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Novel stem-cell therapy continues to show promise for Parkinson's disease

Clinical trial to restore dopamine production posts positive results 18 months after treatment

March 06, 2024
Dr. Claire Henchcliffe, a leading Parkinson's disease expert, is chair of the UCI School of Medicine Department of Neurology.

Dr. Claire Henchcliffe believes a novel neural stem cell therapy may
help stop the progression of Parkinson's disease.
Photo by Michael Neveux.

Orange, Calif. — New clinical trial data for bemdaneprocel, a promising stem cell-derived therapy for Parkinson’s disease, continues to show positive results 18 months after treatment, scientists reported today at an international conference in Portugal.

Data presented by UCI Health neurologist Claire Henchcliffe, MD, at the Alzheimer’s and Parkinson’s Diseases Conference demonstrated that the dopamine-producing neural cell therapy remained safe and well tolerated by participants in the phase 1 clinical trial at the 18-month mark.

The data also showed that the transplanted cells developed by BlueRock Therapeutics survived, have become engrafted in the brain and continued to signal increased dopaminergic activity after immune suppression therapy was curtailed at the 12-month mark.

In addition, both low-dose and high-dose bemdaneprocel patient cohorts showed improvement in motor function tests, with the high-dose participants achieving greater gains.

“It’s exciting that bemdaneprocel met safety and tolerability criteria at 12 months, and now the 18-month results suggest that these allogeneic cells survive and have potentially positive effects even after discontinuation of immunosuppressants,” said Henchcliffe, chair of the UCI School of Medicine Department of Neurology and one of the study’s principal investigators.

“We should not overinterpret results of a phase 1 study, but this is a promising step that deserves to be followed up with further studies.”

The hope is that infusing the brain with these specialized neural cells will restore critical dopamine production and alleviate the progression of Parkinson’s disease, said Henchcliffe, who conducted the clinical trial at the UCI Alpha Clinic in tandem with colleagues at Weill Cornell Medicine in New York City and the University of Toronto.

Parkinson’s disease is a progressive neurodegenerative disorder that damages neurons in the substantia nigra area of the brain. Healthy neurons produce dopamine, a neurotransmitter that allows the brain and nervous system to control and coordinate body movements. As these neural networks become impaired, symptoms of muscle stiffness, tremors, loss of coordination and balance can result.

The incurable disorder affects an estimated 1 million Americans, more men than women and most often in people over age 60. The disease is not fatal but it increases the risk pf pneumonia, falls and other conditions that may lead to death.

The new data revealed that study participants who received bemdaneprocel showed improved symptom control, with those given the higher dose able to control their symptoms an average of 2.7 more hours a day.

In announcing the new findings, BlueRock, a subsidiary of the Bayer AG pharmaceutical company, said bemdaneprocel will be further tested in a phase 2 clinical trial expected to begin later this year.

“We are excited to see the continued positive trends in the data from bemdaneprocel’s phase 1 trial at 18 months,” said Ahmed Enayetallah, BlueRock senior vice president and head of development. “While it is still early days and there is more work to do, we look forward to further investigating bemdaneprocel in clinical studies.”

Henchcliffe, an internationally recognized Parkinson’s expert whose research has included stem cell-based regenerative therapy and gene therapy, hopes the phase 2 trial will also be conducted under the auspices of the UCI Alpha Clinic.

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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