Results from the Phase 3 trial of edaravone (MCI-186) in amyotrophic lateral sclerosis (ALS) were presented by Mitsubishi Tanabe Pharma Corporation at the 68th Annual Meeting of the American Academy of Neurology held April 15-21 in Vancouver, British Columbia.
The Phase 3 MCI-186-19 clinical study has reportedly met its primary efficacy endpoint of mean change in the ALS Functional Rating Scale-Revised (ALSFRS-R) at 24 weeks, with adverse effects occurring at similar rates to placebo.
Edaravone had been granted Orphan Drug status by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in May 2015, but it is not yet currently approved for use in either the U.S. or in Europe.
Edaravone has only been studied in Japanese research institutions for the treatment of ALS — no American research institutions have investigated the effects of MCI-186 in ALS. Nonetheless, two studies (see below) showed positive results in oxidative stress relief, a key factor in ALS onset and progression. Oxidative stress is considered to be an imbalance between the production of free radicals (single reactive electrons) and the body’s ability to react, counteract, or detoxify their consequent harmful effects. ALS patients are found to have consistently increased oxidative stress biomarkers.
“Helping patients enjoy healthier lives is what inspires and motivates us. We recognize the challenges patients and their families face with rare medical conditions such as ALS and are encouraged by these results,” said Dr. Joseph M. Palumbo, M.D., vice president and head of clinical research for American Mitsubishi Tanabe Pharma, in a press release.
For additional reference, the studies on oxidative stress relief are titled “Increased oxidative stress in patients with amyotrophic lateral sclerosis and the effect of edaravone administration,” and “Potential Regulators of Sporadic ALS Development and Alternative Therapeutic Options.”
Researchers have succeeded in using an imaging technique to record changes and inflammation in the brain of a person with amyotrophic lateral sclerosis (ALS), using a potential biomarker of inflammation. The research, conducted at The Neurological Clinical Research Institute at Massachusetts General Hospital, is part of the TRACK ALS project to identify imaging markers of ALS, so as to facilitate early diagnosis and develop new therapies.