substances
derived from embryos where they have shown great promise on a
number of ailments, Parkinson's and heart disease. They are a
veritable fountain of youth with these stem cells being
extracted and then being put into the human body to replace
defective cells.
That issue is soon to come before the Senate on a bill to
eliminate the current prohibition against Federal funding to
extract stems cells from embryos. Controversy arises--and we
have had a series of five hearings on that subject, but it is
relevant to amyotrophic lateral sclerosis perhaps. We will hear
Dr. Fischbach's testimony on that. But it is irrelevant because
at the present time there is a Federal law which prohibits the
National Institutes of Health from using Federal funding to
extract stem cells from embryos.
These embryos have been created for in vitro fertilization
and they are discarded. They are not to be used. If there were
any possibility that they could produce life, I would be
totally against their utilization. But in the situation where
they are going to be discarded and not used for anything and
not produce human life, then it is my view that we ought to try
to save lives with the use of these stem cells.
But that is an issue which will be debated on the Senate
floor in the course of the next several weeks, but I call it to
your attention because these are matters where Members of the
Senate ought to know what the views of their constituents are.
So, if you have a view, you may want to consider presenting it
to your United States Senator.
I am pleased to report that we are moving ahead with
increased funding for the National Institutes of Health. I
frequently say that the National Institutes of Health is the
crown jewel of the Federal Government. I think it may be the
only jewel of the Federal Government.
Senator Tom Harkin, a Democrat of Iowa, and I have worked
collaboratively with this subcommittee and the full committee
on a bipartisan basis to make enormous increases in funding for
NIH. I learned a long time ago if you want to get something
done in Washington, you have to be willing to cross party
lines. And we have increased the funding in the last 3 years by
more than $5 billion so that NIH is now funded at almost $18
billion, and we have put $2.7 billion in this year. Candidly it
is going to be a tough battle to keep it in the budget, but we
are going to try to do that to bring the total funding to
approximately $20.5 billion.
As we have increased funding for NIH generally, we have
increased funding very significantly for amyotrophic lateral
sclerosis, with a funding over the decade from 1990 of $5.8
million to this year to $19.2 million, three to four times as
much as it had been in the past.
I had an inquiry yesterday from national television about
why all the celebrities. And my response is that when people
appear, whom the public associated with, they understand it.
When Michael J. Fox came in to testify about Parkinson's and
how he was leaving the television show, people relate to that.
When Christopher Reeve comes in to testify about the accident
he had, being thrown from a horse, and his paralysis and
severed spinal cord, people say if it can happen to Superman,
it can happen to anyone. And the public attention does help
focus on the Congress the need for increased funding and other
constituent interests such as stem cell research.
With that brief introduction, let me turn to the
distinguished Senator from Nevada, Senator Reid, who was next
to arrive here.
OPENING STATEMENT OF SENATOR HARRY REID
Senator Reid. Senator Specter, first of all, let me say
there are more jewels in the Federal Government than NIH.
But one of those jewels is this subcommittee. I think this
subcommittee has done remarkably good work, and I want to make
sure everyone within the sound of our voices understands that
you talked about not a $5 million increase, but a $5 billion
increase. That is significant, especially when we have been
cutting back and holding--so, I do believe this subcommittee is
one of the jewels of the Federal Government. You and Tom Harkin
have set an example of bipartisanship that most everyone else
in the Congress could take a lesson from.
Many of us know that ALS is the disease that took the life
of the famed Yankee first baseman, Lou Gehrig. Yet, few of us
are aware of the prevalence of this disease and the devastating
effect it has on its victims. We know at least 30,000 Americans
suffer from this diseases, perhaps many more. In Nevada, a
resident is diagnosed every week with this disease. In Las
Vegas, the average age of the ALS is patient is 45, and the
average survival rate is 18 months.
Today we are joined by a distinguished Nevadan, Steve
Rigazio who, like many people who are stricken with this
disease, was at the top of his game, so to speak, when he was
diagnosed with this disease. He was one of our more prominent
business people in Nevada and his story is part of the many
stories that we have told here today. I am glad that the
chairman allowed Mr. Rigazio to be here today.
In the years since Gehrig's death in 1941, very little
progress has been made, and the cause of the disease that bears
his name still remains a mystery. Just last week, this
committee approved $2.7 billion increase for the National
Institutes of Health for this year. I hope that the National
Institute of Neurological Disorders and Stroke will give
scientists the tools and the necessary resources to identify a
breakthrough that will lead to a cure for this devastating
disease. Until research identifies a cure, we must do
everything possible to encourage the development of effective
drugs to treat and improve the quality of life of these
patients.
I have serious concerns--and it will come up during this
hearing--of FDA's handling of one such drug, Myotrophin, and
the fear that this has set a damaging precedent that could
discourage other drug companies from participating in ALS
research.
We also need to fix a flaw in the Medicare program that
requires ALS patients to endure a 2-year waiting period--
remember, I have already established in that in Nevada there is
an average life expectancy of 18 months--to wait until the
final months of their illness to be able to receive Medicare
services. It defies common sense and even human decency.
I am 1 of approximately 20 Senators who support S. 1074
that would correct this problem. I am pleased that the House
companion measure--we have Representative Capps here--has the
support of 230 House Members.
Finally, we cannot ignore the families of ALS patients.
Family caregivers must be recognized for their efforts in this
field and others, and I hope that Congress will pass
legislation that will give a tax credit to family caregivers.
ALS is a disease that strikes at every community with the
potential for striking every American. As my friend Steve
recently learned, no one is immune. Everyone is vulnerable. I
hope that this hearing will help focus important research
efforts on this tragic illness. We must act today because ALS
patients and their families do not have the luxury of time.
I say, Mr. Chairman, I hope that you will excuse me off and
on during this hearing. I just got a beep and I have to go
return to the floor. Thank you.
Senator Specter. Thank you very much, Senator Reid.
You make a pretty good case that there may be some other
jewels in the Federal Government. I am going to reserve
judgment on that about the subcommittee. I still think NIH is
the crown jewel.
STATEMENT OF REPRESENTATIVE LOIS CAPPS
Senator Specter. We have invited to join us today the
Honorable Lois Capps, Congresswoman from California, who has
introduced legislation on amyotrophic lateral sclerosis. She
won election in a special election to succeed her late husband,
Congressman Walter Capps. She serves on the Commerce Committee
and she has this legislative proposal which I thought merited
consideration, and we have decided not to have her as a
witness, but have her join us on the panel, and we are glad to
yield a few minutes to you, Congresswoman Capps.
Ms. Capps. Thank you. Mr. Chairman, I so much appreciate
the opportunity to join you at this important hearing. I have
enormous respect for you, Senator Specter, and also your
colleague, Senator Reid, for your leadership in this area. I
want to congratulate you for your leadership in general in the
area of medical research, as you have both alluded to these
jewels. We would like to increase either their size or their
number, and I think that is a joint effort, bipartisan effort,
in the House as well as in the Senate. And it speaks well for
our Government to be focused in this way.
This hearing is yet another testament to the leading role
that you have played in highlighting the needs in this area. As
a former public health nurse, I want to thank you for those
efforts on behalf of people across the country represented by
those in the audience today.
Most of us know of the famed baseball star for which
amyotrophic lateral sclerosis is named. Many of us are unaware
of the tragic consequences of Lou Gehrig's disease. First
diagnosed over 130 years ago, ALS is a progressive, fatal,
neuromuscular disease afflicting 25,000 to 30,000 individuals
in the United States today. Approximately 5,000 new cases are
reported each year.
This hearing on research efforts into the causes, the
treatments, and the cures for ALS is so very timely. Today we
are on the cusp of medical breakthroughs that will change the
lives of millions of Americans who suffer from diseases such as
ALS and Parkinson's and others. I am so excited about the ALS
Association's new research initiative which is going to be
announced today. I am very interested in hearing from Dr.
Fischbach on advances in research at NINDS and his views on
what we in Congress can do to support this kind of work. We
here in the Senate and the House certainly need to ensure that
the Federal Government meets its responsibilities in NIH
funding and in other areas.
As was mentioned, I am trying to address some of those
needs through legislation I sponsored, the ALS Treatment and
Assistance Act, which is House resolution 353. My bill will
waive the 24-month waiting period for Medicare coverage for ALS
patients. It is only reasonable since the life expectancy for
persons with ALS is, tragically, often shorter than this
waiting period itself, and ALS patients have usually paid into
the Social Security and Medicare systems for years prior to
their diagnosis.
My bill will also provide Medicare coverage for outpatient
drugs and therapies for ALS. This coverage will help ALS
patients and also help spur the development of new treatments
for this disease.
Currently, as was mentioned, more than 230 House Members
are cosponsors of this bill, and I hope that we can enact it
into law this session. I know that Senator Torricelli has
introduced companion legislation here in the Senate, Senate
bill 1074, and I would like to thank him for his leadership on
this issue.
But the biggest thanks of all goes to many people across
this country, PALS. You are in this room. Persons with ALS and
your families. You are the ones who have caused the momentum to
build in the House for those 230 cosigners. I have to tell you
this ALS Day on the Hill that now I have experienced for three
times is one of the most inspiring days for this Member of
Congress, to see you here, tirelessly going from office to
office, personally asking your Representatives. That is
democracy in action. And it has proven to be effective.
Now, this hearing will help build the momentum even
further, and we can expect, hopefully, legislation this year.
Members of Congress and Senators have been educated
regarding the debilitating aspects of this disease and the
critical need for more research. Many times making this
personal connection makes all the difference.
I have a personal connection as well that I just want to
mention briefly. The inspiration for this legislation comes
from just that personal connection. My late husband Walter was
in rehabilitation after a terrible car accident in 1996, and he
struck up a friendship with a Santa Barbara resident, Tom
Rogers, who suffers from ALS. Tom was a compelling and able
legislator, a county supervisor, on a fast track toward great
political success when he was struck down with this disease. He
is a leader still in the environmental movement in Santa
Barbara County in California. His struggle with this disease
has been and still is heroic and an inspiration to all who know
him.
During my husband Walter's campaign for Congress, Tom gave
him his running shoes. He said he could no longer use them
himself due to the toll that ALS was taking on his life. Walter
wore those shoes throughout the months leading up to his
election to the House. It was this gesture of friendship and
support that drew Walter and later me into looking at the costs
that ALS exacts and the desperate need for more research into
this disease.
So, I really do want to thank you, Mr. Chairman, both
personally on behalf of all of these people here for holding
this hearing on an issue we all know is so important. I thank
our witnesses for coming today and I do look forward to hearing
from them. Thank you for the time.
Senator Specter. Thank you very much, Congresswoman Capps.
The subcommittee had invited Senator Torricelli to appear
as well. He is the principal cosponsor of the Senate companion
bill. I am pleased to be a cosponsor. It seems to me that given
the problems of amyotrophic lateral sclerosis and the fatality
and the short life span, that it is too much to have the
regular 24-month waiting period apply. So, that is a
reasonable, even if somewhat costly, provision. So, we will
push hard to see if we can get the bill enacted.
STATEMENT OF GERALD D. FISCHBACH, M.D., DIRECTOR,
NATIONAL INSTITUTE OF NEUROLOGICAL
DISORDERS AND STROKE, NATIONAL INSTITUTES
OF HEALTH, DEPARTMENT OF HEALTH AND HUMAN
SERVICES
Senator Specter. We now turn to our first witness, Dr.
Gerald Fischbach, Director of the National Institute of
Neurological Disorders and Stroke at NIH since July of 1998.
Dr. Fischbach had been Chairman of the Neurobiology Department
at Washington University, Harvard Medical School, and
Massachusetts General. He is a past President of the Society of
Neuroscience, a member of the National Academy of Sciences.
Welcome, Dr. Fischbach, and we look forward to your
testimony.
Dr. Fischbach. Thank you, Mr. Chairman. Thank you for
having this very, very important hearing. I want to add my
thanks to you and this committee for its wonderful and
remarkable support for biomedical science over the years. My
view is NIH is a jewel. My perspective is that it is the jewel
of world biomedical science. This is because we now are on
course for funding that will allow NIH supported investigators
to spread their wings and take advantage of every scientific
opportunity that comes our way.
The subject today is amyotrophic lateral sclerosis, and I
thought I would spend a few minutes defining the disease, say
what we understand about it now, and then say a few words about
hopes for the future and what we may think about in the future
in terms of new therapeutics.
Amyotrophic lateral sclerosis is a difficult name to say.
Amyotrophic because muscles atrophy and waste. The nervous
system can no longer support the development and the health and
the strength of the muscles so essential for movement. Lateral
because the nerve cells that control the muscles are lateral in
the nervous system. They are not in the midline. And sclerosis
because as the nerve cells die, a scar of sorts forms to
replace the disappearing cells. So, ALS is well understood. It
has been studied for 130 years in terms of its pathology, and
we are learning more and more about its causes. That knowledge
will teach us new therapeutic interventions.
The nerve cells that control muscle are very unusual nerve
cells. They are lodged in the spinal cord but they send long
processes that reach out to touch the muscles and control their
contraction and ultimately to support their health. They are
extremely vulnerable cells. One wonders why motor neurons are
affected in this disease. To give you some idea of the burden
that a motor neuron bears and the size of this long extension
that reaches out to muscles, if one nerve cell were the size of
my head, its extension, called its axon, reaching out to
muscles would wrap around this room 20 times. The cell body has
to support that large extension. It has an enormous metabolic
burden to keep it intact and to signal the muscles to contract
in a normal, healthy movement.
The disease does affect about 25,000 to 30,000 people in
this country, but it seems to me and to many people that it is
more prevalent than that. We all know people with ALS and the
presence of the advocates in this room makes one realize that
this has implications far beyond the number of people we now
know are affected by the disorder.
There is a terrible and inexorable march of the disease in
that we do not know how to reverse it. The predictions are
correct of about a 3- to 5-year life span after diagnosis. It
affects men a little more frequently than women, and there are
various clues to what causes the disorder. There are genetic
causes. There are environmental causes and there are some hints
about infectious agents that might be possible inciting causes.
I think there is reason for hope in ALS despite the
terrible current prognosis. The reasons I have for hope are:
First we have learned a tremendous amount in the last 7 years
about the genetics of ALS; second, we know a lot now about how
cells actually degenerate, and everything we learn about that
process offers new opportunities for therapeutic intervention;
third, there are new therapeutics on the horizon. One was
mentioned, a neurotrophic factor. But there are also new
methods for screening for new therapeutics called high
throughput assays, which offer new opportunities for
collaboration between Government and industry; fourth, there is
very promising discussion and early animal experiments on stem
cell replacement therapy and gene therapy. The more we learn
about the defective genes, the more we can attempt to replace
them.
A very small percentage of patients with ALS have inherited
this disorder, passed on in families, but the sporadic cases,
that is, the cases that arise seemingly de novo in the
population are very similar to the cases that are inherited and
passed on in families. So, discovery of a gene that is
defective in the familial form of ALS has enormous implications
for everyone because it may teach us how all cases of ALS
progress.
So, the discovery of a gene that encodes an important
enzyme, superoxide dismutase, 7 years ago, opened enormous
avenues of investigation. This enzyme is responsible for
removing destructive chemicals that build up within cells, and
it offered several insights into possible mechanisms that may
be responsible for nerve cell death, and those investigations
are underway today.
We know how these cells die. We know that there are amino
acids that are toxic in the nervous system and current
therapeutics are attempting to reduce the effect of these amino
acids.
Senator Specter. Dr. Fischbach, we have a very long list of
witnesses and the red light has been on a while. Would you sum
up please?
