By Michelle Koidin Jaffee
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 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 study.
ALS 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 CENTAUR trial was led by Massachusetts General Hospital/Harvard Medical School. The UF arm was led by James Wymer, M.D., a UF professor of neurology who co-authored the study.
The drug, Amylyx Pharmaceuticals’ AMX0035, resulted in slower functional decline as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scaled Revised, or ALSFRS-R, over 24 weeks compared with the placebo group, the investigators reported. The ALSFRS-R is an assessment of bulbar, fine motor, gross motor and breathing function.
The drug “had a 25% slowing in the rate of progression for ALS patients,” Wymer said.
The researchers wrote that larger and longer clinical trials are needed to study the efficacy and safety of the drug in ALS patients.
Nonetheless, the study’s findings were significant, Wymer said.
“The basic science over the last 50 years has been working to try to understand some of the underlying mechanisms, and we’re starting to see the results of that in clinical research with medications that will be directed toward specific pathways involved in ALS,” he said. “I’ve been doing this work for 25 years, and this is the most exciting time that I’ve seen for collaboration, for research options, for the chance to treat an untreatable disease.”