Edaravone for amyotrophic lateral sclerosis: barriers to access and lifeboat ethics

A myotrophic lateral sclerosis (ALS) is a fatal neurodegenerative condition that results in progressive paralysis of limb and bulbar muscles, with an average life expectancy of 3-5 years after diagnosis. Until 2017, riluzole was the only approved medication for patients with ALS, offering a limited...

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Bibliographic Details
Published inCanadian Medical Association journal (CMAJ) Vol. 192; no. 12; pp. E319 - E320
Main Authors Breiner, Ari, Zinman, Lorne, Bourque, Pierre R
Format Journal Article
LanguageEnglish
Published Canada Joule Inc 23.03.2020
CMA Impact, Inc
Subjects
Access control
Amyotrophic lateral sclerosis
Analysis
Clinical trials
Distribution
Dosage and administration
Drug dispensing
Drug therapy
Edaravone
Ethical aspects
FDA approval
Free radicals
Hospitals
Medical ethics
Medical research
Neurology
Review boards
Stroke
Canada
United States > US
Japan
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Summary:A myotrophic lateral sclerosis (ALS) is a fatal neurodegenerative condition that results in progressive paralysis of limb and bulbar muscles, with an average life expectancy of 3-5 years after diagnosis. Until 2017, riluzole was the only approved medication for patients with ALS, offering a limited survival benefit of about 3-5 months. Since the introduction of riluzole in 1994, numerous clinical trials of proposed treatments for ALS have failed. Edaravone is a free radical scavenger that has been available in Japan since 2003 for the treatment of acute stroke,3 but also showed promise in mouse models for ALS. An initial randomized controlled trial (RCT) in patients with ALS did not show benefit for all patients, although a subgroup of patients in the earliest disease phase did benefit. Access to edaravone has varied among countries. Japan, South Korea, the US and Switzerland have approved the medication with relatively broad access.
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ISSN:0820-3946
1488-2329
DOI:10.1503/cmaj.191236