An investigational drug developed to treat a rare, inherited form of amyotrophic lateral sclerosis (ALS) reduced molecular signs of the fatal, paralyzing disease and curbed neurodegeneration—but at six months, the drug did not improve motor control and muscle strength, according to results from a phase 3 clinical trial led by researchers at Washington University School of Medicine in St. Louis.

However, the researchers found evidence that longer-term use of the drug may help stabilize muscle strength and control, a finding that the researchers called encouraging. The trial was sponsored by the pharmaceutical company Biogen, the maker of the experimental drug. The data are published Sept. 22 in The New England Journal of Medicine.

Participants in the trial carry mutations in a gene called SOD1that create a misfolded version of a protein by the same name, which leads to ALS, also known as Lou Gehrig's disease.

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