PREPARED STATEMENT
Dr. Fischbach. Beyond these, the new therapeutics are
promising, and as I mentioned, the animal experimentation
suggests that cell replacement therapy with embryonic stem
cells may very well have a very important function in treatment
in the near future of ALS.
[The statement follows:]
Prepared Statement of Gerald D. Fischbach
Amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, is a
devastating neurological disorder that robs people of their ability to
move, eventually causing death. The progression of weakness and muscle
wasting can follow several different patterns. It may begin, for
example, with difficulty of fine finger movements, such as handling
keys or buttons, and progress to affect muscles of the hand, arm,
shoulder, and legs. Later, muscles that control swallowing and
respiration become involved. Regardless of how it begins, ALS is
relentless in its progression. About 5,000 people in the United States
develop ALS each year, and about 90 percent of them die within 5 years
of when symptoms are first detected.
The symptoms of ALS reflect the death of motor neurons, nerve cells
in the brain and spinal cord that innervate muscles and cause them to
contract, making movement possible. Motor neurons are remarkable
machines. Their cell bodies, which hold the genetic blueprints and
manufacture most cell components, lie in the brain and spinal cord, but
each one extends a long thin fiber called an axon that projects far to
connect precisely to muscle cells. Although the axons are microscopic
in diameter, the total volume of a motor neuron can be 5000 times that
of a typical cell because the axons are so long. The large size of
motor neurons, their high energy requirements, and the extent of axons
out of the protected environment of the brain and spinal cord
contribute to the vulnerability of these cells.
In addition to the spinal cord motor neurons themselves, the so
called ``upper motor neurons'' also die in ALS. These are nerve cells
in the cerebral cortex that activate the motor neurons. The cerebral
cortex helps coordinate the planning and initiation of voluntary
movement. Upper motor neurons, like motor neurons, must support very
long axons, extending as far as from the head to the lower spinal cord,
and this may contribute to their vulnerability.
Although motor neurons are the best studied of all nerve cells, we
don't know why cells die in ALS, why the disease selectively affects
motor neurons and spares other cells, and whether a defect in the motor
neurons themselves, or some other factor, triggers the disease. Most
importantly, we do not yet know how to stop the progress of ALS.
how nerve cells die in als/what causes als
There are many disorders in which nerve cells die. The particular
diseases that result depend on which parts of the nervous system are
affected. Recent research has shown that the final steps leading to
nerve cell death may be the same in many disorders including ALS,
Parkinson's, Alzheimer's and Huntington's disease, and in trauma and
stroke. The discovery that common themes play out in each disease
offers hope because progress against one disorder will very likely help
in the fight against others.
Apoptosis, or ``cell suicide,'' is one unifying theme in
neurodegeneration that has come to prominence in ALS research. Studies
of the development of the nervous system, particularly in simple
organisms such as nematode worms, revealed that many nerve cells take
an active role in their own death. Cells invoke a step-by-step
disassembly process called apoptosis. During apoptosis a cascade of
enzymes takes place, in which one activates the next like the steps in
a computer program, ultimately leading to the destruction of the cell.
Despite the fact that apoptosis is a late step in the progression of
disease, there are now tantalizing suggestions in animal models that
interrupting apoptosis may slow the progress of ALS. Each step in the
cascade offers targets for the development of drugs or other
interventions.
In addition to the cell death cascade itself, a great deal of
research is directed at insults that set it off. Cells enter apoptosis
when they are damaged. Free radicals are among the leading culprits
suspected of causing damage in neurodegenerative disorders including
ALS. Free radicals are highly reactive chemicals that are a byproduct
of normal energy metabolism. If produced in excess or insufficiently
controlled, these chemicals damage critical components of nerve cells.
Because nerve cells, and especially motor neurons, require so much
energy to carry out their electrical and metabolic activities, they are
especially vulnerable to free radical damage.
Molecular genetics is contributing greatly to our understanding of
ALS, and has reinforced the suspicions about a role for free radicals
in the disease process. Although only about ten percent of people with
ALS inherit the disease, studying those familial cases is helping
scientists to understand ALS because genetics can identify the first
step in the disease--a mutant gene--in these cases. Mutations in the
gene for the enzyme superoxide dismutase (SOD) cause some cases of
inherited ALS. SOD normally acts to safely remove free radicals.
However, it appears that the mutation may lead to ALS not because the
enzyme fails to do its job, but because it creates excess free
radicals. This surprising result is an excellent illustration of the
power of genetics to focus attention and generate new hypotheses.
Inherited ALS is clinically similar to the more common forms of the
disease, and scientists are actively investigating the extent to which
the underlying mechanisms of inherited and sporadic ALS are also alike.
Meanwhile, geneticists are searching for other mutations that can cause
this disease and provide additional clues.
The discovery of SOD mutations led to another crucial advance in
ALS research. Scientists leveraged this gene finding by engineering
mice that develop a disease that mimics ALS. These mice are now
critical tools for studying ALS and testing treatments. Several
therapeutic strategies have already shown promise in these animals. For
example, the nutritional supplement creatine, a natural component of
energy metabolism, extended the lives of ALS mice and is now being
tested in people with ALS.
Other processes that damage nerve cells in other neurological
disorders have been implicated in ALS. ``Excitotoxicity'' occurs when
nerve cells are overstimulated by the neurotransmitter glutamate, a
normal chemical signal by which nerve cells electrically activate one
another. In ALS there may be a failure to clear glutamate adequately,
allowing too much to accumulate. Too much glutamate can lead to
abnormal accumulation of calcium within cells and this disrupts many
critical cellular functions. Excitotoxicity and excess calcium can harm
mitochondria, the energy factories of the cell, causing excess
production of free radicals, triggering apoptosis. Free radicals can
damage many parts of cells including neurofilaments, an essential
component of the internal ``skeleton'' of nerve cells that is
especially important in the long axons of motor neurons. Abnormal
aggregation of proteins, including neurofilament proteins, is another
recurring theme in neurodegenerative diseases that has been a focus of
attention in ALS. The role of the immune system in ALS has also been a
target of investigation, with some studies indicating an autoimmune
attack on calcium channels in ALS. (However, a variety of immune based
therapies have been tried and failed to slow the disease.) Similarly,
there have recently--and in the past--been suggestions that a virus may
be associated with the disease in some way, but further investigations
will need to determine whether a virus can actually cause the disease.
Understanding how these processes come together to cause ALS, and what
triggers the harmful interactions to begin, is a complicated puzzle
that must be solved to defeat ALS.
HOW NERVE CELLS LIVE
Despite the accumulating information about what causes nerve cells
to die, we still don't know why neurodegeneration begins in ALS. It is
also a mystery why nerve cell death proceeds so quickly, compared with
disorders like Parkinson's and Alzheimer's. The rapid progression of
ALS may reflect a downward spiral of effects, each one triggering the
next. In addition to processes that damage nerve cells, the disruption
of factors that normally sustain cells may be critical. In other words,
to understand ALS we must attend not only to how nerve cells die, but
also to how nerve cells live.
Nerve cells do not live in isolation, but continually engage in
conversations with other nerve cells, with supporting cells called glia
that greatly outnumber nerve cells, and with target cells, such as
muscles, to which motor neurons connect. Glia, for example, are largely
responsible for clearing excess glutamate to prevent excitotoxicity,
and a deficiency in glutamate clearance has been implicated in ALS. So,
although motor neurons have been the obvious focus of research in ALS,
other cells may also play a crucial role in this disease. Understanding
these complex interrelationships is critical for understanding what
triggers ALS and why it proceeds so rapidly.
The neuromuscular junction illustrates the intricacy of
communication between cells, which includes not only rapid messages
that evoke muscle contraction but also more slowly acting factors that
influence cell growth, survival and specialization. The neuromuscular
junction is the functional connection, or synapse, between the axons of
motor neurons and muscle cells. The motor neuron axon and the muscle
cell each form highly specialized, precisely aligned structures that
together make up the neuromuscular junction. The result allows rapid
and reliable activation of muscles by neurotransmitters released by the
axon. During the development of the neuromuscular junction, the motor
neuron axon and the muscle cell intimately exchange signals that guides
each to form its part of the junction. Likewise, even in the adult,
there is a continual remodeling of the junction with an ongoing
interaction between nerve, muscle and glial cells.
Natural growth and survival molecules called neurotrophic factors
are one token of the slower, nutritive communication between cells. The
receptiveness of cells to these molecules depends on how electrically
active cells are. So, as motor neurons are damaged, for whatever
reason, their interaction with other nerve cells, glial cells, and
muscle may be affected, leading to further problems. Experiments using
neurotrophic factors as therapy have produced some promising results in
animal models of ALS and other neurodegenerative disorders. So far,
success has not followed in people with these diseases, although trials
are continuing. The lack of early success is not surprising, and should
not be discouraging, given how difficult it is to get neurotrophic
factors into the brain and spinal cord where they are needed and how
little we understand about which molecules, perhaps in combination, are
most appropriate.
Other areas of fundamental neuroscience are also likely to have a
bearing on ALS research in the future, and ALS has attracted the
interest of some of the best scientists from many areas of research.
There has been astonishing progress in understanding the steps by which
a primitive embryonic cell becomes a highly specialized motor neuron.
Within cells chemical messengers called transcription factors bind to
specific regions of DNA and turn on and off particular genes, thereby
regulating the fate of the developing cells. Which genes are active in
a cell determines what kind of cell that cell will be. The
transcription factors, in turn, are regulated by chemical signals from
neighboring cells. Insights about how motor neurons specialize to
differ from other nerve cells provide essential clues for understanding
why these cells are selectively lost in ALS. Strategies, such as gene
therapy, might also exploit the gene control elements to target
potential therapeutic genes to motor neurons. Perhaps in the not too
distant future, the developmental pathways might also be invoked to
generate replacement motor neurons from stem cells.
DEVELOPING THERAPIES
The more we understand what causes cells to die in ALS and what
nerve cells need to live, the more rationally we can develop therapies.
Drugs might plausibly target any of the processes implicated in ALS--
free radical damage, excitotoxicity, calcium concentration, apoptosis,
and so on--or therapeutic interventions might supplement sustaining
factors like neurotrophic factors and electrical activity, try to
replace or repair defective proteins such as SOD or even aim to replace
lost cells. A combination of approaches may well be the best strategy.
Pharmaceutical companies use a technology called high throughput
screening to accelerate the development of new drugs. Using robotics,
this approach screens hundreds of thousands of chemicals in a short
time to identify lead compounds for drug development. Although industry
invests heavily in high throughput screening, private companies are
less likely to focus on relatively uncommon disorders such as ALS.
NINDS is trying to find the best ways to put this technology in the
hands of researchers who are focusing on ALS and other neurological
disorders. An important part of the high throughput strategy is the
requirement for simple, repeatable assays, or tests, for the
effectiveness of a potential drug. On April 10-11 NINDS, working
closely with private ALS and SMA groups, held a workshop on ``Assays
for High-Throughput Screening of Drug Candidates for Amyotrophic
Lateral Sclerosis and Spinal Muscular Atrophy'' to help guide that
effort. The meeting brought together experts from academia, large
pharmaceutical companies, small biotechnology organizations, government
and private advocacy groups. NIH will follow up with specific programs
to foster the development of assays and to make the robotics, chemical
libraries, and other requirements for high throughput drug screening
technology accessible to academic investigators.
Several other new therapeutic strategies now on the horizon may
also apply to ALS. NIH is very interested in the development of safe
and effective stem cell and gene transfer therapies, to name two areas
of medicine that have properly captured the public's attention. Stem
cells are developmentally primitive cells that can be coaxed to
multiply and to specialize to form particular cell types, such as motor
neurons. Stem cells might ultimately provide replacements for lost
cells, but these versatile cells can be used for therapy in other ways.
Stem cells, perhaps altered by genetic engineering, might augment the
tissue's ability to clear glutamate or provide neurotrophic factors.
Gene transfer therapy likewise might be employed in several different
strategies, beyond replacing defective genes, for both inherited and
non-inherited forms of disease. Providing neurotrophic factors via gene
transfer therapy is one strategy that has shown promise in animals for
ALS and other neurological disorders. While stem cells and gene
transfer therapy have great potential, each also presents difficulties
that must be resolved before their application in people with ALS, and
both require substantial investments to build a foundation of basic
biological understanding.
Developing better means to deliver therapeutic agents--drugs,
cells, and genes--to where they are needed in the brain and spinal cord
is another focus of attention with implications for ALS and many other
diseases. The blood-brain barrier (and blood-spinal cord barrier)
normally exclude many potentially helpful drugs. Surgical access to the
brain is itself not a trivial matter, even with the dramatic advances
in brain imaging to guide surgeons, and better methods for physically
introducing therapeutics to specific regions of the brain are needed.
Likewise, the ongoing efforts to develop drugs that target free radical
damage, excitotoxicity, and apoptosis may have a broad range of
applications. Just as common mechanisms of damage in many neurological
disorders provide synergies for progress, the shared obstacles to
therapy for many diseases can also have a positive effect as insights
gleaned from each may apply to others.
Although investigator initiated research proposals are at the heart
of NIH strategy, we actively stimulate research in particular disorders
when emerging scientific opportunities or public impact of a disease
warrant such intervention. On both counts we certainly believe ALS
merits special attention. The NINDS extramural program has been
reorganized with creation of a Neurodegeneration Cluster that reflects
the common themes driving neurodegeneration research. We are working to
enhance research on ALS in a number of ways, including grant
solicitations, workshops, and informal discussions with the research
community, and are working closely with ALS advocacy groups in many of
these efforts. In March 2000, NINDS released a request for applications
(RFA NS-01-004) ``Spinal Muscular Atrophy, Amyotrophic Lateral
Sclerosis, and Other Motor Neuron Disorders.'' This RFA sets aside $3
million to fund novel approaches to understanding and treating ALS,
spinal muscular atrophy, and other disorders whose cardinal feature is
a loss of motor neurons. Given the time required for investigators to
write applications and for staff to review and fund new grants,
successful proposals are expected to begin in fiscal year 2001. In
March NINDS also released an RFA (RFA NS-01-003) entitled
``Mitochondrial Function in Neurodegeneration.'' There is compelling
evidence that mitochondria, the energy factories of the cell, play an
important role in the generation of free radicals and in apoptosis in
ALS and other neurodegenerative disorders. Several of the broad NIH
efforts to provide access to emerging technologies, such as gene arrays
and transgenic mice, will also be important for ALS research.
CONCLUSION
The best strategy to find a cure for ALS is to support a broad
research program, including research focused on ALS, on common themes
in neurodegeneration, and on fundamental neuroscience, with an emphasis
on funding the best quality science. It would be a disservice to
patients and families to make promises about when this disease will be
cured. The problems ALS presents are complex and medical progress is
notoriously difficult to predict. However, most researchers, energized
by progress in fundamental neuroscience, about neurodegeneration in
general, and on ALS in particular, feel a cautious optimism that
stopping ALS and other neurological disorders is a realistic goal. We
share that belief, and will continue our efforts to speed the day when
we can better treat, cure, and ultimately prevent ALS.
Senator Specter. Dr. Fischbach, thank you very much.
STATEMENT OF TOM MANIATIS, Ph.D., CHAIRMAN, CURE
ADVISORY COMMITTEE, AMYOTROPHIC LATERAL
SCLEROSIS ASSOCIATION
Senator Specter. We are going to call our second witness--
we would like you to remain at the panel--before we go to a
round of questioning. Our second witness is Dr. Tom Maniatis,
Chairman of the ALS Association's Cure Advisory Committee, and
Professor of Molecular and Cellular Biology at Harvard. He
received his Ph.D. from Vanderbilt, his B.A. from the
University of Colorado. He has had within his own family the
tragedy of a sister who succumbed to ALS. Thank you very much
for joining us, Dr. Maniatis.
