In the first stage of an ongoing phase 2 clinical trial to test the experimental therapy CK-2017357 in amyotrophic lateral sclerosis (ALS), the drug was found to be safe and well-tolerated.
Participants who received the highest dose showed improved scores on tests that measure motor and breathing function, muscle strength and fatigue.
CK-2017357 is designed to work by increasing muscle sensitivity to calcium, which in turn is expected to increase skeletal muscle force and improve muscle function.
The drug is the lead therapeutic candidate for ALS from South San Francisco biotechnology companyCytokinetics, and was granted “orphan drug” status by the U.S. Food and Drug Administration (FDA) in March 2010. (Orphan drug status provides financial incentives for the development of drugs for rare diseases.)
The phase 2 trial results were reported Nov. 30, 2011, at the 22nd International Symposium on ALS/MND in Sydney, Australia, by Jeremy Shefner, professor and chair of the department of neurology at Upstate Medical University, State University of New York in Syracuse. (Shefner directs the MDA/ALS Center at that institution, but MDA is not involved with this study.)