Promise and Potential Challenges With Gene Therapy in Epilepsy: Imad Najm, MD

WATCH TIME: 5 minutes

"Five to ten years from now, if we sit and talk again about epilepsy, where we are, we're going to talk probably the bulk of what we're going to say will be about gene therapy and what is the newest kid on the block, rather than, 'Can we do it or not?'"

An estimated 3.4 million Americans and 65 million people worldwide currently live with epilepsy. Even with the numerous antiseizure medications and surgical options, some patients still struggle to control their seizures. Over the years, the idea of gene therapy has become more highly discussed as a potential option for those with refractory cases. By targeting the underlying genetic causes of epilepsy, the thought is that gene therapy could provide more personalized and precise interventions, potentially reducing or even eliminating seizures.

A gene therapy approach could also minimize the need for invasive surgeries by addressing the root cause of the condition at the molecular level, leading to more effective and long-lasting outcomes. Despite the promise it brings, there are still some barriers with advancing gene therapy, including the need to identify specific genetic mutations and the delivery of gene therapies, specifically crossing the blood-brain barrier.

Ahead of the Cleveland Clinic’s 2024 Epilepsy Summit, held September 11-15 in downtown Cleveland, Ohio, epilepsy expert Imad Najm, MD, sat down to discuss the potential of gene therapy and some of the current barriers with advancing this therapeutic phenomenon. Najm, who serves as director of Cleveland Clinic’s Epilepsy Center at the Cleveland Clinic Neurological Institute, gave perspective on the knowledge and technical barriers of gene therapy in treating epilepsy, as well as some of the targeted treatment approaches to overcome issues with the blood-brain barrier. Furthermore, he provides an optimistic outlook for the future of gene therapy, shifting from broad, non-specific medications to more targeted, genetically informed therapies.

For more information about the Summit, including registration, click here.