Dr. Maniatis. Thank you, Mr. Chairman. I would like to
thank the committee for all their support of NIH which I agree
with everything you have said about the importance of the work
that is being done there.
My name is Tom Maniatis. I am a Professor of Molecular and
Cellular Biology at Harvard University. I teach and direct an
NIH-funded research program in the field of gene regulation. My
lab pioneered the development of gene cloning methods and has
used these methods to study how the readout of genetic
information is regulated. Our work has had a significant impact
on the human genome project and has led to important insights
into human genetic diseases and inflammatory diseases such as
arthritis and asthma.
Recently I have been involved in an effort to identify and
initiate new directions in ALS research. My interest in this
cause was initiated by the recent death of my sister Carol from
ALS. A little over 2 years ago, Carol was an executive
secretary for a computer company in Denver, and at the age of
57 was active, energetic, and full of life. After working and
raising four children, she was at a stage in her life where she
had time to enjoy her grandchildren, spend weekends in the
mountains, and travel.
Suddenly, however, she began to experience the weakness in
her legs and would stumble and fall at work. A series of
medical tests led to the devastating conclusion that she had
ALS. Her next and final 2 years were an unimaginable nightmare
for everyone close to her. Step by step her freedom was taken
away. First, she had to leave the job she loved. This was
followed by a period when she was unable to walk at all, but
could move around controlling the wheelchair with a toggle
switch. However, even that was lost as the muscles in her arms
and legs degenerated. Then she was unable to talk. Her once
articulate and happy voice was gone, replaced by a barely
intelligible noises. Then she could not eat. This necessitated
the insertion of a tube in her stomach. Then she would choke
because she could not swallow. She was ultimately reduced to a
limp and lifeless body with a perfectly good mind inside.
Although her basic needs could be conveyed at first with
the computer and later with her eyes, she was unable to express
the complicated emotions one must feel while the most
fundamental human activities are relentlessly taken from you
day by day. At least with most other diseases, it is possible
to express the feeling of sadness and the fear of dying and to
be comforted in a meaningful way.
A year ago, 2 weeks after her 60th birthday, Carol died of
asphyxiation, leaving emotionally exhausted and deeply saddened
friends and family behind.
I am not a neurologist or even a neurobiologist, but I have
a very broad understanding of biology. I can recognize good
science, and I am familiar with the latest technical advances
that could be applied to the understanding of ALS. I have,
therefore, been involved in establishing a new cure-directed
research initiative for the ALS Association called The Lou
Gehrig Challenge: Cure ALS Initiative. Our mission is to
identify promising new directions in ALS research and to
develop new therapies. Our strategy is to recruit outstanding
investigators and exploit the latest technological advances in
biology and drug development, with a commitment to understand
the disease and find a truly effective treatment for ALS.
The Lou Gehrig Challenge: Cure ALS Initiative is funding a
number of research initiatives, including the establishment of
better animal models for the disease in collaboration with
Jackson Labs, and the development of cell based assays for high
throughput drug screening.
Efforts have also been initiated to identify new genes in
mice and humans that are involved in the familial form of ALS,
and as Dr. Fischbach mentioned, this could lead to major
insights into the mechanisms of the disease.
In addition, we have organized and are funding an
investigation of a recent report which claims an association
between a polio-like virus and the sporadic form of ALS.
Soon the DNA-sequence of the human genome will be
completed, providing exciting new approaches to understanding
ALS. This sequence information will dramatically facilitate
genetic studies and will make it possible to detect small
differences between normal and ALS motor neurons.
Another exciting new direction, which this committee has
discussed extensively, is stem cell research, as Dr. Fischbach
has mentioned. Preliminary experiments suggest that this
technology could provide new therapeutic approaches, but a
great deal of fundamental research is required to assess the
feasibility and the safety of this approach.
The cost of the Lou Gehrig Challenge: Cure ALS Initiative
is provided by a special fund established by the ALS
Association. However, we view this fund as seed capital hoping
that it will lead to exciting breakthroughs that will attract
the interest of the NIH to fund more work on ALS and the
interest of drug companies to increase their efforts to develop
drugs.
I would like to emphasize, however, that we know relatively
little about the disease. So much more basic research is
required. However, it is important to point out the fundamental
understanding of motor neurons gained in the study of ALS
research will be cost effective because of the applicability of
this understanding to other neurodegenerative disorders.
PREPARED STATEMENT
I would like to thank the chairman of the committee for
giving me the opportunity to speak and urge them to
enthusiastically support the funding of efforts to understand
and find a cure for ALS.
[The statement follows:]
Prepared Statement of Tom Maniatis
My name is Tom Maniatis, and I am a professor of Molecular and
Cellular Biology at Harvard University. I teach and direct a NIH-funded
research program in the field of gene regulation. My lab pioneered the
development of gene cloning methods, and has used these methods to
study how the readout of genetic information in the cell is regulated
during embryonic development and in response to infection by pathogenic
microorganisms.
Recently, I have been involved in an effort to identify and
initiate new directions in ALS research. My interest in this cause was
initiated by the recent death of my sister Carol from ALS. A little
over two years ago Carol was an executive secretary for a computer
company in Denver Colorado, and at the age of 57 was active, energetic
and full of life. After working, and raising four children she was at a
stage in her life where she had time to enjoy her grandchildren, spend
weekends in the mountains and travel.
Suddenly, however, she began to experience weakness in her legs and
would stumble and fall at work. A series of medical tests led to the
devastating conclusion that she had ALS. Her next, and final two years
were an unimaginable nightmare for her and everyone close to her. Step
by step her freedom, and her dignity were taken away. First, she had to
leave the job she loved. Then, she could no longer drive her car. This
was followed by a period when she was unable to walk at all, but could
move around by controlling the toggle switch on her electric wheel
chair. However, even that was lost as the muscles in her arms and hands
degenerated. Then she was unable to talk--her once articulate and happy
voice was gone, replaced by barely intelligible noises. Then she could
not eat--this necessitated the insertion of a tube in her stomach,
liquid feeding and the loss of the joy of tasting food. Then she would
chock because she could not swallow--thus, a tube was inserted into her
throat so she could breath. Carol was ultimately reduced to a limp
lifeless body of skin and bones with a perfectly good mind inside.
Perhaps the most tragic aspect of this disease is the inability to
communicate. Although many of her basic needs could be conveyed, at
first with a computer and later with her eyes, she was unable to
express the complicated emotions one must feel while the most
fundamental human activities are relentlessly taken from you day by
day. At least with most other diseases it is possible to express the
feeling of sadness and fear of dying, and to be comforted in a
meaningful way. A year ago, two weeks after her 60th birthday Carol
died of asphyxiation, leaving emotionally exhausted and deeply saddened
friends and family behind.
I am not a neurologist or even a neurobiologist, but I have a broad
understanding of biology, I can recognize good research, and am
familiar with the latest technical advances that could be applied to
the understanding and cure of ALS. I have therefore been involved in
establishing a new cure-directed research initiative for the ALS
Association. Our mission is to identify promising new directions in ALS
research and to develop new therapies. Our strategy is to recruit
outstanding investigators and to exploit the latest technological
advances in biology and drug development, with a commitment to
understand the disease and find a truly effective treatments of ALS.
We are currently supporting a number of new research initiatives,
including the establishment of better animal models for the disease,
and the development of cell based assays for high throughput drug
screening. Efforts have also been initiated to identify new genes in
mice and humans that are involved in the familial form of ALS. In
addition, we have organized and are funded an investigation of a recent
report which claims an association between a polio-like virus and the
sporadic form of ALS.
Soon, the DNA sequence of the human genome will be completed,
providing exciting new approaches to understanding ALS. This sequence
information will dramatically facilitate genetic studies, and make it
possible to detect small differences between normal and ALS motor
neurons.
Another exciting direction is stem cell research. Preliminary
experiments in a mouse model of ALS suggest that this technology may
provide new therapeutic approaches, but a great deal of fundamental
research will be required to asses the feasibility and safety of this
approach.
The expense of our new research initiatives is covered by a special
fund established by the ALS association. However, we view this fund as
seed capital, hoping that it will lead to exciting breakthroughs that
will attract the interest of NIH to fund more work on ALS, and the
interest of drug companies to increase their efforts to develop drugs.
I would like to thank the committee for supporting the recent
congressional appropriations to NIH, which have made it possible for
Dr. Gerry Fischbach the director of the NINDS to consider new research
initiatives in neuromuscular diseases. For example, in collaboration
with the NINDS the ALS association recently held a meeting at NIH to
discuss the development of cell based assays for neuromuscular
diseases. These assays would then be used in conjuction with the latest
advances in combinatorial chemistry and high throughput screening
technology, to search for new drugs for the treatment of ALS. The
meeting ended with two important objectives. First, to promote the
further devolopment of a data base of small molecules (called chembank)
and explore ways of funding the acquisition and distribution of small
molecules for drug screening in an medical/academic setting. Second, to
formulate a contract for the establishment of one or more regional high
throughput screening centers that would make it possible to search for
potential drugs in an academic setting.
Support of ALS research is cost-effective because of applicability
to other neurodegenerative disorders.
Senator Specter. Thank you very much, Dr. Maniatis.
Dr. Fischbach, in the brief period of time we have for
questioning, I would like to focus on the possibilities of
finding a cure for amyotrophic lateral sclerosis. We have
talked a little bit about stem cell research. We have talked
about the genome identification. In this week's edition of the
New England Journal of Medicine, the report was that there has
been a mapping of chromosome 21, which is associated with
amyotrophic lateral sclerosis and some other ailments.
Starting with the issue of stem cells and their extraction
from embryos, what do you see as a possibility if the full
research potential of the National Institutes of Health was
unleashed so that we eliminated the current law which prohibits
Federal funding for embryo stem cell research to really go at
this in a very concerted way? What are the possibilities? I
know you cannot speak with certainty, but what are the
possibilities for stem cell research offering a cure for ALS?
Dr. Fischbach. Certainly the possibilities are much, much
greater than they were just 2 or 3 years ago because we have
learned so much more about human embryonic stem cells. I cannot
put a number on it. It is a very difficult disorder.
Senator Specter. Explain just a bit in lay terms for people
here and people watching on C-SPAN just what the stem cell
does, how it replaces deficient cells and is characterized
accurately as a veritable fountain of youth.
Dr. Fischbach. Well, the stem cell is a cell that can give
rise to many different types of daughter cells, at the same
time renewing itself. So, it is not depleted in principle. The
key is, how many different types of cells can a stem cell give
rise to and how can it reproduce itself and for how long. In
both those categories, it appears that embryonic stem cells are
different from other types of stem cells and have more
potential for a greater diversity of cell types and for
creating large populations of stem cells, which will be needed
for useful therapeutics.
Senator Specter. So, the embryos are necessary in order to
effectively carry out stem cell research?
Dr. Fischbach. Well, ``effectively'' is the key word. In my
view, human embryonic stem cell research is the most promising
avenue of stem cell research today.
Senator Specter. Dealing with the objections which have
been raised from using embryos, is it not true that the only
ones used are those which have been discarded so that if there
is any possibility of the embryo creating a life, there is
absolutely no use of that embryo, but only the ones which have
been discarded?
Dr. Fischbach. To my understanding, that is correct.
Senator Specter. Is this analogous to the controversy which
we had on the use of fetal tissue where many had objected to
the use of fetal tissue on the ground that it would encourage
abortions? And then it was made plain and the procedure was
established to use only discarded fetal tissue so that it was
not a matter of encouraging abortions, but it was a matter of
using discarded fetal tissue where abortions had already taken
place.
Dr. Fischbach. I think the current regulation is that fetal
tissue cannot be obtained for the purpose of research, but that
discarded fetal tissue is available.
Senator Specter. My yellow light is on, so I am going to
conclude with just one more question with respect to the
identification of the gene and chromosome 21. Explain in lay
language just what that means and what the potential is for the
possibility of curing ALS.
Dr. Fischbach. Every gene that is discovered that is mutant
in this disorder offers hope that we will understand the
mechanism of the disease. And once we understand the mechanism
of the disease, we will have a much clearer view of
therapeutics. So, I think discovery of the gene is tremendously
important.
There is one additional tremendous advantage of discovering
a gene, and that is creating an animal model for the disease.
Senator Specter. How do you spell that? Repeat that.
Dr. Fischbach. A second great advantage of discovering the
gene beyond understanding the disease process is the gene can
be used to modify the genome of an animal to create a model for
the disease. And now there is a very effective model for ALS in
mice, and new therapeutics will be screened in the mice. I
think that is a tremendous advance.
Senator Specter. My red light is on, so I will turn now to
Senator Reid.
Senator Reid. Mr. Chairman, I am concerned about this drug
that we have talked about, Myotrophin. It seems the evidence is
clear that it has helped a significant number of patients, and
now we are told that because the FDA did not approve this under
their fast approval track that they are allowed by law, that
the manufacturer is no longer going to make the drug and
notified patients they will no longer be able to get this
product.
Do you have any ideas of what we can do to help this
situation?
Dr. Fischbach. Tom, you should jump in whenever you want,
if you would like.
I am not familiar firsthand with the decision not to
produce the drug any longer. It is a peptide.
Senator Reid. It is a what?
Dr. Fischbach. It is a small protein that is being used as
a medicine. It is not the usual small molecule drug that we
ordinarily take as a pill by mouth.
But the FDA decision does have an effect on industry, and I
think that is all the more reason to support the basic science
labs, to keep modifying this drug, this peptide, and to
encourage further clinical trials so that it will pass FDA
inspection and regulation.
Senator Reid. We have written to the FDA. I wrote to them
in February and got an answer back several months later. During
the months, of course--we do not have a lot of time to wait
around. I am just very disappointed in FDA.
I think that we have to come up with some way to have this
manufacturer and others continue to work on this. I would hope
through your good offices, the National Institutes of Health,
you would reach out to some of these pharmaceutical companies
and individuals who are interested in these experimental drugs.
That is what they are. We know this one has given relief to
people. I have talked to people it helps. Until we find a cure,
we have to look for these kinds of things to relieve pain and
prolong life.
I would hope that you, as a researcher, doctor at Harvard,
and you having this prominent position in the National
Institutes of Health would gather your colleagues and try to
give some ray of hope to the manufacturer. The reason they
stopped, it is a small number of people that it helps, because
we have established that there is at most at this time 30,000
patients.
Without repeating myself, we really need to come up with a
plan. As a legislator, I am having a difficult time doing that,
and I would hope that the research community would give us some
assistance.
Dr. Fischbach. Senator Reid, I would like to respond to you
in writing after we have a chance to make further inquiries and
try and understand what we can actually do right now to make
sure that research does not disappear.
Dr. Maniatis. I would agree with that. I am not fully
familiar with the details of the clinical trials, but I think
it is really important to look at those very carefully, as Dr.
Fischbach says. If improvements could be made that would
increase the efficacy, every effort should be made to do that
and support the companies who are trying to develop the drug.
Senator Reid. Thank you.
Senator Specter. Congresswoman Capps, would you care to
question?
Ms. Capps. I would appreciate the opportunity. Thank you.
I want to start out by congratulating the ALS Association
and Dr. Maniatis for your leadership in this new cure-directed
research effort of the association and commend you for focusing
in the way that you have on this targeted kind of response.
Hopefully there might be time to hear a little bit more about
how you are doing that.
As a segue, I use that to ask Dr. Fischbach if NIH is
focused in the same way. I applaud all of the efforts that have
been expressed here and fuller funding for it in general, the
genome study, all of these interrelated areas that are
benefitted by the research in gene efforts and stem cell and
all of it because it does not just affect ALS. It affects a
broad range of neurological disorders at least, others as well.
