Stem-cell therapy is a 'big leap' for Parkinson's treatment

With safety proven, Nature calls for bigger trials to prove efficacy

Orange, Calif. — Calling it a "big leap in the field" of Parkinson's disease treatment, the journal Nature reported today that stem-cell therapy improved symptoms in a clinical trial conducted by researchers at the University of California, Irvine, and centers in New York and Toronto.

Data from the phase 1 exPDite clinical trial, published in Nature today, demonstrated that injections of dopamine-producing neurons derived from human embryonic stem cells was not only safe 18 months after treatment, but also that some participants "experienced visible reductions in tremors," the Nature reviewer noted. Results of second trial of human-induced pluripotent stem cells conducted in Japan reported similar results.

The journal review said larger trials are now needed to prove the efficacy of these stem cell-based therapies on the progressive, incurable disorder, which affects more than 1 million Americans and 10 million people worldwide.

“The concept of 'rebuilding’ brain networks that have been lost to disease is compelling,” said exPDite senior study investigator Claire Henchcliffe, MD, DPhil, chair of the UC Irvine School of Medicine’s Department of Neurology and a nationally renowned Parkinson's disease specialist. “The results of this early phase clinical trial demonstrate the promise of regenerative medicine and should provide hope for Parkinson’s disease patients and their families.”

The 18-month trial results showed that surgical transplantation of bemdaneprocel, a dopaminergic neuron progenitor cell product developed by BlueRock Therapeutics and derived from human embryonic stem cells, in the brains of 12 participants was “generally well tolerated” and had caused no serious adverse events related to cell therapy.

Imaging studies demonstrated continued neuron cell engraftment in the brain after immunosuppression therapy ended 12 months after treatment. In a secondary outcome of the trial, which was designed to test only the cell therapy’s safety, participants also showed some improvement in motor function.

Bemdaneprocel is the first allogeneic, pluripotent stem-cell therapy developed for Parkinson’s disease in the United States and the most clinically advanced, according to BlueRock, a clinical-stage cell therapy company and subsidiary of the German multinational pharmaceutical and biotechnology company Bayer AG.

“Cell therapy is a potential new treatment option for individuals with Parkinson's disease and our team is immensely proud of having bemdaneprocel’s 18-month phase 1 data published in such a prestigious journal as Nature,” BlueRock Therapeutics Chief Medical Officer Amit Rakhit, MD, MBA, said in a news release. “Our efforts are now focused on maintaining our forward momentum to initiate and enroll the phase 3 trial as we advance bemdaneprocel’s development to this exciting next stage.”

The phase 3 trial, called exPDite-2, is expected to open in later this year, said Rakhit, Bluerock's head of development. Henchcliffe said the trial will be offered at UCI Health and the UCI Alpha Clinic, the clinical trial arm of the UC Irvine Sue and Bill Gross Stem Cell Research Center.

Parkinson’s disease is the second most common U.S. neurodegenerative disorder after Alzheimer’s disease, seen usually in adults over age 60. Current treatments are focused largely on drugs that replace dopamine or dopamine-like substitutes. They can provide relief for movement-related symptoms — tremors, stiffness, loss of motor control and balance — but their effectiveness wanes as the disease progresses.

Some Parkinson's patients may also benefit from deep brain stimulation, which involves implanting a device that delivers electrical impulses to parts of the brain that control movement.

For the phase 1 trial, bemdaneprocel was surgically transplanted in the putamen, a part of the mid brain where dopamine-producing neural networks are progressively destroyed by the disease.

This approach, which restored neural networks in animal studies, could radically change the trajectory of the disease. If successful in humans, Henchcliffe believes it could be a “one-size-fits-all, single treatment that could potentially provide lifelong relief from Parkinson’s symptoms."

Preliminary phase 1 trial data of bemdaneprocel at the 24-month mark show similar promising results, Henchcliffe noted. Those findings were presented earlier this month at the American Association of Neurology (AAN) conference by Harini Sarva, MD, an associate professor and neurologist specializing in Parkinson’s disease at Weill Cornell Medical Center in New York.

Other authors of the abstract presented at the San Diego conference include: Viviane Tabar, MD, chair of Neurosurgery at Memorial Sloan Kettering Cancer Center in New York and Canadian Parkinson’s disease specialists Andres M. Lozano, MD, PhD, chair of Neurosurgery at the University of Toronto; neurologist Alfonso Fasano, MD, PhD, and neurosurgeon Suneil Kalia, PhD, MD, also with the University of Toronto.

For more information about Parkinson’s disease stem-cell clinical trials, please email the UCI Alpha Stem Cell Clinic at stemcell@uci.edu or call 949‐824‐3990.

Related information

• Learn about our comprehensive Parkinson's Disease and Movement Disorders Program ›
• Read more about deep brain stimulation procedures offered at UCI Health ›
• Promising Parkinson’s therapy gets FDA nod ›
• Novel stem-cell therapy posts gains in promising Parkinson's disease clinical trial ›

About the UCI Alpha Clinic

The UCI Alpha Clinic is the clinical trial arm of the UC Irvine Sue & Bill Gross Stem Cell Research Center (SCRC) and part of a network of the state’s leading medical centers funded by the California Institute of Regenerative Medicine (CIRM). The UCI Alpha Clinic specializes in delivering leading-edge stem cell clinical trials to patients and seeks to accelerate the development of treatments through partnerships with patients, medical providers and clinical trial sponsors. The clinic’s network supports both CIRM-funded clinical trials and those funded by academic and industry sponsors. Visit www.stemcell.uci.edu to learn more about stem-cell clinical trials and regenerative medicine research at UC Irvine.

About UCI Health

UCI Health, one of California’s largest academic health systems, is the clinical enterprise of the University of California, Irvine. The 1,317-bed system comprises its main campus UCI Medical Center, its flagship hospital in Orange, Calif., the UCI Health — Irvine medical campus, four hospitals and affiliated physicians of the UCI Health Community Network in Orange and Los Angeles counties and a network of ambulatory care centers across the region. UCI Medical Center provides tertiary and quaternary care and is home to the only Orange County-based National Cancer Institute-designated comprehensive cancer center, high-risk perinatal/neonatal program and American College of Surgeons-verified Level I adult and Level II pediatric trauma center, gold level 1 geriatric emergency department and regional burn center. Powered by UC Irvine, UCI Health serves nearly 4 million people in Orange County, western Riverside County and southeast Los Angeles County. Follow us on Facebook, Instagram, LinkedIn and X (formerly Twitter).