[Senate Hearing 109-257]
[From the U.S. Government Printing Office]



                                                        S. Hrg. 109-257
 
                  AMYOTROPHIC LATERAL SCLEROSIS (ALS)

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

                                HEARING

                                before a

                          SUBCOMMITTEE OF THE

            COMMITTEE ON APPROPRIATIONS UNITED STATES SENATE

                       ONE HUNDRED NINTH CONGRESS

                             FIRST SESSION

                               __________

                            SPECIAL HEARING

                      MAY 11, 2005--WASHINGTON, DC

                               __________

         Printed for the use of the Committee on Appropriations


  Available via the World Wide Web: http://www.gpoaccess.gov/congress/
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                               __________

                      COMMITTEE ON APPROPRIATIONS

                  THAD COCHRAN, Mississippi, Chairman
TED STEVENS, Alaska                  ROBERT C. BYRD, West Virginia
ARLEN SPECTER, Pennsylvania          DANIEL K. INOUYE, Hawaii
PETE V. DOMENICI, New Mexico         PATRICK J. LEAHY, Vermont
CHRISTOPHER S. BOND, Missouri        TOM HARKIN, Iowa
MITCH McCONNELL, Kentucky            BARBARA A. MIKULSKI, Maryland
CONRAD BURNS, Montana                HARRY REID, Nevada
RICHARD C. SHELBY, Alabama           HERB KOHL, Wisconsin
JUDD GREGG, New Hampshire            PATTY MURRAY, Washington
ROBERT F. BENNETT, Utah              BYRON L. DORGAN, North Dakota
LARRY CRAIG, Idaho                   DIANNE FEINSTEIN, California
KAY BAILEY HUTCHISON, Texas          RICHARD J. DURBIN, Illinois
MIKE DeWINE, Ohio                    TIM JOHNSON, South Dakota
SAM BROWNBACK, Kansas                MARY L. LANDRIEU, Louisiana
WAYNE ALLARD, Colorado
                    J. Keith Kennedy, Staff Director
              Terrence E. Sauvain, 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
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
MIKE DeWINE, Ohio                    MARY L. LANDRIEU, Louisiana
RICHARD C. SHELBY, Alabama           RICHARD J. DURBIN, Illinois
                                     ROBERT C. BYRD, West Virginia (Ex 
                                         officio)
                           Professional Staff
                            Bettilou Taylor
                              Jim Sourwine
                              Mark Laisch
                         Sudip Shrikant Parikh
                             Candice Rogers
                        Ellen Murray (Minority)
                         Erik Fatemi (Minority)
                      Adrienne Hallett (Minority)

                         Administrative Support
                              Rachel Jones


                            C O N T E N T S

                              ----------                              
                                                                   Page

Opening statement of Senator Richard C. Shelby...................     1
Opening statement of Senator Tom Harkin..........................     2
Opening statement of Senator Patty Murray........................     3
Statement of Story C. Landis, Ph.D., Director, National Institute 
  of Neurological Disorders and Stroke, National Institutes of 
  Health, Department of Health and Human Services................     4
    Prepared statement...........................................     6
Statement of Lucie Bruijn, Science Director and Vice President, 
  ALS Association................................................    19
    Prepared statement...........................................    21
Statement of Eric Obermann.......................................    24
    Prepared statement...........................................    28
Statement of Robert Borsellino...................................    30
Statement of Tommy John..........................................    39
    Prepared statement...........................................    41
Statement of David Cone..........................................    41
    Prepared statement...........................................    43
Statement of Kate Linder.........................................    44
    Prepared statement...........................................    46
Statement for the record from Adrienne Hallett...................    47


                     AMYOTROPHIC LATERAL SCLEROSIS
                                 (ALS)

                              ----------                              


                        WEDNESDAY, MAY 11, 2005

                           U.S. Senate,    
    Subcommittee on Labor, Health and Human
         Services, Education, and Related Agencies,
                               Committee on Appropriations,
                                                    Washington, DC.
    The subcommittee met at 10 a.m., in room SDG-50, Dirksen 
Senate Office Building, Senator Richard C. Shelby presiding.
    Present: Senators Shelby, Harkin, and Murray.


             OPENING STATEMENT OF SENATOR RICHARD C. SHELBY


    Senator Shelby. I want to take this opportunity to thank 
all of you for being here to discuss the issues surrounding 
ALS. This month is ALS awareness month and today, the ALS 
Association's advocacy day. So I can think of no better time to 
be holding this hearing.
    I want to take a minute to thank Senator Specter and also 
Senator Harkin because I am not the chairman of this 
subcommittee. I am a chairman of another subcommittee in the 
Appropriations chain and so I am indebted to Senator Specter 
and the ranking member Senator Harkin, for letting us conduct 
this hearing today.
    This is an important issue that affects many of our 
constituents. I am pleased that so many folks were able to join 
us here today including some of my constituents from my home 
State of Alabama.
    Appearing before us today are two ALS patients, a number of 
their advocates, both local and celebrity, as well as a leading 
researcher in the field. I look forward to hearing from each of 
you.
    ALS is a tragic disease that we know little about and have 
no treatment for at this time. I have seen firsthand how 
rapidly this disease robs one's ability to function and how 
quickly it can take our loved ones from us.
    Just last year, I met with some Alabamians about ALS and I 
am sad to report that one of the individuals that we spoke 
about during that meeting is no longer with us. His wife, who 
is here today, continues her efforts to promote increased 
research for ALS and I for one greatly appreciate her efforts 
on behalf of those suffering from ALS.
    Federal funding for ALS research is critical. While we work 
diligently to increase funding, there is much more to do. I am 
hopeful that this hearing today will provide a forum to discuss 
the issues that must be addressed by researchers.
    There is so much basic research that must first be done if 
we are to find the cause and the cure of ALS. I also believe 
that we must continue to work to find immediate opportunities 
to slow its progression and provide patients with every option 
available.
    Again, I hope this hearing will serve to increase awareness 
of ALS and our need to do more on behalf of the ALS patients 
and their families.
    Thank you all for being here today and I look forward to 
hearing from each of you.
    Senator Harkin.


                OPENING STATEMENT OF SENATOR TOM HARKIN


    Senator Harkin. Mr. Chairman, thank you very much, Senator 
Shelby, for calling this hearing today.
    Senator Shelby and I have been friends for a long time. We 
served in the House together many--well, a few years ago. 
Probably longer ago than we care to admit, I suppose. But we 
have been great friends for many, many years.
    I want to thank you for calling this hearing today.
    Let me also thank the hundreds of patients and their loved 
ones who have packed this room today to demonstrate their 
support for ALS research. I just shook the hands of a man from 
Alaska and I said you get the prize for coming the farthest. He 
said, no, there is someone here from Hawaii.
    So people have come a great distance and I know the 
difficulty of traveling today, so we just really appreciate 
this show of support.
    Later today this morning, you will be fanning out across 
Capitol Hill to make your case for more aggressive ALS 
research. I want to tell you how important it is that you are 
here. You remind us and those that you will be seeing that ALS 
is not just about statistics. It is about the suffering of real 
human beings, our friends and our family.
    It makes all the difference in the world for you to bring 
that message here today in person. Your presence could not come 
at a more crucial time. It has been 6 decades since Lou Gehrig 
died, but we still have only one FDA approved drug to treat 
this disease and that drug only extends life for a few months. 
This is unacceptable.
    Five thousand Americans are diagnosed with ALS each year. 
For some reason, no one knows why, our military veterans are 
especially susceptible to this disease. So we have a 
responsibility to do more to find an effective treatment and 
cure.
    Regrettably, the administration has taken a hard line 
against one of the most promising avenues of research, stem 
cells. Scientists tell me that ALS is exactly the kind of 
disease that could benefit from stem cell research.
    They have already figured out how to direct stem cells to 
develop into motor neurons. If they could solve that next 
critical step, replacing motor neurons that have died off in a 
person with ALS, then we would be looking at a cure.
    Instead the administration has put a choke hold on research 
by limiting the number of stem cell lines that federally-funded 
scientists can study.
    We were initially told that more than 70 lines would be 
eligible for federally-funded research. Today there are just 21 
and all of them, every single one, are contaminated with mouse 
feeder cells, meaning that there is really no chance that they 
will ever be used for any human therapy.
    That is why Senator Specter here in the Senate, Congressman 
Castle on the House side have introduced bipartisan legislation 
to expand the number of stem cell lines that are eligible for 
federally-funded research.
    The House will vote on this measure sometime in May. I was 
talking with Congressman Castle who is a member of the 
Republican party on the House side and he believes that they 
have the votes to pass it, but, please, any help you can give 
on the House side, I would appreciate that. Then we will take 
it up later on.
    So I hope that in your meetings today with senators and 
their staffs that you will also push for this legislation.
    In the meantime, I was pleased to learn that NIH is 
supporting five clinical trials on ALS that are either ongoing 
or about to start. I am sure we will hear about that from Dr. 
Landis. That is more than we have ever had before. So these are 
clearly more hopeful times for ALS and I look forward to 
hearing more about the trials this morning from Dr. Landis.
    Again, I want to join Senator Shelby in welcoming an 
outstanding group of witnesses. In particular, I would just 
take the time right now to recognize Rob Borsellino, a renowned 
columnist for the Des Moines Register.
    Rob has been in the news business for 30 years in TV and 
radio and newspapers. He has worked for a number of newspapers 
across the country from New York to Florida and decided that 
Iowa was the best place to live, so settled in Iowa.
    In the mid nineties, he was a talk show host on ABC, a TV 
affiliate in Des Moines. He and his wife have two sons, one 18, 
one 15. So Rob, I just want to say, Rob Borsellino has an 
intensely loyal following all across Iowa. I am one of his 
biggest fans.
    So it came as a blow to all of us when we opened the 
newspaper one morning and Rob announced in his column earlier 
this year that he has ALS. Since then, he has been writing and 
speaking about this disease with his customary humor and grace.
    So I want to thank you, Rob, for the effort you showed to 
be here with us in person today.
    I also want to recognize Roger Gold and his wife who are 
here from Iowa, also from Ames. Roger has had ALS now for 10 
years and is one of those that has fought this disease. I just 
want to recognize him for coming here today also.
    So with that, Mr. Chairman, again, thank you for having us 
here.
    Senator Shelby. Senator Murray.


               OPENING STATEMENT OF SENATOR PATTY MURRAY


    Senator Murray. Well, Mr. Chairman, I want to thank you for 
scheduling this really important hearing on ALS. I think it is 
really important for this subcommittee to hear from the 
National Institute of Neurological Disorders and Stroke as well 
as all of those who are suffering from this devastating 
disease.
    It is important that everyone affected is informed about 
what we know today about ALS, about what we can hope for in the 
future, and what we need to do to find a cure.
    We know that ALS is a tragic neurological disease that 
often strikes the patient in the prime of life and causes 
tremendous suffering and pain both for the victims and for 
their families.
    Yet, while we all know this disease can be overwhelming and 
challenging, there is hope. I think it is really appropriate 
that the ALS Association has chosen their theme this year to be 
a flickering light of hope.
    I believe that the source of that light is the work being 
done by so many dedicated research and health care 
professionals who each day bring us closer to a cure.
    We have a responsibility to fuel the light of hope by 
funding research through NIH and avoiding political or 
ideological battles that might stand in the way of facilitating 
ethically sound research and progress on this really crucial 
issue.
    I hope that this hearing today, Mr. Chairman, serves to 
raise awareness of this disease and the impact on both the 
patient and their families.
    I want to thank Senator Shelby for chairing this and I want 
to thank Chairman Specter as well and Senator Harkin for their 
work on behalf of NIH funding. I look forward to working with 
everyone at the table to maintain our investment in sound 
biomedical research.
    Thank you, Mr. Chairman.

STATEMENT OF STORY C. LANDIS, Ph.D., DIRECTOR, NATIONAL 
            INSTITUTE OF NEUROLOGICAL DISORDERS AND 
            STROKE, NATIONAL INSTITUTES OF HEALTH, 
            DEPARTMENT OF HEALTH AND HUMAN SERVICES

    Senator Shelby. Dr. Landis, welcome to the committee.
    Dr. Landis is the director of the National Institute of 
Neurological Disorders and Stroke. She earned her Master's and 
Ph.D. from Harvard University. She later served on the faculty 
of the Harvard Medical School and was the chairman of the 
Department of Neurosciences at the Case Western Reserve 
University School of Medicine. She has been the director of 
NINDS since 2003.
    We welcome you. We understand you have a written statement 
and you also have an opening statement. You proceed as you 
wish.
    Dr. Landis. Right. Thank you.
    Mr. Chairman and members of the committee, I am Dr. Story 
Landis, Director of the National Institute of Neurological 
Disorders and Stroke, one of the components of the National 
Institutes of Health. I want to thank you for offering me the 
opportunity to speak with you about our research programs in 
ALS.
    As you have already heard and will hear from other panel 
members, ALS is a devastating disease, results from the death 
of motor neurons in the brain, brain stem, and spinal cord.
    These nerve cells relay control signals from the brain to 
the muscles throughout the body and when these nerve cells die, 
patients lose the ability to move. They lose the ability to 
swallow, to speak, and ultimately to breathe. For many 
patients, 5 years from the diagnosis of ALS, they die. It is a 
terrible disease.
    Scientists do not fully understand what triggers motor 
neuron death in this disease. Most believe that it is an 
interaction between genes, environmental influences, and aging.
    There are a number of specific hypotheses that include 
oxidative stress, over-excitation, lack of trophic support, 
and/or aberrant signaling within a nerve cell or between nerve 
cells.
    About 10 percent of ALS cases are genetic in origin and the 
identification of these disease genes has actually given us 
very important clues as to why motor neurons die in this 
disease and also have given us tools to increase our ability to 
understand the disease and to develop therapies.
    There is a desperate need for new therapies for ALS. At 
present, as you have already heard, there is only one drug 
approved by the FDA for ALS. That is Riluzole and it is not 
very effective.


                            CLINICAL TRIALS


    We have four clinical trials now. It is rare for a disease 
that we are able to say this. We have four clinical trials that 
are already enrolling patients and a fifth that will start 
enrolling patients in the fall.
    These clinical trials are based on very promising pre-
clinical data and are a result directly and indirectly of the 
doubling of the NIH budget. We did actually do something good 
with those monies.
    One of them is a phase three clinical trial looking at a 
trophic factor, IGF-1. A second is a phase three clinical trial 
looking at minocycline, an antibiotic which turns out to have 
an additional activity which is reducing inflammation and nerve 
cell loss.
    We have a phase two clinical trial that is looking at 
CoQ10, an antioxidant which works in pre-clinical studies.
    A fourth trial is actually one aimed at helping patients 
who are in mid stages of ALS which is an effort to look at when 
nutritional therapy and respiratory therapy is best undertaken.


                         BASIC SCIENCE RESEARCH


    Now, in order to have good clinical trials, you really have 
to have a very good understanding of the basic underpinnings of 
motor neuron biology and cell death. A very useful strategy in 
understanding motor neuron survival and the death of these 
motor neurons in ALS has been the identification of genes.
    We now have three genes which are known to cause ALS and 
the first and most informative of these is one in copper/zinc 
superoxide dismutase or SOD1. This accounts for about 2 percent 
of ALS patients. There are over 100 mutations in this gene 
which can cause ALS.
    So the discovery of this gene and the protein which is 
influenced has given us very interesting and testable 
hypotheses about why motor neurons die and examination of a 
mouse model that has been created has informed us that, in 
fact, normal glial cells, support cells can help motor neurons 
which have the mutation survive. An interesting finding that is 
relevant to stem cell therapy.
    It also turns out that this mouse model has been a critical 
piece in the pre-clinical studies that allow us to look at what 
drugs might be useful for human trials.
    We believe that discovering other ALS genes is critical to 
increasing our understanding of why motor neurons die and to 
develop additional pre-clinical models. It is not enough to 
just have one.
    So using funds that we got from the doubling, the increase 
in NINDS funding, we created a repository for genetic samples 
of patients with ALS so that those samples can be shared and 
genes can be discovered more quickly. We are working in 
particular with ALSA to encourage investigators to deposit 
samples in the repository.
    So I have given you an example of clinical trials we are 
running and of very basic research. One of the challenges for 
NIH has been how you bridge the gap between those two.


                         TRANSLATIONAL RESEARCH


    We have with our doubling invested funds in a number of 
translational research programs helping investigators move 
promising leads from basic research to the point where they 
would be ready for clinical trials.
    ALS has been a particular benefactor from this new program 
and because of monies invested in this program, the fifth 
clinical trial is going to look at an antibiotic which no one 
would have ever guessed would be useful in ALS. We are very 
optimistic that we will see benefit from that.


                           PREPARED STATEMENT


    So I have focused on a very small number of efforts that 
NINDS is making in the area of ALS with seven other institutes 
and centers at the NIH and in collaboration with a number of 
very effective voluntary organizations.
    I would be pleased to take questions. Thank you.
    [The statement follows:]

                 Prepared Statement of Story C. Landis

    Mr. Chairman and Members of the Committee, I am Dr. Story Landis, 
Director of the National Institute of Neurological Disorders and Stroke 
(NINDS), a component of the National Institutes of Health (NIH) within 
the Department of Health and Human Services (HHS). The NINDS is 
investing aggressively in new approaches to understand and treat 
amyotrophic lateral sclerosis (ALS), and I am pleased to be here today 
to share our research priorities, plans, and advances with you.
    Without question, ALS--or Lou Gehrig's disease--is one of the most 
debilitating and devastating of all diseases, and NINDS takes the need 
for treatments in this community very seriously. As many of you already 
know, ALS is caused primarily by the loss of nerve cells called motor 
neurons. These cells reside in the brain and spinal cord, and relay 
control signals from the brain to muscles throughout the body--
including those of the limbs, face and respiratory system. Although the 
clinical presentation varies widely, the death of motor neurons in ALS 
eventually leads to increasing difficulties with movement. These often 
occur first in the hands or feet, but occasionally begin in muscles 
such as those that control the tongue and swallowing. Regardless of the 
site of onset, the disease is relentless, and it gradually robs 
affected individuals of all motor function over a period of months or 
years. Approximately 5,000 people in the United States are diagnosed 
with ALS each year, and only 10 percent survive beyond five years after 
the onset of symptoms. Despite the advances made in ALS research and 
continued improvements in supportive therapy, current options for 
disease-modifying treatment are quite limited. Only one drug--
Riluzole, an inhibitor of the excitatory neurochemical glutamate--is 
approved by the U.S. Food and Drug Administration (FDA) for treating 
ALS, and it only extends survival by a few months.
    Researchers do not fully understand what triggers motor neurons to 
die in people with ALS, but several factors have been implicated, 
including the increased cellular stress caused by the need for these 
neurons to maintain tremendously long connections with distant cells; 
overstimulation by excitatory nerve chemicals, like glutamate; and the 
activation of specific signals inside the cells that lead to their 
destruction. In 1991, researchers funded in part by NINDS published the 
initial discovery of a genetic link to ALS, specifically the 
chromosomal mapping of a gene that was believed to contribute to the 
hereditary form of ALS. This discovery later led to the identification 
of more than 100 mutations in this gene, along with the recognition 
that it normally codes for an enzyme called superoxide dismutase 
(SOD1), which helps clear damaging free radicals from cells. Only ten 
percent of ALS cases have been found to be associated with inherited 
genetic mutations, and mutations in SOD1 only a small fraction of 
those. In addition, researchers still do not fully understand how SOD1 
mutations make people more susceptible to developing ALS. However, the 
discovery of this first gene energized investigators by providing the 
scientific community with new insights into possible mechanisms of 
disease, and by offering a means to create useful animal models of ALS 
that could be used for studying disease causality and testing 
treatments.
    To adequately address all of the issues that impact the ALS 
community, NINDS supports a continuum of research: clinical research to 
rapidly test available therapies; translational research to move basic 
science towards clinical applications; and basic science research to 
expand our understanding of the causes of ALS.

