Pilot Study to Screen FDA-approved Compounds in the SOD1 Mouse Model
PsychoGenics Inc. New York

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.

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.