Drug Plan Hatched by Brown Undergrads Blossoms into Potentially Helpful Therapy for Amyotrophic Lateral Sclerosis (ALS) (Lou Gehrig’s Disease); Experimental Two-Drug Combination Slows Disease Progression in Small Study Published in NEJM

An experimental combinational medication slows the progression of the neurodegenerative disease called amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease (named for baseball star (photo) who died of ALS at age 37, according to recently released results from a clinical trial run by investigators at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) and Amylyx Pharmaceuticals, Inc., the company that manufactures the medication. The findings, reported in the September 3, 2020 issue of the New England Journal of Medicine, offer hope that a treatment may one day be available for patients with ALS, a fatal condition, with no known cure, that attacks the nerve cells in the brain and the spinal cord to progressively hinder individuals’ ability to move, speak, eat, and even breathe. Called AMX0035, the oral medication is a combination of two drugs, sodium phenylbutyrate and taurursodiol, that each target a different cell component important for protecting against nerve cell death. AMX0035 targets endoplasmic reticulum- and mitochondrial-dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases. The NEJM article, titled “Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis” (https://www.nejm.org/doi/full/10.1056/NEJMoa1916945?query=featured_home), is accompanied by an editorial titled “Incremental Gains in the Battle Against ALS” (https://www.nejm.org/doi/full/10.1056/NEJMe2021144?query=recirc_curatedRelated_article). In the so-called “CENTAUR” clinical trial, 137 participants with ALS were randomized in a two-to-one ratio to receive AMX0035 or placebo.