                           CLINICAL RESEARCH

    Despite the difficulties presented by this disease, NINDS is 
greatly encouraged by the enhanced interest and creativity among 
researchers in exploring approaches that may reduce the burden of ALS 
by intervening in the progression of the disease and/or improving the 
supportive care of affected individuals. Not only do these different 
approaches have the potential to extend survival and improve quality of 
life, but by targeting different aspects of the disease, they may 
ultimately be explored as part of a combination therapy approach.
    Several ALS clinical trials are actively recruiting subjects, 
including a NINDS-funded Phase III trial of insulin-like growth factor-
1 for ALS, for which enrollment is nearly complete. This trial takes 
advantage of the potential for a naturally occurring protein that 
promotes nerve cell growth and survival to delay loss of muscle 
strength, improve function, and extend survival in people with the 
disease. Enrollment is also underway for a Phase III trial of the 
antibiotic minocycline for treating ALS. In addition to its antibiotic 
properties, minocycline can also suppress cell death signals and 
inflammation--and has shown promise in delaying disease progression in 
several animal models of neurodegenerative disease, including ALS. The 
data from preclinical work in animals, in combination with safety and 
tolerability information provided in a Phase I/II human study of 
minocycline, provided support for its advancement into Phase III 
testing.
    In addition to these ongoing studies, the Institute is also 
supporting several new translational research programs and clinical 
studies that herald a new era of patient-focused research at NINDS. As 
a prime example of the momentum in the field, ALS researchers have 
recently participated in the screening of a number of drug candidates 
with possible effectiveness in treating the disease. NINDS conceived of 
this novel program for academic researchers, and designed it to enable 
rapid screening of potential therapeutic compounds with known 
bioactivity and/or safety in humans, so that the most viable candidates 
could be quickly moved forward into clinical trials. Initiated in 2001 
in collaboration with the Amyotrophic Lateral Sclerosis Association 
(ALSA) and two other private funding organizations, the program 
supported the screening of more than a thousand bioactive compounds in 
nearly 30 laboratory models of nerve cell degeneration. Over 75 percent 
of these drugs were already FDA-approved, which means that researchers 
could also access some information on the toxicity of these compounds 
in humans. The availability of toxicity information is a significant 
advantage for the research community, since it can save years of time 
in the drug development process for any agents that are moved forward 
into subsequent testing. In the models that were most relevant to ALS, 
a group of antibiotics related to the penicillins emerged as the 
candidates with the most potential for further study. These antibiotics 
are not only effective in killing bacteria; they were also found to 
protect cells from the toxicity of mutant SOD1 and to activate a gene 
for a glutamate transporter--a protein found on the surface of glial 
cells (non-neuronal cells that provide support and nutrition to nerve 
cells) that helps remove excess glutamate from the spaces surrounding 
nerve cell connections. Too much glutamate in these spaces can stress 
cells via overexcitation; and this process may occur in people with 
hereditary ALS as well as the sporadic form of the disease. After 
identifying this family of compounds in the large drug screen, 
researchers moved quickly to evaluate the most promising member of this 
family--ceftriaxone--in additional laboratory tests and preclinical 
studies of neuroprotection. Recent results indicate that ceftriaxone 
can stimulate the glutamate transporter gene in intact animals, and can 
protect neurons both in animal models of oxygen deprivation injury and 
ALS. Moreover, NINDS, in partnership with ALSA and Project ALS, 
contracted a study that demonstrated that ceftriaxone can delay the 
loss of muscle strength and death in an animal model of ALS as well.
    With these results in hand, clinical investigators designed an 
integrated clinical trial to explore the safety, tolerability, and 
ultimately the efficacy of ceftriaxone in people with ALS. Typically, 
NINDS relies on investigator-initiated research proposals to attain 
most of the Institute's clinical research goals. In most cases, these 
investigators conduct Phase I and Phase II studies to explore dosing, 
safety, and tolerability, then analyze the results before submitting a 
separate application for a Phase III trial to explore the efficacy of a 
particular therapy. In the ceftriaxone trial, NINDS worked with the 
applicant to use a design feature that is new to ALS and neurology, but 
not to the field of clinical research, that allowed the separate trial 
phases to be combined into one integrated study. In the three-step 
ceftriaxone trial, investigators will first determine the optimal 
dosage of ceftriaxone in a small group of 60 subjects, and will 
continue in a second step to examine the safety and tolerability of the 
drug in these same individuals. If sufficient levels of the drug are 
well tolerated in these participants, the researchers will expand the 
trial to 600 participants, in order to determine if the drug prolongs 
survival. The advantages of this approach are that it can eliminate the 
9-22 months that are often required for the review of a Phase III trial 
application, and the study can still be stopped early if the equivalent 
of the Phase II results are negative or discouraging. NINDS has 
recently initiated funding for this trial and expects to begin 
recruitment in the fall; the Institute hopes this trial will serve as a 
first step in the successful translation of this therapy into clinical 
practice.
    Many researchers exploring potential treatments for 
neurodegenerative diseases have also considered the antioxidant and 
health supplement coenzyme Q10 (CoQ10) as a promising candidate. Its 
ability to penetrate the nervous system and protect cells from 
oxidative stress, combined with its excellent safety profile, has 
stimulated interest in the drug for the treatment of Parkinson's 
disease, Huntington's disease, and ALS. In April 2005, enrollment began 
for a Phase II trial supported by NINDS that is designed to examine the 
potential of high-dose CoQ10 to treat ALS. Like the ceftriaxone study, 
this trial will also be conducted in several sequential steps. In the 
first part of the trial, investigators will identify the optimal dose 
of the drug; in the second, they will collect preliminary evidence of 
efficacy using a number of different outcomes, including functions 
needed for daily living, and measures of lung capacity, fatigue, and 
quality of life. Although a conclusive determination of efficacy may 
not be available at the end of this study, NINDS hopes that it will 
facilitate the collection of data needed to plan a phase III trial.
    Although the need for therapies designed to intervene in the 
cellular events that cause ALS is essential, NINDS also supports 
strategies to prolong survival by improving the clinical care of ALS 
patients. Specifically, the clinical literature suggests that 
respiratory and nutritional support can independently improve survival 
in people with ALS. However, issues such as the identification of the 
optimal timing for initiating respiratory support; the best method for 
improving the tolerability of appliances that facilitate respiration; 
and the development of better techniques to assess the balance of 
energy consumption and use have not been addressed in well-designed 
clinical studies. NINDS has recently funded a Phase II trial to collect 
data on these and other issues; this information will enable 
investigators to design a Phase III trial of combined respiratory and 
nutritional therapy. This trial is slated to begin enrollment in the 
very near future, and NINDS is enthusiastic about the possibility that 
these two approaches might have synergistic effects in treating ALS, 
and may in the future be combined with therapies that target the 
cellular mechanisms of the disease.

                         TRANSLATIONAL RESEARCH

    Across its areas of responsibility, NINDS actively promotes 
translational research, which links findings in basic science 
laboratories with early-phase clinical trials. Specifically, NINDS has 
designed a large translational research program to facilitate the 
development of goal-directed, milestone-driven research projects, and 
issued three Program Announcements (PAs) in July 2002 as part of this 
project. These PAs target high-risk exploratory studies; large research 
programs that could be conducted under cooperative agreements with 
NINDS; and mentored research scientist awards--all focused solely on 
translational research goals. NINDS also established special review 
panels to evaluate applications received in response to these PAs, to 
ensure that the unique needs of translational research would be taken 
into consideration. To date, this program has provided support for a 
cooperative agreement that is enabling the research group responsible 
for the preclinical data on ceftriaxone to identify additional 
antibiotic and non-antibiotic compounds that may also have stimulatory 
effects on the glutamate transporter gene. In addition, NINDS is also 
supporting an exploratory project designed to evaluate another series 
of promising compounds originally tested in the drug screening program 
described above. In the latter project, investigators will assess eight 
compounds that can potentially protect neurons by enhancing glutamate 
uptake, validate any observed cellular effects in intact mice, and then 
test their downstream impact on disease in a mouse model of ALS.
    As a complement to the translational grants program, NINDS has also 
established a facility at the Southern Research Institute in 
Birmingham, Alabama, that is miniaturizing laboratory tests relevant to 
neurodegeneration; automating them via robotic technology; and then 
using them to rapidly screen a collection of approximately 100,000 
chemically diverse, non-proprietary compounds. By enabling the academic 
research community to have access to the type of drug screening 
resource that is normally only available to the pharmaceutical 
industry, the Institute is hoping to accelerate the identification of 
potentially useful therapeutics for a number of neurodegenerative 
diseases, including ALS. To date, two ``test-tube'' models of ALS are 
already being used at the facility to screen for drugs that may be 
useful in treating ALS, and researchers at the facility have already 
identified several drugs as possible ``hits'' in these screens.
    In addition to these specific programs, NINDS-funded researchers 
continue to independently explore potential therapies for ALS that 
target a wide range of cellular processes. As suggested above, 
inflammation is one potential contributor to ALS that is gaining 
attention among translational and clinical investigators. Along these 
lines, recent data have suggested that a novel anti-inflammatory 
compound called pioglitazone can improve motor performance, delay the 
death of motor neurons, and extend survival in a mouse model of ALS. 
Further work will be required to confirm these effects in animals, but 
the continued success of these researchers in identifying novel 
therapeutic compounds is encouraging.

                         BASIC SCIENCE RESEARCH

    As discussed earlier, research has implicated multiple cellular 
mechanisms--including cellular stress, overexcitation, and activation 
of cell death signals--in the motor neuron degeneration that causes 
ALS. Researchers are actively investigating these and many other leads 
in an effort to understand the disease and develop new strategies for 
treatment. In one example, they have found that in animals that are a 
chimera--having some cells with the SOD1 mutation, and others without 
it--the presence of normal glial support cells can delay the 
degeneration of motor neurons that harbor the mutation. This finding 
not only helps to explain the cause of ALS, but also suggests that 
glial support cells might be a reasonable target for the development of 
therapeutics.
    Mitochondria, the energy generators of the cell, are also emerging 
as a target of enhanced interest among ALS researchers, including those 
studying how mutations in the SOD1 gene cause motor neurons to be 
uniquely vulnerable in ALS. In 2004, two independent groups of 
researchers discovered important clues that suggest a prominent role 
for these cellular structures in the death of motor neurons. In one 
study, investigators explored the effects of SOD1 mutations on 
mitochondria, and found that the vulnerability of motor neurons in ALS 
may be linked to an unexpected buildup of mutant SOD1 proteins inside 
the mitochondria of the spinal cord, where they can cause the 
subsequent degeneration of affected neurons. In a second study, a 
separate group of researchers explored the function of the mutant SOD1 
protein and found that within spinal mitochondria, these proteins may 
bind to and specifically trap other proteins that are necessary for 
cells to survive. By taking the cell survival proteins out of 
circulation, the mutant SOD1 proteins may be contributing to the 
neuron's ultimate demise.
    NINDS-funded investigators also continue to study the genetics of 
ALS--specifically the genetic changes that lead to inherited forms of 
disease. In addition to the mutations in SOD1 that cause an adult-onset 
form of ALS (ALS1), researchers also discovered in 2001 that a mutation 
in a different chromosome can cause a rare juvenile-onset form of ALS 
(ALS2). Then, in 2004, investigators found a link between a second form 
of childhood-onset ALS (ALS4)--one with a clinical syndrome distinct 
from ALS2--and a known gene called Senataxin that is believed to play a 
role in the control of protein production following the activation of 
specific genes. This discovery involved the analysis of genetic 
material from four different families in the United States and Europe, 
highlighting the importance of the contributions from affected 
individuals to the research process.
    Although researchers now have a better understanding of the genetic 
contributors to ALS, the search for genes that play a role in this 
disease is not yet over. For many years, NINDS has recognized that 
genetic researchers would benefit from having access to many well-
characterized DNA samples from people with specific neurological 
conditions. To address this need, the Institute established a Human 
Genetic Resource Center at the Coriell Cell Repositories in New Jersey, 
in September 2002. As part of their contract, Coriell maintains a 
repository of data, cell lines, and DNA samples for the study of the 
genetic factors contributing to neurological diseases, including 
conditions like ALS that affect motor neurons. Genetic information is 
absolutely critical for the study of these conditions, as an 
understanding of the mutations in genes linked to ALS can help 
clinicians identify who is at risk for the disease, and can aid 
researchers in characterizing the disease process and identifying 
potential points of intervention. The positive effects of the 
repository on research in other neurological conditions are already 
evident, and NINDS is working with ALSA and the extramural research 
community to try to accelerate the rate of contributions of samples 
that are useful for ALS research.
    As mentioned above, an understanding of the genes that play a role 
in the development of ALS can serve as a springboard for researchers 
searching for new strategies to treat the disease. The promising 
technique of RNA interference (RNAi) is one such strategy that is 
receiving a great deal of attention across many fields of medical 
research for its potential in treating diseases caused by known genetic 
mutations. Investigators both here in the U.S. and overseas have 
already begun to explore its applicability to inherited forms of ALS. 
Researchers initiate RNAi by delivering small pieces of genetic 
material that match those coding for an unwanted protein in a cell. By 
``binding up'' the genetic intermediates that lead to toxic protein 
production, these proteins can be reduced or eliminated in the target 
cells. Although U.S. researchers are still in the very early stages of 
exploring this therapy for ALS, results from a recent study suggest 
that a well-designed RNAi strategy might be capable of simultaneously 
counteracting the more than 100 possible mutations in the SOD1 gene 
that can contribute to the development of hereditary forms of ALS. This 
proof-of-principle study is encouraging, and will hopefully lead to 
additional tests of this approach in animal models of the disease.
    Though much of the basic science research described above is 
focused on improving our understanding of the causes of ALS, NINDS also 
supports other areas of fundamental neuroscience that may have an 
impact on the disease. For example, nervous system plasticity and stem 
cell research are particularly promising fields of study for ALS 
researchers, since the replacement of the motor neurons lost to the 
disease and the stimulation of these replacement cells to make contact 
with their original targets offer a reasonable therapeutic strategy.
    It is widely recognized that the brain and spinal cord of adult 
mammals, including people, show a very limited capacity to regrow 
following injury. However, in recent years, researchers have shown that 
even the brains of adult mammals can generate new nerve cells under the 
right conditions. As an example, investigators have recently found that 
the brains of adult mice can generate new corticospinal motor neurons--
which control voluntary movement via long nerve fibers they extend from 
the brain to the spinal cord--if the normal motor neurons are destroyed 
in a particular way. Importantly, these new nerve cells were also able 
to regrow their long extensions and make distant connections with their 
spinal cord targets. These findings are encouraging, as they suggest 
that in some cases, the body may be able to produce its own replacement 
cells, and that these cells may be able to make the contacts needed to 
restore lost function. Further understanding of what controls the 
generation of these cells and the growth of their long nerve fibers may 
facilitate the development and optimization of repair strategies for 
conditions like ALS and spinal cord injuries.
    While promising, using ``internal'' replacement cells is only one 
approach to restoring the nerve cells lost in ALS. Investigators are 
continuing to explore other possible approaches as well, such as 
stimulating stem cells to produce a pool of motor neurons for nerve 
cell replacement therapy. Recently, one such group of researchers has 
found that motor neurons derived from human fetal neural stem cells can 
survive transplantation, make connections with target muscles, and 
support improvements in motor function in a rat model of motor neuron 
degeneration. In addition, a separate group of investigators has shown 
that they can successfully cause a line of federally-approved human 
embryonic stem cells to specialize to become motor neurons. Their step-
wise procedure involved a sequential application of growth-stimulating 
molecules to the cells--molecules that researchers had previously 
identified as being important during nervous system development. 
Potential uses for these types of cells include additional studies on 
the development of motor neurons, the screening of drugs that could be 
useful in treating ALS, the testing of therapeutics in animal models of 
disease, or ultimately, the replacement of motor neurons in people with 
ALS.

                        TOPICS FOR FUTURE STUDY

    While ALS researchers understand more than ever about the causes of 
and potential treatments for this disease, many questions remain 
unanswered. Are there additional genes involved in hereditary ALS? How 
are the rare inherited forms of the disease linked to the more common 
sporadic forms? What triggers cell death in the sporadic cases? How can 
we intervene early in the disease process to protect motor neurons when 
they first become vulnerable to the cellular events underlying ALS? Why 
are these cells uniquely vulnerable? Are there additional drugs already 
available that might reduce the burden of ALS?
    Recently-developed programs at NINDS, including the accelerated 
drug screening efforts and DNA repository, are fueling a more 
aggressive approach to these questions within the research community. 
To complement these efforts, NINDS also sponsored a workshop in January 
2003 to explore the remaining gaps in our understanding of ALS and 
motor neuron biology, and released a Request for Applications (RFA) in 
August 2003, jointly with the Department of Veterans Affairs and ALSA. 
This solicitation encouraged studies of ALS in a broad range of 
research areas, including the causes of disease across broad 
populations, including genetic and environmental causes; the cellular 
interactions that contribute to the disease; the cellular and sub-
cellular problems in affected tissues; novel approaches to delivery of 
therapies; and biomarkers for early disease detection. NINDS funded 
five applications that were responsive to this solicitation, and NIH's 
National Institute of Environmental Health Sciences contributed funds 
to support two additional awards.

                              CONCLUSIONS

    NINDS has a history of funding strong basic science research on 
ALS, but the translation of these findings into effective clinical 
therapies for this disease has been extremely challenging. We realize 
that these challenges must be overcome, and we believe that the 
multifaceted approach we have taken will be the key. While still 
maintaining a vibrant program of preclinical research, we have now 
complemented this work with multiple opportunities for translational 
research to flourish, an active drug screening program that is leaving 
no stone unturned in the search for readily available drugs with 
promise against ALS, and several new and exciting clinical trials. 
While it is still not possible to guarantee when a cure for ALS will be 
developed, we are extremely encouraged by the progress of the research 
community, and we hope that this excitement extends to our most 
important constituents--the community of people with ALS and their 
families.
    Mr. Chairman and Members of the Subcommittee, thank you for the 
opportunity to share this information with you. I will be happy to 
answer any questions you may have.

