The Promising Future of Gene Therapy in Treating Neurological Diseases: Michael Kaplitt, MD, PhD


The professor of neurological surgery at Weill Cornell medicine talked about how gene therapy may offer a more direct and efficient pathway to develop treatments for neurological diseases like Parkinson disease. [WATCH TIME: 6 minutes]

WATCH TIME: 6 minutes

"I think the fact that there's so much work going on will create a universe of advanced biological therapies over the next several years, that could become available to more general practitioners and not just in experimental trials."

For clinicians, neurodegenerative disorders present a unique challenge for treatment because of the slow progression of the disease and the profound neuron loss before the onset of clinical symptoms. Options for treatment are additionally constrained by the post-mitotic nature of the central nervous system (CNS) neurons and confined ability of these cells to perform regeneration. Moreover, the blood brain barrier hinders peripheral access to the brain and so there are some limitations inherent in respect to treatment especially for protein and peptide-based therapeutics.1

To combat these constraints, investigators continue to expand the therapeutic platform based on the delivery of genes engineered for efficient CNS expression. Researchers first assessed gene therapy over 20 years ago and since then have continued to evolve it for neurodegenerative diseases like Parkinson disease (PD). Despite incremental advancements realized for PD so far, clinicians believe there is hope for the technology. Gene therapy in PD may offer the opportunity to alter dopamine production and neuronal phenotype permanently, which would be a significant therapeutic advancement even if there isn’t a cure found yet.

Michael Kaplitt, MD, PhD, professor of neurological surgery and vice chairman for research in the department of neurological surgery at Weill Cornell Medicine, recently sat down with NeurologyLive® in an interview to discuss how gene therapy streamlines the process of developing treatments compared with traditional drug discovery methods. He also spoke about the key factors that would make a patient a suitable candidate for specific gene therapies in neurological disorders. Furthermore, Kaplitt talked about the advancements in gene therapy that are currently being tested for PD.

1. Feng LR, Maguire-Zeiss KA. Gene therapy in Parkinson's disease: rationale and current status. CNS Drugs. 2010;24(3):177-192. doi:10.2165/11533740-000000000-00000
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