Could you comment please on whether you believe the NIH is
targeting ALS sufficiently and are there ways that we could
help in that arena?
Dr. Fischbach. First, Representative Capps, let me join you
in the sentiment that it is wonderful to see the ALS
Association bring scientists from other fields, prominent
scientists, wonderful scientists like Dr. Maniatis, and a group
that is now working with them. One of the greatest challenges
is to bring people from other fields to think about this
problem in fresh and new ways, and that is happening. We are
hopefully part of it at the NIH.
The NINDS is focusing on ALS. You have put it very well,
that we are focusing on it because it is an example of a
neurodegenerative disorder that has enormous implications for
all neurodegenerative disorders, Huntington's disease,
Parkinson's disease, even Alzheimer's disease. Similar
processes may be at work here.
But we are focusing on ALS specifically, and we have
recently released an RFA, a request for applications, dealing
with motor neuron diseases and ALS in particular. And we
sponsored a conference with the ALS Association that Dr.
Maniatis chaired on looking for new therapeutics. I believe
that our group within the Institute interested in neurorepair
and regeneration and interested in neurodegeneration spend a
great deal of their day thinking about ALS and stimulating
research in this area.
Dr. Maniatis. I would like to comment that we have been
working with NINDS. In particular, I think one of the really
exciting initiatives is an RFA on high throughput mutagenesis
screening at Jackson Labs to look for new mutations that affect
motor neurons. This could really be an extremely exciting
approach that would identify a pathway leading to the disease.
In addition, this conference that Dr. Fischbach mentioned
involved bringing people together from industry, from academic
science, and from medicine to begin to look at how one could
begin to establish new assays for detecting ALS in vitro in a
cell-based assay, and then to employ the high throughput
screening methods that drug companies have used in an academic
setting which would interface the medicine, the science, and
the drug screening. That was a very exciting conference. I
think it led to a number of proposals that are now being
followed up on.
So, from my perspective, this is an extremely exciting time
in biology, and this technology and these ideas have not been
applied to ALS, and that is what we are trying to do, to bring
together people who are experts in these new areas and focus
them on the disease.
Ms. Capps. The yellow light is on. I just want to commend
you for that kind of fresh insight, and I think that is, as you
said, Dr. Fischbach, a model for all of us. Thank you.
Senator Specter. Thank you very much, Congresswoman Capps.
Thank you very much, Dr. Fischbach and Dr. Maniatis. We are
going to continue to support you through the NIH funding, but
we are going to look for results.
Dr. Fischbach. That is fair.
STATEMENT OF STEVE BEUERLEIN, QUARTERBACK, CAROLINA
PANTHERS
Senator Specter. I would like to call our next panel, Mr.
Steve Beuerlein, Mr. Steve Garvey, and Mr. Dick Schaap. Would
you gentlemen step forward please?
This distinguished panel of sports personalities will be
adding to our knowledge base on amyotrophic lateral sclerosis.
We are going to lead off with Steve Beuerlein, starting
quarterback for the NFL's Carolina Panthers. His season this
year was highlighted with a Pro Bowl appearance. Before
starting his professional career, Mr. Beuerlein was a 4-year
starter at Notre Dame, where he set numerous records for
passing and total offense.
Mr. Beuerlein's high school friend, Jeff Sherer, age 34--
Mr. Sherer is seated in the front row--talk just a bit about
another medical problem facing quarterbacks, and that is the
excessive punishment in the National Football League. And I
asked for Mr. Beuerlein's expert opinion, as we asked not too
long ago for Troy Aikman's expert opinion, on the approach that
there are too many late hits and too many pounding into the
ground after it was plain that the ball has gone. If we have
any time after ALS, Mr. Beuerlein, we may ask for your expert
opinion on that.
Mr. Beuerlein. Let the record show that I agree with you
100 percent.
Senator Specter. In the anteroom before we started, Mr.
Beuerlein suggested legislation which I think would have fit in
with our jurisdiction over the Commerce Clause.
Thank you for joining us, Mr. Beuerlein, and the floor is
yours.
Mr. Beuerlein. Thank you, Mr. Chairman, distinguished
members of the subcommittee. It is truly an honor to be here
testifying on behalf of ALS today.
I would also like to say before I get going, that my
reception here in Washington, D.C. has been a little different
than it normally is as an opposing quarterback coming into
these parts. Generally speaking, it is not a very warm
reception, but I have been treated well in the 24 hours or so
that I have been here. I am not naive enough to think, though,
that on September 3rd, when we come here to open our season
against the Washington Redskins, that the reception will be
quite as warm. In fact, there are probably several people
sitting here today that will be getting pretty excited every
time I get knocked to the turf in that game. But I can handle
that and deal with that. That is part of my job.
My reason for being here today is very straightforward. I
am here on behalf of my friend, Jeff Sherer, who has ALS, one
of my best friends from high school. I am here to encourage
this subcommittee and all the relevant Federal agencies, as
well as the researchers who testified earlier today, and the
ALS Association to pursue all avenues possible to find a cure
for this dreadful disease. I know, Mr. Chairman, that you and
the other members of the subcommittee share this objective, and
I respect you tremendously for that.
I met Jeff back in 1979 on a football field in southern
California at Servite High School. Over the 4 years that we
played together, we developed a very close relationship. We won
a State championship my senior year, had a tremendous year. And
I have got three more of my teammates sitting over here that
came out to support this cause today as well.
But during the course of that season, I was very fortunate
to not get hit very often by anybody from the other team, and
one of the main reasons, in fact the biggest reason for that,
was my right tackle, Jeff Sherer. He was 6 foot 2 and 300
pounds, conservatively as a freshman in high school.
As he caught up with his body, he became a tremendous
football player. I never worried about the right side of my
offensive line because I knew that Jeff had it taken care of.
He played with tremendous heart. Every time I knew that I could
count on him no matter what the situation was, and he today he
plays with tremendous heart every time he wakes up in the
morning and fights this terrible disease.
Away from the football field, Jeff always lived his life
with tremendous passion, and he still does. He loves to smile,
loves to make other people smile. If I could give just one
example. I always remember every time we got together as a
group, all the ladies would always fight to find a way to get
close to Jeff, and I never could figure out why. But the reason
was because they knew if they were close to Jeff, they probably
had the first chance of getting that famous Jeff Sherer back
rub.
I used to find myself scooting in there once in a while as
well.
But the point of the matter there is that Jeff has always
been a tremendous friend, always thinking about other people.
He had dreams like all of us, big dreams, and when he met his
wife and married her, Marya--she is here today as well--he
started to live out a lot of those dreams. She is an
unbelievable woman. She is a rock, and I have no idea where
Jeff would be without her today. But they have three kids: a 4-
year old, a 22-month old, and a 5-month old.
When Jeff was diagnosed with ALS 2\1/2\ years ago, a lot of
those dreams were put on hold for obvious reasons. As you know,
Mr. Chairman, this horrible disease is incurable and it is
relentless. I believe that Jeff knows, as well as anybody, that
he is fighting for his life. He is battling for his life
against a disease that in its most tragic sense has never been
defeated.
At the age of 34 today, this once tremendous athlete, Jeff
no longer has the use of his arms or his legs. His 5-month-old
son he has never been able to pick up and hold and tell him
that he loves him. Imagine the frustration and the pain that
goes along with that.
What can we do? Well, every night my wife and I say our
prayers and we think about Jeff and his family. We pray that
the Lord will guide them through this difficult disease, this
difficult time. But while we hope for a miracle from heaven, it
is up to us to use all of our resources possible to find a cure
here on earth for this disease.
In conclusion, I would like to say, Mr. Chairman, that I am
here for my friend Jeff Sherer. I am also here for all those
people living with ALS in this country and their families and
those who care about them, and I am here for the 14 Americans
that today will find out that they too have ALS.
My testimony today began with a little bit of football
humor. My relationship with Jeff Sherer began on a football
field back in 1979, and I would like to submit to this
committee that we take an aggressive football-style attacking
approach to finding a cure for this disease. We need to pursue
the cure. We need to hunt the disease down and stop it cold in
its tracks.
PREPARED STATEMENT
Senator Specter, from what I have seen today and from what
I have read and observed about you and your past, your
aggressive style of leadership, I have no doubt that you are
the right man to be sitting in this chair and leading and
orchestrating this effort. On behalf of all the people that are
affected with ALS and those that care about them and their
families, this is a desperate situation. In football terms, we
could say it is fourth in goal, the clock is running, we have
got our backs up against the wall. Just ask Jeff Sherer or any
of the other ALS patients that are here today.
Thank you and God bless you.
[The statement follows:]
PREPARED STATEMENT OF STEVE BEUERLEIN
Thank you, Mr. Chairman, and the other distinguished members of the
Subcommittee, I am both humbled and honored to testify before you this
morning.
I appear before you today for a reason that is both straightforward
and profound. My best friend--or one of my best friends from
childhood--Jeff Sherer, has ALS. I am here to do my small part to
encourage this Subcommittee, all of the relevant federal agencies, the
researchers who testified earlier, and the ALS Association to join
forces to find a cure for ALS. And, Mr. Chairman, let me say up front
that I know you have the same objective because it is only through your
leadership that we are here this morning,
Let me talk for a few moments about my friend, Jeff.
He and I played football together in high school. Jeff was the
starting offensive right tackle on a team that won the California state
championship.
Jeff was 6-foot-3, 280 pounds. He was so large that we
affectionately nicknamed him, ``the Coke Machine.'' When he blocked
someone, the other guy would basically disappear . . . Jeff would just
kind of engulf him.
I never worried about Jeff as my right tackle, He was so big and so
powerful and so smart, he always came through.
Jeff went on to college . . . began his career . . . got married to
a wonderful woman, and began to raise a family. Today, Jeff and his
wife, Marya, have three small, children--a daughter, Madison, and two
sons, Jeff, who is 22 months old, and A.J., the baby who is just 12
weeks old.
Jeff had big dreams, just like all of us have dreams for our lives.
Jeff had begun to live his dreams.
And then he was diagnosed with this horrible, incurable disease
known as amyotrophic lateral scleroses, or ALS. Today, all of Jeff's
dreams have been put on hold. He is, quite literally, battling for his
life against an opponent that is relentless. Jeff is up against a
disease that, in the most horrible and tragic sense, is undefeated.
As you know, Mr. Chairman, there is no known cause, prevention or
cure for ALS.
And so, my best friend and former teammate, Jeff Sherer, at age 34
is confined to a wheelchair, This once strapping athlete is now unable
to move his arms or his legs. This father of three young children
breathes with the aid of a machine at night to help him sleep. Jeff now
even has trouble speaking.
I pray for Jeff and his family every night. I pray for the Lord to
guide and comfort, all of them through this terrible disease.
But I also know that while we must pray for a miracle from heaven,
we must work together on Earth for a cure. That's why I am encouraged
by the testimony I have heard from Dr. Fischbach and Dr. Maniatis, and
by the discussions I have had with the leadership of the ALS
Association,
I am here for Jeff Sherer. But I am also here for all of the ALS
patients, family members, and other ALS activists who have filled this
hearing room. I am here for the Americans who already are living with
ALS. And I am here for the 14 Americans who will be called into a
doctor's office today and be told that they have been diagnosed with
ALS.
Mr, Chairman, I began my testimony today by talking briefly about
football. After all, my friendship with Jeff Sherer began on the
football field. It has been said that the perfect middle linebacker
must be ``AGILE, HOSTILE and MOBILE''--pronounce each with a long ``I''
so the words rhyme.
Mr. Chairman, I would submit to you that we need the same approach
to find a cure for ALS. We need an approach that is AGILE, HOSTILE, and
MOBILE. We need to pursue a cure for ALS, hunt this disease down, and
stop it in its tracks. Senator Specter, from what I have seen and heard
today . . . from what I have read and observed about your style of
aggressive leadership, I know that you are the right Senator to be
sitting in that chair to help lead and orchestrate this effort.
Thank you, and God bless you.
Senator Specter. Thank you very much, Mr. Beuerlein.
The subcommittee is going to have to take a very brief
recess because there is a simultaneous Judiciary Committee
executive session down the hall taking up the issue of
subpoenas on a subcommittee which I chair. The recess is going
to be very brief, and I will return in just a moment or two to
pick up with the testimony of Mr. Garvey and Mr. Schaap. So,
the committee stands in brief recess.
The appropriations Subcommittee on Labor, Health and Human
Services, and Education will now resume. That may set the
record for the briefest recess of a Senate subcommittee.
When I walked back in and saw all of the photo taking and
all the autographs and all the handshaking, the thought crossed
my mind that that was probably the best part of the hearing, to
have the recess and give you a chance to get the Schaap,
Garvey, Beuerlein autograph session and the photographs.
STATEMENT OF STEVE GARVEY, FORMER FIRST BASEMAN, LOS
ANGELES DODGERS
Senator Specter. We now turn to one of baseball's great
sports personalities, Mr. Steve Garvey, 19 years as first
baseman for the Los Angeles Dodgers and San Diego Padres, 5
World Series, 4 Gold Glove Awards, nationally MVP, most
valuable player, in 1974 and the most valuable player in the
1978 and 1984 National League Championship Series.
He retired from baseball in 1987, ending a streak of 1,207
consecutive games. That is quite a record, quite a streak, not
quite up to Lou Gehrig's 2,130, but phenomenal.
The biographical material I have also lists Mr. Garvey
having played in 10 All Star games. I think that probably omits
the Softball All Star game which was played in Philadelphia in
1995 before the regular All Star game after you had retired.
That was a Softball All Star game of particular moment to me
because, as a Philadelphian, I was on the team. Based on having
played second base for the Russell, Kansas Junior American
Legion baseball team when I was 15, which got to the semi-
finals, I felt a little bit at home at second base until the
ground balls started coming my way. And Keith Hernandez was
playing first--I am taking more time than I should, but this
was quite a day for me. There was a sharp grounder hit at me. I
did not know quite what to do until Keith Hernandez came over
from first base and scooped it up and got the player out.
You may not remember this, Mr. Garvey, but you got the base
winning hit. You hit one to my right and Hernandez could not
quite handle it.
But that was an exciting All Star game for me.
I know there is a lot of excitement in having you here and
the others to talk about amyotrophic lateral sclerosis. So,
thank you for joining us and the floor is yours.
Mr. Garvey. Mr. Chairman, you still maintain your quickness
with that brief break, as we noticed back in 1995. It is nice
to be with you again, members of the subcommittee. Steve, Mr.
Schaap, and I now realize that we are a little too close to
you. A Panther in Redskin country is a little perilous here.
Your presentation was wonderful.
I am Steve Garvey, former first baseman with the Los
Angeles Dodgers and San Diego Padres, and part of Lou's team.
Dick and I and Steve a number of other committed people are
part of Lou's team, along with this wonderful group of men and
women behind us. We are here simply as foot soldiers. The two
committed doctors who made presentations to you are at the
front line in fighting this disease.
It is ironic that such a crippling disease as ALS would hit
another first baseman, one whose incredible strength, stamina
and consistency earned him the name ``The Ironman.'' I am
speaking, of course, about Lou Gehrig, whose name will forever
be linked with this disease. ALS struck Lou Gehrig at age 36,
and he expired 2 years later.
I have been blessed that no one in my family has been
affected by ALS. However, I do know that ALS has been a death
sentence. It affects the nerves and muscles, making them
unusable. One day you find yourself tripping over your own
feet. Then you seem to have trouble lifting small objects. Your
speech starts to slur. You cannot swallow. Eventually your body
becomes paralyzed while your mind stays alert. You and your
family prepare for the inevitable. Essentially you become a
prisoner in your own body.
