Krystof Bankiewicz, MD, PhD, on Dialing Back Progression of Parkinson Disease With GDNF Gene Therapy


The professor at Ohio State University and University of California – San Francisco discussed updated data from a phase 1 study of GDNF gene therapy in patients with PD.

“It really takes a lifetime for an investigator to get anything meaningful done in the field of medicine. It's a painstaking, long process... very complicated, highly regulated, for good reason. So, these processes take a long time and the products that we are delivering to patients are very complicated and hard to generate. So those things make that progress slow. But nevertheless, if one is determined to bring it to the finish line, then it'sa very rewarding journey.”

Updated data from a trial evaluating glial cell-derived neurotrophic factor (GDNF) gene therapy for Parkinson disease (PD) were presented at the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting, held May 16-20, in Los Angeles, California, by KrystofBankiewicz, MD, PhD, professor and vice chair of research, department of neurological surgery, Ohio State University, and professor emeritus and vice chair of research, University of California – San Francisco, and cofounder, Brain Neuropathy Bio.

CGTLive spoke with Bankiewicz to learn more about the phase 1b trial (NCT04167540) of the GDNF gene therapy, its progress, and the updated data presented. The trial is supported by the California Institute for Regenerative Medicine. He stressed “fascinating” results from the trial in which patients with moderate PD seemed to have their disease dialed back to early stages of PD, effectively erasing progression of the disease for up to 5 years of follow-up.He also touched on the phase 1 trial (NCT04680065) of GDNF gene therapy for patients with multiple system atrophy.

Click here to read more coverage of ASGCT 2023.

Bankiewicz K. Developing AAV Gene Therapies for Movement Disorders. Presented at: ASGCT 2023 Annual Meeting; May 16-20; Los Angeles, California.
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