    Senator Shelby. Dr. Landis, you have touched on this 
already, but specifically has the doubling of the NIH budget 
and the significant increase in ALS specific funding helped 
accelerate clinical research on ALS and, if so, just briefly 
tell us how?
    Dr. Landis. It absolutely has. Before these five new 
clinical trials were undertaken, we had only ever been able to 
do one clinical trial in ALS. We now have four underway and 
recruiting patients. A fifth that will begin.
    That fifth trial is a direct result of a new program in 
translational research that we funded with the doubling. It 
involved a screen of a thousand FDA-approved compounds. Out of 
that, a number of hits arose. Through this translational 
research program that we were able to initiate with additional 
funds, we have three separate projects that are looking at 
different strategies for treating ALS.
    Senator Shelby. How many Americans are being treated for 
ALS roughly?
    Dr. Landis. So the best figures that we have are that there 
are 5,000 patients that are diagnosed each year. Because this 
is a very rapidly-progressing disease, probably any one time, 
there are between 15,000 and 20,000 patients with ALS in this 
country.
    Senator Shelby. What are you doing to slow the progression 
of ALS? I know you said basically there is no cure yet----
    Dr. Landis. Right.
    Senator Shelby [continuing]. For it. But you have made 
some--you have had a head start in that direction.
    Dr. Landis. So all of the--out of the clinical trials that 
we are running, four are directed at neuroprotection, at 
slowing the progression. These drugs which are being tested in 
these clinical trials showed efficacy in this mouse model, SOD1 
mouse model that I told you about.
    Senator Shelby. Doctor, is it true that only one drug, one 
FDA approved drug is on the market to treat ALS?
    Dr. Landis. Yes. Just one.
    Senator Shelby. Just one?
    Dr. Landis. Riluzole was approved 10 years ago. I would say 
that it is always depressing when you look at a disease as 
devastating as this one to have not made further progress.
    The identification of the SOD1 mutation has allowed us to 
come up with better hypotheses and better animal models which 
has resulted in this significant increase in the number of 
strategies to develop therapies for ALS and these clinical 
trials.
    We anticipate if we had more genes, we would have better 
ideas about how to design therapies and more clinical trials.
    Senator Shelby. What would you need to accelerate the 
research? I know you need more resources. We understand that 
and this is an appropriations committee----
    Dr. Landis. Right.
    Senator Shelby [continuing]. There is a big fight 
everywhere for dollars today and always has been. But what 
could you do with additional money and what would you be 
thinking about?
    Dr. Landis. So I have already indicated gene discovery 
which allows one to develop better pre-clinical models. We have 
a number of investigators who have assays which can be used for 
pre-clinical screening of potential therapeutic molecules.
    The problem is oftentimes those initial screens, the 
compounds in those initials screens are not appropriate for 
direct movement to people. The FDA drug screen was important 
because all those were FDA approved. But in these new screens, 
we are coming up with molecules that are known to be safe, 
efficacious and get to the brain. So medicinal chemistry is a 
very important piece of what we would spend money on.
    Senator Shelby. Additional money would be very helpful.
    Dr. Landis. Absolutely. We would put those monies to very 
good use. I think if I had been asked to testify at a hearing 
like this 5 years ago, I would have not had nearly as much 
positive to report.
    I can tell you that we have invested the dollars, as you 
can see from the progress in ALS, very wisely. I hope that the 
fruits of that investment will make a difference for patients 
now and patients in the future.
    Senator Shelby. Doctor, you referenced translational 
research.
    Dr. Landis. Right.
    Senator Shelby. Research that bridges basic science 
findings with clinical therapies. Is any of this research 
relevant to ALS and, if so, how?
    Dr. Landis. Absolutely. I will give you an example of one 
project which will begin funding on June 1.
    We know that in some animal--in culture, tissue culture and 
in some animal models, insulin-like growth factor can make a 
difference for motor neuron survival. We have had one clinical 
trial that was successful, a second that was unsuccessful. We 
are conducting a third where it is given systemically.
    But there is very good evidence that if you deliver it 
specifically to the terminals of motor neurons it helps 
survival. We are funding a translational project which will 
help investigators take this basic science finding to the point 
where they could apply for an IND for the FDA and begin a 
clinical trial.
    Senator Shelby. Thank you.
    Senator Harkin.
    Senator Harkin. Thank you, Mr. Chairman.
    Dr. Landis, my first question is very simple. Why has it 
taken so long to do anything about this disease? Forty years--
60 years now since Lou Gehrig had it. We just know very little 
about it, what causes it, how to cure it.
    Now I am finding out there is a whole subset of veterans 
who seem to be more susceptible to it. Why? I mean, why do we 
know so little about it? After all these years, why do we know 
so little about it?
    Dr. Landis. Well, I do not know that I would say we do not 
know so little. It is that we do not know enough to actually 
make as big a difference as we would like to for patients.
    Any of the neurodegenerative diseases, and we are 
responsible for a host of them, target specific populations of 
neurons. A puzzle has been why are motor neurons susceptible in 
ALS, why are dopamine neurons susceptible in Parkinson's. It is 
difficult to come up with strategies that allow you to look at 
individual populations of neurons and understand why they are 
susceptible and also to understand how to make them better.
    For any nervous system disease, a major problem in 
delivering therapy is something called the blood-brain barrier. 
So there is a cellular barrier between the blood and the brain 
parenchyma which in most cases is protective, keeps toxins out. 
But if you want to deliver a therapy to the brain, you have to 
figure out a way to get it across the blood-brain barrier. This 
has been very challenging.
    Therapy that we need that would be given systemically needs 
to be something that would get across the barrier or it needs 
to be delivered directly to parenchyma. We have made 
extraordinary strides and we will continue to make them. It is 
just not enough yet.
    Senator Harkin. One of the things I have learned about ALS 
is there is no real single test to determine whether someone 
has ALS.
    Again, referring to my friend, Rob Borsellino, he talks 
about how you go from doctor to doctor to doctor and no one 
really knows what is going on until a lot of time passes. Are 
we making any progress in terms of early detection?
    Dr. Landis. This is a very challenging problem. Again, it 
is not unique to ALS. It is a problem for many of our 
neurological diseases. There is a lot of redundancy and extra 
capacity in the nervous system and oftentimes symptoms do not 
appear until 20, 30, 40, 50, or 60 percent of the nerve cells 
are gone.
    People often go to a general practitioner. They complain of 
weakness and it is not until they get to a neurologist that 
they actually get the appropriate tests done to determine what 
is wrong. If we had a blood test, it would be spectacular and 
we do not have that. So let me give you----
    Senator Harkin. Is your department looking at this?
    Dr. Landis. So let me give you an example of one of the 
things that we could do if we had more money. In these ALS 
clinical trials, we will be enrolling a thousand or more 
patients. If we could collect biological samples from----

                     AMYOTROPHIC LATERAL SCLEROSIS

    Senator Harkin. How much more money do you need?
    Dr. Landis. We would need $9 million.
    But the biomarkers are a very interesting strategy. If we 
could collect blood, biological samples from each of these 
patients who were in these trials, and that is not part of the 
funding presently planned for those trials, we could then use 
proteomics and genomics to look at what markers are present, 
what markers are present in these patients that are absent from 
controls.
    Senator Harkin. Let me ask you another question about 
clinical trials. A couple of people have asked about clinical 
trials. If you enter a clinical trial, you do not know whether 
you are getting the drug or not.
    Dr. Landis. Right.
    Senator Harkin. When my brother was dying of cancer and he 
was looking around for different things, there were some 
experimental drugs at the National Cancer Institute.
    Dr. Landis. Right. Different institute.
    Senator Harkin. I know it is not yours. I understand that.
    But my point is they were doing some clinical trials on 
some new drugs. My brother said, well, wait a minute. I am 
dying anyway. Why put me in a control group. Why not just give 
me the drug and see what happens.
    So I mean, ALS, let us be frank about it, I mean, except 
for a few cases like Roger who has lived for 10 years, I mean, 
people are facing a death sentence.
    Dr. Landis. Right.
    Senator Harkin. My brother knew he was dying of cancer and 
he said I do not care. If I die of the drug, I die of the drug. 
I am going to die one way or the other. I might as well take 
the drug.
    My point is for this class of people, why--I can see doing 
some things--why not if someone really says, look, I am willing 
to be a guinea pig--my brother, for example, was willing to be 
a guinea pig--why not let them be a guinea pig? Why not?
    Dr. Landis. So one of the basic tenets of clinical trials 
research is that you need to have a double blind control 
group----
    Senator Harkin. I understand that.
    Dr. Landis [continuing]. To determine whether or not the 
effects that you are seeing are a function of placebo. There 
are very good examples where patients receiving the placebo 
have their health status improve as much as patients who are 
receiving a drug because of wiring in the brain that is able to 
influence health. The only way we are going to know if 
something is efficacious is to compare it to either best 
treatment or a control group. There is no simple way around 
that.
    Senator Harkin. I understand that. But I am just saying 
that if someone was willing, of sound mind, and could make that 
informed decision, why not let them? If there is an 
experimental drug out there that may have some hope and someone 
is willing as my brother was--I mean, he took a chance. Did not 
help him, but--and they did. They gave him an experimental drug 
because he was willing to do it.
    Dr. Landis. So that is a strategy whereby the person could 
receive the drug knowing that they were receiving it.
    Senator Harkin. Right.
    Dr. Landis. But not be part of a clinical trial because in 
the clinical trial, you have to----
    Senator Harkin. That is right. That is what I am saying.
    Dr. Landis. Right. But then that person would not be 
participating in a clinical trial. If one of the goals----
    Senator Harkin. I am saying can that--I am asking a 
practical question. Could someone who is willing to take an 
experimental drug that someone has at least indicated may have 
some hope, could they get--we used to call it a----
    Dr. Landis. Compassionate use of the drug.
    Senator Harkin. Compassionate release or whatever. Could 
that happen?
    Dr. Landis. That could happen. That participation, taking a 
drug under circumstances like that could--and having access to 
drugs like that, through that kind of mechanism could slow the 
development of therapeutics because one of the most difficult 
problems in clinical research is patient recruitment. We would 
have much more information about therapies for diseases if we 
were able to speed recruitment.
    We have on occasion closed trials because people will not 
volunteer. If you could get what you perceived to be the drug 
that would be effective outside of the trial, I think it would 
decrease recruitment.
    Senator Harkin. Thank you. I see my time is up.
    Mr. Chairman, my time is up. But I hope that before Dr. 
Landis is excused, I would like to have one follow-up question.
    Senator Shelby. Senator Murray.
    Senator Murray. Thank you, Mr. Chairman.
    I have worked very hard to increase access to health care 
for our veterans because I believe we owe them nothing less 
than quality affordable health care for the service to our 
country. I think that is especially true of our veterans with 
service connected injuries or illness.
    Recently the VA recognized that ALS as a possible service 
connected disability for our Persian Gulf veterans. I really 
applaud their decision to quickly recognize that link and to 
provide the necessary benefits for the veterans who have been 
affected.
    I have also worked very hard to support expanded DOD 
research, Department of Defense research on surveillance of 
neurological diseases within the population of people who have 
served our country.
    Can you tell me if NINDS has been working with the VA and 
the DOD on these efforts?
    Dr. Landis. Absolutely. In the case of ALS, we have 
sponsored grant solicitations with the VA. We have a group of 
institutes and centers that interact with the DOD and with the 
VA more generally for neurodegenerative diseases. We have 
strong ties to them.
    It is very clear when money is tight, we need to make sure 
that there is not duplication and also that all possible 
opportunities for advances are exploited.
    Senator Murray. Is anybody at NINDS or any other institute 
at NIH working on research involving potential toxic exposure 
triggers for ALS?
    Dr. Landis. Yes. This is within the purview of the National 
Institute of Environmental Health Sciences and they have 
recently funded a grant that will be looking at exposures and 
genetic susceptibilities to exposures that veterans might have.
    Senator Murray. What are we learning?
    Dr. Landis. It just got funded. So it will be unfortunately 
several years before we have results of that.
    Senator Murray. Is there anything we can do to help better 
coordinate NIH research and DOD and VA research?
    Dr. Landis. I think that we recognize the importance of 
coordination and that each part of the government has 
opportunities and strategies for expediting research and that 
we try to communicate often enough to make sure that each of us 
is doing the things that we can do best.
    This also is true for the health voluntary organizations. 
We have significant coordination with them. They can sometimes 
be a bit more nimble to undertake novel and innovative 
strategies which then the NIH can pick up.
    Senator Murray. Okay. If you can help us do the right thing 
so we can better coordinate this because I think this has 
tremendous potential. I think we have a responsibility 
obviously to make sure we are doing it correctly.
    Chairman Shelby asked you a question about funding and what 
you could do with more funding. Let me ask you kind of the 
reverse of that. President's budget calls for 1 percent 
increase for NIH. I am deeply concerned about how the 
Appropriations Committee is going to be able to deal with the 
difficult task of balancing a lot of very, very difficult 
requests this year.
    Can you tell us how this limited budget that has been 
proposed is going to impact your ongoing research?
    Dr. Landis. So during the time when we received significant 
increases, it was possible for my institute to undertake a 
number of novel programs including translational research, 
expanding our support for clinical trials, increasing the 
number of physicians, scientists that we are training, loan 
repayment, developing strategies for clinical trials to be 
undertaken in rare diseases.
    At the present time with the President's budget, any time 
we do something new, a creative, innovative program, we are 
going to have to stop doing something that we have been doing. 
For example, some of the compounds that are being tried in 
clinical trials for ALS are also being looked at for other 
neurodegenerative diseases. They could have wide applicability.
    Senator Murray. This budget, if it is enacted, is going to 
be a step backwards for you?
    Dr. Landis. Yes.
    Senator Murray. Will any researchers who are currently 
working be reduced? Are you going to have to lay off any 
researchers as a result?
    Dr. Landis. If we are to fund new programs, we will have to 
stop funding old programs. One of the issues that the NIH is 
dealing with now is we know that we are not going to solve, sad 
to say, our diseases which are devastating, difficult, 
challenging diseases in the next 5 years.
    To have investigators who will be able to carry the banner 
forward, we need to be investing in new and young 
investigators. For every young investigator, a senior 
investigator will be unfunded. For every senior investigator 
who is refunded, it means a junior investigator----
    Senator Murray. So we know, if the President's budget is 
enacted, will any of the research that is currently being done 
be delayed?
    Dr. Landis. We have gone from being able to fund 25 percent 
of grants at the height of the doubling to being able to fund 
16 percent. What this means is that investigators will 
oftentimes have to submit their grant two or three times before 
they get money.
    Senator Murray. So it will have a significant impact?
    Dr. Landis. It will have a significant impact.
    Senator Murray. Thank you. I really appreciate that.
    Dr. Landis. You're welcome.
    Senator Murray. Thank you, Mr. Chairman.
    Senator Shelby. Just one quick follow-up, Dr. Landis. Would 
it be helpful for ALS research if NIH funded researchers could 
access more stem cell lines than are currently allowed?
    Dr. Landis. Scientists believe and make very compelling 
arguments that more stem cell lines would be better. Up until a 
year and a half ago when I took this job, I myself was a 
practicing scientist and know that it would be more useful. You 
have already outlined some of the issues: Mouse feeder layers, 
change in the stem cell lines with time. Not all stem cell 
lines are equally able to be turned into different derivatives. 
An issue which has not been discussed here is that it would be 
very helpful to have stem cell lines that have the same 
mutations in them that cause disease in humans. Right now the 
models that we have are at best models.
    In order to get a mouse to have an ALS-like syndrome, you 
have to significantly over express the SOD1 mutation. Well, in 
people, one copy of a mutant SOD1 mutation causes disease. So 
we could make significant use of those lines were they to be--
if more lines were to be available.
    Senator Shelby. Thank you, Dr. Landis, and we are going to 
continue to work with you to get appropriate funding----
    Dr. Landis. Thank you very much.
    Senator Shelby [continuing]. To find some breakthrough in 
this disease.
    Dr. Landis. We will put the money to good use.
    Senator Shelby. I know you will. Thank you very much.
    Our second panel will be composed of Dr. Lucie Bruijn. She 
is the ALS Association science director and vice president. 
Before coming to the association, Dr. Bruijn focused her 
research on the treatment of neurodegenerative diseases. She 
received her Master's in Neuroscience and a Ph.D. in 
biochemistry from the University of London Institute of 
Psychiatry.
    Dr. Bruijn, thank you for being here today. We look forward 
to your testimony.
    We also will have on the second panel Mr. Eric Obermann of 
Huntsville, Alabama. He was diagnosed with ALS when he was 20 
years old. Prior to his diagnosis, Eric was a student at 
Georgia Tech University where he studied computer science and 
played in the University symphony.
    Now 23 years of age, Eric is unable to walk or talk and 
requires the use of a ventilator to help him breathe. I 
consider Eric to be an extraordinary young man and I am glad to 
have here today to hear his testimony.
    Eric's father, Stewart Obermann, will accompany him. Mr. 
Obermann is the Chairman of the ALS Association, the North 
Alabama Friends Group. He is also the former chairman and CEO 
of Mobular Technologies, a Huntsville, Alabama based Internet 
company.
    Mr. Obermann, I appreciate your willingness to sit at the 
table with Eric. I believe you can provide us all some 
important perspective on what effect ALS has had on the 
families of ALS patients.
    Eric, I understand that you have prepared an opening 
statement today. From what I understand, it was only with 
significant time and effort. We all greatly appreciate your 
willingness to come today to tell us about your experience and 
to help us understand what ALS is and what it does.
    Eric will be using a computer to give his testimony today. 
I understand it has taken him hours to put this together.
    Then we have--you want to introduce Mr. Borsellino? You 
alluded to him earlier.
    Senator Harkin. I can run through the whole thing again, 
but I think I gave you Rob Borsellino's background and the fact 
that, again, what really strikes me is he has got two young 
kids. As I said, he has got a dedicated following in the State 
of Iowa.
    We have all just been shaken by this happening to someone 
that we know and we follow daily. I guess we all have the same 
question: Why? Why just out of the clear blue sky does this 
happen to someone? I just want to thank Rob for being here 
today.
    Senator Shelby. Dr. Bruijn.
STATEMENT OF LUCIE BRUIJN, SCIENCE DIRECTOR AND VICE 
            PRESIDENT, ALS ASSOCIATION
    Dr. Bruijn. Senator Shelby, Senator Harkin, and members of 
the subcommittee, thank you for inviting me today to appear 
before you. I commend you for holding this important hearing 
and for your efforts to support people with ALS in your home 
States and across the country.
    My name is Lucie Bruijn. I am the science director and vice 
president for the ALS Association. The ALS Association is the 
only national not-for-profit health association dedicated 
solely to the fight against ALS or Lou Gehrig's disease.
    In addition to serving as a resource for people with ALS 
and their families, the association and more than 41 chapters 
and affiliates across the country advocate for increased 
funding for ALS research and other health care reforms that 
respond to the needs of people with ALS.
    The ALS Association is the largest private source of 
funding for ALS specific scientific research in the world and 
having awarded nearly over $30 million since 1995 to fund the 
best research to identify the cause, means of prevention, and 
cure for ALS.
    ALSA funded scientists are currently looking at over 15 
areas of research relevant to the disease including stem cell 
therapy, gene therapy approaches to treatment, biomarkers for 
early diagnosis of the disease, and identification, as Dr. 
Landis so well described, of new genes linked to the disease.
    Currently ALSA is funding over a hundred promising research 
projects in 14 different countries in the world.
    I am particularly pleased today to be appearing before you 
with my colleague, Dr. Story Landis, the Director of the 
National Institute of Neurological Disorders and Stroke. Dr. 
Landis is one of the world's leading experts in neurological 
disorders and has been one of ALSA's champions through the 
years.
    It is through collaboration and with ALS experts throughout 
the world and together with the NIH that we can and we will 
make real progress in the disease.
    I am pleased to say that through the efforts of the 
subcommittee and the Congress as a whole the ALS community has 
been able to benefit from vital funding provided to the NIH.
    In fiscal year 2005 NIH and NINDS will provide more than 
$48 million in funding for ALS specific research and we hope 
that both the Congress and NIH will continue to make this 
funding a priority again in fiscal year 2006.
    Collaborations between ALSA and NINDS have been extremely 
successful as Dr. Landis so clearly put today. As she 
mentioned, the screening of a thousand FDA approved compounds 
and model systems of ALS has led to one important compound 
being tested in clinical trials which the NINDS is funding.
    In addition, I will emphasize again the importance of the 
establishment of this repository that the NINDS has done and 
this will provide us with important genetic clues. This is a 
resource that will be available widely for all investigators 
here and internationally to learn more about the genetics of 
the disease and ultimately to have new targets for therapies.
    Why do we only have one FDA approved compound? We need to 
have much better and novel compounds and this is a huge 
challenge.
    I am pleased, therefore, today to announce that the ALS 
Association is launching a new program, TREAT ALS, 
Translational Research Advancing Therapy for ALS.
    Researchers have made tremendous progress over the years 
both scientifically and in technological fields. This knowledge 
has enabled us to design the laboratory models as described and 
lead to innovative ideas. The time is right to translate these 
more rapidly into clinical trials for patients.
    TREAT ALS will be led by a steering committee of biologists 
and chemists and by noted Dr. Tom Maniatis of the Molecular and 
Cellular Biology Department of Harvard who lost his sister to 
the disease and testified here in May 2000.
    So tangible progress will be turned towards the treatment 
of cures for the patients. Translational research and clinical 
trials will find the drugs that will prevent, halt, or 
significantly slow the disease. We will support the development 
of lead compounds.
    We are extremely excited about this program, but it will 
need significant financial resources. We hope that both 
Congress and the NINDS will work with us so that this program 
will be a success and will benefit the ALS community in the 
United States and around the world.
    In addition, we are currently working with Senator Reid and 
Members of the Congress to offer the establishment of a 
national ALS registry at the Centers for Disease Control.
    A national patient registry does not exist today. The value 
of such a registry cannot be overstated. It is vital to 
understand the disease, its management, and the development of 
standards of care.
    Another issue mentioned today is the need for more research 
of the veterans. One way in which the Congress can not only 
help veterans with ALS but all people with ALS is to support 
funding through the peer reviewed medical research program at 
the Department of Defense and recommend that ALS continue to be 
a disease that is studied under the program.
    I have before you today a summary or white paper of ALS and 
the military for the records.
    Finally, we hope that the promise of stem cell research 
will open up new avenues for ALS to understand the disease, as 
a drug screening tool, and in the future as a therapy for ALS.
    We are very excited about the new and promising avenues for 
ALS research. Thank you again for this opportunity to appear 
before the subcommittee.

