ALS Prevalence Studies Underway Near Hazardous Waste Sites
People with ALS encouraged to identify themselves in study areas
August 3, 2004
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Responding
to community concerns, the Agency for Toxic Substances and Disease Registry (ATSDR),
a public health agency of the U.S. Department of Health and Human Services,
is funding an environmental health program to determine the prevalence of
amyotrophic lateral sclerosis (ALS) in areas surrounding hazardous waste sites
in five states.
The neurodegenerative disease, commonly referred to as Lou Gehrig’s disease, attacks nerve cells in the brain and spinal cord resulting in muscle weakness and atrophy. Patients usually survive between two and five years from the time of diagnosis.
Researchers are focusing on communities
near hazardous waste sites in
The ALS Association (ALSA) and its
network of chapters is providing assistance in these studies through the
coordination of efforts to identify people with ALS in the study areas. In
most cases, patients are being identified through their neurologists.
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read this article in its entirety.
New
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The potential is described in a new
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Research Update...
We're Funding The Way To A Cure!
From the ALS NY & Northern New Jersey Newsletter
Summer/Fall 2001, Issue 13
In the first year of the new millennium, ALSA and its chapters raised almost $25
million for ALS-specific research and patient services. With the exception
of the government, this sum exceeds that of any group in the United States
dedicated to fighting ALS.
Focusing just on research, ALSA funded 28 new investigator-initiated research
grants in 2000 in an amount close to $3 million. This is in addition to 48
projects already underway, bringing the total currently funded projects to 76.
Additionally, The Lou Gehrig Challenge created by ALSA in 2000, is committed to
raising another $25 million in five years to accelerate new directions in
research. This is the largest, ALS-specific research program ever
conducted by a non-government health organization and to-date has raised $5.4
million. Your Chapter has already raised $200,000 to the Challenge.
Chapter Funds Ambitious Research
In our [Greater NY] Chapter's short history, we've contributed close to $1.7
million to ALS research. In 2000, as part of The Lou Gehrig Challenge, the
Chapter contributed $100,000, of which $65,000 supported an exciting
investigation by William Dauer, M.D., Columbia University. Using recent
technology, Dauer will develop a new mouse model with "inducible expression
of mutant SOD1" to test whether the disease process can be reversed by
turning off the SOD1 protein. Using the same strategy, Dr. Dauer's
colleagues, working in the same group, have published encouraging results for
Huntington's disease.
Other projects funded in 2000 include three studies undertaken locally, two by
Cornell University scientists. Mikahil Bodganov, Ph.D., was awarded
$74,000 for a study concerning oxidative DNA damage and repair, and $27,000 went
to Flint Beal, M.D. for a project which delves into matrix matalloproteinases as
they relate to cell survival and death in ALS. Saud Sadiq, M.D., St.
Luke's-Roosevelt Hospital, received $40,000 for an investigation of neuronal
proteins involved in the development of ALS.
The remainder of the research funded by the Chapter includes $80,000 from
Michael Zaslow's ZazAngels Fund to Martina Berger, Ph.D., University of
California, Irvine, in collaboration with the Centers for Disease Control and
Prevention, to examine the possible role of viral factors in ALS; $35,000
from The Andersens' Fight Back campaign for a genetic linkage analysis to
identify new gene defects in familial ALS conducted by Cheng Zhang, M.D., Sun
Yat-sen University, China, in collaboration with Robert H. Brown, Jr., D.Phil.,
M.D., Massachusetts General Hospital; and $5,000 from the Adele Zinberg, M.D.,
ALS Research Fund, to David Fink, M.D., University of Pittsburgh, for working to
develop a system that will deliver a therapeutic gene to protect motor nerve
cells from premature death.
Your [Greater NY] Chapter supports leading researchers and their most promising
projects. In short, with your continuing contributions, we're funding the
way to a cure!
Reversible ALS-like Disorder in
HIV Infection;
ALSA-Funded Follow-up Study Being Conducted at Beth Israel
Two studies published in the September [2001] issue of the journal Neurology* describe
an ALS-like syndrome associated with HIV that is responsive to antiretroviral
therapy. A viral mechanism for ALS has long been hypothesized and publications
describing the presence of certain viruses in post-mortem spinal cord tissue of
ALS patients, but not in control tissue, have rekindled an interest in
understanding the possible link between viruses and ALS
The two studies report seven cases of ALS-like syndromes accompanying HIV
infection. In one study led by Dr. Antoine Moulignier of Adolphe de Rothschild
Foundation in Paris, six cases of ALS-like syndromes among 1700 HIV patients
with neurologic manifestations were identified. All six of these patients either
stabilized or partially recovered from the ALS-like
syndromes after starting antiretroviral therapy. The other study was led by
Daniel J.L. MacGowan, MD, MRCPI, Beth Israel Medical Center, New York.
