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Stem-cell therapy for Parkinson’s disease shows promise in early study

Study of dopamine-producing neural cells raises hope for patients, UCI Health expert says

August 29, 2023

IN THE NEWS: Results of a first-in-human trial of a stem-cell-derived therapy for Parkinson’s disease show signs of promise, scientists reported at the International Congress of Parkinson's Disease and Movement Disorders in Copenhagen, Denmark.

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The trial of bemdaneprocel, a dopamine-producing neural cell therapy, proved safe, well tolerated and showed evidence of clinical improvement in the small study of just 12 participants, UCI Health neurologist Dr. Claire Henchcliffe, a leading Parkinson’s disease expert and principal investigator of the clinical trial at the UCI Alpha Clinic, told the Guardian.

“The data from this phase 1 … study are extremely encouraging,” said Henchcliffe, chair of the UCI School of Medicine’s Department of Neurology.

“While this is a small, open-label study, meeting [its] primary endpoints of safety and tolerability, along with initial improvements seen in clinical outcomes, represents a great step forward. The hope now is that these trends continue and translate into meaningful benefit for people with Parkinson’s disease in controlled clinical trials.”

Developed by BlueRock Therapeutics, a subsidiary of the pharmaceutical company Bayer AG, bemdaneprocel is injected into the brain in the hope that the dopamine-producing neurons derived from human embryonic stem cells will rebuild damaged neural networks and reverse effects of the disease. BlueRock,in announcing the study results, said planning is underway for a phase 2 trial in early 2024.

Henchcliffe is an internationally recognized clinician and scientist in neurological disorders whose research has focused on developing new treatments for Parkinson’s disease, including stem cell-based regenerative therapy and gene therapy. While at Weill Cornell Medicine, she led a clinical trials network team as part of an initiative of the National Institute of Neurological Disorders and Stroke (NINDS) to improve treatment for neurological diseases in partnership with academia, private foundations and industry.

The UCI Alpha Clinic is the clinical trials arm of the UCI Sue & Bill Gross Stem Cell Research Center (SCRC) and part of a network of the state's CIRM-funded leading medical centers. It specializes in delivering leading-edge stem cell clinical trials and gene therapy to patients, and it seeks to accelerate the development of new treatments through partnerships with patients, medical providers and clinical trial sponsors. Visit www.stemcell.uci.edu to learn more about clinical stem-cell trials and regenerative medicine research at UCI.

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