The life expectancy of an ALS patient averages 2 to 5 years
from time of diagnosis. A simple example was the time that we
have sent our children to college for a 4 or 5 year span.
ALS can strike anyone. It knows no ethnic or racial
boundaries, although men are 20 percent more likely than women
to get ALS. You are probably aware of numerous celebrities have
died from ALS like actor Robert Niven, Michael Zaslow, Sesame
Street creator Jon Stone, fellow major leaguer Catfish Hunter,
and someone who was special to Congress, Jacob Javits.
But ordinary people, those close to your heart, could be
ALS' next victim. It might be your grandmother, your daughter,
your son, or your wife. No one is immune. And the devastation
of watching a loved one die is coupled with the fact that the
medical costs for treating ALS can reach $200,000 a year.
The ALS Association's ultimate goal is to find a cure for
ALS. But until then, we want to improve the quality of life for
those who have been diagnosed with ALS. Currently the ALS
Association is funding 80 live research projects.
A woman like Shelbie Oppenheimer, an ALS patient, who will
speak to you today. ALS patients like Corinne Werdel. Let me
tell you just briefly about Corinne. She has a pacemaker, a
feeding tube, and has been on a ventilator for more than 2
years. She has lost all movement except for the muscles in her
face which she uses to work a computer. Corinne has the courage
to continue her life. She is a true heroine.
PREPARED STATEMENT
We are with you today because we believe in you. Using
baseball terms, we have stepped up to the plate, we have made
our pitch, even for an old first baseman, and it may only be 80
miles per hour, but we are asking you, the committee, to hit a
home run for us. We stand on guard for you. We are your foot
soldiers. We are here to support you.
May God bless you and as the Apostle said, ``We shall run
the good race, fight the good fight, do good deeds from a good
heart, be kind and just, fair in principle, and in the end we
shall succeed.'' Thank you.
[The statement follows:]
Prepared Statement of Steve Garvey
Mr. Chairman and Members of the Subcommittee, thank you for
providing me with the opportunity to testify. I am Steve Garvey and I
am a former first baseman with the Los Angeles Dodgers and the San
Diego Padres.
It's ironic that such a crippling disease as ALS would hit another
first baseman, one whose incredible strength, stamina, and consistency
earned him the nickname, ``The Ironman.'' I am speaking, of course,
about Lou Gehrig, whose name will forever be linked with the disease.
ALS struck Lou Gehrig at age 36. He was dead two years later.
I have been blessed that no one in my family has been affected with
ALS. However, I do know that ALS has been a death sentence. It affects
the nerves and muscles, making them unusable. One day, you find
yourself tripping over your own feet. Then, you seem to have trouble
lifting the smallest objects. Your speech starts to slur. You can't
swallow. Eventually, your body becomes paralyzed, while your mind stays
alert. You and your family prepare for the inevitable.
The life expectancy of an ALS patient averages two to five years
from the time of diagnosis. Think of it this way: In the time it took
for you to go through college, that would be how long you'd likely have
left to live after being diagnosed.
ALS can strike anyone. It knows no ethnic or racial boundaries,
although men are 20 percent more likely to get ALS than women. You are
probably aware of numerous celebrities who have died from ALS like
actor Robert Niven, Sesame Street creator Jon Stone and fellow major
leaguer Jim ``Catfish'' Hunter. These celebrities' tragic deaths helped
to bring ALS into the spotlight. But ordinary people--those close to
your heart--could be ALS's next victims. It might be your grandmother .
. . your daughter . . . your wife. No one is immune. And the
devastation of watching a loved one die is coupled with the fact that
the medical costs for treating ALS can reach $200,000 a year.
The ALS Association's ultimate goal is to find a cure for ALS. But,
until we can find a cure, there should be ways to improve the quality
of life for those who have been diagnosed with ALS. Currently, the ALS
Association is funding 80 ``live'' research projects.
Because of what was learned from such research, Rilutek, a drug
approved by the FDA, was developed and was found to modestly slow down
the progression of ALS. Rilutek enables ALS patients to live a few
months more.
You may think, what are a few more months of life if you are
inflicted with this disabling disease? Ask that question to Shelbie
Oppenheimer, an ALS patient who will speak to you today. Ask one of the
thousands of ALS patients like Corinne Werdel. Currently, Corinne has a
pace-maker, a feeding tube, and has been on a ventilator for more than
two years. She has lost all movement except for the muscles in her
face, which she uses to work a computer. Corrine has the courage to
continue her life.
With the Lou Gehrig Challenge/Cure ALS research initiative--the
largest research effort ever undertaken to cure ALS--there is more that
can be accomplished. Funded by ALSA's $25 million, five-year research
initiative, scientists, using powerful computers, will be able to test
thousands of new chemical combinations simultaneously and assess their
potential as a treatment or as a cure for ALS.
With the Lou Gehrig Challenge, the ALS Association and a selected
committee of researchers will actually take the initiative themselves
to determine what questions need to be answered to find a cure for the
disease. Then, the Committee will decide who are the best researchers
and institutions to conduct particular research projects.
With increased government interest in ALS, including the king of
partnering the National Institute of Neurological Disorders and Stroke
has already undertaken in the ALS Association's research--for which we
are most grateful--and advanced technologies available to research, a
cure could be found for ALS in this decade.
That is why I traveled here to Capitol Hill and volunteered my time
to speak for just five minutes. Although ALS has not affected my life
directly--it could. There are 14 new cases of ALS each day. That's a
new case every one hundred minutes. We need to find out why ALS
happens. And, of course, we need to find the cure. That's why I am
here.
That's why these ALS patients, their family members, and other ALS
activists have packed this hearing room.
Of course, Mr. Chairman, none of us would have had this opportunity
without your great leadership of this Subcommittee. We respect you, the
other members of the Subcommittee, and the fine work of your staff led
by Betti Lou Taylor.
Thank you for the opportunity to add my voice of support to
everyone who is working so hard to find a cure for ALS.
Senator Specter. Thank you very much, Steve Garvey, for
those inspirational words.
STATEMENT OF DICK SCHAAP, HOST, ESPN'S THE SPORTS
REPORTERS
Senator Specter. We now turn to Mr. Dick Schaap, host of
ESPN's The Sports Reporters. Mr. Schaap began his broadcasting
career in 1969 and is cohost of the Joe Namath Show. Perhaps he
will tell us who the other cohost was. During his distinguished
career, he has reported on sports and non-sporting events with
many features on 20/20 and on ABC's World News Tonight,
received numerous Emmy Awards for his reports on AIDS, the
Olympics, cultural events, authored 31 books. A Brooklyn
native. He is a graduate of Cornell University and the Columbia
University Graduate School of Journalism.
Thank you for joining us, Mr. Schaap and the floor is
yours.
Mr. Schaap. Thank you, Mr. Chairman, for this opportunity
to share my thoughts at this critical ALS hearing.
First of all, my cohost was from Pennsylvania, from western
Pennsylvania, Mr. Namath.
Was that the year the Jets won the championship?
Mr. Schaap. Yes, it was. I was a front runner and I hope I
am here too.
Second, if all the athletes I covered were as articulate as
Steve Beuerlein and Steve Garvey, I would have a very easy job.
I am a journalist. I cover mostly sports, but I have
written books about murderers, drug addicts, comedians, and
even politicians.
I am fortunate that no one in my own family has been
directly affected by amyotrophic lateral sclerosis. But as a
sports writer I know how devastating the disease can be, how it
can strike people as sturdy as Lou Gehrig and Jim Catfish
Hunter, each of whom was an Ironman in his own way. I have
worked for several years, side by side, with Mitch Albin, whose
eloquent and moving account of his college mentor living and
dying with ALS, his professor, Morrie Schwartz, who set a noble
example for all of us. One quote from Morrie that Mitch used
was he was intent on proving that the word dying was not
synonymous with useless, and I think the people here today
prove that eloquently.
For the past 5 years, I have been the master of ceremonies
at the Lou Gehrig's sports banquet, the Nation's largest fund
raising event for the ALS Association. There I have met so many
wonderful and brave men and women at this banquet. The
following year I have come back and seen their husbands or
their wives alone, and I have known, without asking, that those
people have died of ALS, as so many people do each year.
I could reiterate some of the things that the doctors said,
but they are far more expert on that than I am. My field is
people and I worry about people who suffer from all illnesses,
and particularly from this dreaded illness. I hope that you and
your subcommittee, with your help and with your leadership,
through you people, that some day when I emcee one of these Lou
Gehrig dinners, I will inquire into a wife or a husband and I
will be told he or she has recovered. When that happens, I will
consider myself one of the luckiest men on the face of the
earth.
Thank you.
[The statement follows:]
PREPARED STATEMENT OF DICK SCHAAP
Thank you, Mr. Chairman. I appreciate the opportunity to appear
with these other panelists at this important hearing on ALS.
As you may know, I am a reporter. Although I have covered a variety
of news stories over the years, I am probably best known as a sports
reporter.
ALS has not directly affected my family. But as a journalist for
the past five decades, I have covered the careers of such athletes as
George Brett, Kent Hrbek, Terry Steinback, and Jim ``Catfish'' Hunter.
George Brett lost his best friend to ALS . . . both Kent and Terry
lost their fathers to ALS . . . and, of course, Catfish Hunter died of
ALS last year.
Mr. Chairman, I believe in the words of the poet, John Dunne, who
wrote: ``No man is an island, entire of itself . . . any man's death
diminishes me, because I am involved in mankind.''
The death of those men diminishes me . . . they diminish all of us.
That's in part, why, for the past five years, I have served as the
Master of Ceremonies at the Lou Gehrig Sports Banquet in New York City.
It is the nation's largest annual fund raiser for the ALS Association.
During the past five years, we have raised a total of more than $5
million.
I support the ALS Association because it is the only national, non-
profit voluntary health organization dedicated exclusively to the fight
against ALS. ALSA aggressively encourages the identification and
funding of research into the cause, means or prevention, and cure of
ALS. ALSA helps patients and families cope with the day-to-day
challenges of living with ALS. Some of these patients and families have
traveled to be here today.
As some of the other panelists have stated, these men and women are
extremely courageous. They deserve our commitment in the fight against
ALS.
Mr. Chairman, I began by alluding to my career as a journalist. I
am trained to report the facts. In closing, I would like to share a few
important facts for your Subcommittee to consider as you work through
the appropriations process this year.
Fact Number One: ALS and other neurodegenerative disorders already
present a major public health challenge. That challenge will grow in
the years to come. Within the next generation, neurodegenerative
disorders will rival heart disease as the leading cause of disability
and death in this country.
Fact Number Two: Current research indicates that a seemingly
diverse group of syndromes, from epilepsy to depression, share key
features and mechanisms. Research into ALS will yield benefits for all
neurodegenerative disorders. Likewise, advancements in our
understanding of neurodegeneration will benefit Americans with ALS.
Fact Number Three: There are now new opportunities for study, and
much shorter time frames for converting research findings into clinical
applications. It's clear from the testimony today that if NIH, NINDS,
CDC, and other relevant federal agencies continue to join forces with
private organizations such as the ALS Association, we will find better
treatments and, ultimately, a cure for ALS.
Mr. Chairman, your Subcommittee is in a position to be the catalyst
for quantum leaps forward. Thank you for your continued leadership in
this area.
Senator Specter. Well, thank you, Mr. Schaap, for those
profound comments.
This subcommittee is determined to do the maximum for
funding for biomedical research. We have undertaken the cause
because we now know that out of every 100 applications which
are filed for research grants, only 30 are accepted, and it is
impossible to know what is behind those other 70 doors out of
the 100 which might be produced.
We are a very wealthy Nation. Our budget this year for the
Federal Government is $1,850,000,000,000. Do you know how much
money that is? I do not think anybody else does either.
They say if you had an enormous auditorium like this one,
there is insufficient space to stuff $10,000 bills, not enough
room. Now, that is the Federal budget, and we have an economy
which is in the $7 trillion to $8 trillion. That means we have
the potential to do what we choose once we establish
priorities. The National Institutes of Health have had
remarkable results on what they have done.
These are a line of ailments which affect everybody. When
you talk about cancer, if you live long enough, they say you
are bound to get it, and the problem is breast cancer in young
women or middle-age women or even old women or prostate cancer,
or heart ailments or Parkinson's disease or Alzheimer's.
And it is a real battle, as we find that there are
opportunities, like on stem cells, to get the full medical use.
They have already shown the effectiveness on Parkinson's, and
the question is about amyotrophic lateral sclerosis. That is
why we focused on that and the identification of the genomes,
all the genes in the body. So, those doors can be opened.
Senator Harkin and I and this subcommittee have had a tough
job on getting the funding. This year was the first time we got
a majority of Senators to approve our resolution for increased
NIH funding, 54 to 46. 3 years ago, we lost 63 to 37, and then
we lost 59 to 41, and then about 55 to 45. And for the first
time, this year we got a majority of the votes because we have
been talking in this hearing room, which is carried on C-SPAN
and some of the national networks, about what it can mean, and
people are motivated to call their Senators and call their
Congressmen, tell them how important they think it is because
it touches everyone and it is a matter of priorities. There is
nothing we cannot do on this subject if we make up our minds to
do it and set the priorities.
So, let me ask you three men, with some experience that you
have had as celebrities in attracting attention. Sometimes
these hearings are criticized. Why are you bringing in
celebrities? What is the point? And I was interviewed yesterday
by ABC. Why are you bringing in celebrities?
And I make a very simple point, and it is the point that
when Michael J. Fox comes in and testifies about his own
problem with Parkinson's, people see some of the incipient
symptoms, and they know he is leaving his television show, and
they associate with him. He is the guy next door. They say,
hey, that is serious. If we can cure Parkinson's, let us do it.
Let us put our money there instead of somewhere else. There are
a lot of priorities, but this ought to be elevated.
Or as I said before, Christopher Reeve comes in, and we all
see Christopher Reeve on his high flyer in his Superman
uniform. And he comes in and he is in the wheelchair, as so
many men and women who are here today with ALS, and his neck is
in a very rigid position because his spinal column was severed.
So, if it happened to Superman, it can happen to anybody.
And Elizabeth Taylor comes in to testify about AIDS, and
that is sort of a tough item. It is of epidemic proportion, but
there is just a little bit of public resistance to funding for
AIDS. But if Elizabeth Taylor says something is worthwhile, a
lot of people give it a little extra consideration.
Now, maybe it should not be that way. Maybe if Betty Brown
came in, they ought to pay as much attention, or if Steve Mills
came in, they ought to pay as much attention as Steve Garvey or
Steve Beuerlein. But they do not.
So, let me start with you, Mr. Schaap. You are the author
of 31 books. You corralled Joe Namath to cohost his show. ABC
may run you tonight. They will not run my question of you, but
they may run you tonight on why Dick Schaap is an effective
witness to get funding for amyotrophic lateral sclerosis. How
about it?
Mr. Schaap. Well, I think all three of us up here have been
associated with many different causes and with many diseases
that need help from your committee and from the medical
community. This is one disease that is particularly close to us
because of its roots in the sports community, because of the
fact that it is named after an athlete. It is commonly called
Lou Gehrig's disease. And because all of us have seen Steve
Beuerlein up close with a friend and me with people who I just
meet casually and people Steve knows. How devastating this
disease can be. To me it is frightening if there is any disease
in this day and age for which there is no known cure, no known
cause, and no real treatment.
With the state of science today, I almost feel there is no
excuse for that, and the only excuse is the one, of course, you
mentioned, the shortage of available funds. I think this is a
disease that can be defeated and it is a disease that, in
defeating it, will also help defeat other diseases.
