ALS Advances and Hot Topics for MDA 2023: Matthew B. Harms, MD

“I think what's not as commonly recognized is that some of these therapies have a good chance of working in nongenetic ALS, or in ALS where we haven't yet found a gene mutation, because it's addressing a root factor that's shared by most ALS patients as opposed to this specific gene mutation in 1 of these more common causative genes. It's a very exciting time as we think about being able to manipulate the genome in order to accomplish what we think needs to happen to slow down motor neuron loss.”

Amyotrophic lateral sclerosis (ALS) has been a challenging disease to treat for some time, but recent progress has chipped away at this difficulty, with another FDA approval occurring in late 2022 of Amylyx’s phenylbutyrate/taurursodiol (Relyvrio) to add to the total of therapeutic options.¹ Additionally, recently published data suggest another investigational therapy, CNM-Au8, from Clene Nanomedicine, might be similarly on its way to helping bolster the armamentarium of treatments.²

Gene therapy is an emerging area of interest in ALS, with multiple investigational products currently in development. Several of these candidates will be discussed at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held in Dallas, Texas, March 19-22, 2023. At the meeting, Matthew B. Harms, MD, an associate professor of neurology at Columbia University and a medical consultant and care center director for MDA, will be chairing a session at the conference entitled “Gene Therapy in ALS”. The session will be held from 2:00 PM to 4:00 PM on Tuesday, March 21, 2023, and is coupled with a morning session in the same track.

In an interview with NeurologyLive^®, Harms provided his expectations for this year’s meeting and gave an overview of the various topics that will be discussed in his session. Additionally, he pointed to the benefits of the variety of clinicians who attend the meeting, the knowledge he gleans from other specialists, and the challenges in care that the community is still seeking to address.

REFERENCES
1. Amylyx Pharmaceuticals announces FDA approval of Relyvrio for the treatment of ALS. News release. Amylyx Pharmaceuticals. September 29, 2022. Accessed March 9, 2023. https://www.amylyx.com/media/amylyx-pharmaceuticals-announces-fda-approval-of-relyvriotm-for-the-treatment-of-als
2. CNM-Au8 associated with delayed time to key clinical progression events at six months supporting a survival benefit in the Healey ALS Platform trial. News release. March 9, 2023. Accessed March 9, 2023. https://www.globenewswire.com/news-release/2023/03/09/2623829/0/en/CNM-Au8-Associated-With-Delayed-Time-to-Key-Clinical-Progression-Events-at-Six-Months-Supporting-a-Survival-Benefit-in-the-Healey-ALS-Platform-Trial.html