CALABASAS, Calif., April 16 /PRNewswire/ -- A pilot study has been
launched to test three FDA-approved compounds for their effect in treating ALS
to help determine whether clinical trials are warranted. The two-phase study
is a collaboration of The ALS Association (ALSA), The Robert Packard Center
for ALS Research at Johns Hopkins and Project A.L.S.
The unprecedented effort to screen drugs in the mutant G93A SOD1 mouse
follows a 2002 study in which ALSA partnered with The National Institute of
Neurological Disorders and Stroke (NINDS) to screen a set of over 1000
FDA-approved compounds, termed the NINDS collection, in neurodegeneration
"This is an important follow-up to the previous study as it will show
whether the compounds with an apparent effect in those assays will increase
the lifespan of the ALS mouse model. This investigation will provide strong
support for moving to clinical trials," said Dr. Lucie Bruijn, science
director and vice president of The ALS Association.
"Project A.L.S. is pleased to join forces in screening these promising
drugs aggressively and thoroughly, with the necessary scientific
oversight -- which is crucial. Our goal is to work together intensively, as
committed organizations, build on the initial findings of the NINDS screening,
and identify medicine that will make a difference to ALS patients," said
Jenifer Estess, chief executive officer of Project A.L.S. Estess has ALS.
The three test compounds chosen for this animal study are among those that
show a significant effect in assays representing various potential disease
mechanisms in ALS (models in a dish).
-- Phase 1. Three test compounds and one control compound will be
screened in mice beginning at five weeks of age.
-- Phase 2. Compounds that show a significant effect will be tested in
mice beginning at 12 weeks when animals begin to show signs of
"We were eager to get on with this next phase of testing," says Dr.
Jeffrey Rothstein, who directs the Packard Center. "It's certainly the best
way we know to determine whether these drugs should move on to clinical
The ALS Association's Greater New York and Bay Area Chapters along with
The Robert Packard Center for ALS Research at Johns Hopkins and Project A.L.S.
are providing support for the study, which will be conducted by New York-based
PsychoGenics Inc, an independent testing facility.
The ALS Association (ALSA) is the only national not-for-profit voluntary
health organization dedicated solely to the fight against ALS. Its mission is
to find a cure for and improve living with ALS.
SOURCE The ALS Association