Clinical efficacy of edaravone for the treatment of amyotrophic lateral sclerosis. (3rd May 2017)

• Record Type: Journal Article
• Authors: Sawada, Hideyuki
• Is Part Of: Expert opinion on pharmacotherapy. Volume 18:Number 7(2017)
• Page Start: 735
• Page End: 738
• Publication Date: 2017-05-03
• Subjects: Oxidative stress -- nitric oxide -- motor neuron -- clinical trial -- radical scavenger; Chemotherapy -- Periodicals; 615.5805
• DOI: 10.1080/14656566.2017.1319937
• Languages: English
• ISSNs: 1465-6566

Abstract

ABSTRACT: Introduction : Amyotrophic lateral sclerosis (ALS) is a progressive, fatal, neurodegenerative disease. Although the pathogenesis remains unresolved, oxidative stress is known to play a pivotal role. Edaravone works in the central nervous system as a potent scavenger of oxygen radicals. In ALS mouse models, edaravone suppresses motor functional decline and nitration of tyrosine residues in the cerebrospinal fluid. Areas covered : Three clinical trials, one phase II open-label trial, and two phase III placebo-control randomized trials were reviewed. In all trials, the primary outcome measure was the changes in scores on the revised ALS functional rating scale (ALSFRS-R) to evaluate motor function of patients. Expert opinion : The phase II open label trial suggested that edaravone is safe and effective in ALS, markedly reducing 3-nitrotyrosine levels in the cerebrospinal fluid. One of the two randomized controlled trials showed beneficial effects in ALSFRS-R, although the differences were not significant. The last trial demonstrated that edaravone provided significant efficacy in ALSFRS-R scores over 24 weeks where concomitant use of riluzole was permitted. Eligibility was restricted to patients with a relatively short disease duration and preserved vital capacity. Therefore, combination therapy with edaravone and riluzole should be considered earlier.