Researchers report experimental drug may prolong survival in ALS patients

By Todd Taylor

A new trial drug combination that previously showed promise for slowing the progression of ALS symptoms now also shows potential to prolong survival of ALS patients by more than six months, researchers report in Muscle and Nerve.

Doctor James Wymer
Dr. James Wymer

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

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects motor neurons and, over time, the ability to move, speak, eat and breathe. There is currently no cure.

The oral drug, Amylyx Pharmaceuticals’ AMX0035, is a combination of sodium phenylbutyrate and taurursodiol. It was tested in a placebo-controlled, randomized, double-blind trial of 137 ALS patients within 18 months of onset of symptoms. The compounds, which previously were found to reduce neuronal death in preclinical models, resulted in statistically significant slowing of disease progression in the human trial, according to the first study in the New England Journal of Medicine. Ninety of the trial’s participants then took part in an open-label extension trial.

The subsequent survival analysis spanned nearly three years and included 135 of the original participants, regardless of whether they participated in the open-label extension. The analysis showed that those originally randomized to receive the drug lived for 6.5 months longer than those originally randomized to receive the placebo — a median survival of 25 months compared to 18.5.

Read the article in Muscle and Nerve.

Read a story about the study in The New York Times.