Riluzole, the first treatment to alter the course of ALS, was approved by the FDA in late 1995. This antiglutamate drug was shown scientifically to prolong the life of persons with ALS by at least a few months.
Lithium in patients with amyotrophic lateral sclerosis (LiCALS): a phase 3 multicentre, randomised, double-blind, placebo-controlled trial
From the UKMND-LiCALS Study Group
Summary article posted online in The Lancet Neurology, February 27, 2013 •
Lithium has neuroprotective effects in cell and animal models of amyotrophic lateral sclerosis (ALS), and a small pilot study in patients with ALS showed a [...]
“Some may look at recent failures and be discouraged,” wrote Richard Bedlack of the Duke ALS Clinic in Durham, North Carolina, in an e-mail to Alzforum. Thanks to advances in genetics and biomarkers, and a list of potential target pathways, “I think this is a time of unprecedented hope and excitement in ALS research”
While some progress has been made in the treatment of ALS, combination therapies with drugs that target other pathways, alongside stem cell technology, are sorely needed
Cytokinetics Announces Opening of BENEFIT-ALS, a Phase IIb Clinical Trial of Tirasemtiv (CK-2017357)
“Patients who suffer from this devastating disease are in critical need of a novel therapy that addresses the functional deficits that limit their activities of daily living,” stated Jeremy M. Shefner, MD, PhD, Professor and Chair of the Department of Neurology at the Upstate Medical University at the State University of New York and Principal Investigator of BENEFIT-ALS. “If successful, this novel mechanism therapy could improve the lives of many patients living with ALS.”
Current treatment of ALS is aimed at symptomatic relief, prevention of complications and maintenance of maximum optimal function and optimal quality of life.
Results of the first stage showed that receiving dexpramipexole appeared to slow the progression of symptoms measured both by the ALS Functional Rating Scale and by pulmonary capacity. The second stage had similar results, with slower disease progression and a reduced risk of death in participants receiving the higher dosage.