Matthew B. Harms, MD, on Gene Therapy in ALS, MDA 2023

“I think what's not as commonly recognized is that some of these therapies have a good chance of working in non-genetic 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.”

Gene therapy is an emerging area of interest for treatment of amyotrophic lateral sclerosis (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. 

Matthew B. Harms, MD, associate professor of neurology, Columbia University, and medical consultant and care center director, 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.

In an interview with CGTLive’s sister publication, NeurologyLive, Harms gave an overview of the various topics that will be discussed in this session. He also touched on the other aspects of the conference that he is most excited about and highlighted a topic that he hopes gets more attention this year: the pricing of gene therapies for rare diseases such as ALS. Beyond discussing the conference itself, Harms also gave a general overview of the current state of gene therapy as a potential treatment approach for ALS and pointed out several investigational products that he will be paying close attention to in 2023.