                           PREPARED STATEMENT

    The ALS Association appreciates your previous support for 
our cause, urges your continued support, and we look forward to 
a Nation that commits itself to finding a treatment and 
ultimately a cure for this horrific disease.
    I'm very happy to answer any questions you may have.
    [The statement follows:]

                   Prepared Statement of Lucie Bruijn

    Chairman Specter, Senator Harkin, and Members of the Subcommittee, 
thank you for inviting me to appear before you today. I commend you for 
holding this important hearing and for your efforts to support people 
with ALS in your home states and across the country.
    My name is Lucie Bruijn and I am the Science Director and Vice 
President for The ALS Association. The ALS Association (ALSA) is the 
only national not-for-profit health association dedicated solely to the 
fight against ALS, or Lou Gehrig's disease. In addition to serving as a 
resource for people with ALS (PALS) and their families, The Association 
and our more than 41 Chapters and affiliates across the country 
advocate for increased funding for ALS research and other health care 
reforms that respond to the needs of people with ALS. The ALS 
Association also is the largest private source of funding for ALS-
specific scientific research in the world, having awarded nearly $30 
million since 1995 to fund research seeking to identify the cause, 
means of prevention and cure for ALS.
    ALS is a progressive, neurodegenerative disease that attacks nerve 
cells and pathways in the brain and spinal cord. Motor neurons reach 
from the brain to the spinal cord and from the spinal cord to muscles 
throughout the body. It is through these neurons that we are able to 
control all muscle movement, whether it be moving our arms and legs, or 
simply breathing or opening and closing our eyelids. As the disease 
progresses and these motor neurons cease to function and die, our 
ability to initiate and control muscle movement is lost, ultimately 
resulting in total paralysis in the later stages of the disease.
    However, what makes ALS particularly devastating is that as people 
progressively lose the ability to walk, move their arms, talk and even 
breathe, their minds remain sharp; acutely aware of the limits ALS has 
imposed on their lives. That is to say, people in the later stages of 
the disease continue to think, reason, and have the same emotions, but 
they are trapped inside a body they no longer can control or use to 
communicate.
    Early symptoms of ALS often include increasing muscle weakness, 
especially involving the arms and legs, slurred speech, and difficulty 
swallowing or breathing. When muscles no longer receive the messages 
from the motor neurons that they require to function, the muscles begin 
to atrophy (become smaller) and our arms and legs also begin to look 
thinner.
    The average life expectancy for a person with ALS is two to five 
years from the time of diagnosis. We currently do not know what causes 
ALS or how it can be prevented and cured. Moreover, only one drug, 
approved by the FDA in late 1995, currently is available to treat ALS. 
Thus far, the drug, Rilutek, only has shown limited effects, prolonging 
life by just a few months.
    As I mentioned, The ALS Association is the largest private source 
of funding for ALS-specific scientific research in the world. ALSA-
funded scientists currently are looking into 15 different research 
areas relevant to ALS including stem cell and gene therapy approaches 
to treatment, biomarkers for early diagnosis of the disease and 
identification of new genes linked to the disease.
    In May 2000, ALSA announced an aggressive, new initiative to 
rapidly accelerate the search for a cure for ALS--The Lou Gehrig 
Challenge: Cure ALS. In just 4 years, $16 million has been raised 
toward the initiative's $25 million goal. The strategy of this program 
is to recruit outstanding investigators, identify the most promising 
directions in ALS research and develop new ALS therapies. Currently, 
ALSA is funding more than 100 promising research projects around the 
world.
    ALSA also regularly convenes scientific workshops to examine new 
trends in ALS research, providing vital leadership in areas ranging 
from examining research on ALS and the environment to engaging and 
educating young investigators.
    ALSA's clinical management research program focuses on managing the 
care of ALS patients in areas such as nutrition, respiration, mobility, 
quality of life, and psychosocial needs. Currently, ALSA is funding ten 
clinical management research grants, representing a commitment of more 
than $414,000.
    Although The Association continues to initiate, fund and support 
ALS research, our most effective and promising programs have occurred 
as the result of collaboration with ALS experts--individuals and 
institutions--throughout the world. I am pleased to say that through 
the efforts of this Subcommittee specifically, and the Congress as a 
whole, the ALS community has been able to benefit from vital funding 
provided to the National Institutes of Health and, within NIH, to the 
National Institute of Neurological Disorders and Stroke (NINDS). In 
Fiscal Year 2005, NIH and NINDS will provide more than $48 million in 
funding for ALS specific research and we hope that both the Congress 
and NIH will continue to make this funding a priority again in Fiscal 
Year 2006.
    I am particularly pleased to be appearing before you today along 
side my colleague Dr. Story Landis, the Director of NINDS. As you know 
Dr. Landis is one of the world's leading experts on neurological 
disorders and has been one of ALSA's champions through the years.
    Collaborations between ALSA and NINDS, including funding for 
research projects, have been extremely successful in recent years. For 
example, NINDS and ALSA partnered to screen 1,040 Food and Drug 
Administration (FDA) approved compounds in model systems of ALS, one of 
which will be funded by the NINDS in clinical trials later this year. 
In addition we are working with NINDS to establish a repository of 
genetic materials from ALS patients. This will be a vital resource 
internationally for investigators to understand more about the 
mechanisms leading to cell death in ALS.
    As you can see, the leadership and expertise provided by NIH and 
NINDS have enabled us to move forward in the fight against ALS. But 
much more work remains to be done to learn more about the disease, its 
method of action, what causes ALS, and how it can prevented, treated, 
and ultimately cured.
    It is with those goals in mind that The ALS Association today is 
launching TREAT ALS, a comprehensive new initiative that will 
concentrate efforts toward the rapid discovery of new therapeutics for 
ALS. TREAT ALS stands for Translational Research Advancing Therapy for 
ALS, and entails a two-pronged approach to discovering new treatments 
for the disease. First, the initiative will prioritize existing drugs 
that may be candidates for clinical trials, and second, it will take on 
the task of drug discovery by identifying new compounds with promise 
for the disease.
    Researchers have made tremendous scientific and technological 
advances in the ALS field. We understand far more about the biological 
basis of the disease. This knowledge has enabled design of laboratory 
models of ALS that have yielded innovative ideas and clinical 
candidates. The time is right to translate these advances into 
effective therapeutics for ALS patients.
    Already partnerships have formed, between The ALS Association 
(ALSA), other ALS organizations, and biotech to bring new and existing 
compounds to clinical trials. The National Institutes of Health in 2003 
launched its own road map program that emphasizes the search for new 
medicines. ALSA is building on this momentum.
    A steering committee is in place to guide TREAT ALS of prominent 
biologists, chemists and business advisors including noted biologist 
Tom Maniatis, Ph.D., of the Molecular and Cellular Biology Department 
at Harvard University, who lost his sister to ALS and testified before 
this Subcommittee in May 2000.
    Now tangible progress will be turned towards patients to produce 
treatment success. Translational research and clinical trials will find 
the drugs which will prevent, halt or significantly slow down disease 
progression.
    We anticipate supporting the development of lead compounds which 
can be ready for large scale, U.S. Food and Drug Administration 
approved clinical trials. This means the identification of small 
molecular entities that demonstrate bioactivity in animal models of 
ALS, and an acceptable safety profile, for evaluation in a Phase 1 
clinical trial. At the same time, ALSA seeks to engage in one or two 
small pilot trials each year, of known or existing compounds, such as 
FDA-approved drugs.
    TREAT ALS will align closely with existing trial centers as well as 
the National Institute of Neurological Disorders and Stroke clinical 
trials groups. NINDS and ALSA are already discussing the need for 
trials that span a larger geographic region and recognize the 
importance of involving many more clinical centers.
    To create a powerful clinical trials process, ALSA will partner 
with the NINDS and other disease organizations. TREAT ALS will fund an 
annual clinical investigator award, will seek collaboration by multiple 
centers, and will sponsor educational workshops on effective design of 
clinical trials. ALSA can play a leading role in recruiting expertise 
outside the ALS field to help improve clinical trials design and 
outcome measures.
    Concrete steps along the TREAT ALS path are already taken. ALSA has 
established a partnership with NINDS to identify and prioritize 
existing compounds with relevance to ALS and to move them forward 
expeditiously into clinical trials. ALSA is already funding several 
biotech companies with programs in ALS. Treat ALS enables further 
support of those efforts that are most likely to develop promising 
leads for ALS.
    Treat ALS will consolidate these efforts, launch from them a series 
of clinical trials, and will translate a decade of progress into real 
promise for ALS patients.
    We are extremely excited about TREAT ALS and we hope that both 
Congress and NINDS will work with us on the program so that together we 
can realize its full potential to benefit the ALS community in the 
United States and around the world.
    In addition to research funding, this Subcommittee will, or soon 
will, have before it several other opportunities to help us make 
progress in defeating ALS. For example, we currently are working with 
Senator Reid and other Members of Congress to authorize the 
establishment of a national ALS registry at the Centers for Disease 
Control and Prevention. As you may know, a single national patient 
registry does not exist in the United States today. However it is 
urgently needed for ALS research, disease management and the 
development of standards of care. In addition to collecting data on the 
number of people living with ALS and the rate at which ALS occurs in 
the United States, the registry would collect other data, including 
information on environmental factors that may be associated with the 
disease, the age, race and ethnicity of individuals with ALS, family 
history, and additional information that will promote a better 
understanding of the disease. The registry would identify, build upon, 
and coordinate with existing data, surveillance systems and registries, 
such as state-based ALS registries, the Department of Veterans Affairs 
ALS registry and the National Institute of Neurological Disorders and 
Stroke (NINDS) repository. We hope that when the opportunity arises, 
the Committee will support funding for this critical effort.
    While not under the jurisdiction of this Subcommittee, but under 
the jurisdiction of the full Committee, are other initiatives supported 
by ALSA that are focused on learning more about the apparent connection 
between ALS and military service. As you may know, several studies, 
including studies funded by the Department of Defense and the Veterans 
Administration, have found that veterans of the 1991 Gulf War are 
nearly twice as likely to die of ALS as veterans who did not serve in 
the Gulf War. Another study, published earlier this year by 
epidemiologists from the Harvard School of Public Health, found that 
men with a history of any military service, whether Vietnam, Korea, or 
World War II, are nearly 60 percent more likely to die of ALS than men 
in the general population.
    We believe additional funding is necessary to further explore the 
questions that arise as a result of these studies, including to 
definitively determine what is the risk of ALS for our military men and 
women, and what is causing these elevated risks. The Committee can help 
in this endeavor by supporting funding for the Peer Reviewed Medical 
Research Program (PRMRP) at the Department of Defense, and recommending 
that ALS continue to be a disease that is studied under the program, 
funding for which is provided in the Senate through the DOD 
Appropriations bill. You also can support other research and programs 
at the DOD and VA, including the VA ALS registry and other initiatives 
that will respond to the recommendations to Congress given by the 
Research Advisory Committee on Gulf War Veterans' Illnesses. Those 
recommendations called on Congress to set a national goal to develop 
treatments for the diseases affecting Gulf War veterans within five 
years.
    Finally, we are hopeful that funding stem cell research will allow 
us to explore the potential it holds for ALS. While it remains unclear 
whether this research will produce immediate treatments for ALS, we 
believe that any effort which may further our understanding of the 
disease must be explored. Moreover, as is the case with all research, 
advances in stem cell research may lead to unexpected developments that 
could help us bring new treatments from the lab to the patient earlier 
than if this research was discouraged.
    Clearly we have challenges before us if we are to continue the 
advances we have made in ALS research. There are many areas of research 
to explore and many opportunities for us as a country to make progress 
in improving our understanding of the disease, how it can be prevented, 
treated and cured. The work of The ALS Association and our new TREAT 
ALS program is one example of what can be done. The work conducted and 
supported by NIH and NINDS is another as are the efforts of the 
Department of Defense of the Department of Veterans Affairs. However, 
it is your work here, in this Subcommittee--in Congress--that can make 
possible our collective goal of relegating ALS to a disease of the 
past. And I urge you provide the support that is needed in this effort. 
Equally important, I urge you and the Congress to provide the 
leadership that is needed to encourage and facilitate collaboration 
among these varied interests who share the common goal of finding a 
treatment and cure for ALS. Together we can light the way in finding a 
treatment and cure for ALS.
    Thank you again for inviting me to appear before the Subcommittee. 
The ALS Association appreciates your previous support for our cause and 
we look forward to continuing to work with you as the nation commits 
itself to finding a treatment and cure for this horrific disease.
    I am happy to answer any questions you may have.

    Senator Shelby. Mr. Obermann, could you just tell the 
audience, if you could, how your son put this statement 
together. I think this would be interesting.
    Mr. Obermann. Certainly, Mr. Chairman. Under difficult 
circumstances. Certainly Eric has been asked in the past to 
write several articles, mostly in medical journals.
    He is a ventilator user. There is great interest in how 
people can try to proceed with as normal a daily life as 
possible doing things like traveling which just a few years was 
not possible. So he had written some articles previously for a 
journal there. He had actually done a brief article that the 
ALS Association had used as part of their fund raising.
    We pieced together parts of those previous stories that he 
had written along with some comments and responses that he gave 
in response to some questions that my wife, Marcia, and I asked 
Eric. Then I did most of the word processing, but all of the 
thoughts and the emotions that you are able to hear are from 
Eric.
    Senator Shelby. Thank you.