Senator Specter. Well, Mr. Schaap, why will people listen
to Dick Schaap more so than Dick Smith or Dick Brown?
Mr. Schaap. They probably should not, as you pointed out,
but over the years I have developed a certain amount of
credibility, and I would think that is probably rare among
journalists these days.
Senator Specter. How do journalists rate with elected
officials on the unpopularity scale?
Mr. Schaap. When I was covering politics, Senator Robert
Kennedy once asked me if I missed covering sports, and I said,
not really, it was a lot like covering politics, except that
the athletes were not smart enough to lie.
But his response to me was everybody in your profession
lies too.
Senator Specter. We may have some athletes who will
challenge you, at least on the smart point.
Mr. Schaap. Some have become Senators.
Senator Specter. And some Senators have become athletes.
The athletes here may challenge you on the issue of how
smart they are. They will not challenge you on that lie issue.
They will agree that athletes do not lie.
Mr. Schaap. Since we have built up a certain following, if
we say what we believe, some people are going to pay attention.
Even if it is only a few people, even if it is people who have
misplaced faith in my credibility, still it will help.
Television is a tremendously powerful medium. People believe
what they see on television. When I was working on a book with
Peter Falk and he was playing a lawyer at the time on
television, we had people turn themselves into him while we
were on the streets. People believe, maybe not as much now as
they did 20 years ago, but there is a great deal of trust and
credibility that goes back and forth. I hope that can be used
for causes that deserve it, such as this.
Senator Specter. Well, I think you put your finger on the
point of credibility. People think they know Dick Schaap, and
people trust Dick Schaap. So, when Dick Schaap says something,
it has some resonance.
Steve Beuerlein, is it possible to bring Jeff Sherer up to
the table into the camera range? I think people would be
interested in your talking a little bit more about Jeff Sherer.
Welcome, Mr. Sherer.
I see from your prepared statement, Mr. Beuerlein, that you
say that Jeff Sherer was so large that he was affectionately
known as the Coke Machine.
Mr. Beuerlein. Yes.
Senator Specter. And when he blocked someone, you said here
the other guy would basically disappear. Jeff would just kind
of engulf him.
Jeff, are you in a position to confirm or deny that?
Mr. Beuerlein. I think he is agreeing that he was a very
large man.
Senator Specter. Just nod yes if you agree.
So, how about it, Steve? What did he do to those opposing
players? And this was high school?
Mr. Beuerlein. High school, and he went on to play college
football as well at Long Beach State in southern California. He
became a very good football player. But he was a large man. He
used his strength very well. He would literally swallow up a
defensive lineman a lot of times. If he had a chance to get a
guy down, he did not hesitate to throw that big body on top of
him either.
Senator Specter. Is that Mrs. Sherer? Would you step
forward please? Mrs. Sherer, why do you not have a chair next
to Mr. Beuerlein and sit down and relax.
Tell us a little bit about Jeff. First of all, how long
have you two been married?
Ms. Sherer. 6 years this April.
Senator Specter. And Steve referred to a son.
Ms. Sherer. We actually have two sons and a daughter.
Senator Specter. And when did Jeff first know he had ALS?
Ms. Sherer. Jeff started showing symptoms in April of 1997.
He had slurred speech. He has what they call the bulbar onset.
Actually it was quite humorous because we would go places and
people would think that he had been drinking. So, they would
threaten to cut him off, but it was just because he had this
slur.
After about 6 months, after a lot of doctors' visits, it
was finally diagnosed in January of 1998.
Senator Specter. Of 1998. So, the diagnosis was just 2\1/2\
years ago, and the onset was just 3 years ago.
Ms. Sherer. Correct.
Senator Specter. Tell us a little bit about what happened
to Jeff during the course of the last 2\1/2\ years physically.
Ms. Sherer. Well, as we found out through our experience
with other members of the ALS Association, it really affects
everybody differently. So, Jeff lost his speech first. It then
moved into his hands and then down to his legs. So, he
currently has no use of his arms. He speaks mostly through me.
A lot of his close friends can understand him with a little
interpretation. He basically has no use of his legs either.
Right now he is starting to have respiratory problems. So, he
has moved into that stage, which is a pretty serious stage.
Senator Specter. Well, he is a real fighter though. He is
still in there battling, and you are at his side.
Are your children here today?
Ms. Sherer. Our children are not here today. We chose to
make the trip without them, but we do bring pictures, though.
If you would like to see them, I would be more than happy to
show them to you.
We definitely brought many family members and friends, a
lot of our teammates.
Senator Specter. Do you still live in California?
Ms. Sherer. Still live in California, yes.
Senator Specter. Well, we appreciate your being here
because when people see Jeff Sherer and hear Steve Beuerlein
talk about his decimating opposing lineman and see the
situation today, it produces an inevitable reaction that we
ought to be doing something about it, if it is humanly possible
to do it.
Ms. Sherer. Thank you very much for the opportunity to let
us be here.
Senator Specter. If it is possible to prevent people from
getting ALS in the future, we ought to be doing it.
Mr. Garvey, let me come back to you with thoughts you might
have. I know you have been associated with many causes. You
have had a phenomenal career and people look up to you in all
walks of life. What is your recommendation about how to develop
more public understanding and support for medical research to
try to cure ailments like ALS?
Mr. Garvey. Mr. Chairman, just a brief footnote. There was
I think one time back in the 1970's or early 1980's where seven
athletes ran for office and seven journalists ran for office.
With due respect to Mr. Schaap, the seven athletes won.
We can draw our own conclusion. If Mr. Schaap would have
won----
Mr. Schaap. I draw my conclusion from that.
Mr. Garvey. Quite simply, my wife Candace, our children,
and myself have a simple family philosophy, and that is life is
God's gift to us. What we do with it is our gift to God. For
those of us who have been blessed with strong minds and strong
bodies, whether it is the ability to write, the ability to
lead, the ability to play a sport, to be a business leader or
religious leader and then to be able to take the recognition
from that and stand in front or sit in front of a subcommittee
or thousands of people in front of television cameras and be
able to speak what you truly believe in with your heart and
your soul and to be able to introduce people like Jeff and his
wife and the other patients here today, it is a wonderful
opportunity to give back. We do not always have that
opportunity. Your words today have reinforced to us your
commitment and your dedication and your influence on helping us
find a cure for ALS.
When we talk to our children, we always say either they get
it or they do not get it. For those in the media that question
why people like Dick and myself and Steve and Blair and anyone
else would take the time to advocate and stand up for something
we believe in, I challenge them to take their name and their
visibility and their opportunity to reach millions of people
and join Lou's team or stand up for another cause they believe
in because it is very easy to criticize. Critics are many. It
is those people that actually stand up, that look beyond the
critic, and see the end in sight are the ones that my family
and everyone here applaud. That is our philosophy.
Senator Specter. Well, thank you very much, Mr. Garvey.
I have just one final question for you. Who was the
toughest pitcher you ever faced?
Mr. Garvey. I have had the opportunity to face many Hall of
Famers, but I always found Phil Niekro the thoughest because he
threw the knuckle ball. He did not know where it was going. The
catcher did not know where it was going. So, it was extremely
difficult to hit, very tough.
Just one final question for you, Steve Beuerlein. Who was
on the other end of the toughest sack or knockdown you ever
sustained?
Mr. Beuerlein. The toughest one. There have been so many.
We were talking earlier in the chambers in the back about Troy
Aiken having four known concussions, and I made the point that
those are the only ones that he knows about. He probably had
several more. I have not had any documented in the NFL, but a
lot of hits I do not even remember. So, it would be hard for me
to put my finger on it.
If I had to put my finger on one, it goes back to when I
was playing at Notre Dame. A man named Cornelius Bennett was
playing for the University of Alabama, and I was running the
naked bootleg where I was faking the run one way and trying to
sell----
Senator Specter. We all know what a naked bootleg is.
Mr. Beuerlein. I know you do, but I came out of there. And
then Cornelius Bennett did not bite on the fake, and he met me
in the back field, put his on my chin and drove my head into
the artificial turf which is like playing on this carpet right
here. So, it did not feel very good.
Senator Specter. Well, you made a mistake. You should have
Jeff Sherer as your offensive linebacker.
Mr. Beuerlein. I would have loved to have had that
opportunity.
Senator Specter. Well, thank you very much, Mrs. Sherer,
Mr. Jeff Sherer, Steve Beuerlein, Steve Garvey, and Dick
Schaap.
Those who may be watching on C-SPAN, which is carrying
this, we would ask you if you think more money ought to be
directed to ALS, call up or write to your Senators or to me. We
will tabulate them and we will put them up on the big board.
Thank you all very much.
We now call our final panel: Mr. Blair Underwood, who stars
as Dr. Ben Turner; Ms. Shelbie Oppenheimer, and Mr. Steve
Rigazio. If you would come forward.
Mr. Hickock. Excuse me, sir. Excuse me. I have ALS. I know
I am out of order. I am very sorry. You need to hear from
somebody who has got ALS who has a mouth and can speak.
Senator Specter. We would be pleased to hear from you. Will
you identify yourself?
STATEMENT OF GREG HICKOCK, ALS PATIENT, JACKSON, MI
Mr. Hickock. My name is Greg Hickock. I am from Jackson,
MI.
I want to fire you up so hard that you lose weight, you got
5 hours of a sleep of night, whatever you need to do. We do not
need you to take action before you need to or whatever, but we
need to get it through there. We need to have people aware of
what is going on.
You talk about stem cells. I come across the country in my
wheelchair. I heard at Johns Hopkins University from a
researcher who is working with stem cells. It would cover a
whole lot of areas. It would cover Alzheimer's, Parkinson's. It
would cover the nerves. The stem cells--the research shows that
they can grow new organs with it. It would cover a whole lot.
Senator Specter. So, Mr. Hickock, you are in favor of stem
cell research.
Mr. Hickock. I am in very favor of it.
Senator Specter. Tell us a little about yourself, about
your own background. First of all, where do you live?
Mr. Hickock. I am sorry. I might appear as a scum bag. I am
sorry.
I am from Jackson, MI. We live northwest of Jackson, MI.
Senator Specter. OK, Mr. Hickock, you look to me like a
citizen and a taxpayer.
Mr. Hickock. I am a citizen and a taxpayer.
Senator Specter. We have got time to hear you.
Mr. Hickock. Thank you.
Senator Specter. Tell us when you developed ALS?
Mr. Hickock. It was diagnosed in November of 1995. Symptoms
before that--I do not know for sure when it started.
Senator Specter. In 1995.
Mr. Hickock. Yes, sir. In 1995 I was working as a field
engineer at the time. I was working as a field engineer and
they told me I needed to quit because I was working with high
voltage and they did not need the liability. I do not want them
to have the liability.
Senator Specter. What were the first symptoms that you
felt?
Mr. Hickock. The first symptoms that I felt was I felt
myself not being able to walk any farther. I had to stop in the
middle of a parking lot and rest.
Senator Specter. And what happened after that?
Mr. Hickock. It was very cold. I figured if I was going to
live, if I was going to survive, I needed to pick my suitcases
up and get to my car.
Senator Specter. And go someplace else to live do you mean?
Mr. Hickock. No. I was on a business trip at the time. I
needed to get back to the apartment where I was staying. I just
started a new job.
Senator Specter. Mr. Hickock, how long have you been in a
wheelchair?
Mr. Hickock. I have been in a wheelchair since the first
part of 1997. I used my engineering background. I had a
computer program I could help design my house, so I am one of
the fortunate ones. Believe me, I am fortunate to be here. I am
fortunate to talk to you.
But I designed my house so that I could make my bathroom
big enough to get my wheelchair in. I designed ramps so I could
get around my house. I designed a ramp so I could get out my
door.
Senator Specter. How are you feeling at the present time?
Mr. Hickock. Very nervous.
Senator Specter. So am I, but how are you feeling?
Mr. Hickock. I am very sorry. I do not mean to put you on
the spot.
Senator Specter. You have already done that, but that is
OK.
Mr. Hickock. I do not mean to put any of these other guys
out.
Senator Specter. That is within my pay grade.
Mr. Hickock. I am very sorry.
Senator Specter. Well, that is OK.
What I would like you to tell us about, what your symptoms
are, how you are feeling, whether the situation is getting
worse, whether it is staying about the same. I understand it
does not get better.
Mr. Hickock. It is definitely getting worse. No, it does
not get better.
Senator Specter. How much deterioration or getting worse do
you actually feel?
Mr. Hickock. Well, I would like to tell about something
else first, if I could. I am sorry. But the stem cell. They are
talking about even possibly reversing the effects of ALS. They
do not know for sure, but looking at the research data that was
presented to us, it is about six or eight times more effective
than anything they have got right now. We need to fund that
research.
I tell you what. You talk about spending money. I
understand. Trillions. That has got too many zeroes in it for
my mind even to conceive. But what is being spent on ALS and
ALS research is about a one-hundredth of what is being spent on
other things. I could name names, but I do not want to name
names. I do not want to do that. I do not want to drive that in
the ground. I just want to increase ALS spending.
Senator Specter. Mr. Hickock, you can name names if you
want to. We will listen to whatever you want to say.
But I would like to know about your condition.
Mr. Hickock. My condition is getting worse.
Senator Specter. I would like to know how it is getting
worse and how you are feeling, so people can have some idea as
to what it is like to have ALS.
Mr. Hickock. Thank you. I appreciate it.
To have ALS, I notice myself my hands getting weaker, my
legs getting weaker, my breathing getting weaker. I notice
myself choking more. I notice myself not being able to do the
things that I like to. I am mechanical. I really like to be
able to fix things.
Senator Specter. Do you have family?
Mr. Hickock. Yes.
Senator Specter. Children?
Mr. Hickock. I have got four boys, 13, 16, 18, and 20.
Perry is with me today here, my youngest one. He is 13. My
oldest one had a--I have got a granddaughter. I am one of the
fortunate ones.
Senator Specter. And tell us a little bit about the impact
on the family. We can well understand how tough it is on the
family, but let us hear it from you.
Mr. Hickock. We really put them through heck.
Believe me, there is a guy that came from New York to
Washington in his wheelchair to be here today. I see a lot of
people doing super things, just unbelievable things. You would
not believe the jurisdictions that you got to go through, the
logistics, and all of that. Just getting here this morning, we
had a guy that parked right in front of one of the--we were
late today because this guy parked right in front of one of the
wheelchair access to get down off the curb, even in front of
the old Capitol Building. We were going to call President
Clinton and see if he would come down and lay down before us so
we could get down off the curb.
Senator Specter. Did you get a busy signal when you called?
Mr. Hickock. We did not get a hold of him, no. He would not
answer his phone.
Senator Specter. Did you call him?
Mr. Hickock. I did not even know where to start.
Senator Specter. His number is 456-1414.
Mr. Hickock. Just a minute. Let me get a piece of paper and
a pencil. Somebody will write it down, I am sure.
Senator Specter. Well, Mr. Hickock, we gave you the full
time. The red light has been on a little bit. We appreciate
your coming. We know how difficult it is. We thank all the
people who have come under very adverse circumstances for the
ALS Awareness Month March, which is in May of this year, and
that is why you are here and we scheduled this hearing
specially to focus attention on ALS because we knew you would
all be in town. When you step forward and wanted to be heard,
we are glad to hear you. We are glad that although some of your
faculties may be a little skittish, that you still have got a
lot of guts, a lot of courage, and you have stepped forward and
you speak your piece.
Mr. Hickock. I did. Thank you.
Senator Specter. I admire and respect that.
Mr. Hickock. Thank you.
