“Some may look at recent failures and be discouraged,” wrote Richard Bedlack of the Duke ALS Clinic in Durham, North Carolina, in an e-mail to Alzforum. Thanks to advances in genetics and biomarkers, and a list of potential target pathways, “I think this is a time of unprecedented hope and excitement in ALS research”
Cytokinetics Announces Opening of BENEFIT-ALS, a Phase IIb Clinical Trial of Tirasemtiv (CK-2017357)
“Patients who suffer from this devastating disease are in critical need of a novel therapy that addresses the functional deficits that limit their activities of daily living,” stated Jeremy M. Shefner, MD, PhD, Professor and Chair of the Department of Neurology at the Upstate Medical University at the State University of New York and Principal Investigator of BENEFIT-ALS. “If successful, this novel mechanism therapy could improve the lives of many patients living with ALS.”
South San Francisco biotechnology company Cytokinetics announced Sept. 10, 2012, that it plans to conduct a new phase 2b clinical trial of its oral drug tirasemtiv (formerly CK-2017357) in people with amyotrophic lateral sclerosis (ALS). The trial is set to launch later this year.
Both patients and investigators perceived a dose-dependent benefit of CK-2017357 as measured by global impression of change. Maximum voluntary ventilation and submaximal handgrip endurance also improved.
Parone and Da Cruz concluded that muscle cells could not be the most crucial source of ALS pathology.
CK-2017357 previously demonstrated potentially clinically relevant pharmacodynamic effects in a completed Phase IIa Evidence of Effect clinical trial in ALS patients.
Some trial participants who were treated with the highest dose showed measurable improvement in motor function and muscle strength.