Highlighting Genetic Discoveries and Therapeutic Developments in ALS: Matthew B. Harms, MD

WATCH TIME: 5 minutes

“There's a tremendous amount of enthusiasm and excitement in ALS at this time. We have more phase 3 trials ongoing, and expectations for some important phase 2 studies to release results over the next 6 to 9 months. We know of 80 drugs that are in clinical development and being brought forward to target these very important genetic molecules that we know are playing a role in ALS.”

In April 2023, the FDA approved Biogen’s tofersen (Qalsody), a therapeutic designed to treat patients with SOD1 mutation-mediated amyotrophic lateral sclerosis (ALS), a genetic form of ALS for which there are no other approved products^.1 The decision was supported by the treatment's demonstrated reduction of plasma neurofilament lights, a blood-based biomarker of axonal injury and neurodegeneration. Since its approval, the field of ALS has had many ongoing developments for treatments that target genetic forms of the disease.

At the 2024 Muscular Dystrophy Association (MDA)Clinical and Scientific Conference in Orlando, Florida, held March 3-6, Matthew B. Harms, MD, medical advisor and care center director at Muscular Dystrophy Association (MDA), will chair a track session on genetic discovery, and mechanisms and therapeutic development in ALS. This track will cover topics including rare variant analyses validating known ALS genes, translation of genetic discoveries into therapies for ALS, UNC13A cryptic exon skipping antisense oligonucleotide as a treatment for ALS, and an investigation into ALS caused by mutant KIF5A.

Harms, who also serves as associate professor of neurology at Columbia University Irving Medical Center - Eleanor and Lou Gehrig ALS Center, sat down with NeurologyLive® prior to the meeting to discuss how the MDA's mission has expanded to include ALS care and research. He also talked about the role genetic understanding has played in propelling therapy development for ALS in the last 5 to 10 years. In addition, Harms spoke about how the recent approval of tofersen for ALS provides a blueprint for future drug development in the field.

Registration for the 2024 MDA Conference is now open! The meeting is set to be held at the Hilton Orlando, in Orlando, Florida, from March 3 to 6, 2024. To register and for more information, head to www.mdaconference.org

REFERENCES
1. FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene. News release. FDA. April 25, 2023. Accessed February 20, 2024. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-amyotrophic-lateral-sclerosis-associated-mutation-sod1-gene