Matthew B. Harms, MD, on Gene Therapy’s Potential to Address Unmet Needs in ALS

“Coupling those [currently approved] medications to hopefully slow down the disease, with physical therapy, breathing interventions, nutritional interventions—things that we know improve quality of life and help keep patients alive for longer—are the mainstays of ALS treatment now, but we all recognize that those are inadequate and they're not bringing about the changes in life expectancy and functionality for our patients [that we would like to see]. And so, we're hopeful that gene therapies may be a breakthrough in that respect.”

While there are several approved medications currently available for amyotrophic lateral sclerosis (ALS), significant unmet needs remain for patients with the disease. Several gene therapies are currently in development which are intended to address these unmet needs.

Matthew B. Harms, MD, associate professor of neurology, Columbia University, and medical consultant and care center director, Muscular Dystrophy Association (MDA), chaired a session covering this topic entitled “Gene Therapy in ALS” at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held in Dallas, Texas, March 19-22, 2023.

In an interview with CGTLive™, Harms spoke about the current standards of care for patients with ALS and the potential of gene therapy to address needs not currently met by these options. He noted that the earliest breakthroughs are likely to come about for patients with identifiable gene mutations related to ALS, such as SOD1 and C9orf72. Harms also discussed the implications for patients if any of the gene therapies currently in development for ALS are approved, emphasizing that an approval could inspire increased interest in ALS research from neuromuscular neurologists. In addition, he touched on his thoughts on the MDA conference in general, pointing out that it was an opportunity for clinicians working in the ALS gene therapy space to learn from neuromuscular disease experts specializing in other diseases for which gene therapies are in development.

Read more coverage of the 2023 MDA Conference here.

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.