RIDE FOR LIFE EDITOR'S NOTE:
We are continuing to update this story as new information becomes available. Stay tuned.

April 11, 2003

The ALS Association is actively investigating thrombopoietin (rhTPO) and
its potential benefit for people with ALS. In aggressively collecting
information over the past several weeks, ALSA's president, research
director and vice president, patient services have initiated multiple
discussions with several ALS science and clinical experts, representatives
from the FDA, Genentech - the company that apparently holds the available
rhTPO - and George Schwartz, MD, the physician who reported the ALS
one-patient case study using rhTPO. We recognize the time-sensitive nature
of the rhTPO issue.

How is ALSA helping?

We are actively urging more review and discussions about the study of
rhTPO for ALS while we continue to gather more information about rhTPO,
about Dr. Schwartz's patient experience and about the scientific rationale
for testing rhTPO in ALS. Additionally, we remain in frequent
communication with all involved parties including the drug companies as we
look into what next steps are necessary. ALSA staff is pursuing all
avenues related to rhTPO on behalf of people with ALS while maintaining
the highest scientific standards and concern for patient well-being.

A summary of the information we have gathered to date is included here in
the format of frequently asked questions (FAQs).

What clinical trials have been conducted with rhTPO?

There have been a number of clinical studies evaluating the potential
benefit of rhTPO for use in cancer - related to possible treatment for the
platelet reduction that can come with some chemotherapy. Other conditions
for which rhTPO has been studied are those that cause thrombocytopenia.
Thrombocytopenia is a medical term for a reduction of blood platelets.
These clinical trials have not led to FDA approval for any indication, and
rhTPO remains an experimental compound.

What is the scientific rationale for how rhTPO might provide a benefit for
people with ALS?

RhTPO is known to be a growth factor that stimulates the production of
platelets and other primitive blood cells. It is theorized that platelet
growth factors might have a neuroprotective effect on the motor nerves.
This has not been proven yet in an ALS animal model or in humans.

The rationale for testing rhTPO in a clinical study of ALS is that
elevation of the platelet counts may result in a release from platelets of
several different growth factors with possible subsequent diffusion into
the central nervous system. A working hypothesis of this study is that
these platelet growth factors might exhibit trophic and protective
features for motor neurons.

What studies have been done with rhTPO in ALS and what is planned?

· Through our extensive Internet research, we have found citations for a
few scientific studies that may tangentially support the rationale for
testing rhTPO in ALS. We have forwarded these citations to ALS experts and
are in the process of obtaining copies of these articles and reviewing
them as possible supportive literature for defining the science basis for
rhTPO in ALS.

· An ALS one-patient case study from Dr. Schwartz using rhTPO has been
reported at a recent scientific meeting. ALSA is obtaining the abstract
and any publications related to this case study report, and has requested
additional information and literature from Dr. Schwartz.

· Mouse TPO is now being tested in an ALS mouse model at the Massachusetts
General Hospital (MGH). This study is in its early stages of investigation
and ALSA maintains close communication with the investigators.

· These same ALS investigators at MGH have a protocol prepared for a
10-patient, safety study of rhTPO. Moving forward with this human trial is
dependent on obtaining rhTPO, which is not currently available to the
investigators. ALSA is aggressively working to make the existing supply of
rhTPO available for this human trial.

Why is rhTPO not available for the small human ALS study?

Although a small amount of rhTPO exists, the company Pharmacia holds the
rights for further development and production of rhTPO. ALSA is in contact
with both Genentech and Pharmacia concerning rhTPO and how to find a way
to make the existing supply available for a clinical trial.

What is thrombopoietin?

TPO is a hormonal substance, made largely in the liver and bone marrow of
humans and other mammals, and is found in the body's plasma. Identified in
the 1950s, TPO was recognized as acting to increase the number of
platelets in the blood in situations in which the body's level of
circulating platelets drops below a key level.

Recombinant human thrombopoietin (rhTPO) is an investigational agent
(meaning that it is not FDA approved). It stimulates megakaryocyte
production and raises the circulating platelet count. Megakaryocytes are
large cells in the bone marrow that release mature platelets. Platelets
contain several different growth factors that have demonstrated in vitro
and in vivo to have trophic actions for motor neurons. In vitro means in
a laboratory environment such as a test tube or dish, and in vivo means in
a living body - animal or human.

