Research highlight

Potential therapy for ALS

Nature Medicine

November 21, 2011

A drug, dexpraminpexoile, may have therapeutic potential for amyotrophic lateral sclerosis (ALS), in patients, reports a preliminary study published this month in Nature Medicine. ALS is a neurodegenerative disease that primarily affects motor neurons throughout the body, leading to progressive muscle weakness and eventually death. Though the cause is not fully known, mitrochondrial dysfunction has been shown to contribute to the progression of ALS. Present therapies show only modest benefit, helping to extend patients’ lifespan, but not helping with muscle weakness or loss of function. In a small placebo-controlled trial, Valentin Gribkoff and colleagues found that dexpramipexole, a drug with as-yet unknown function, may have some preliminary indications of a clinical effect in ALS patients. Further research, with a bigger patient group, is needed to confirm these results.

doi: 10.1038/nm.2579

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