Safety and Efficacy of Rho-Associated Kinase Inhibition, a Novel Target in Amyotrophic Lateral Sclerosis

Preclinical models of neurodegeneration and amyotrophic lateral sclerosis (ALS) suggest that Rho-associated protein kinase (ROCK) inhibition reduces neuronal degeneration, modulates inflammation, and promotes axonal regeneration. This multicenter, double-blind, placebo-controlled study investigated the safety and efficacy of fasudil, a ROCK inhibitor, in 120 patients with at least probable ALS randomized to 30 mg or 60 mg of intravenous fasudil or placebo daily for 20 days. Participants had a slow vital capacity (SVC) >65% of normal and continued riluzole and edaravone use. The coprimary endpoints were safety to day 180 and tolerability during treatment.

The study met the coprimary endpoints, with no treatment-related serious events at 180 d…