What is recombinant?

This is the technology of making combinations of materials using human DNA
sequences. This technology process is used to make novel products or
compounds or to develop a commercial product of a naturally occurring
human biologic or chemical substance. The "r" indicates it is made by
recombinant technology and the "h" refers to human.

When is rhTPO not recommended or not suitable and what are known side
effects?

Cautions for the potential use of rhTPO in humans include those people
with known prior hypersensitivity to recombinant forms of thrombopoietin,
and active thromboembolic disorder. Care should be taken with people who
have blood clotting disorders, prior blood clots, cardiovascular disease,
kidney or liver insufficiency, and prior pelvic radiation.

The risks of blood clots and thromboembolism (clots traveling to the legs,
lungs, brain, heart or other body areas) are not known and are under
investigation. Previous trials with rhTPO for cancer patients may not
provide the same side effect information as a trial in ALS would provide
with rhTPO, due to potential differences in dosage and/or length of
treatment.

ALSA will continue to keep the ALS community informed of new information
regarding rhTPO.

Contact:
alsinfo@alsa-national.org
Toll Free (800) 782-4747

*******************

Before you read the story below, we would like to preface it with some remarks from Ken O'Donnell, who is an ALS patient and Research Chairman of Ride for Life. Ken is also an ALS advocate.

Dear friends,

As interested people in finding a cure for ALS, your hearts must have lept in your mouth to read of one ALS sufferer who was actually getting better. In 2003 this was the second report of an ALS patient seeming to recover lost function. The first was a report by a Canadian physician who reported that an ALS sufferer was actually gaining strength on an AIDS cocktail and that buoyed by her success another 18 patients would be beginning a clinical trial. The second report by Dr. George Schwartz was even more encouaging with the exception that the drug company, Genentech, was not interested in supplying the drug for further study. Dr. Schwartz's existing supply was very low and the drug's efficacy degrades over time.

Incensed by the press release, I called Dr. Schwartz and discussed the issues with him. After discussing this with him it was obvious I needed to make a few more calls.

My next call went to Mike Havilchek at ALSA National. Mike had spoken with Dr. Schwartz and assured me that ALSA was going to use all resources to bring about the next step to prove the benefits of this drug to more than a single person. The next person I talked to was Dr. Farangrazi at ALSHOPE who had also spoken with Dr. Schwartz. She had spoken with her contacts at Genentech who gave her some insights as to why, from a business standpoint, Genentech may not be interested and they gave her some ideas as to alternative drugs which she is following up on.

So what is it I would recommend for an ALS ADVOCATE to do?

I would pressure the drug company via Washington D.C. by writing an email or letter with a copy of the press release to the head of Health and Human Services, Tommy Thompson, with copies to your Senators and Representative, briefly explaining why the action of this drug company is unacceptable, what you want him to do (supply funds for the study through NIH and pressure Genentech to provide the drug) and your intention of discussing this with your elected officials on ALS advocacy day on May 1st, 2003 .

Regards,

Ken O'Donnell,
Advocate for a cure for Amyotrophic Lateral Sclerosis(Lou Gehrig's Disease)

Annual Meeting: American Spinal Injury Association, April 2-6, 2003 - Hotel Inter-Continental Miami, Florida

MIAMI, April 2 /PRNewswire/ -- Reversal of Lou Gehrig's disease may now be possible using thrombopoietin and thyroid hormone to cause regeneration of endogenous stem cells of the central nervous system. Evidence suggests spinal cord injury can be reversed through regeneration by stem cells. This research will be presented by George R. Schwartz, M.D., a senior researcher at Neuroregeneron Co., this week at the annual meeting of the American Spinal Injury Association held in Miami, Florida.

The Food and Drug Administration (FDA) biologics division approved a unique clinical trial in May 2002, authorizing the use of thrombopoietin for Lou Gehrig's disease (amyotrophic lateral sclerosis) in a 40-year-old mother of three small children whose clinical condition was deteriorating rapidly. She was more than 90% paralyzed, with minimal speech capability, tremendous difficulty swallowing, and rapidly failing respiratory function.

