Consortium Undertakes Collaborative Initiative to Screen FDA-Approved Compounds for Treatment of ALS in Mice

Apr 16, 2003, 01:00 ET from The ALS Association

    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
 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
     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