[Senate Hearing 106-855]
[From the U.S. Government Printing 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
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                                 ______

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                                 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.]