Senator Specter. It is just a little late for you to file
for the Senate race in Michigan, but there is another election
coming up in a year or 2.
Mr. Hickock. I cannot guarantee I will be here in year or
2.
Senator Specter. Well, if you are, fine.
Mr. Hickock. I would love to meet you then. Maybe I will.
Senator Specter. I am not up for a while yet. You do not
live in Pennsylvania or I would not have been so generous with
all this committee time.
Just kidding. We have been glad to hear from you under any
circumstance.
I will tell you a short story. Paul Tsongas, Senator
Tsongas, in 1984 was up for reelection and he had lymphoma.
Paul decided he did not want to run because he might not live
out his term. In my own background, I have had some diagnoses
which were stark. 7 years ago I was given 3 to 6 weeks. I said
to Tsongas back in 1984, when I had also had the problem before
that, you owe duty to tell the people your condition. You ought
not to keep it a secret if you are running for reelection, but
you do not owe them a duty to serve out your term if they elect
you and lymphoma takes you. He disagreed with me and did not
run.
He lived out past 1990, ran for President in 1992. If he
had run for the Senate in 1984, and then had run for the
presidency in 1992, and you wanted to call up the President
today, you might have been calling somebody else. He might have
been elected in 1992. So, stay tuned. If you feel good, run.
STATEMENT OF BLAIR UNDERWOOD, ACTOR
Senator Specter. Let us go back to our regular witness
list. Our first witness is Mr. Blair Underwood, who stars as
Dr. Ben Turner in the new TV hit drama, City of Angels. He is
also currently starring in the box office hit, Rules of
Engagement, which I saw and liked very much. He made his
professional debut in the Cosby Show and since that time has
played many leading roles. Received the NAACP Image Award for
the outstanding actor in a drama series in 1994. A graduate of
Carnegie Mellon University, Pennsylvania. His grandmother died
of ALS.
Thank you for joining us, Dr. Turner, Mr. Underwood. The
floor is yours.
Mr. Underwood. Thank you, Mr. Chairman. I am pleased and
honored to be here this morning, and I thank you for your
leadership on this subcommittee.
And, Mr. Hickock, thank you for your initiative. I very
much appreciate it.
I also, Mr. Chairman, wanted to thank you for defending
your right to invite celebrities to this hearing because before
we are so-called celebrities, we are sons and daughters and
fathers and grandsons. As you alluded to, my grandmother died
from ALS in 1978. Out of all the roles I have been blessed to
play in my career, the one that affected me the most, of
course, was that role of caretaker to my grandmother. Her name
was Betsy Scales from Buffalo, New York. She was a sweet,
hardworking, loving woman, a single mother in the 1930's. She
raised my mother who was born in 1932 by herself. So, she was a
very strong woman. So, you can understand how and why we were
awestruck when she was diagnosed with ALS.
The experience of living with someone with ALS is like Mr.
Hickock said. It puts the family through heck, but that is
absolutely nothing compared to what the patients are going
through, like Mr. Hickock. She was very able to use her vocal
cords, and it was her body that was ravaged initially. Her
right hand atrophied first, and then her left arm and hand was
completely limp. The best analogy or metaphor I can give you to
watch this happen to a loved one is if you think of a strong
ice sculpture, it is akin to watching that ice sculpture melt
away. And it is devastating to watch.
In a sense, though, I am preaching to the converted because
you are leading the charge, and I thank you for that.
Amyotrophic lateral sclerosis was a word I learned when I
was 14 years old. It is a word and a medical term that no 14-
year-old or 4-year-old or 40-year-old should ever have to know
or learn unless it is in a history book of something that used
to be.
My mother, Betsy Scales' daughter, Marilyn Scales, later to
be Marilyn Underwood, now has multiple sclerosis. So, this
neurodegenerative disease process runs in the family. So,
coming here today is very personal to me and to hear the
testimonies has been very moving to me as well.
PREPARED STATEMENT
So, I will not belabor the point or be redundant, but I
encourage you and the members of the subcommittee and the
viewers, of course, on ESPN watching this all over the world to
take note of the testimonies and the faces and the experiences
in front of us and take effort to give us more funding for
research because we must find a cure. As Mr. Schaap said, it is
almost embarrassing in this day and age, in this climate of
medical research, to not have a cure, to have no known cause,
not have a cure, or not have any real, viable treatment.
So, again, thank you for your leadership.
[The statement follows:]
Prepared Statement of Blair Underwood
Mr. Chairman, thank you. I have great respect for the medical
researchers who have testified before me today. I may play a medical
doctor on TV, but Dr. Fischbach and Dr. Maniatis are the real McCoy,
and I applaud their work.
I grew up as an ``Army brat'' so my family moved all over the
world, but I call Virginia home. It's good to be just across the river
today, here in the District. I began acting as a way to deal with my
family's transient lifestyle and I have been studying acting, directing
and producing ever since.
During my career, I have portrayed a U.S. Marine captain, a
psychotic stalker, a space shuttle navigator, a death row inmate, a
geneticist, a corporate banker, a lawyer, a police officer, a social
worker, and a newspaper reporter, among others. I have portrayed Jackie
Robinson, I am right now preparing for a movie role as the heavyweight
boxing champion Floyd Patterson, and I once even played Jesus Christ in
the movie ``Second Coming.''
But I am not here to tell you about my acting career. In fact, the
most difficult role I was ever given to play did not involve a movie
script. It was not make believe. My most difficult role was all too
real. I was a caregiver to my wonderful grandmother who died of ALS.
My mom's mom was diagnosed with ALS when I was 14 years old. I
remember that her left hand was crippled with arthritis, and her right
arm was completely limp due to the ALS. My grandmother passed away
while I was in high school. Because I loved her so much, it was ironic
that she died on Valentine's Day. I was rehearsing a play at school
when I got the news that she had died. She had been diagnosed with ALS
less than a year before. After she was diagnosed, she just gradually
slowed down, her muscles wasting away. It was like seeing a beautiful
ice sculpture melting away before my eyes.
Now, my mother has multiple sclerosis, or MS. Mr. Chairman, I
understand that you and your Subcommittee already have held hearings
this year on MS and other degenerative disorders. Thank you for that.
My mother's diagnosis with MS, combined with my grandmother having died
of ALS, means that coming to Capitol Hill to speak out about ALS is
very important to me. It is my chance to do something to stop this
disease once and for all. This cause is very personal to me. The
genetic lineage of my grandmother to my mother means that this could
affect my children or in the future, their children.
Of course, ALS could affect me directly.
Naturally, I question whether my mother having MS and my
grandmother having ALS is somehow related. One thing you come to learn
when portraying a doctor in a television series is that doctors can't
solve every medical problem. There is only so much funding for research
for diseases. We need more funding if we are going to fight a disease
like ALS and other neurodegenerative disorders.
Mr. Chairman, the tremendous efforts of this Subcommittee, and the
work of such distinguished scientists as Dr. Maniatis and Dr.
Fischbach, is too late for my grandmother. However, my strong sense is
that we are on the threshold of great progress in the treatment of ALS.
All of the encouraging testimony that I have heard today confirms this
belief
As Dr. Maniatis testified, the ALS Association is formally
announcing today its largest and most aggressive research initiative.
The Lou Gehrig Challenge will get to the heart of the matter. It will
answer those questions that need to be answered now so that effective
and viable treatments for ALS can be developed more rapidly.
But what I find most encouraging is that the ALS Association is not
alone. Several federal agencies are rallying to fight ALS as well.
Just weeks ago, the Department of Veterans Affairs and the
Department of Defense launched a nationwide study to determine the rate
of ALS among military veterans who were on active duty during the Gulf
War.
Dr. Fischbach's agency, the National Institute of Neurological
Disorders and Stroke, also has joined with the ALS Association to
search for new therapies for motor neuron diseases such as the purchase
or creation of chemical libraries, and the funding of highthroughput
screening facilities.
These research partnerships will accelerate the discovery of
clinical treatments and drug development. These discoveries will,
ultimately, lead to a cure for ALS and other neurodegenerative
disorders.
Mr. Chairman, the future is now. We've entered a new millennium.
Let's enter a new era of prevention, treatment, and cure.
Thank you, Mr. Chairman. My hopes and prayers will be with you,
your staff, the researchers, the ALS patients, and their families.
Thank you.
Senator Specter. Thank you very much, Mr. Underwood.
STATEMENT OF SHELBIE OPPENHEIMER, ALS PATIENT, NEW
HOPE, PA
Senator Specter. We turn now to Ms. Shelbie Oppenheimer,
who was diagnosed with ALS at the age of 21. Since that
diagnosis, she has become an ALS advocate working with local
groups, particularly in the greater Philadelphia area. She is
the mother of a 2-year-old daughter, Isabel, resides in New
Hope, Pennsylvania.
Thank you for joining us, Ms. Oppenheimer, and we look
forward to your testimony.
Ms. Oppenheimer. Thank you, Senator Specter, and it is good
to see you again. I was present when you received the first
Jacob Javits Award 3 years ago, and I would like to thank you
for sticking with our fight.
This gathering today is a step forward in our journey
toward a cure for Lou Gehrig's disease. I thank you all for
making this happen and I am honored to be here today to tell
you how ALS has affected my life.
My name is Shelbie Oppenheimer, and for so long my life had
been a fairy tale. As the daughter of Gloria and Jerry
Wasserman and the sister of Brian Wasserman, I grew up in a
loving and nurturing family, the kind of family that laughs
together over supper. I have also been fortunate enough to have
the same best friend since I was 3, and I married my soul mate
and the love of my life, Jeff Oppenheimer.
We soon, after we got married, relocated to Bucks County,
Pennsylvania. The plan was to buy a house in the suburbs and
start a family, but as we were to about to embark on this
beautiful and fulfilling life's mission, an old Yiddish
expression became relevant to my story: A mench tracht and Gott
lacht. We plan and God laughs.
On the same day we discovered and put a deposit on our
dream home, in the call that we made to my parents to share
this exciting news, we learned that my mother had been
diagnosed with stage IV cancer. As I sat at her bedside for
weeks, while she fought and lost this brief and brutal bout
with lung cancer, I thought nothing could be worse. Only now
can I appreciate that through this horrible struggle, she knew
that thousands of researchers and billions of dollars had
beaten forms of her disease, were making progress on others,
and because of this, she had hope that maybe, just maybe, she
would be the beneficiary of all this research. Sadly, that did
not happen for her, but that hope gave her comfort and kept her
strong.
When I began to feel strong enough mentally, my husband and
I had decided to start a family, but I was not quite ready
after the shock of losing my mom. But when I began to feel
better mentally, there was a matter of some weakness and
twitching in my left arm that my family physician had
recommended I see a specialist about.
Little did I know that from this innocent-enough
consultation with our family physician that the terrifying,
surreal process of elimination had begun. I had noticed
twitching and weakness in my left arm. Since I was a healthy,
active 28-year-old, my doctor assumed it was a pinched nerve
and sent me to a neurologist.
Well, it was not that simple. After numerous x-rays, MRI's,
EMG's, it was determined that this definitely was not a pinched
nerve. I was told that it most likely was amyotrophic lateral
sclerosis. My neurologist also told me that they were testing
for other possibilities and urged me not to read anything about
ALS until they were sure.
The next day at the bookstore, I looked up ALS in a medical
encyclopedia, and that is when I learned that ALS is Lou
Gehrig's disease. The book said that this is a degenerative
nerve disorder that causes nerves to die, muscles to atrophy,
and eventually leads to the inability to breath and imminent
death. As I read on, I learned that this usually happens within
2 to 5 years after diagnosis, and all the while the patient is
mentally alert and aware. I read that it affects 30,000
Americans and there is no cure. I read on and I cried right
there in the store.
It was several months later at Johns Hopkins University it
was confirmed that, yes, I have ALS. At age 28, I was diagnosed
with a terminal illness.
Every good fairy tale has its moment of dark, impending
tragedy. For me the tragedy was not just that my life would be
cut short, but that I may never hear the words, ``Mommy, I love
you.'' I am happy to share with you today that I hear these
words every day. It has been 2 years since my husband and I
adopted Isabel from Guatemala. Although I devote myself every
day to caring for, loving, and nurturing my daughter and not
wasting them away consumed by what may be, sometimes I cannot
help but worry which muscle will fail me next and how will that
effect my ability to care for her. When will my physical
limitations become too big to hide from her? Will she need to
feed me as I once fed her?
Instead of weekend plans, what to serve for dinner, which
preschool for Isabel, I cannot help but be angry that I must
think about slowly fading away physically and being completely
aware of it mentally. I cry at the thought of not being able to
tell my husband and daughter that I love them, and I weep at
the thought of my father burying another child.
PREPARED STATEMENT
I am tougher than I look and I am going to do what it takes
to beat this. And I am asking you to do the same. With your
commitment of support, someday, somewhere a young woman still
not yet diagnosed, will look up ALS in the medical
encyclopedia, much like I did. Her heart will pound as she
reads about its devastating effects on her body. But her
spirits will soar as she reads on. There in black and white she
will read about a research breakthrough that you, by being here
today, helped to make happen. And I will be damned if I am not
browsing a couple of aisles over.
Thank you.
[The statement follows:]
PREPARED STATEMENT OF SHELBIE OPPENHEIMER
Thank you, Chairman and distinguished Subcommittee members: This
gathering today is a step forward in our journey toward a cure for Lou
Gehrig's disease. I thank all of you for making this happen. I am
honored to be here today to tell you how ALS has effected my life.
My name is Shelbie Oppenheimer. For so long, my life had been a
fairy tale. As the daughter of Gloria and Jerry Wasserman and the
sister of Brian Wasserman, I grew up in a loving, nurturing family. The
kind of family that laughed together over supper. I have been fortunate
enough to have the same best friend since I was three years old, and I
married my soul mate and the love of my life.
Three years after Jeff and I were married, the company he and a
friend had started in the basement was now large enough to attract the
attention of a much larger competitor. After his company was purchased,
we relocated to Bucks County, Pennsylvania.
Our plan was to buy a house in the suburbs and start a family. But,
as we were about to embark on this beautiful and fulfilling life's
mission, an old Yiddish expression unfortunately became relevant to my
story: A mench tracht und Gott lacht . . . We plan, and God laughs.
On the day we discovered and put a deposit on our dream home, we
made a call to my parents to share this exciting news. What was to be
happy conversation turned tragic. We learned that my mother had been
diagnosed with stage four Cancer. I sat at her bedside for weeks while
she fought a brief and brutal bout with lung cancer.
As I watched my mother die, I thought nothing could be worse. Only
now can I appreciate that through this horrible struggle, my mother
knew that thousands of researchers and billions of dollars had beaten
forms of her disease, were making progress on others and that maybe,
just maybe, she would be the beneficiary of all these resources. Sadly,
that didn't happen for her. But that hope gave her comfort and kept her
strong.
Since then, I've learned that daughters don't ever fully recover
from losing their mothers. I did not feel emotionally up to carrying
and caring for a child just then, but soon, very soon. And when I began
to feel strong enough mentally, there was a matter of this pinched
nerve that my family doctor had recommended I see a specialist about
once we settled into our new area.
Little did I know that from this innocent-enough consultation with
our family physician that the terrifying, surreal process of
elimination had begun.
I had noticed twitching and weakness in my left arm. Since I was a
healthy, active 28 year old my doctor assumed it was a pinched nerve
and sent me to a neurologist. Well, it wasn't that simple. After
numerous x rays, MRIs and EMGs, it was determined that it was
definitely not a pinched nerve. I was told that most likely it was
amyotrophic lateral sclerosis. My neurologist also told me that they
were testing for other possibilities and urged me not to read anything
about ALS until they were sure.