STATEMENT OF ERIC OBERMANN
    Mr. Obermann. Good morning. Can everyone hear me?
    My name is Eric Obermann and I live in Huntsville, Alabama. 
I am 23 years old and I have ALS.
    I would first like to thank you all for giving me the 
opportunity to talk with you about this disease. I think it is 
very important for all of you to hear and see firsthand how ALS 
affects both patients and their families. I am truly honored to 
be here.
    To begin my story, I am going to take you back to May 2000. 
I was a typical 18-year-old, excited to graduate from high 
school and nervous about going to college.
    I went to Grissom High, a fine public school named in honor 
of astronaut Gus Grissom. Grissom is known throughout the State 
for its outstanding bands and I was a first chair clarinetist 
and enjoyed playing with the marching band. I worked hard in 
high school, got good grades, and was accepted into Georgia 
Tech.
    There have been computers in our home since I was 7 or 8 
years old, so I became an avid computer user from early on. 
Computer science seemed to be a very promising field, so that 
fall I enrolled at Tech as a CS major. I expected to graduate 
in about 4 years with a Bachelor's Degree in computer science 
and management.
    I also enjoyed a lot of outdoor activities, such as helping 
my parents with landscaping projects, riding my bike, and 
taking backpacking trips with my dad. Life was good and my 
future looked great.
    The first signs of trouble came in the fall of 2000 just as 
I was entering my freshman year at Georgia Tech. I had noticed 
that my ability to play clarinet was beginning to slip as I was 
developing weakness in my mouth and tongue.
    Soon I also started to have a speech impedient, very subtle 
at first, but it quickly became quite noticeable. My words were 
slurred and I could not enunciate. It was very frustrating as 
people started to not understand what I was saying.
    We decided to seek medical advice, starting with an ear, 
nose and throat doctor. He noticed my tongue was atrophied or 
shrinking which explained my speech problem.
    Fearing a brain tumor or disease, he scheduled an MRI for 
that same day and referred us immediately to a neurologist. It 
was then that I realized that this may be a serious issue. But 
my family remained optimistic that it was something the doctors 
could fix and my life could go on as normal.
    The first neurologist ran a few tests and referred us to a 
neuromuscular specialist who understands what he called these 
unusual cases.
    Since I was in Atlanta going to school, we made an 
appointment at Emory University. The specialist completed a 
thorough examination, reviewed the notes from the other 
neurologist, and left the room.
    When he came back, he delivered the bad news. Eric, I think 
you have ALS. Do you know what that is? I had no idea and 
looked at my mom, a nurse, who was with me. She looked stunned, 
blank.
    The doctor continued, it is a progressive neuromuscular 
disease. It is fatal, but you probably have 3 to 5 years to 
live. Keep doing what you are doing. Get exercise and build up 
your body and muscles. That may allow you to last a little 
longer.
    My mom and I left in total silence. I felt as though I had 
just received a death sentence. Mom took me out to eat at one 
of our favorite places. But feeling sick to our stomachs, 
neither of us could eat.
    We just looked at each other wondering what had just 
happened to us. We were in shock. An hour later, mom took me 
back to the dorm to drop me off. I had to go back to class that 
afternoon.
    Over the next few weeks, we thought about what the doctor 
at Emory had said. It did not make sense. We were sure the 
doctors were all wrong. So we decided to seek a second opinion 
and then a third and a fourth.
    We went to four neurology specialists across the country. 
None of them said the same thing other than I was a very 
unusual case. They all essentially told me, I am sorry. There 
is nothing we can do for you. Check back in 6 months and we 
will see where you are then.
    I was in denial, unwilling to accept the fact that I had 
this beast of a disease they call ALS.
    Over time, we concluded that even if what I had was not 
typical ALS, it was progressing as they had predicted. My 
speech became unintelligible. I had shortness of breath. I had 
a hard time chewing and swallowing my food. Eating a normal 
meal became a huge effort, taking almost an hour.
    I often would have a choking fit when I ate right there in 
front of my new college friends. I started losing weight 
rapidly.
    Through it all, I kept going to class full time, hanging 
out with my friends at school and was starting to really enjoy 
my new life as a college student. But there was no ignoring the 
obvious signs of deterioration that my body was undergoing. My 
friends noticed it too, although none of them knew exactly what 
was wrong. I could not bring myself to tell them.
    When I came home for Christmas break during my sophomore 
year, we decided to have a feeding tube inserted so I could 
take my food without having to chew or swallow. This worked 
fine for a while, but soon I began choking on my saliva. 
Sometimes I would cough and gag for several minutes before I 
could breathe again.
    In the spring of 2002, I came down with severe pneumonia 
caused by aspiration of fluids from my mouth. I spent my spring 
break in the ICU.
    After a few weeks, I recovered and we drove over to Atlanta 
to move me out of my dorm room. I realized at that time that I 
would never be going back to school at Georgia Tech. This was a 
very painful experience for me.
    Living back at home again, I had repeated bouts of 
pneumonia that spring and was hospitalized three times, 
spending a total of 45 days in the ICU. The cause each time was 
aspiration of fluids from my mouth.
    The only solution was to do a laryngectomy, a radical 
procedure involving removal of my voice box and routing my 
trachea out through a hole in my neck. This was the only way to 
prevent the pneumonia, my doctors told me.
    On June 1, 2002, I had the surgery. I knew I would never 
speak, drink, eat, or smell again. I then started using a 
respirator, also called a ventilator, at night as I could no 
longer breathe on my own when I was lying down.
    My disease progressed rapidly in distinct downward steps. 
After each step, there would be a temporary halt to the 
progression and my condition would stabilize. I remember that 
with each of these phases, my family and I would think, okay, 
we can deal with this. If it only would stop here, we will be 
all right. But it never stopped. There was always another slide 
coming. We just did not know exactly what it would be or when.
    Concealing my laryngectomy stoma with a foam filter, I went 
back to school that fall at the University of Alabama at 
Huntsville to continue my degree. I soon had to begin wearing a 
neck brace as my neck could no longer support my head.
    My face became paralyzed so I could not laugh, smile, or 
grimace from pain. Without the ability to speak, I got a speech 
synthesis computer similar to the one I am using today, but 
with which I could talk by typing in words on a keyboard.
    My family and I also took American Sign Language courses 
which allowed us to communicate without a machine. 
Unfortunately, the next symptom was that my hands and arms 
began to lose strength and dexterity.
    Soon I was unable to type and my sign language became as 
garbled as my speech had been.
    My new computer which I operate using a switch under my toe 
was given to me by Jimmy McDonald, one of my friends from our 
ALS support group who passed away 1 year ago. Jimmy's wife, 
Bonnie, who along with Jimmy started our support group in 
Huntsville 3 years ago is here with me today.
    In 2003, I had to begin using the ventilator 24 hours a day 
as I could no longer breathe at all without assistance. 
Fortunately we found a portable ventilator which can be 
strapped to the back of my wheelchair. Very soon my legs also 
gave out. No longer able to drive my car, I had to withdraw 
from school again.
    That was it. I could no longer walk, talk, eat, or even 
breathe without assistance. Trying to be optimistic, I 
concluded that having lost so much there is not really much 
else that can happen.
    That brings us up to the present, May 2005. I am a 
ventilator-dependent quadriplegic who requires 24 hour skilled 
nursing care and I am fully dependent on my parents. This is 
certainly not what I had in mind as I first headed off to 
school only 5 years ago.
    I was just starting a new life as a young adult. By now I 
thought I would have my degree, a new profession in perhaps a 
new city, maybe a girlfriend, and the financial independence 
that every young person dreams of. Obviously ALS changed all 
those plans dramatically.
    But two things ALS cannot take from me are my mind and my 
spirit. Despite the radical changes my body has undergone, I am 
still the same guy inside. I am actually much tougher mentally 
and have learned to appreciate many things I used to take for 
granted.
    My faith has grown stronger as I have a lot of time to 
listen to what God has to say to me. I now have many wonderful 
friends in the ALS community that I might not have met. These 
are a few of the many blessings that I have received during my 
illness.
    Despite my severe disability and thanks to the 
determination and commitment of my loving parents and the 
support of my sister, Lauren, I am able to do things that few 
people in my condition ever dreamed of.
    I can travel and go on trips with my family, go to movies 
and concerts, attend swim therapy, and take walks in the woods. 
I am very blessed that there is technology to enable me to do 
these activities. My family has dedicated themselves to helping 
me live with ALS rather than simply waiting to die from it.
    Which brings me to the reason I am here today. I have never 
been a very outgoing person or one who seeks the limelight. 
Rather, I have always been quiet, analytical, and somewhat 
introspective.
    But my situation today is such that having an opportunity 
to speak out on behalf of all ALS patients is very important to 
me. This is something I can do and something I want to do 
because it is so important to so many people who are suffering.
    There are hundreds of advocates from the ALS Association 
who traveled here this week to emphasize to you what a 
devastating disease ALS is. Hopefully, after hearing my brief 
story, you will have a better understanding of how the patient 
as well as the family is impacted in such a devastating way.
    I come here with hopes that we can make a difference in how 
Congress will provide for ALS patients and their families. We 
are all very fortunate to live in a country that has risen to 
great challenges time after time and conquered them.
    In 1960, our country embarked on a program to put a man on 
the moon and return him safely and did this in less than a 
decade. ALS was first identified in 1869, 136 years ago. Yet, 
the prognosis today is exactly the same as it was then.
    How can this be? The answer is simple. We need to dedicate 
more funding to researching treatments and a cure. We have 
brilliant researchers working on this disease, but they simply 
need more resources to accelerate the progress they are making.

                           PREPARED STATEMENT

    It may be too late for me and for many of my friends here, 
but hopefully any positive results from our visit this week 
will help hundreds of thousands of Americans with ALS in the 
future.
    Thank you again for your time. I am very grateful that I 
have had this opportunity to speak to you.
    [The statement follows:]

                  Prepared Statement of Eric Obermann

    Good morning. Can everyone hear me?
    My name is Eric Obermann and I live in Huntsville, Alabama. I am 23 
years old, and I have ALS. I would first like to thank you all for 
giving me the opportunity to talk with you about this disease. I think 
it is very important for all of you to hear and see first hand how ALS 
affects both patients and their families. I am truly honored to be 
here.
    To begin my story, I am going to take you back to May of 2000. I 
was a typical 18 year old, excited to graduate from High School, and a 
bit nervous about going to college. I went to Grissom High, a fine 
public school named in honor of astronaut Gus Grissom. Grissom is known 
throughout the state for its outstanding bands, and I was a first chair 
clarinetist and enjoyed playing with the marching band. I worked hard 
in High School, got good grades, and was accepted into Georgia Tech. 
There have been computers in our home since I was 7 or 8 years old, so 
I became an avid computer user from early on. Computer Science seemed 
to be a very promising field, so that fall I enrolled at Tech as a CS 
major. I expected to graduate in about 4 years with a bachelors degree 
in computer science and management. I also enjoyed a lot of outdoor 
activities, such as helping my parents with landscaping projects, 
riding my bike, and taking backpacking trips with my Dad. Life was 
good, and my future looked great.
    The first signs of trouble came in the fall of 2000, just as I was 
entering my freshman year at Georgia Tech. I had noticed that my 
ability to play clarinet was beginning to slip, as I was developing 
weakness in my mouth and tongue. Soon, I also started to have a speech 
impediment; very subtle at first, but it quickly became quite 
noticeable. My words were slurred and I could not enunciate, it was 
very frustrating as people started to not understand what I was saying. 
We decided to seek medical advice, starting with an ear nose and throat 
doctor. He noticed my tongue was atrophied, or shrinking, which 
explained my speech problem. Fearing a brain tumor or disease, he 
scheduled an MRI for that same day, and referred us immediately to a 
neurologist. It was then that I realized that this may be a serious 
issue. But my family remained optimistic that it was something the 
doctors could fix, and my life could go on as normal.
    The first neurologist ran a few tests, and referred us to a neuro-
muscular specialist, who understands what he called ``these unusual 
cases''. Since I was in Atlanta going to school, we made an appointment 
at Emory University. The specialist completed a thorough examination, 
reviewed the notes from the other neurologist, and left the room. When 
he came back, he delivered the bad news. ``Eric, I think you have ALS. 
Do you know what that is?'' I had no idea, and looked at my Mom, a 
nurse, who was with me. She looked stunned, blank. The doctor 
continued, ``It is a progressive neuro-muscular disease. It is fatal. 
But you probably have 3 to 5 years to live. Keep doing what you're 
doing, get exercise and build up your body and muscles. That may allow 
you to last a little longer.''
    My Mom and I left in total silence. I felt as though I had just 
received a death sentence. Mom took me out to eat at one of our 
favorite places, but feeling sick to our stomachs, neither of us could 
eat. We just looked at each other, wondering what had just happened to 
us. We were in shock. An hour later, Mom took me back to the dorm to 
drop me off. I had to go back to class that afternoon.
    Over the next few weeks, we thought about what the doctor at Emory 
had said. It didn't make sense, we were sure the doctors were all 
wrong. So we decided to seek a 2nd opinion, and then a third, and a 
fourth. We went to 4 neurology specialists across the country. None of 
them said the same thing, other than I was a very unusual case. They 
all essentially told me,'' I'm sorry, there is nothing we can do for 
you. Check back in 6 months and we'll see where you are then.'' I was 
in denial, unwilling to accept the fact that I had this beast of a 
disease they call ALS.
    Over time, we concluded that even if what I had was not typical 
ALS, it was progressing as they had predicted. My speech became 
unintelligible. I had shortness of breath. I had a hard time chewing 
and swallowing my food. Eating a normal meal became a huge effort, 
taking almost an hour. I often would have a choking fit when I ate, 
right there in front of my new college friends. I started losing weight 
rapidly. Through it all, I kept going to class full-time, hanging out 
with my friends at school, and was starting to really enjoy my new life 
as a college student. But there was no ignoring the obvious signs of 
deterioration that my body was undergoing. My friends noticed it too, 
although none of them knew exactly what was wrong. I couldn't bring 
myself to tell them.
    When I came home for Christmas break during my sophomore year, we 
decided to have a feeding tube inserted so I could take my food without 
having to chew or swallow. This worked fine, for a while, but soon I 
began choking on my saliva. Sometimes I would cough and gag for several 
minutes before I could breathe again. In the spring of 2002, I came 
down with severe pneumonia, caused by aspiration of fluids from my 
mouth. I spent my spring break in the ICU. After a few weeks I 
recovered, and we drove over to Atlanta, to move me out of my dorm 
room. I realized at that time that I would never be going back to 
school at Georgia Tech. This was a very painful experience for me.
    Living back at home again, I had repeated bouts of pneumonia that 
spring, and was hospitalized 3 times spending a total of 45 days in the 
ICU. The cause each time was aspiration of fluids from my mouth. The 
only solution was to do a laryngectomy, a radical procedure involving 
removal of my voice box and routing my trachea out through a hole in my 
neck. This was the only way to prevent the pneumonia, my doctors told 
me. On June 1, 2002, I had the surgery. I knew I would never speak, 
drink, eat or smell again. I then started using a respirator, also 
called a ventilator, at night, as I could no longer breathe on my own 
when I was lying down.
    My disease progressed rapidly, in distinct downward steps. After 
each step, there would be a temporary halt to the progression, and my 
condition would stabilize. I remember that with each of these phases, 
my family and I would think, ``Okay, we can deal with this. If it only 
would stop here, we'll be all right.'' But it never stopped. There was 
always another slide coming, we just didn't know exactly what it would 
be, or when.
    Concealing my laryngectomy stoma with a foam filter, I went back to 
school that fall at the University of Alabama at Huntsville to continue 
my degree. I soon had to begin wearing a neck brace as my neck could no 
longer support my head. My face became paralyzed so I could not laugh, 
smile, or grimace from pain. Without the ability to speak, I got a 
speech synthesis computer, similar to the one I'm using today, but with 
which I could talk by typing in words on a key board. My family and I 
also took American Sign Language courses which allowed us to 
communicate without a machine. Unfortunately, the next symptom was that 
my hands and arms began to lose strength and dexterity. Soon, I was 
unable to type, and my sign language became as garbled as my speech had 
been. My new computer, which I operate using a switch under my toe, was 
given to me by Jimmy McDonald, one of my good friends from our ALS 
support group who passed away one year ago. Jimmy's wife Bonnie, who 
along with Jimmy started our support group in Huntsville 3 years ago, 
is here with me today.
    In 2003, I had to begin using the ventilator 24 hours a day, as I 
could no longer breathe at all without assistance. Fortunately we found 
a portable ventilator, which can be strapped to the back of my 
wheelchair. Very soon my legs also gave out. No longer able to drive my 
car, I had to withdraw from school, again. That was it. I could no 
longer walk, talk, eat, or even breathe without assistance. Trying to 
be optimistic, I concluded that, having lost so much, there is not 
really much else that can happen.
    That brings us up to the present, May 2005. I am a ventilator 
dependent quadriplegic who requires 24 hour skilled nursing care, and I 
am fully dependent on my parents. This is certainly NOT what I had in 
mind as I first headed off to school only 5 years ago. I was just 
starting my new life as a young adult. By now, I thought, I'd have my 
degree, a new profession in perhaps a new city, maybe a girlfriend, and 
the financial independence that every young person dreams of. 
Obviously, ALS changed all those plans. Dramatically.
    But two things ALS cannot take from me are my mind, and my spirit. 
Despite the radical changes my body has undergone, I am still the same 
guy inside. I am actually much tougher mentally, and have learned to 
appreciate many things I used to take for granted. My faith has grown 
stronger, as I have a lot of time to listen to what God has to say to 
me. I now have many wonderful friends in the ALS community that I might 
not have met. These are a few of the many blessings that I have 
received during my illness.
    Despite my severe disabilities, and thanks to the determination and 
commitment of my loving parents, and the support of my sister Lauren, I 
am able to do things that few people in my condition ever dreamed of. I 
can travel and go on trips with my family, go to movies and concerts, 
attend swim therapy, and take walks in the woods. I am very blessed 
that there is technology to enable me to do these activities, and my 
family has dedicated themselves to helping me LIVE with ALS, rather 
than simply waiting to die from it.
    Which brings me to the reason I am here today. I have never been a 
very outgoing person, or one who seeks the limelight. Rather, I have 
always been quiet, analytical, and somewhat introspective. But my 
situation today is such that having an opportunity to speak out on 
behalf of all ALS patients is very important to me. This is something I 
CAN do, and something I want to do, because it is so important to so 
many people who are suffering.
    There are hundreds of Advocates from the ALS Association who 
traveled here this week to emphasize to you what a devastating disease 
ALS is. Hopefully, after hearing my brief story, you will have a better 
understanding of how the patient, as well as the family, is impacted in 
such a devastating way. I come here with hopes that we can make a 
difference in how Congress will provide for ALS patients and their 
families. We are all very fortunate to live in a country that has risen 
to great challenges, time after time, and conquered them. In 1960, our 
country embarked on a program to put a man on the moon and return him 
safely, and did this in less than a decade. ALS was first identified in 
1869, 136 years ago. And yet the prognosis today is exactly the same as 
it was then. How can this be? The answer is simple, we need to dedicate 
more funding to researching treatments and a cure. We have brilliant 
researchers working on this disease, but they simply need more 
resources to accelerate the progress they are making.
    It may be too late for me, and for many of my friends here, but 
hopefully any positive results from our visit this week will help 
hundreds of thousands of Americans with ALS in the future.
    Thank you again for your time, I am very grateful that I have had 
this opportunity to speak to you.

    Senator Shelby. Thank you.
    Mr. Borsellino.

