New Trial Drug Combination Shows Promise for Slowing Progression of ALS

A new drug combination tested in a multisite human clinical trial shows promise for slowing the progression of ALS, often called Lou Gehrig’s disease, researchers at the University of Florida and partner institutions reported in The New England Journal of Medicine.

The oral drug, a combination of sodium phenylbutyrate and taurursodiol, was tested in the Phase 2 CENTAUR trial, a placebo-controlled, randomized, double-blind trial of 137 ALS patients within 18 months of the onset of symptoms. The compounds, which previously were found to reduce neuronal death in preclinical models, resulted in a statistically significant slowing of disease progression in the human trial, according to the study.

The CENTAUR trial was led by Massachusetts General Hospital/Harvard Medical School, and UF was the No. 2 enroller of patients, said James Wymer, M.D., a UF professor of neurology who co-authored the study.

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