Advancing Therapeutic Knowledge of Genetic Influence in ALS: Matthew B. Harms, MD

WATCH TIME: 3 minutes

"We think it’s time to reevaluate and go back to the basics—where’s the genetics telling us to go, and what’s it highlighting as therapeutic targets?"

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that primarily affects motor neurons, leading to muscle weakness, paralysis, and eventually death. While the exact causes of ALS remain unclear, genetics play an important role in both familial and sporadic forms of the disease. To date, some of the most notable and common ALS-associated genes include C9orf72, SOD1, TAR DNA-binding protein 43, and FUS, while other genes, such as VCP, OPTN, and TBK1, have also been implicated, though less frequently.

At the upcoming 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held March 16-19, in Dallas, Texas, Matthew B. Harms, MD, will chair a track focused on ALS and frontotemporal dementia, two neurodegenerative disorders that share common genetic, pathological, and clinical features. In that track session, Harms, an associate professor of neurology at Columbia University Irving Medical Center, an MDA Care Center, will give a talk on leveraging genetics for ALS therapeutics. To date, tofersen (Qalsody; Biogen), an antisense oligonucleotide, is the only FDA-approved treatment for SOD1-ALS, considered one of the most common familial form of the disease.

Prior to the meeting, NeurologyLive^® sat down with Harms to discuss the details behind the track session, and why clinicians should consider attending. He spoke about how the session will highlight the latest work on genetic risk factors in ALS, including the use of big data to identify new targets, the role of sphingolipid metabolism in ALS, and drug repurposing efforts. Harms noted that key speakers will present on efforts to aggregate global ALS genomic data to uncover actionable genetic variants, as well as new insights into how genetic abnormalities lead to ALS and potential therapeutic approaches.

Registration for the 2025 MDA Conference is now open! The meeting is set to be held at the Hilton Anatole, Dallas, Texas, from March 16 to 19, 2025. To register and for more information, head to www.mdaconference.org