STATEMENT OF ROBERT BORSELLINO
    Mr. Borsellino. Well, Eric, need I say, was about as 
articulate as one can be about this illness.
    My name is Rob Borsellino. I do live in Iowa, but I am from 
the Bronx, so I just want to point out when a U.S. Senator 
calls and asks you to testify, if you are an Italian from the 
Bronx, there's that moment of hesitation, uncomfortableness.
    But Senator Harkin's staff assured me that they basically 
wanted to, you know, put a face on this. I would not have to 
name names.
    I am 55. I have a wife, two teenage sons, and I have been 
in pretty good health up until now. I do not drink. I do not 
smoke. I cannot remember the last time I had a joint.
    Anyway, back in November, I started slurring my words. I 
was tired all the time. I felt weak. Friends kept asking me if 
I was drinking again. Eventually I went to see a doctor. He ran 
some tests and he asked me to go see another doctor, a nerve 
specialist.
    That guy ran more tests. At the end of the session, he is 
telling me I have this fatal, incurable, exotic-sounding 
disease, ALS, also called Lou Gehrig's disease. Then he is 
telling me most people live 2 to 5 years with this.
    It has been 6 months. I have been to see three other nerve 
specialists in various parts of the country. I am hearing the 
same thing and every time I hear it, I am in denial. I find 
myself sitting there avoiding the important stuff.
    I will not be there for my kids' weddings. I will not know 
my grand kids. I have got to do a will. You have got to deal 
with life insurance, all that stuff.
    What is most shocking to me is that conventional medicine 
has nothing to offer people in my situation except this one FDA 
approved medicine that will keep you alive another 2 or 3 
months.
    So, you know, I am doing--and I would not like this to be 
public--I am doing Yoga and acupuncture and massage therapy. I 
am taking Chinese herbal medicines, anti something or others. 
It is the only way I feel I can engage in this stuff rather 
than just sit and wait for things to get worse.
    Something else that is making me nuts. I am hearing about 
and from Americans with these new degenerative diseases, they 
have to go to Portugal. They got to go to India for surgery. 
ALS patients in Iowa are going to China for stem cell surgery 
because they cannot get it here in the United States.
    Anyway, and when you talk to people about that, most people 
do not know what this is, so they are stunned. They are 
appalled that this is going on and it is a complete mystery to 
them and to me why this illness remains a death sentence which 
is pretty much what it is.
    One theory is that it is not that so few people have it. It 
is that people live such a short time that there has not been a 
movement in effect to deal with it. That is something we got to 
look at.
    One other quick thing. You go through life. You talk to 
your kids. You say, you know, be careful, use sun screen, 
mosquito repellant, do not drink and drive, practice safe sex. 
With ALS, that stuff is worthless. It is always a death 
sentence.
    We need more information about what factors are common 
among ALS patients. I am told--and, again, I am not an 
authority on this sort of thing, but we need to have a database 
to keep track of those things that are affecting people. We 
need hopeful, meaningful treatment. We need a cure.
    One last thing I did want to say real quick. When Senator 
Harkin was saying he wanted me to put a face on this, I have 
been looking for some sort of analogy and the best thing I 
could come up with from me personally is the fact that it is 
little league season right now and I cannot play catch with my 
15-year-old.
    Thank you.
    Senator Shelby. Dr. Bruijn, in your opinion, what is the 
most promising research currently being conducted with respect 
to ALS in your judgment?
    Dr. Bruijn. I think there are a few if I may mention. I 
think one exciting thing is the potential of gene therapy as 
Dr. Landis mentioned. We know that the motor neurons need to be 
nurtured by the right factors. We are working hard on the pre-
clinical work to try and get the first gene therapy trial for 
ALS. It is difficult and challenging.
    I think the other area is the potential of finding new 
genes. So I do think that the most promising would be to 
capture all the samples from the population of ALS through a 
registry and through the repository that we are working so hard 
on because I do believe that we are close, but there are a lot 
of tools that we still need to make that difference.
    Senator Shelby. What do you need to do to put that database 
together? I know you need money.
    Dr. Bruijn. One is money and a commitment from the 
community to participate. I think that the patient community--
--
    Senator Shelby. How much money roughly?
    Dr. Bruijn. I know that the Veteran Affairs Registry is 
something in the order of $5 million, at least now, for 5 
years. So we recognize that that is only the beginning and the 
Veteran Registry is only a part of our ALS population.
    So I think it is going to be several million. But to even 
get it started, a million or so. The exact figures are 
difficult, but to get it started would be a huge commitment.
    Senator Shelby. The ALS Association has had a lot of 
experience working cooperatively with the NIH. From that 
experience, could you tell us what forms of specific 
cooperation work well and where, if anywhere, are the 
impediments? Where are the problems?
    Dr. Bruijn. I think what works well is that we are able to 
through our programs and our granting get very exciting new and 
sometimes risky projects on the table. Certainly those small 
amounts of funding seed the ideas that then enable larger 
funding from the NIH.
    Our partnerships as well in asking for proposals has been 
great. The biggest impediment is going to be the lack of 
increased funding because I think that we have now an 
investigator pool both young and more established that are 
outstanding. The thought that those could be restricted would 
be a real shame.
    Senator Shelby. What conditions have come together to make 
it possible? You are talking about your research and the 
doubling of the NIH budget. Has that enabled you to get to this 
point?
    Dr. Bruijn. I think it has made a huge impact. I think that 
what has also helped is the awareness through the patients and 
through the community, the recognition that the veterans are 
affected, and the excitement that we have the first tantalizing 
models to be able to do the research.
    So I am proud to say that we have investigators worldwide, 
people that would maybe not have thought about researching ALS 
that are now working on the disease.
    Senator Shelby. Is there cooperative research now between 
the VA medical researchers, DOD researchers, NIH people?
    Dr. Bruijn. I hope that there could be an increase. An 
example is that the VA Registry is a very exciting resource and 
it is not entirely clear how we can capitalize on that and 
combine that repository that is being done there with the NINDS 
repository.
    I think with leadership such as Dr. Landis, we can make it 
happen. But I do think that we have to all be talking very 
seriously.
    Senator Shelby. Senator Harkin has spoken to me about this 
and he is in a position and hope Senator Specter will be to 
lead that effort.
    Dr. Bruijn. Excellent. Thank you.
    Senator Shelby. Doctor, would you give us a couple of 
examples of the type of research that would be possible maybe 
if an ALS registry were created? In other words, what do you 
get from that?
    Dr. Bruijn. Well, one thing that is so striking about the 
disease--and I wanted to touch on the question you have, why do 
we not have therapies--it is a very challenging one because as 
you see here in the room today, we have those that have 
impaired speech, those that have less mobility, and those that 
live a long time and a short time.
    It is a real mystery to us that this is all really quite a 
variety under one umbrella. So if we had a registry, we could 
start to understand how we could perhaps subdivide the groups 
and maybe the failure in getting good therapies is that we are 
not targeting the right population with the right therapy.
    So I think that this is going to be a critical and 
important need that will take time because it will take several 
years and commitments over several years to get all that data 
together.
    Additionally, if we can get the genetics, we would confine 
new targets. So the mystery of the disease and the tardiness is 
because of its challenge.
    Senator Shelby. Thank you.
    Mr. Obermann, when you first learned of your son's 
diagnosis, did you have the information you needed to make 
choices regarding his care? In other words, what type of 
information was available to you and was it adequate or was it 
trial and error?
    Mr. Obermann. It was probably really the latter. We turned 
initially to the Internet and we found tons of information 
about the disease and probably too much information. It became 
very difficult to sort out what might be useful or reasonable 
information from--there is a lot of stuff out in the Internet 
that truly is both inaccurate and not useful at all for patient 
care. We spent time going to these clinics and we got good 
information there as well.
    But I would say that even though we are a small 
organization, our ALSA chapter is relatively young, serving 
about 10 families right now, that the exchange of information 
we do in our monthly support group probably was the best source 
of real practical information about where to get DME, you know, 
which physicians are doing which prescriptions and things of 
that sort.
    So I think turning to the other patients is where the real 
bulk of valuable information was available to us.
    Senator Shelby. What has been the impact--it has had to 
have had an impact--of your son's diagnosis and care on your 
family?
    Mr. Obermann. I guess to try to sum it up in a word, it is 
profound. As Eric said, he said he requires 24-hour skilled 
nursing care. I do not think it would shock any of you to know 
that neither insurance nor Medicare will provide that for a 
family. So my family like most of the families that you see in 
this room here, the primary caregiver, the bulk of that burden 
falls to the spouse, the parent, or the children.
    I think we are very fortunate in our family that Eric has 
both parents available to care for him and actually the 
intensity of his care as his disease has progressed has 
required both my wife and I to leave our full-time professions 
and spend more time at home working with him.
    Senator Shelby. Thank you.
    Mr. Borsellino, how do we encourage ALS patients to 
participate in clinical trials if only half of them will 
receive the medication? You know, they have got to have a 
database. They have got to have some way to manage.
    Mr. Borsellino. Well, I am the wrong one to ask because up 
to now I have avoided clinical trials for that very reason.
    The weird thing about this illness is that there is so 
little in terms of hope, in terms of positive, good news. To 
the extent that we can come up with some good news--and I 
realize if you do not do the clinical trials, you are not going 
to have the results. I understand that.
    But as I say, I am the wrong one to ask that particular 
question. I am not going to try to finesse it.
    Senator Shelby. Dr. Bruijn, you want to comment on that?
    Dr. Bruijn. I would like to make a comment and I think 
really to honor as well a good colleague and friend, Dr. Rick 
Ulney, who is a physician who was struck by ALS. He himself is 
in a controlled double-blinded study, recognizing the need of 
it.
    But I also do think that a solution to this might be better 
design of clinical trials and we are committed to working with 
investigators on that. There are cross-over trials in which 
there is a much shorter period of time that a patient needs to 
be on that placebo and then they can go onto the drug.
    I think that by creative design, we can meet both 
challenges, that we really find a drug that makes a difference 
and help the patients.
    Senator Shelby. Mr. Borsellino, you have tried alternative 
medicines in your struggle. Any of them seem to help and what 
has helped, if any?
    Mr. Borsellino. No. I mean, I have tried these herbal 
Chinese anti something or other and, you know, drinking 
vegetable juice and, you know, washing down the soy, whatever. 
I have tried whatever was put in front of me, whatever I heard 
was out there.
    Again, I do not know if it works. I do not know if I would 
be sitting here in a wheelchair unable to speak had I not done 
some of the stuff.
    Senator Shelby. Sure.
    Mr. Borsellino. So I will say that I have rapidly 
deteriorated in my voice in the past 6 months, weakness in my 
hands, a lot of the traditional problems with ALS. So, again, I 
do not know if it has helped.
    Senator Shelby. Okay. Thank you.
    Senator Harkin, thanks for your indulgence.
    Senator Harkin. No. Thank you, Mr. Chairman. Those are all 
good questions and many of those I wanted to ask myself.
    But following up, Dr. Bruijn, just on that last question 
and what Rob was saying, does your association have any even 
anecdotal types of information from other countries, China, 
other countries, where obviously they have people that have ALS 
too that they may have treated with other kinds of 
interventions? Do you have any information on that at all?
    Dr. Bruijn. Sir, we have an international research program. 
In fact, many of our investigators are funded worldwide. We do 
keep a close eye on what is going on.
    I think it is important to note that although intriguing 
and, of course, I understand in a desperate situation without 
therapies, we would try and go anywhere, I am concerned about 
the kinds of therapies going on abroad which are not done in 
any kind of trial and are not providing any kind of data and at 
huge cost, $20,000 plus to a patient.
    So I think that our challenge here is not that there are 
better therapies out there that we cannot get to, but to be 
able to learn from it. I think that it is not appropriate to 
suddenly be doing these kinds of things in patients here. But 
we do need to engage in the laboratory's research, especially 
for stem cells in an aggressive way.
    So I think if there was something really promising, we will 
go out there and find it. I think that these kinds of studies 
are difficult without any rigorous follow-up.
    Senator Harkin. I am glad you mentioned stem cells. As you 
heard from Dr. Landis earlier, after we doubled--and, again, as 
often as I can, I pay tribute to Senator Specter for helping 
lead that charge here and the two Senators you see here, 
Senator Murray and Senator Shelby, both very supportive of 
doubling the funding for NIH. Got the job done. We were at this 
plateau where, as you heard Dr. Landis say, about one out of 
four peer reviewed research were funded and now it is down to 
16 percent and falling.
    So, again, whatever you can do to help us get the funding 
back up for NIH would be very helpful. We did not double the 
funding to then fall of a cliff. We doubled the funding to keep 
going.
    Basically the budget that was sent to us is the lowest 
increase, I think, in NIH in, well, a long time. I do not know 
how many years it is, but we need to do better than that.
    Second, on stem cells, you mentioned that--you heard Dr. 
Landis' response to me--we have these clinical trials and 
everything going on. But, again, it just seems to me a lot of 
these drugs that we are investigating are interventions that 
will keep you alive a couple more months or 5 more months or 
maybe relieve some of the symptomatic symptoms of ALS.
    In order to get at the root cause, many scientists believe 
that stem cells really hold the answer and that this could be, 
as my staff person said, the low hanging fruit for stem cell 
research. Even Dr. Wilmot who cloned dolly, has said that of 
all of the candidates for stem cell intervention, ALS could be 
the primary one.
    So, again, if you would just address that just for a couple 
minutes about the need to more aggressively have stem cell 
research, I would appreciate that.
    Dr. Bruijn. I absolutely agree that it has huge promise. I 
think that we must not lose the fact that we have many other 
avenues that we need to be focused on.
    I think that the challenge of stem cells as a therapy, we 
need to diagnose the disease earlier because as we heard, at 
the point that 50 percent our motor neurons are lost, it is a 
hard task then to replace all those neurons with stem cells.
    Having said that, I absolutely endorse that stem cells are 
a vital opportunity not to replace the neurons themselves. I 
think that is a bigger challenge. If we remember that we have 
got to connect our spinal cord to our muscle in meter and 
length. We have done it in dishes not on patients.
    But the stem cells are an absolutely vital tool as a pump 
and a resource like gene therapy to put the right things around 
those motor neurons. So there is a huge promise and the biggest 
problem is the lines are available because we cannot even use 
them as a pump to facilitate the right factors with the 
contamination of the mouse feeders.
    So what I find so frustrating as a scientist and with my 
colleagues is that we have to have separate buildings to be 
able to do studies that are either government funded or 
privately funded. I think that there has to be an openness to 
move exciting avenues with appropriate ethical guidelines 
forward. And so the stem cell lines are a real limitation at 
this point.
    Senator Harkin. Thank you, Dr. Bruijn.
    Again, I just want to thank Eric for your testimony, for 
taking the effort I know that it took to be here today and to 
your family.
    Let me just express my high esteem for you, Mr. Obermann, 
and your wife and your family for what you have done to support 
your son. It takes its toll and I do not know you personally. I 
think is probably the first time we have ever met. But, again, 
just my highest esteem for what you are doing. You provide a 
great example of what a family ought to be about. Appreciate it 
very much.
    Mr. Obermann. Thank you.
    Senator Harkin. Again, Eric, you mentioned about we did 
this moon shot. We did the moon thing in the sixties. Well, we 
need that kind of moon shot again in medical research. We could 
if we set the goals and we funded it.
    See, biomedical research is basically like opening doors. 
If you've got 10 doors to open, you do not know what is behind 
them.
    Now, if you are going to open five doors and leave five 
unopened, then what are your odds? I mean, maybe the answer 
lies behind one of the doors you have not opened. If you could 
open 8 out of 10, your odds go up that you are going to find 
what you are looking for.
    So when we say we are down to 16 percent, we are opening 1 
out of 7 doors. You see what the odds are. So that is why we 
have got to get the research. You have to open more of those 
doors. A lot of them will come to dead ends. But the more we 
open, the more our chances are that we are going to find 
something.
    So I hope that we can sort of use what Eric said about a 
moon shot and look at it in that way, that within 10 years, we 
could open more of these doors and try to get at some of the 
answers.
    To my friend, Rob Borsellino, thank you.
    I just have to admit in front of everyone here that when I 
found out about Rob--I had had dinner with he and his wife last 
summer and no indication about anything. When I had read in his 
column that he had ALS, I could not even call him up.
    Here is a guy that has been poking fun at me a long time, 
see. Usually when I call him, I try to get back at him. As you 
can tell, he is a man of great humor. But how do you respond to 
something like this?
    So, again, a lot of us feel we know Rob better than we know 
others because we read him every day and we read his columns 
and his columns are always very funny and sometimes at our 
expense. So you really know someone like this. You feel that it 
is a part of your family that something has happened to them.
    So I do not know if I have any questions, Rob, other than 
just, you know, as all of you can tell, here is someone still 
who can express humor. To me, I just think that is a great 
attribute.
    Mr. Borsellino. I was not trying to be funny.
    Senator Harkin. No. I know that.
    You just are naturally. But you have great courage and I 
hope you keep writing about it.
    Mr. Borsellino. Thank you, Senator. I appreciate it.
    Senator Harkin. Keep writing about it. Keep telling people 
what is happening out there.
    Mr. Borsellino. Is it appropriate to put in a pitch with 
the book I have coming out----
    Senator Harkin. Absolutely.
    Mr. Borsellino [continuing]. Next month, at a time like 
this?
    Senator Harkin. You have a book coming out?
    Mr. Borsellino. Yeah.
    Senator Harkin. See, that is how little I know.
    Senator Shelby. Talk about it. When is it coming?
    Mr. Borsellino. It should be available May 25. On the Des 
Moines Register, web site, you can find what you need. I cannot 
believe I am doing this.
    Senator Harkin. Rob, thank you very much for being here. 
Thank you.
    Senator Shelby. Thank you.
    Senator Murray.
    Senator Murray. Well, thank you, Mr. Chairman. Thank you to 
all of our witnesses today for your courage and coming and 
sharing with us what we need to know to help make life better 
for those that are here, but really for those that are coming 
after as we know the history of this disease. But your being 
here does make a difference.
    I know we have a vote coming up in a minute and we also 
have another panel that we want to hear from today. So I will 
just be really brief. I just want to focus on one area and 
that's respite care.
    My own father had multiple sclerosis and I know well that 
when a patient gets a diagnosis of a disease like that, it is 
the entire family that gets the diagnosis. I know what a 
struggle it was caring for my dad throughout the rest of his 
life and for my own mother and for all of us. We were seven 
young kids when he was diagnosed.
    I often think that the family gets left behind, forgotten, 
but mostly because families, I know how hard it was for us to 
say we need help because you want the person who has been 
diagnosed to know you are there every single minute and you do 
not want to look like you need help.
    But I do think respite care is especially important. I 
think those who are suffering from ALS want to know that their 
families are well cared for as well. I think for families, it 
is extremely important so you can be there 100 percent when you 
have to.
    Mr. Obermann, you are here with your son and I know you 
just said that you and your wife are both taking leaves from 
your jobs to care for him.
    Can you talk for a minute just about what we can do as 
Members of Congress? We have a bill called ``Respite Care for 
Life,'' things like that that will help families be able to be 
the best they can for the person they are caring for.
    Mr. Obermann. I think that bill is probably the brightest 
thing on the visible horizon for families like ours and like 
yours earlier. You really cannot overstate how challenging it 
is on a family.
    You are dealing with this devastating disease of a loved 
one. It has great emotional toll obviously, also great 
financial toll. They estimate that I think in the later stages 
of the disease, it costs roughly $200,000 per year to maintain 
a person.
    Eric is certainly at that stage being ventilator dependent. 
We just cannot up and leave him with anybody. People are 
intimidated by a ventilator. Even skilled nurses and even 
physicians just have not had that type of experience. We 
obviously have.
    I am not a trained medical professional, but my wife 
fortunately is a registered nurse and so that is another 
blessing that our family has had because she can train even an 
engineer to take care of my son.
    But it is very difficult to, even if you can pay for 
respite care, it is very difficult to find qualified 
individuals to do that.
    My sister flies down from Massachusetts once or twice a 
year to give us a break. She does not have to do that because--
she does that because she loves Eric and realizes how important 
it is that Marcia and I have a few days away now and then.
    I can tell you, and my wife would say the same thing, that 
when we come from a long weekend or, you know, 3 or 4 days just 
out of town doing nothing, our ability to take care of Eric 
is--it increases 10-fold.
    Sometimes you do not realize how raw and fatigued and just 
emotionally drained you are until you go away for a few days 
and come back. Suddenly you have got tolerance and patience 
again. Eric understands that too.
    In fact, I remember over a year ago, he got down on his 
computer, you know, dad, are you and mom getting enough time 
away. This is coming from a kid that requires our help every 
day. We were touched by that and we tried to understand that as 
much as we want to be with him every day, we do need to get 
away now and then in order to be good caregivers.
    Senator Murray. Thank you very much for that.
    Mr. Chairman, thank you.
    Senator Shelby. Thank you. We thank the panel very much for 
your appearance today and I think we are getting a lot out of 
what you had to say and your experiences. Thank you very much.
    Our third panel will consist of Mr. Tommy John, everybody 
knows him, former major league baseball player; Mr. David Cone, 
former major league baseball player; Kate Linder, actress.
    If you will proceed to the table, I am going to talk about 
all of you for just a minute.
    First, Mr. Tommy John is a former major league baseball 
player. During his 26-season career, he played for the New York 
Yankees and the Los Angeles Dodgers. In 1976, he earned the 
Come-back Player of the Year Award. His career continued with 
three trips to the World Series.
    Currently he is a minor league coach in the Yankee 
organization and an ALS advocate. Mr. John along with his wife 
and son are the 2005 recipients of the ALS Association's All 
Star Award.
    We appreciate, Mr. John, you being here today.
    Your written testimony, if any, will be made part of the 
record.
    Our second panelist is Mr. David Cone. Mr. Cone is also 
well-known in America, all over the world. He is a former major 
league baseball player who played for 16 seasons with the 
Kansas City Royals, the New York Mets, the Toronto Blue Jays, 
and the New York Yankees.
    Over that period, he earned five World Series champion 
rings. He is an honorary board member of the Greater New York 
Chapter of the ALS Association and was recognized as the 2004 
ALS Association All Star Award recipient.
    We appreciate you being here.
    We also have with us today Ms. Kate Linder. Ms. Linder is a 
star of the daytime television drama ``The Young and the 
Restless.'' Ms. Linder is helping to lead the fight against ALS 
through Kate's Club, a new public awareness campaign for the 
ALS Association. In December 2004, Kate's 49-year-old brother-
in-law was diagnosed with ALS.
    We appreciate all of your testimony here today. Our problem 
is going to be in a few minutes. We just got notice that we 
have a vote on the floor of the U.S. Senate.
    So I'm going to start with you, Mr. John. You sum up 
whatever you want to say as quickly as you can here today. 
Thank you.
STATEMENT OF TOMMY JOHN
    Mr. John. Thank you, Mr. Chairman.
    Members of the subcommittee, my name is Tommy John. I 
appreciate this opportunity to appear today alongside another 
great Yankee, David Cone, to talk about a disease I care about 
so deeply, a disease which was named after a former Yankee, Lou 
Gehrig.
    The reason I mention my affiliation with the Yankees is 
because of my career with the Yankees is the reason I am 
involved in this fight. It is because I was aware of Lou 
Gehrig's disease during my playing days, but I did not know 
what the disease was or what it would do to one of my 
teammates, Jim Catfish Hunter. That is how I got involved in 
this fight. It is a personal fight with me.
    Many of you remember Catfish as a great pitcher, Hall of 
Fame pitcher, who like David and one of our colleagues, Jim 
Bunning, threw a perfect game back in 1968. You may remember 
him as a larger-than-life figure with his North Carolina drawl 
and his big handlebar mustache that Charlie Finley gave him the 
nickname Catfish.
    Catfish was a great teammate and a great person. ALS took 
his life in 1999 at the age of 53. As a friend of Catfish's 
back in North Carolina, I saw firsthand what ALS can do to a 
body that was just a strapping young man, that would hunt every 
day, and would make him totally powerless. It is a horrific 
disease and we must find a cure for it.
    My entire family including my son, Taylor, and my wife, 
Sally, who are here with me today, we are committed to finding 
a cure for this disease.
    As you said, I am an honorary member of the New York 
Chapter and each year, I am proud to attend the annual New York 
Sports Banquet. We do a fund raiser in Raleigh, North Carolina, 
the Jim Catfish Hunter Chapter.
    We are also co-chairing a little league baseball tournament 
to benefit ALS, the first ever. That is out in Los Angeles July 
23 to July 30.
    However, what I want to do is I want to focus the remainder 
of my remarks today not on what I have done or what my family 
has done to raise the awareness of ALS. What I want to discuss 
is why military veterans, people who seem to be so strong like 
Catfish, are diagnosed with ALS and dying from the disease at a 
greater rate than other Americans.
    Although I am not a researcher, I am aware of at least 
three recent studies which have found that military veterans 
are at an increased risk of dying from ALS. Two of those 
studies focused on the 1991 Persian Gulf War.
    One found that those who served in the gulf were nearly 
twice as likely to contract ALS as veterans who did not serve 
in the gulf.
    The other study found that young gulf veterans, those under 
the age of 45 were more likely, twice as likely to develop ALS.
    The third study which was conducted by researchers at 
Harvard found out that people with a history of any military 
service, Vietnam, Korea, World War II, are at a more than 50 
percent greater risk to get ALS than people who never served in 
the military.
    As a veteran myself having served in the Air Force from 
1966 to 1973, I believe that we owe it to our Nation's veterans 
to find out why there seems to be an increased risk of ALS with 
military service.
    We owe it to people like Daniel Borsen, a veteran diagnosed 
with ALS who is sitting behind me with his daughter, Erica. We 
owe it to people like Charles Diser, a veteran who is also 
here, to the other veterans here today and across the country 
who are fighting ALS.
    In this effort, we should also remember that ALS can strike 
anyone at any time as we have seen here earlier today. 
Therefore, I urge the subcommittee while it is important to 
discover what may be causing the increased risk of ALS in the 
military, we still must focus on ALS research as a whole for 
any progress we realize in ALS research certainly will benefit 
the entire ALS community.
    Catfish Hunter was a pitcher back in his era and like David 
and like myself, when we took the mound, we took the mound to 
go nine innings to do a complete game. Catfish could not finish 
this game. He has turned the ball over to David, myself, Curt 
Schilling. We ask the subcommittee to take part in this and 
help us out of the bullpen to get that game complete so that we 
can find a cure for ALS.