In the article, authored by McGowan, Stephen Scelsa, MD and M. Waldron, MD, the
researchers describe a patient diagnosed with HIV with a progressive ALS-like
disease. Antiviral therapy led to complete and sustained recovery from ALS-like
symptoms.
These intriguing findings warrant further investigation of how viruses may play
a role in ALS. Determining how antiretroviral agents may be involved in
reversing the ALS-like symptoms may lead to a better understanding of ALS, said
Lucie Bruijn, PhD, Science Director of The ALS Association. In fact, she adds,
ALSA is funding a follow-up study led by Dr. Scelsa at Beth Israel Medical
Center.
Funding support for the Scelsa grant comes from The ALS Association Greater
New York Chapter with specific support from ZazAngels and the Adele
Zinberg, M.D., ALS Research Fund. Beth Israel Medical Center is a certified
ALSA Center supported by the Greater New York Chapter.
The study, which begins October 1, is a placebo-controlled investigation with 46
patients to assess whether Indinavir, a protease inhibitor used in the treatment
of HIV infection, slows the progression of ALS in patients that are negative for
HIV and to investigate the possible mechanism of the compound. Indinavir
is similar to one of the compounds in the cocktail of
drugs that showed reversal of symptoms in the patients with ALS-like symptoms
and positive for HIV. Indinavir, rather than the protease inhibitor used in the
published study, has been chosen for the clinical trial as it best penetrates
the cerebrospinal fluid (fluid which surrounds the brain and spinal cord). One
mechanism proposed by Dr. Scelsa is that
the protease inhibitors may be effective in preventing caspase-dependent
apoptosis (programmed cell death) of motor neurons. Caspases are a group of
enzymes that are involved in regulating apoptosis. Protease inhibitors inhibit
apoptosis of CD4 lymphocytes (a white blood cell) in culture although there is
currently no data to indicate that they inhibit apoptosis in motor neurons.
These HIV-related cases of an ALS-like syndrome suggest that a viral mechanism
may underlie some forms of ALS, however, there is currently no evidence that
reversal of symptoms with treatment of these antiviral agents will be effective
in ALS patients that are negative for HIV. At this point HIV treatment for ALS
patients is not recommended, as there are
no animal or human studies to support their use in ALS patients who do not have
HIV infection. These medications, unfortunately, may have serious adverse
effects.
*Neurology 2001; 57: 945-946; 995-1001; 1094-1097.
------------------------
Questions and Answers
*What is the relationship between ALS and HIV?
There is currently no known relationship between ALS and HIV. What has been
observed in the seven patients described in the Neurology articles is an ALS-like
syndrome accompanying HIV that reverses or is improved with treatment for the
HIV infection.
Further investigation into what causes ALS-like symptoms in a sub-set of HIV
patients may provide important clues to understand the causes of motor neuron
death in ALS. The current study led by Dr. Scelsa, and funded by ALSA, will
determine whether a drug, Indinavir, similar to the protease inhibitor that was
used in the cocktail HIV therapy will benefit people with ALS that are negative
for HIV.
*How can I get more information about or enroll in the new Indinavir clinical
trial?
Information about the Indinavir clinical trial and patient enrollment can be
found on ALSA' s web site at http://alsa.org/research/drugdev21.cfm.
*Since Indinavir is an FDA-approved drug for HIV, can I take it or other HIV
medications now?
Dr. Scelsa, investigator of the Indinavir study, offers these considerations: At
this point patients with ALS should not be started on HIV treatment, as there
are no animal or human studies to support its use in ALS patients who do not
have HIV infection. Our current study is only exploring a hypothesis spurred by
the encouraging outcome in the patient report cited above. These medications,
unfortunately, may have serious adverse effects. It is best to discuss this
question with your own physician.
*What are the side effects of Indinavir?
Common side effects of Indinavir are mostly gastrointestinal, including
diarrhea, abdominal discomfort, nausea and vomiting. More serious, but less
common side effects include: kidney stones (approx. 5%), kidney failure (rare),
liver abnormalities or failure (rare), anemia, rash, diabetes, fat
redistribution and others. The following drugs should not be taken with
Indinavir: seldane, versed, orap (pimozide), propulsid, halcion, rifampin and
ergot medications (i.e. cafergot). These drugs may
cause life-threatening problems such as irregular heartbeats or excessive
sedation.
For further information:
Lucie Bruijn, PhD, Science Director, The ALSA Association, for science questions
lucie@alsa-national.org
(203) 453-4580
ALSA's National Office Patient Services Department for clinical questions
alsinfo@alsa-national.org
(800) 782-4747
Copyright © 1999 by Michael
Zaslow's ZazAngels. All rights reserved.
01/04/06 05:14:40 PM