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Research Highlights
ALS clinical trial follows promising animal study
March 21, 2005
From VA Research Currents Vol.5, No.3/Mar.2005
 Patients with amyotrophic lateral
sclerosis (ALS), or Lou Gehrig's
disease, will be recruited starting in
late March for a VA-funded nationwide
study testing the safety of sodium
phenylbutyrate as a life-extending
treatment. The drug, which has been used for years
as a cancer therapy, significantly extended the lives of ALS mice in a recent study.
ALS involves the destruction of
cells in the brain and spinal cord that
control muscle movement, resulting in
gradual muscle wasting and loss of
movement. Only 20 percent of ALS
patients live longer than five years
after onset. The disease usually strikes
people between ages 40 and 70. There
is no cure, but several potential new
treatments are being investigated,
including sodium phenylbutyrate, which
is thought to work by correcting the
"death messages" transmitted to the
neurons affected in ALS.
"We do not have a cure, but we do
have a therapy that we hope will slow
the disease," said Robert J. Ferrante,
PhD, MSc, a neurology researcher at
the Edith Nourse Rogers Memorial
Veterans Hospital in Bedford, Mass.
Ferrante and two colleagues-Dr.
Merit Cudkowicz of VA and Massachusetts
General Hospital (MGH), and
Dr. Robert Brown Jr. of MGH and
Harvard Medical School-conducted
the animal study and are coordinating
the new clinical trial, to take place at
six VA sites and two non-VA sites
nationwide. The effort is part of a
"bench to bedside" translational
program focused on motor-neuron
disease that Ferrante and colleagues
have built over the past 15 years.
The only approved drug for ALS is
riluzole, which Ferrante said offers
only "marginal benefit." He said
support for the sodium phenylbutyrate
trial reflects the increasing willingness
of VA and other funding agencies to
invest in translational research aimed
at fast-tracking potentially valuable
new treatments.
The VA sites that will host the ALS
trial are Bedford, Houston, Lexington,
KY; Syracuse, Durham and Iowa City.
The non-VA sites are MGH and Johns
Hopkins University in Baltimore. For
more information call (781) 687-2884.
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