The next day, at the bookstore, I looked up ALS in a medical
encyclopedia. That's when I learned that ALS is Lou Gehrig's disease.
The book said that this is a degenerative nerve disorder that causes
nerves to die, muscles to atrophy and eventually will lead to the
inability to breath and imminent death. As I read, I learned that this
usually happens within two to five years after diagnosis and all the
while, the patient is mentally alert and aware. I read that it affects
30,000 Americans and there is no cure. I cried, right there in the
store.
It was several months later at Johns Hopkins University it was
confirmed that, yes, I have ALS. At age 28 I was diagnosed with a
terminal illness.
Every good fairy tale has its moment of dark, impending tragedy.
For me the tragedy was not just that my life would be cut short, but
that I may never hear the words, ``Mommy, I love you.'' I'm happy to
share with you today that I hear these words every day. It has been 2.5
years since my husband and I adopted Isabel from Guatemala.
Although I devote myself every day to caring for, loving, and
nurturing my daughter and not wasting them away consumed by what may
be, sometimes I can't help but worry . . . which muscle will fail me
next and how will that effect my ability to take care of her? When will
my physical limitations become too big to hide from her? Will she need
to feed me as I once fed her?
Instead of thinking about weekend plans, what to serve for dinner,
which preschool for my daughter, I can't help but be angry that I must
think about slowly fading away physically and being completely aware of
it mentally. I cry at the thought of not being able to tell my husband
and daughter that I love them, and I weep at the thought of my father
burying another child.
I'm tougher then I look and I will do what it takes to win this.
I'm asking you to do the same. With your commitment of support,
someday, somewhere a young woman still not yet diagnosed, will look up
ALS in a medical encyclopedia much like I did. Her-heart will pound as
she reads about its effects. But her spirits will then sore, as she
reads on. There, in black and white, she will read about a research
breakthrough that you, by being here, helped to make happen. And I'll
be damned if I'm not browsing the bookshelves a couple of aisles away.
Senator Specter. Thank you very much, Mrs. Oppenheimer, for
those very moving statements. We will come back to you for some
questions in a moment or two.
STATEMENT OF STEVE RIGAZIO, ALS PATIENT, LAS VEGAS, NV
Senator Specter. I would like now to turn to our final
witness, Mr. Steve Rigazio, diagnosed with ALS just last year
at the age of 44. Senior Vice President of Energy Delivery of
the Nevada Power Company. Holds a master's from the University
of Nevada at Reno, and a bachelor's degree from Eureka College,
Illinois.
Thank you for joining us, Mr. Rigazio, and we look forward
to your testimony.
Mr. Rigazio. Thank you very much. My name is Steve Rigazio,
and I am a family man and businessman from Las Vegas, Nevada,
constituent of Senator Reid. And I appreciate his comments this
morning, very kind comments, and I appreciate very much his
office and staff and Senator Reid. Since I have been diagnosed,
he has been there every minute for me, and I appreciate it very
much.
I am 45 years old. I have been married for 22 years to my
wife Annette and have two children, Bethanie who is 16, a
junior in high school, and my son David is 13. Like any
concerned parent, we are afraid to take the kids out of school
for 3 days, so Annette and the kids are back in Las Vegas
attending school. 16-year-old girls have a tendency to--at this
stage, you have to watch them a little and make sure they do
attend school.
But I do have my brother here today, Mark, and my brother-
in-law Pete and my sister Mary and their 9-year-old daughter
Elizabeth, my niece, who is out here today. I appreciate them
being here.
I actually started out in the electric utility industry as
an iron worker and began iron work in a power plant for 2
years, went back to school, saved enough money, earned money
and went on for my master's degree at the University of Nevada,
came back to the industry as an analyst. I was very fortunate,
worked my way up the corporate ladder, and last year, upon a
merger of two power companies, I was named Senior Vice
President of Operations of both companies.
I am very active in the Las Vegas community. I am on a
number of boards. I am on the Board of Regents of Bishop Gorman
High School, the only Catholic high school in Las Vegas,
Salvation Army, Sunrise Children's Hospital, Channel 10 public
broadcasting, New Horizons Academy, which is a school for
children with learning disabilities where my son attends.
I have always been physically active and athletic. As a
child growing up in a small town of Oglesby, Illinois, my
brother Mark and I played baseball morning till night as my mom
called us in for dinner. In the wintertime, I played ice hockey
and played competitively. 12 months ago, May of last year, I
was on the ice getting banged around by a bunch of big guys and
enjoying every minute of it. Today I cannot even put on my
coat.
All that changed for me on August 25th, 1999. I was
diagnosed with ALS, at the Baylor College of Medicine. It
obviously shattered me, my wife, and kids and our dreams. The
most disappointing thing to me, after receiving the news, was
to find that there is no cure and very few medications that can
even slow the disease down. Myotrophin, one of those, was
pulled off the market 4 months after I was diagnosed. I never
had that opportunity to even try it.
When you have something like this, you are grasping for
straws. Basically I enjoy life. I have a deep faith and family
values. I just want to be with my family and friends in my
community of Las Vegas and live life to its fullest for a long
time.
I really want to see my daughter go on to college and
graduate from college. I want to see my son get through grade
school and high school. I want to come back here in 9 years and
see Lizzy graduate from high school, if I can.
PREPARED STATEMENT
When you have this, you can take different attitudes. I
have chosen to try to put a positive spin on this as best I
can, and shortly after diagnosis and the initial trauma, some
friends of mine in Nevada started a foundation called Nevadans
for the Prevention of ALS, and we are trying to raise money for
awareness, research, and provide money to the local ALS chapter
for patient care. We had our first fund raiser last week, a
dinner. Senator Reid's staff was there. We raised over
$100,000. In some small way, maybe this will lead to a cure and
a new life for those afflicted with this horrible disease.
Thank you very much.
[The statement follows:]
PREPARED STATEMENT OF STEVE RIGAZIO
My name is Steve Rigazio. I am 45 years old. I have been married
for 22 years to my wife, Annette and we have two children. Bethanie is
16 years old and David is 13 years of age.
I am currently a Senior Vice President for Nevada Power Company, an
electric utility company in Las Vegas, Nevada.
I am very active in the Las Vegas Community. I am on a number of
boards including Bishop Gorman Regents, a local catholic high school,
the Salvation Army, the Sunrise Children's Hospital, a local public
broadcasting TV station, the Nevada Taxpayers Association, New Horizons
Academy. a school for children with learning disabilities and the
Chamber of Commerce Board of Advisors.
I was always physically active and athletic. As a child growing up
I played baseball and ice hockey and continued doing so as an adult.
On August 25, 1999, 1 was diagnosed with Amyotrophic Lateral
Sclerosis (ALS--Lou Gehrig's disease). Obviously, that shattered me, my
wife and kids and our dreams. The most disappointing thing to me is
after receiving the news, was to find there is no cure and few
medications that slow the disease down. One of these is Myotrophin
which was pulled off the market shortly after my diagnosis. When you
have something like this, you are grasping for straws. Basically, I
enjoy life. I have a deep faith and family values. I just want to be
with my family and friends in my community of Las Vegas, and live life
to its fullest for a long time.
Finally, shortly after diagnosis and after the initial trauma, a
group of friends and prominent Nevadans approached me about starting a
foundation in order to help in some way. We formed Nevadans for the
Prevention of ALS (NPALS). Our goal is to raise funds for awareness of
ALS, patient care, and research. Last week in Las Vegas we had our
first fundraiser, a dinner that raised $110,000. In some small way,
maybe this will lead to a cure and new life for those afflicted with
this horrible disease.
In closing, I would like to thank this committee, in particular
Senator Harry Reid from Nevada, for his support, and you for the
opportunity to speak before the Senate Appropriations Subcommittee on
Labor, Health and Human Services and Education.
Senator Specter. Thank you very much, Mr. Rigazio, for
sharing those personal feelings, experiences with us.
Mrs. Oppenheimer, I was interested in your quotation of ``A
mench tracht, Gott lacht.'' My grandmother taught me that one
as well, but your written statement has the translation as ``we
plan and God laughs.'' I was taught the translation was,
``People plan and God laughs.'' It is about the same. Somehow
it has a lot more pizazz and oomph in Yiddish, ``A mench
tracht, Gott lacht,'' than it does in the translation.
Ms. Oppenheimer. Everything does.
Senator Specter. Tell us a little bit about what happened
to you at the onset of ALS. You were 28. We are going to have
ask you how long ago that was. I do not like to ask a woman her
age.
Ms. Oppenheimer. Well, my symptoms of ALS started 4 years
ago, so I am very fortunate that the progression in my case is
considered slow.
Senator Specter. What were the first symptoms?
Ms. Oppenheimer. The first symptoms were weakness in my
left arm and twitching, the inability to do little things like
buttoning. It just became more difficult.
Senator Specter. And what happened next?
Ms. Oppenheimer. I was seen by several neurologists. I
think when you are 28, that is not something that comes to mind
right away when they are looking at you. So, lots of tests,
EMG's, MRI's, blood tests. There is not one specific test for
ALS, so they rule out a lot of other things.
Senator Specter. This is somewhat personal, but perhaps all
of it is. You adopted a daughter. You had testified about your
plans to have a family, move to the suburbs, have a family. You
decided not to do it but go the adoption route?
Ms. Oppenheimer. No. Well, at the time I was diagnosed, we
did not realize that I was slow progressing, and we really did
not want to waste a lot of time. So, we opted to go the
adoption route just for certainty purposes.
Senator Specter. And how are you feeling now?
Ms. Oppenheimer. On a day-to-day basis, I feel pretty good.
But the disease progresses. Even though it is slow in me, it is
always progressing, so everyday there are new challenges.
Senator Specter. To what extent has ALS disrupted your
normal living activities?
Ms. Oppenheimer. Well, because of my weakness and fatigue,
I can no longer work. So, I am a stay-at-home mom, but I am not
even a full-time stay-at-home mom because I cannot care for her
by myself all the time. So, my daughter goes to day care for 2
days a week so that I can rest and do heavy chores that I
cannot do while she is around.
Senator Specter. Mr. Rigazio, you felt the symptoms just in
the course of the past year, and what kind of progression have
you felt?
Mr. Rigazio. Very similar to Ms. Oppenheimer. I started in
my right arm and hand, twitching in muscles, about January of
1999, and went through about a 6-month period thinking I had a
pinched nerve, a weakness in my arms and hands. After the whole
battery of tests in August of last year, everything else was
ruled out, and it was ALS. My case is progressing also,
hopefully slow, but every day I can feel the difference, and
weakness in my hands and arms and shoulders.
Senator Specter. To what extent has it interfered with your
work?
Mr. Rigazio. Quite a bit. It is interesting. I got the
promotion to Senior Vice President of the company 25 days
before this diagnosis. I still go to work every day. However, I
am unable to drive a car anymore, so I need assistance in
getting to and from work and need assistance at work.
Particularly I need assistance dressing, which my wonderful
wife Annette and my children help out, and assistance eating. I
am unable to cut steak with a knife, things such as that.
Senator Specter. Who is that, Mrs. Oppenheimer?
Ms. Oppenheimer. This is my daughter, Isabel.
Senator Specter. OK, Isabel. Be careful. You are on camera
now.
How old is she, Mrs. Oppenheimer?
Ms. Oppenheimer. She is 2.
Senator Specter. At 2, I do not suppose she has much
awareness of what you are going through.
Ms. Oppenheimer. No. She is the only person that does not
see me as having any physical limitations.
Senator Specter. Well, she just ran right up and jumped on
your lap.
Tell us, Mr. Rigazio, about the impact on your family. How
has that been?
Mr. Rigazio. I am a very lucky person. I have a wonderful
family. My parents are married I think 46 years. My in-laws
have been married 48 years. I come from a large family. We had
a lot of fun growing up. I married a wonderful lady. For 22
years we have been married. Two kids.
It changed a lot of things. I was looking forward to
retiring in 10 years at age 55. All that is going to change.
Annette and I were looking forward to our 25th wedding
anniversary and taking an Alaskan cruise. We decided to change
that and make it about 2 months from now on our 22nd
anniversary.
Around the house I am unable to do the things I used to do,
but I still have the love of my wife and my children and my
family. So, I am a very fortunate person from that standpoint.
Senator Specter. Mr. Underwood, you talked about ALS
striking your grandmother. Can you tell us what you observed,
how her situation was, what deterioration, if any?
Mr. Underwood. Well, Mr. Chairman, her situation started
very much like Mr. Rigazio and Mrs. Oppenheimer, in her hands,
twitching in her left finger. And then it progressed to the
point where, as I said, her right hand had atrophied and the
left became very limp. She was still able to walk and she was
still able to speak when she expired in her sleep.
Senator Specter. She died in her sleep?
Mr. Underwood. Yes, she did.
Senator Specter. And how old was she?
Mr. Underwood. She was 68 years old at that time.
Senator Specter. Tell us about the impact on the family.
You were 14 at the time?
Mr. Underwood. I was 14 at the time, and the impact was
just a tremendous sense of helplessness. You feel like an
innocent, helpless bystander. There is nothing you can do but
give support and love and encouragement and be there.
Another aspect of this disease is sometimes the mental
pressure and sometimes depression which may mean therapy and
medication, but that is another side of this that has not been
brought up today. So, that support of family is vital.
Senator Specter. Ms. Oppenheimer, I think I know the answer
to this question, but tell us your views with respect to hope,
faith that we will find a cure for ALS to save your situation.
Ms. Oppenheimer. Well, I am much more hopeful than I was
when I was first diagnosed 4 years ago because there has really
been an explosion in research. Just some of the most brilliant
minds in medical research are working on ALS, but
unfortunately, as you know, it just all requires money. I guess
as long as they are properly funded, I think that there is
definite hope for patients with ALS.
Senator Specter. Mr. Rigazio has been diagnosed for just a
little less than a year. What is your sense of hope with
respect to the possibility of curing your disease in time?
Mr. Rigazio. I have tremendous hope and I believe a lot of
the reason for that is the awareness brought to this disease
over the last several years, which is significantly more than
it has ever been. And events like today with the people that
are here today telling their stories from all walks of life, I
have a great deal of hope, a great amount of faith. I am
thoroughly convinced that the cure will be found and I am going
to be cured. I really believe that.
Senator Specter. Well, Mr. Underwood, we will give you the
last word. You are a doctor in the TV drama, City of Angels,
but you are a surgeon. You are not a neurologist.
Mr. Underwood. That is correct.
Senator Specter. I do not know what weight we should give
to the surgeon's views. But how do you evaluate the situation?
What can people like you do to bring public attention to focus
the Congress on providing enough money to get a breakthrough?
Mr. Underwood. Well, Mr. Chairman, I think Steve Garvey
said it so eloquently, that we are blessed to do what we enjoy
doing and to use our God-given abilities, and if that brings
you some sense of awareness from people and for whatever
reason, right or wrong, earned or unearned, people will listen
to what you have to say, then so be it. That is why I wanted to
be here today.
But you are doing it, Senator, and this subcommittee is
doing the work and the testimonies of people coming here today.
Senator Specter. Well, thank you, Mr. Underwood. Thank you,
Mr. Rigazio. Thank you, Mrs. Oppenheimer, and Miss Isabel
Oppenheimer.
Ms. Oppenheimer. Thank you.
CONCLUSION OF HEARING
Senator Specter. Thank you all very much for being here,
that concludes our hearing. The subcommittee will stand in
recess subject to the call of the Chair.
[Whereupon, at 11:40 a.m., Thursday, May 18, the hearing
was concluded, and the subcommittee was recessed, to reconvene
subject to the call of the Chair.]