                           PREPARED STATEMENT

    Thank you for providing me with this opportunity and I am 
pleased to answer any questions you may have.
    [The statement follows:]

                    Prepared Statement of Tommy John

    Mr. Chairman, Members of the Subcommittee, my name is Tommy John. I 
appreciate the opportunity to appear before you today, alongside 
another former New York Yankee, to talk about a disease I care so 
deeply about--a disease which was named after a former Yankee--Lou 
Gehrig.
    The reason I mention our affiliation with the New York Yankees is 
because it is through my career with the Yankees that I am involved in 
the fight to find a cure for ALS. While I was aware of Lou Gehrig's 
disease during my playing days, what I did not know was that the 
disease would take one of my teammates--Jim ``Catfish'' Hunter. That's 
why I'm involved in this fight. It is personal.
    Many of you may remember Catfish Hunter as a great pitcher--a hall 
of fame pitcher who, like David and one of your colleagues, Jim 
Bunning, threw a perfect game back in 1968. You may remember him as a 
larger than life figure with his trademark mustache and Carolina 
accent. Jim was a great teammate. ALS took his life in 1999, at the age 
of 53.
    As a friend of Jim's back in North Carolina, I saw first hand what 
ALS is and how the disease can whittle away at the human body and how 
it can take a once powerful man and make him powerless. It is a 
horrific disease and we must find a cure for it.
    My entire family, including my son Taylor and my wife Sally, who 
are with me here today, is committed to finding a cure for the disease 
that took the life of Catfish Hunter and thousands of others. I am an 
honorary member of the Greater New York Chapter and each year I am 
proud to attend the annual New York Sports Banquet, which is the single 
largest fund raising event for The ALS Association. My family and I 
also work closely with The ALSA Chapter in North Carolina--the Catfish 
Hunter Chapter--to increase awareness of the disease and raise funds 
for ALS research. In fact, Taylor, who is a professional singer, 
performed at the Candlelight Vigil that was held on Monday evening at 
the Jefferson Memorial. He performed at last year's vigil as well.
    However, what I want to focus the remainder of my remarks on today 
is not what I have done or what my family has done to raise awareness 
of ALS. What I want to discuss is why military veterans--people who 
seem to be so strong, like Catfish Hunter--are being diagnosed with ALS 
and dying from the disease at a greater rate than other Americans.
    Although I am not a researcher, I am aware of at least three recent 
studies, which have found that military veterans are at an increased 
risk of dying from ALS. Two of those studies focused on the 1991 
Persian Gulf War. One found that those who served in the Gulf were 
nearly twice as likely to contract ALS as veterans who did not serve in 
the Gulf. The other study found that young Gulf War veterans--those 
under age 45--were more than twice as likely to develop ALS. The third 
study, which was conducted by researchers at Harvard, found that people 
with a history of any military service--Vietnam, Korea, and World War 
II--are at a more than 50 percent greater risk of ALS than people who 
have never served in the military.
    I believe that we owe it to our nation's veterans to find out why 
there seems to be an increased risk of ALS with military service. We 
owe it to people like Daniel Bourson, a veteran diagnosed with ALS who 
is sitting behind me with his daughter Erika. We owe it to people like 
Charles Dysart a veteran who also is. And to other veterans here today 
and across the country who are fighting ALS.
    In this effort, we should also remember that ALS can strike anyone 
of us at anytime. Therefore, I urge the Subcommittee that while it is 
important to discover what may be causing an increased risk of ALS in 
the military, we still must focus on ALS research as a whole, for any 
progress we can realize in ALS research certainly will benefit the 
entire ALS community.
    Thank you for providing me with the opportunity to appear before 
you today. I am pleased to answer any questions you may have.

    Senator Shelby. Mr. Cone, you are welcome here, both of 
you. I just think if we could put you on the mound at the same 
time in your prime, would that not be a spectacle? Go ahead.
    Mr. Cone. That would be nice.
    Senator Shelby. Thank you for being on this mound today 
though.
STATEMENT OF DAVID CONE
    Mr. Cone. Senator Shelby, Senator Harkin, thank you for 
helping make this happen today and doing your part.
    I have had the privilege of working with and meeting a 
great many people in the ALS community through my association 
with the Greater New York Chapter. You know, I was also a 
Yankee spokesman. You know, there has always been a Yankee 
player connected with ALS back to Lou Gehrig and I certainly am 
humbled to be one of those players along with Tommy John.
    I am proud to have helped raise awareness of the disease 
and funding for ALS research and patient services. But I have 
to say that each time I participate in one of the chapter's 
events, each time I meet a person with ALS or a family whose 
lives have been touched by ALS, I am truly humbled.
    After all, I am just an ex-ball player who is willing to 
help out. The people out there on the front lines living with 
ALS helping to fight this disease are the true heros. Everyone 
you see sitting here, all the families behind us. Many of the 
people in this room with us today and who are meeting with 
their Members of Congress as we speak are heros and they are 
the reason why I am so honored to be here today.
    Before I continue with my remarks, I want to mention 
another baseball player and his wife who have been tremendous 
supporters in the fight against ALS, Curt Schilling of the 
Boston Red Socks and his wife, Shanda, who I would like to 
introduce is here.
    They have helped raise millions of dollars for ALS research 
and patient services throughout Curt's career from Philadelphia 
and Arizona to Boston. They have also raised awareness of the 
disease.
    You probably saw the KALS written on Curt's shoe during the 
World Series last year. Every time the camera focused on Curt's 
shoe and the now infamous bloody sock, you saw the words strike 
out KLS or KALS.
    As you know, ALS is a disease that can strike anyone 
whether a seemingly invincible Hall of Fame ball player like 
Lou Gehrig or your next-door neighbor. It does not 
discriminate.
    Unfortunately, we do not know what causes ALS, how it can 
be effectively treated, how to prevent the disease, or how to 
cure it. But we have an opportunity to change that.
    In addition to increased funding for ALS research, which I 
strongly urge the subcommittee to support, we need to arm ALS 
researchers with the information and data they need to identify 
and pursue promising avenues of research that may provide new 
insights into the disease and lead us closer to finding 
treatment and cure.
    The Greater New York Chapter is working with the New York 
State legislature on an effort that would identify and collect 
information about people living with the disease in New York 
State. While this is an important step in providing researchers 
with valuable information about ALS, more needs to be done on a 
national basis.
    As Dr. Bruijn mentioned earlier, a national ALS registry 
will allow us to learn more about ALS, not only in terms of how 
many people have ALS in the United States today, but also those 
people and their ages, the family history and other information 
that is so vital to gaining a better understanding of the 
disease.
    By collecting this information, we may be able to learn 
what causes ALS and why people like Lou Gehrig, Catfish Hunter, 
or Eric Obermann, the young man who testified before me, 
contracted the disease, why ALS chose to strike them.
    Equally important, this information may help us to prevent 
ALS from occurring in the first place or to discover new and 
better ways to treat the disease and improve the quality of 
life of people with ALS.
    Although I am not a scientist, it is apparent to me that a 
national ALS registry could yield important clues about ALS and 
bring some significant benefits to the people sitting behind me 
in this room and as well as people living with ALS back home in 
your States.
    I hope that this committee will provide the funding that 
not only makes additional research possible but also empowers 
those who conduct this research with the information and data 
they need to find a treatment and cure for ALS.

                           PREPARED STATEMENT

    Senator Shelby, Senator Harkin, obviously thank you again 
for inviting me to testify today and I welcome the opportunity 
to work with you and the subcommittee to fight to strike out 
ALS. Thank you.
    [The statement follows:]

                    Prepared Statement of David Cone

    Good morning, Chairman Specter, Senator Harkin, Senator Shelby, and 
members of the Subcommittee. My name is David Cone, and I am pleased to 
be here today.
    I have had the privilege of working with and meeting a great many 
people in the ALS community through my association with the Greater New 
York Chapter of The ALS Association and as the New York Yankee 
spokesman for The ALS Association. I am proud to have helped raise 
awareness of the disease and funding for ALS research and patient 
services.
    But I have to say that each time I participate in one of the 
Chapter's events. Each time I meet a person with ALS or a family whose 
lives have been touched by ALS, I truly am humbled. After all, I am 
just an ex-ballplayer who's willing to help out. The people out there 
on the front lines living with ALS and helping to fight this disease 
are heroes. Many of the people in this room with us today and who are 
meeting with their Members of Congress as we speak are heroes. And they 
are the reason why I am so honored to testify before you today.
    Before I continue with my remarks, I wanted to mention another 
baseball player and his wife who have been tremendous supporters in the 
fight against ALS. Curt Schilling of the Boston Red Sox and his wife 
Shonda, who is joining us today, have helped raise millions of dollars 
for ALS research and patient services throughout Curt's career--from 
Philadelphia to Arizona to Boston. They've also raised awareness of the 
disease. You probably saw the K-ALS written on Curt's shoe during the 
World Series last year. Every time the camera focused on Curt's now 
famous bloody sock, the world also saw strikeout ALS.
    As you know, ALS is a disease that can strike anyone--whether a 
seemingly invincible hall-of-fame ballplayer like Lou Gehrig, or your 
next door neighbor. It does not discriminate. Unfortunately, we do not 
know what causes ALS, how it can be effectively treated, how to prevent 
the disease or how to cure it. But we have an opportunity to change 
that.
    In addition to increased funding for ALS research, which I strongly 
urge the Subcommittee to support, we need to arm ALS researchers with 
the information and data they need to identify and pursue promising 
avenues of research that may provide new insights into the disease and 
lead us closer to finding a treatment and cure.
    The Greater New York Chapter is working with the New York State 
legislature on an effort that would identify and collect information 
about people living with the disease in New York State. While this is 
an important step in providing researchers with valuable information 
about ALS, more needs to be done on a national basis.
    As Dr. Bruijn mentioned earlier, a national ALS registry will allow 
us to learn more about ALS, not only in terms of how many people have 
ALS in the United States today, but also who those people are, their 
ages, family history, and other information that is so vital to gaining 
a better understanding of the disease. By collecting this information 
we may be able to learn what causes ALS and why people like Lou Gehrig, 
Catfish Hunter or Eric Obermann--the young man who testified before 
me--contracted the disease. Why ALS chose to strike them.
    Equally important, this information may help us to prevent ALS from 
occurring in the first place or to discover new and better ways to 
treat the disease and improve the quality of life for people with ALS. 
Although I am not a scientist, it is apparent to me that a national ALS 
registry could yield important clues about ALS and bring significant 
benefits to the people sitting behind me in this room as well as the 
people living with ALS back home in your states.
    I hope that this committee will provide the funding that not only 
makes additional research possible, but also empowers those who conduct 
this research with the information and data they need to find a 
treatment and cure for ALS.
    Mr. Chairman, thank you again for inviting me to testify today. I 
welcome the opportunity to work with you and the Subcommittee in the 
fight to strikeout ALS.

STATEMENT OF KATE LINDER
    Ms. Linder. Good morning, Mr. Chairman, members of the 
subcommittee. Thank you for inviting me to participate in this 
important hearing today. I am truly honored to be here.
    As you know, my name is Kate Linder. I am an actress. I 
play the role of Esther Valentine on the number one daytime 
drama ``The Young and the Restless'' for the past 23 years.
    But I am also an advocate for people with ALS, their 
families, and caregivers. I am pleased to be here today to 
share with you my personal relationship with this terrible 
disease.
    I also hope that through this hearing and in our meetings 
with your colleagues in both the Senate and House we will be 
able to raise awareness of the disease and support for our 
cause.
    Five months ago, on December 8, 2004, my 49-year-old 
brother-in-law was diagnosed with ALS in Seattle, Washington. 
Now, at the time, I only had a passing knowledge of ALS. That 
is to say I had heard of ALS or Lou Gehrig's disease. But like 
many Americans, I did not know the nature of the disease or how 
it would change the lives of our entire family.
    I did not know that ALS robs a person of the ability to 
move their arms and legs, to speak, and to breathe, nor was I 
aware that there was no cure for the disease. That is why I am 
here today, to raise awareness and to help advance the effort 
to find a treatment and cure for ALS.
    Scott and his wife have two daughters and his diagnosis has 
devastated our entire family. But we will continue to fight 
ALS. It is still really hard for me to believe that even now, 5 
months later, how someone so young and so full of life could be 
stricken with such a terrible disease.
    Since Scott's diagnosis, I have made it my mission to do 
everything I can to support Scott, his wife, Georgianne, his 
daughters, Kristin and Sandra, in their fight against ALS.
    Working with the ALS Association, ALSA, we have created a 
new public awareness campaign called Kate's Club which will 
focus much-needed attention on the disease. Through the ALS 
Association and Kate's Club, I am networking with fellow 
actors, fans, and volunteers to raise awareness and encourage 
them to join us in this important fight.
    I have also filmed a public service announcement to promote 
one of ALSA's signature events, the Walk to Defeat ALS. This 
year, ALSA chapters are staging more than 147 walks in cities 
across the country to raise funding to support ALS research and 
patient services.
    Last year, these walks collectively raised more than $9 
million and more than 90,000 people participated in the events.
    I hope that our work with Kate's Club will help raise even 
more funding to support programs and research that are so 
essential to finding a treatment and cure for ALS and to 
improving the quality of life for people with ALS and their 
families and caregivers.
    I also wanted to praise the efforts of a fellow actor, 
Allen Rosenberg, who is also with us today for this hearing. 
Allen who portrayed a person with ALS on the hit show, ``The 
Guardian,'' also has helped raise awareness of ALS and funding 
for our fight, having filmed a recent public service 
announcement and participated in numerous ALSA events and 
events organized by the Greater Los Angeles Chapter of the ALS 
Association.
    But as actors and actresses, people in my profession are 
used to playing many roles as part of our every-day life. But 
what I would also like to focus attention on here today is the 
important role that family caregivers play in the lives of 
people with ALS.
    Specifically I would like to highlight the need for respite 
care. As people with ALS lose the ability to walk, move their 
arms, talk, and even breathe, the disease requires them to rely 
on caregivers, usually their families, to provide the care and 
assistance that is needed to perform normal activities of daily 
living.
    In many cases, particularly in the latter stages of the 
disease, people with ALS have a need for continuous care 24 
hours a day, 7 days a week. The burden placed on caregivers and 
family members is tremendous.
    Therefore, these families and caregivers have a significant 
need for respite care services. Respite care provides temporary 
relief to caregivers of individuals with chronic illnesses and 
disabilities and is a key component of quality long-term care.
    Respite care may take place in the home or in a facility 
and allows caregivers much needed time off while providing 
quality care for the loved one. Respite care helps keep 
families together, helps prevent abuse and neglect, and 
forestalls premature costly institutionalization and possible 
impoverishment. Virtually every family who is touched by ALS 
has a need for respite care services.
    Legislation such as ``Life Span Respite Care Act,'' which 
passed the Senate in 2003, is needed to help expand the 
availability of respite care services for people with ALS and 
their families. The bill is expected to be introduced again 
this year by Senators Clinton and Warner and I hope Congress 
will pass this much-needed legislation.
    When it does pass, I also hope that this subcommittee will 
support funding for the programs included in the bill. They 
would be a tremendous benefit to people with ALS and their 
families across the country. They would benefit Scott and his 
family as they continue their fight against ALS.

