Great news from the Hope Center – Tim Miller, MD, PhD, who is one of the nation’s foremost researchers on ALS, developed an experimental drug (tofersen) for a form of genetic ALS that is now moving into a Phase 3 clinical trial. Phase 3 is designed to further evaluate the safety and efficiency of the new drug. Dr. Miller is the director of the Christopher Wells Hobler Laboratory for ALS Research at the Hope Center, and he received our Spirit of Hope Award in 2018.

An experimental drug for a rare, inherited form of amyotrophic lateral sclerosis (ALS) has shown promise in a phase 1/phase 2 clinical trial conducted at Washington University School of Medicine in St. Louis, Massachusetts General Hospital in Boston and other sites around the world and sponsored by the pharmaceutical company Biogen Inc.

The trial indicated that the experimental drug, known as tofersen, shows evidence of safety that warrants further investigation and lowers levels of a disease-causing protein in people with a type of amyotrophic lateral sclerosis, or ALS, caused by mutations in the gene SOD1.

The results of the study, published July 9 in The New England Journal of Medicine, have led to the launch of a phase 3 clinical trial to further evaluate the safety and efficacy of tofersen.

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