The Development of New Gene Therapies to Address Unmet Needs in ALS: Matthew B. Harms, MD


The associate professor of neurology at Columbia University spoke at the 2023 MDA conference about the unmet needs of patients with ALS and the potential of new gene therapies. [WATCH TIME: 5 minutes]

WATCH TIME: 5 minutes

“I think [one] unmet need in this field is trying to speed diagnoses. Patients diagnosed earlier in the disease, when few motor neurons have already been lost, are able to save more of the ones that are there are still in a healthier state. Additional funding and efforts to very closely identify and follow patients who are carrying ALS gene mutations is another unmet need.”

In the field of amyotrophic lateral sclerosis (ALS), there are a handful of approved treatments; however, there are none approved to treat genetic forms of the disease. Research shows there are more than 40 genes have been identified as having a relationship with ALS. Among these, C9orf72, SOD1, TARDBP, and FUS severe as the4 key ones that account up to 70% of standard familial cases in ALS.1 This has led to emerging interest in the field in identifying causative gene mutations in ALS and tracking the patients who carry them to address unmet needs.

A session on the topic of gene therapy in ALS was presented at the recent 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 19-22, in Dallas, Texas. Matthew B. Harms, MD, chaired the session and presented a talk focused on the 30 years of ALS genetic research previously done and separating the successes from the current unknowns about genetics.2

In an interview with CGTLive®, a sister publication to NeurologyLive®, Harms shared his thoughts on the conference in learning from other specialties in neuromuscular diseases and the excitement surrounding potential gene therapies for ALS. Harms, associate professor of neurology at Columbia University, and medical consultant and care center director at the Muscular Dystrophy Association (MDA), discussed the implications of potential approved future gene therapies and the current challenges in the field.

Click here for more coverage of MDA 2023.

1. ALS Genes and Mutations. ALS Association. Accessed April 6, 2023.
2. Harms M. 30 Years of ALS Genetic Research: Separating Wheat from Chaff. Presented at: MDA Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX. Gene Therapy in ALS session.

Editor’s Note: Harms disclosed that he has board memberships in the Northeast ALS Consortium and Muscular Dystrophy Association. He also disclosed consultancies or paid advisory boards for Biogen and Ionis; grants/research funding pending for Biogen, Ionis, and UniQure; and grants received/research funding for Biogen and Ionis.

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