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April 16, 2003
Consortium Undertakes Collaborative Initiative
to Screen FDA-approved Compounds for Treatment of ALS in Mice
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 assays (models).
"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 disease
progression.
"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 trials."
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.
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