Dexpramipexole Safe but Not Effective for ALS

Amyotrophic lateral sclerosis (ALS) is a progressive, ultimately fatal neurodegenerative disease for which effective treatments are almost entirely lacking. Based on a mechanism of action of enhancing mitochondrial function (a biological mechanism that has been implicated in ALS), evidence of a neuroprotective effect in in vitro assays, improved survival and retention of motor function in in vivo models of ALS, and promising phase II human data, researchers undertook a manufacturer-sponsored, premarket clinical trial of dexpramipexole in patients with ALS. EMPOWER was a randomized, double-blind, placebo-controlled, phase III trial conducted across 81 academic medical centers in 11 countries. The trial enrolled 943 participants, randomized 1…