                           PREPARED STATEMENT

    I just want to personally thank you so much for inviting me 
to participate in this hearing. Thank you.
    [The statement follows:]

                   Prepared Statement of Kate Linder

    Good morning. Mr. Chairman, Members of the Subcommittee, thank you 
for inviting me to participate in this important hearing today. I am 
honored to be here.
    My name is Kate Linder and I am an actress, having played the role 
of Esther Valentine on the daytime television drama, The Young and the 
Restless, for the past 23 years. I also am an advocate for people with 
ALS, their families and caregivers and I am pleased to be here today to 
share with you my personal relationship with this terrible disease. I 
also hope that through this hearing, and in our meetings with your 
colleagues in both the Senate and House, we will be able to raise 
awareness of the disease and support for our cause.
    Five months ago, on December 8, 2004, my 49 year old brother-in-law 
was diagnosed with ALS in Seattle, Washington. At the time, I only had 
a passing knowledge of ALS--that is to say, I had heard of ALS, or Lou 
Gehrig's disease. Like many, many Americans, I did not know the nature 
of the disease or how it would change the lives of our entire family. I 
did not know that ALS robs a person of the ability to move their arms 
and legs, to speak and to breathe. Nor was I aware that there was no 
cure for the disease. And that is why I am here today: To raise 
awareness and to help advance the effort to find a treatment and cure 
for ALS.
    Scott and his wife have two daughters and his diagnosis has 
devastated our entire family. But we will continue to fight ALS. Yet, 
it still is hard for me to believe--even now, 5 months later--how 
someone so young and so full of life could be stricken with such a 
terrible disease.
    Since Scott's diagnosis, I have made it my mission to do everything 
I can to support Scott in their fight against ALS. Working with The ALS 
Association (ALSA), we have created a new public awareness campaign 
called ``Kate's Clubsm,'' which will focus much needed 
attention on the disease. Through The ALS Association and Kate's Club I 
am networking with fellow actors, fans, and volunteers, to raise 
awareness and encourage them to join us in this important fight. I also 
have filmed a public service announcement to promote one of ALSA's 
signature events, the Walk to D'Feet ALS. This year, ALSA's Chapters 
are staging more than 147 walks in cities across the country to raise 
funding to support ALS research and patient services. Last year, these 
walks collectively raised more than $9 million and more than 90,000 
people participated in the events. I hope that our work with Kate's 
Club will help raise even more funding to support programs and research 
that are so essential to finding a treatment and cure for ALS and to 
improving the quality of life for PALS and their families and 
caregivers.
    I also wanted to briefly praise the efforts of a fellow actor, Alan 
Rosenberg, who also is with us today for this hearing. Alan, who 
portrayed a person with ALS on the hit show The Guardian, also has 
helped raise awareness of ALS and funding for our fight, having filmed 
a recent public service announcement and participated in numerous ALSA 
events, and events organized by the Greater Los Angeles Chapter of The 
ALS Association.
    As actors and actresses, people in my profession are used to 
playing many roles as part of our everyday lives. But what I also would 
like to focus attention on here today is the important role that family 
caregivers play in the lives of people with ALS. Specifically I would 
like to highlight the need for respite care.
    As people with ALS lose the ability to walk, move their arms, talk 
and even breathe, the disease requires them to rely on caregivers, 
usually their families, to provide the care and assistance that is 
needed to perform normal activities of daily living. In many cases, 
particularly in the later stages of the disease, people with ALS have a 
need for continuous care, 24 hours a day, seven days a week. The burden 
placed on caregivers and family members is tremendous. Therefore, these 
families and caregivers have a significant need for respite care 
services.
    Respite care provides temporary relief to caregivers of individuals 
with chronic illnesses and disabilities and is a key component of 
quality long-term care. Respite care may take place in the home or in a 
facility and allows caregivers much needed time off, while providing 
quality care for the loved one. Respite care helps keep families 
together, helps prevent abuse and neglect, and forestalls premature, 
costly institutionalization and possible impoverishment. Virtually 
every family who is touched by ALS has a need for respite care 
services.
    Legislation, such as the Lifespan Respite Care Act, which passed 
the Senate in 2003, is needed to help expand the availability of 
respite care services for people with ALS and their families. The bill 
is expected to be introduced again this year by Senators Clinton and 
Warner and I hope Congress will pass this much needed legislation. When 
it does pass, I also hope that this Subcommittee will support funding 
for the programs included in the bill. They would be a tremendous 
benefit to people with ALS and their families across the country. And 
they would benefit Scott and his family as they continue their fight 
against ALS.
    Thank you again for inviting me to participate in this hearing.

    Senator Shelby. Thank you.
    I want to thank the panel.
    I want to recognize again Mrs. Schilling. Thank you for 
coming. We all appreciate the courage and the competitiveness 
of your husband and the fact that he is involved in this fight, 
too, ALS, and you too.
    Mr. John, you know firsthand the benefit of biomedical 
advances. I will tell Dr. Andrews, who is a constituent of 
mine, that you were here today. He would rebuild your arm if 
you were probably a couple years younger, you know.
    Mr. John. I need somebody to--a body would due right now.
    Senator Shelby. A body.
    Mr. John. The arm is fine. The body is gone.
    Senator Shelby. Mr. Cone, we appreciate your involvement 
here and the background of both of you.
    Ms. Linder, you are very articulate.
    So we are going to do everything we can to properly fund 
this research to try to find a cure to a dreaded disease.
    We have got a vote on the floor. They are holding the vote 
for us. Senator Harkin and I are going to have to leave.
    Senator, you may have the last word.
    Senator Harkin. I just want to thank you, Mr. Chairman. I 
want to thank you, Mr. John, Mr. Cone, Ms. Linder. Obviously a 
lot of people look up to you. You are heros or heroines to a 
lot of people in this country, rightfully so, and your 
leadership in this really has a great impact.
    So I just want to thank you for lending your names and your 
status to this effort. It means a lot. It really does. It 
motivates a lot of people. They look up to you and they respect 
you. When they hear that you are involved in this fight, that 
gives them courage too. So thank you for doing this.
    Finally, I ask unanimous consent to include a written 
statement from Adrienne Hallett in the hearing record.
    [The statement follows:]

             Statement for the Record From Adrienne Hallett

    There's no question about when my father, Albert Arthur Hallett, 
lost his battle with ALS. He died early in the morning on August 5, 
2004, at the age of 67. What's harder to determine is when his battle 
began.
    Did he carry a nefarious gene when he was born in 1936, the first 
son of a farmer and a teacher in a small town in northeast Iowa? Did it 
have something to do with the chemicals and pesticides he was exposed 
to as a teenager on a struggling family farm?
    Did ALS creep in when he enlisted in the Army, filled with all of 
the cocky pride of an 18-year-old about to see the world? He trained at 
Fort Hood, Texas, and spent four years in Korea. Was there something in 
the Army rations--those industrial meals he reminded me of every time I 
as a child ``forgot how good I had it'' with my mother's cooking?
    Was ALS lurking when he got back from the Army and courted Shirley 
Ann Schnieders over dinner at the Cozy Inn? My father liked a 
challenge, and this strong, spunky young woman was a challenge worth 
winning. She used to say that by making her a Hallett, my father took 
the ``SASS'' out of her. He would wink at me and say, ``not by half.'' 
Her feistiness would come in handy when his body started failing, but 
no one had any answers, and when someone needed to stand up to the HMO, 
and he didn't have the strength to do it himself.
    Did he already have ALS as he raised two children? Three times in 
his life, my father made a beeline from a doctor's office to a bank. In 
1971, the doctor informed him he was going to have a son. He went to 
the bank to start a college fund. Three years later, a little girl was 
on the way, and college was on his mind again. The third trip to the 
bank was the day of the ALS diagnosis. It was no surprise to anyone 
that Al Hallett's first thought was to make sure his family would be 
taken care of.
    Did ALS take root in the manufacturing plants where my father 
walked the floors before returning to the office where he worked as an 
industrial purchaser? Or was the toll taken in the back-breaking years 
of laying carpet in people's houses on Saturdays--a side business he 
ran to make ends meet?
    One thing I know now, ALS was there when he retired in 1998. An 
Internet search at the time convinced him that the choking feeling in 
his throat was the result of a hiatal hernia. He would regularly 
complain that his left thumb tingled, as he spent countless hours 
volunteering with the Optimist Club, SCORE, the Boy Scouts, and any 
other organization willing to put up with his corny phrases in return 
for his service. As I think back on his symptoms, my father's voice is 
in my head: ``Adrienne, hindsight is always 20/20.''
    But in 1998, no one thought twice about those symptoms. How could 
they? This was the tough man whose right index finger was permanently 
bent at the first joint because of a disagreement with a corn husker. I 
used to joke that my father was clearly enjoying retirement because he 
spent his Saturday morning at the diner with all of the other retired 
guys, complaining about their various aches and pains. Meanwhile, he 
had eight years to wait until his wife could retire. He would delight 
in saying, ``That's what I get for robbing the cradle.'' He planned the 
trips they would take and the property they might buy.
    Soon, however, he would tell us that his hand would periodically go 
numb. He visited his primary care doctor, an otolaryngologist for his 
throat, a neurologist for his hand and finally a neurosurgeon. We never 
thought to tell the neurologist about the otolaryngologist, or vice 
versa. No one ever asked. We knew nothing about ALS and no health 
professional even mentioned the possibility. An MRI turned up a small 
bone spur in his fourth vertebrae. There was no accompanying loss of 
strength or range of motion, and so my mother was adamantly opposed to 
a surgery that would cut open her husband's spine.
    Yet, five years after retiring, his left arm had further weakened 
and his feet would sometimes drag. Yes, Dad, hindsight is always 20/20. 
He convinced my mother that he needed to get the surgery before she 
retired and their health insurance changed. So in October 2003, a 
surgeon removed a section of his fourth vertebrae and replaced it with 
a bone graft. A metal plate held it all in place. We were told it was a 
common procedure--no reason to gather the family. With a son in 
Seattle, working for a genetics company, and a daughter in Washington, 
working for this subcommittee, Al Hallett underwent a procedure that 
would change our world forever.
    When my father woke up from surgery, his left hand felt no better. 
More alarming, he couldn't move his right arm at all. While this wasn't 
typical, the doctors assured us that there was nothing to worry about. 
The nerve was simply bruised from the procedure, they said; with 
physical therapy, motion would return in four to six weeks.
    He tried to comply, but his growing fatigue made physical therapy 
all but impossible. His feet began losing function and he began having 
falls. My mother hammered at the neurosurgeon in follow-up visits, 
convinced that something dreadful had happened in the surgery. Whatever 
it was, it was getting worse.
    A month later, just before Thanksgiving, the neurosurgeon finally 
agreed and sent him to a neurologist. Brain scans, MRIs, and spinal 
taps ensued. Tests ruled out everything. Weeks went by with doctor 
appointments galore and no answers. When my father called me after an 
MRI, he sounded ecstatic. In keeping with the Optimist oath he took 
every Saturday at the diner, my father cheerfully announced that he had 
a vitamin deficiency. He passed the phone to my mother, who left the 
room and told me tearfully that he had multiple sclerosis. Further 
conversation revealed that the doctor had told them it could be 
anything from a vitamin deficiency to MS. The optimist and the 
pessimist--opposites will forever attract.
    The final diagnosis came in March 2004, five months after the 
surgery. A neurologist who had defensively brushed off the questions 
posed by my mother, my brother and me for months delivered the 
devastating news to my mother in the middle of a crowded waiting room: 
``I'm sorry but there is no treatment. I suppose you will want a second 
opinion but I assure you this is it. There is a drug out there but I 
think it is a waste of money for him.'' Words can't describe that 
moment. My strong, spunky mother crumpled in front of me and I couldn't 
seem to find oxygen in the room. Anger: who was this doctor, this 
indifferent man sitting there throwing this news at us like an 
executioner in front of 50 total strangers? Fear: we cannot do this, it 
is too much, it is too hard and we will lose in the end. Disbelief: a 
terminal diagnosis based on ruling everything else out? How do they 
know? They can't know for sure, there wasn't a test, it could be 
something else. It must be something else, anything else. Shock: 
please, God, this isn't real. Please let it not be true. I want my 
father back.
    The University of Iowa, located just 45 minutes away from our home 
in Cedar Rapids, had an expert in ALS and a comprehensive Center on 
Disability. Unfortunately, the HMO agreed to cover a visit there for a 
second opinion only; as far as they were concerned, there were plenty 
of neurologists in town. But none of those doctors offered the 
comprehensive approach of the UI center, and my parents couldn't pass 
up its services, even if it meant draining their retirement savings.
    In one afternoon at the center, my father saw a neurologist who 
immediately confirmed the diagnosis, a nutritionist, an assistive 
technology specialist and a mental health coordinator who checked both 
my father and mother for signs of depression. A follow-up appointment 
was scheduled for three months later, the normal timeframe for ALS 
patients.
    But the ALS took over so quickly, and I learned that progressive 
diseases don't fit the HMO model. My father's HMO wouldn't approve the 
purchase of any equipment until after he had lost the skill or function 
it served. Once that happened, we had to go to the doctor for a 
prescription, then submit it to the insurance company, then purchase 
the equipment--a process that could take weeks. In the meantime, he 
suffered and went without.
    The two bright stars in this very dark night were the University 
Center on Disability and the local Muscular Dystrophy Association. The 
Center gave my father respect and choices. There was so much he 
couldn't control, but they told him of the limited options he had and 
let him decide. The MDA group loaned us equipment when we asked for it, 
so it was there when he would finally swallow his pride enough to use 
it. Leg braces were replaced by a walker, which led to an electric 
scooter. It took multiple trips to the emergency room with injuries 
from falling down before my dad submitted to using the scooter. 
Persuading him to use a wheelchair would take longer, but as is the 
case with ALS, it was inescapable.
    My father, who had built our deck and repaired almost everything in 
our house, had to watch others build an accessible entrance, raise the 
chairs so it was easier to get in and out, and widen the doorways for a 
wheelchair. The only part of it the former purchaser liked was picking 
out the wheelchair lift and haggling with the salesman over gears and 
resistance and loads. None of it was eligible for insurance.
    The HMO insisted that my father go to an approved physical 
therapist. This therapist ran him through a series of activities such 
as getting up from a lying position, standing, sitting, and negotiating 
stairs. My father was helped to the floor and directed to stand up. He 
tried in vain. When finally he was humbled enough to admit defeat, the 
therapist told my father to drag himself across the floor to a chair to 
pull himself up. With his wife watching, he labored to make his arms--
the source of his initial symptoms--pull his body weight across the 
floor. The report to the insurance company indicated that my father was 
able to take care of himself.
    My father went home that night exhausted and utterly humiliated. 
The therapist seemed totally unaware that fatigue is dangerous for ALS 
patients. Whenever my father tired himself out, it would take days to 
get merely a semblance of his energy and function back. We lost a 
little piece of him each time he became fatigued.
    Meanwhile, the University nutritionist helped my mother as she 
tried in vain to keep weight on my father. She tried to make things as 
normal as possible, even putting a bratwurst in the blender on the 
Fourth of July.
    In late June, the doctor appointment showed that he was losing lung 
capacity and his weight loss was unsustainable. He wasn't quite to the 
designated marker for the insertion of a feeding or breathing tube, but 
the next appointment wouldn't be for three months. Out of fear and 
frustration, my mother arranged for a consultation with the ALS center 
scheduled for the second week of August. Hospice workers checked on 
him, but said he wasn't sick enough for their services.
    A week later, on August 4, my father informed his nurse that he 
wanted to go to the SCORE volunteer awards. He had had to quit the 
group months before, but he wanted to cheer on his friends. He even 
insisted that my mother take him to the barber for a haircut--an Army 
man to the end.
    After the haircut, he went home and went to sleep. My mother woke 
him twice for meals. He quietly died in the middle of the night. A week 
after being too well for hospice, a week before his chance for a 
breathing tube, my father went to sleep and never woke up.
    A day later, the postman brought another denial from the HMO: The 
condition was not serious enough to warrant the June doctor 
appointment.
    Did ALS come in from chemicals, from the environment, from the 
Army, from a gene? Or was it simply random? Can a good man, who does 
everything our society tells him is the right and responsible way to 
live, be struck down by the toss of the dice? I don't know the answer 
to that. But a man who told me repeatedly ``Can't never did nothin', 
Adrienne. I don't want to hear that word from you'' was eventually 
faced with a room full of doctors who had nothing for him but the word 
he so despised. No treatment, no options--we can't help you. He 
deserved more.
    My father never complained and never let the rest of us complain. 
At his funeral, one of his buddies told me with tears in his eyes that 
my father had spoken to them just once about ALS. As he was walking out 
of the diner for the last time, just after he had come back from the 
emergency room following a fall, my father looked back, sadly shook his 
head and said, ``What did I ever do that was so wrong?''
    He did nothing wrong. He was a good man. He helped his parents, he 
served his country, he saved for his kids' education and his 
retirement, and he volunteered in his community. Like everyone here 
today, I have a thousand more questions than answers. I leave 
conclusions to be drawn by this Subcommittee and by the test of 
history. I will say though, that for all of the years my father talked 
about it, ALS taught me once and for all to hate the word can't. We can 
find a cure for ALS. We just have to try.

                         CONCLUSION OF HEARING

    Senator Shelby. Thank all of you. That concludes our 
hearing.
    [Whereupon, at 11:50 a.m., Wednesday, May 11, the hearing 
was concluded, and the subcommittee was recessed, to reconvene 
subject to the call of the Chair.]

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