TRIALS OF TREATMENTS FOR ALS
FOCUS OF MDA CONFERENCE
TUCSON, Ariz., May 22, 2003 — More than 150 experts on
amyotrophic
lateral
sclerosis (ALS, or Lou Gehrig's disease) will convene next month to
set
plans for testing new treatments against the deadly neuromuscular disorder.
The conference is organized by the
ALS
Division of the Muscular Dystrophy Association, which has led the
fight to conquer the disease for 50 years.
A faculty of some 50 ALS experts from the United States, England, Canada
and France will present the latest information on clinical research on
the
disease, which affects some 30,000 Americans. The speakers include
directors of several of
MDA's
29 ALS centers.
ALS attacks the body's muscle-controlling nerve cells, typically leading
to
paralysis and death within three to five years of diagnosis. It's best
known for taking Gehrig's life in 1941.
To speed the development of new treatments and to mark the legendary New
York Yankee's 100th birthday on June 19, MDA is convening the conference,
titled ALS Clinical Trials: The Challenge of the Next Century. The
conference, to be held in Tarrytown, N.Y., June 13-15, will focus on ways
to improve the design of human ALS trials.
Today, as in Gehrig's day, there's no cure for ALS. Dozens of therapies
have shown promising effects in mice with the disease, only to yield
disappointing results in human trials. A single drug, riluzole, is approved
for use against ALS by the Food and Drug Administration (FDA), but it
prolongs survival by only a few months.
The mice most commonly studied in ALS have a genetic form of the disease,
but humans probably have various forms not suited to a "one treatment
fits
all" approach. MDA-funded researchers are currently involved in trials
of
several potential ALS treatments, including the arthritis drug Celebrex
and
the antibiotic minocycline.
At the conference, ALS experts will discuss how to test multiple drugs
in
combination, and explore ways to detect subtle changes in nerve cells
that
might forewarn of the disease, said conference co-director Hiroshi
Mitsumoto, medical director of the Eleanor and Lou Gehrig MDA/ALS Center
at
Columbia University in New York, which is hosting the conference.
"We'll also have representation from officials from the FDA
and the
National Institute of Neurological Disorders and Stroke, and experts on
human trials for other neurological diseases, AIDS, cancer and heart
disease," Mitsumoto said. "Our goal is to apply the experiences
of a vast
array of experts to the task of conquering this extremely challenging
disease."
The conferees will examine the pros and cons of establishing a national
ALS
study group to coordinate upcoming trials, a task that's so far been left
to regional groups.
In 2002, MDA devoted $13.8 million to ALS research and services, and this
year, that amount will increase to $16.6 million. MDA's ALS Division also
provides a steady stream of updated ALS information, which can be seen
online at
www.als-mda.org.
Mitsumoto and conference co-director Serge Przedborski of the Gehrig center
are members of MDA's Medical Advisory Committee. The conference is partly
funded by the National Institutes of Health and the NIH Office of Rare
Diseases.
ALS is one of more than 40 neuromuscular diseases covered by MDA. The
Association's programs are funded almost entirely by individual private
contributors.
Exclusive reports on ALS Clinical Trials: The Challenge of the Next Century,
from the international gathering of experts on
amyotrophic
lateral sclerosis in Tarrytown, N.Y., June 13-15:
