Investigational drug for genetic form of ALS improves disease’s molecular signs

Washington University School of Medicine in St. Louis • September 21, 2022September 28, 2022

Timothy Miller, MD, PhD, talks with an amyotrophic lateral sclerosis (ALS) patient at Washington University School of Medicine in St. Louis. Miller led an international phase 3 clinical trial for a rare, inherited form of ALS. The trial, sponsored by the pharmaceutical company Biogen, showed that an investigational drug, known as tofersen, reduced molecular signs of the fatal disease, but at six months did not improve motor control and muscle strength. However, Miller and colleagues found evidence that longer-term use of the drug may help stabilize muscle strength and control.

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 the six-month mark, 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.