Ronald Crystal, MD: Developing Gene Therapy in Alzheimer Disease

October 17, 2020

The chairman of the Department of Genetic Medicine at Weill Cornell Medicine detailed the ongoing research in developing gene therapy for patients with Alzheimer disease.

"You can use gene therapy to put genes directly into the nervous system, so that bypasses the blood-brain barrier problem, and if it works, it’s a single administration to cure the disease forever.”

Gene therapy has come to some of the forefront conversations in medicine across a wide spectrum of disorders. This is, in part, because it offers a number of advantages for patients with chronic diseases when it is safe and effective, allowing a single administration of therapy to essentially cure the disorder. As well, it often allows for direct-to-target engagement, offering a workaround for some of the challenges in administering therapies to patients.

For Ronald G. Crystal, MD, chairman, Department of Genetic Medicine, Weill Cornell Medicine, an expert in gene therapy, these 2 particular facets provide solid reasoning—as well as interest—for the potential of these types of treatment to make an impact in genetic forms of Alzheimer disease that are influenced by APOE4. This research began with genes intended to treat lysosomal storage diseases, which were delivered intravenously, and has since advanced to intravenous and intracisternal delivery.

To find out more about the state-of-the-art research being done in this field for Alzheimer disease, NeurologyLive sat down with Crystal. He recently gave a talk at the Alzheimer’s Drug Discovery Foundation International Conference about this very topic, and in this interview, he detailed some of his major talking points from that presentation, including how his research is targeting those individuals who are APOE4 heterozygotes.