The new treatment was approved for a clinical trial after it was demonstrated that platelet growth factors could be tremendously increased through use of thrombopoietin. Platelet growth factors act as stimulants for the growth and development of glial cells which act as repair cells for dying nerve cells. In addition, platelet growth factors can stimulate immature cells to differentiate into cells which act as neurons. Thyroid hormone was added to the trial after experimental evidence demonstrated that thyroid hormone acted as a signaling substance helpful for repair cells to function.

Platelets were raised in cycles to more than 10 times the normal level resulting is blood serum rich in platelet growth factors.

At day 42 of this clinical trial, this patient showed remarkably improved head and neck control and strength. At day 45, she exhibited improvement in tongue strength and motion with improved swallowing functions. As a result, a feeding tube was not necessary.

At day 60, increased leg muscle strength was clearly evident. Along with this motion, the patient was able to turn her arms and hands which had been paralyzed for more than a year. At day 110, she began to move her hands. Muscle strength throughout her body increased and her pelvic muscles could support more weight.

The patient showed clear reversal of a previously deteriorating condition, and return of functions. Her downhill course stopped.

The nerve cell regeneration and reversal of paralysis in this patient with Lou Gehrig's disease suggests that spinal cord injury and paralysis can also be treated with re-growth of the nerve cells of the spinal cord.

Further trials are urgently needed since the average length of life in ALS patients is 3-5 years after diagnosis. There is also some indication that regeneration in cases of spinal cord injury would be more effective soon after the injury.

The FDA has been extremely supportive of this clinical trial and has urged that other trials be conducted as soon as possible. "If this proves out, it is a very exciting result indicating a new treatment and approach to Lou Gehrig's disease and spinal cord injury," said a senior neurologist with the FDA in Rockville, Maryland.

However, despite the encouraging results and excitement generated by this clinical trial, the Genentech company has decided not to release the drug thrombopoietin for any further trials in neurologic disease or injury.

"We will not proceed with any further trials at this time," said Mary Stutts, director of corporate relations at Genentech. The medication was manufactured in substantial quantity in the late 1990s and the current stock of clinical grade thrombopoietin will expire in the year 2003. "Remanufacture is not planned at this time," confirmed Heather Mccauley, spokeswoman at Genentech. She offered no other explanation for the decision not to conduct any further clinical testing.

A director of the Lou Gehrig's clinic at the Massachusetts General Hospital and a professor at the Harvard Medical School, has prepared a trial for ten people with this disease. "I am totally puzzled," commented the doctor, who was rebuffed when he approached Genentech with his proposal. "This defies all common sense and scientific responsibility. We have no other treatments for these conditions," he explained.

Dr. Schwartz, who has been following his patient closely with the FDA approved trial, is also puzzled. "Are they blind to the implications of this drug for use in neurologic diseases or injury?" he remarked.

Monica Collier, one of the researchers who has been following this patient's ground-breaking clinical course, expressed amazement at the lack of compassion shown by the spokespeople at Genentech. "I cannot understand their approach," she said. "It would seem to be in their interest to try to develop this medication, and the patients just cannot wait."

Dr. Schwartz added, "I know the people at Genentech would be happier if there was a large amount of animal experimentation before the clinical trial. However, the reality is that animal models are not suitable to test for regeneration of nervous system cells at this time. We have a treatment which is ground-breaking and which is working in our patient. Let us go forward with further testing. Re-manufacture will take years. Meanwhile all the medication for clinical testing is literally going to waste. Patients are suffering and family and spouses are watching tragic deterioration in their loved ones."

Contact:
George R. Schwartz, M.D.
Senior Researcher, Neuroregeneron Company (a Division of Schwartz
Pharma LLC)
Tel: 505-610-8243
http://www.healingresearch.org

SOURCE Neuroregeneron Company

CONTACT: George R. Schwartz, M.D., Senior Researcher, Neuroregeneron Company, +1-505-610-8243/