Isis Pharmaceuticals has initiated Phase I studies of ISIS-SOD1Rx to treat patients with an inherited aggressive form of Lou Gehrig's disease, also known as familial amyotrophic lateral sclerosis (ALS).
Approximately 20% of all familial ALS cases are caused by a mutant form of superoxide dismutase, or SOD1, which the new antisense drug is designed to inhibit.
The ALS Association and the Muscular Dystrophy Association are providing funding for the development of ISIS-SOD1Rx.
Isis Pharmaceuticals senior vice-president of research Frank Bennett said that this study is the first step in demonstrating the applicability of antisense drugs to treat severe neurodegenerative diseases.
"This is our first antisense drug to enter clinical trials to treat a neurodegenerative disease and our first antisense drug to be administered directly into the central nervous system," Bennett said.
The Phase I study of ISIS-SOD1Rx will take the shape of a placebo-controlled, dose-escalation study designed to assess the safety, tolerability and pharmacokinetic profile of ISIS-SOD1Rx.
In December 2007, the US Food and Drug Administration granted ISIS-SOD1Rx orphan drug designation for the treatment of ALS.