Keck Medicine of USC Starting Parkinson’s Stem Cell Therapy Trial

Key findings

• Keck Medicine of USC is testing an implanted stem cell therapy for Parkinson’s disease in an early-phase clinical trial.
• The approach uses induced pluripotent stem cells to generate dopamine-producing brain cells.
• The multisite Phase 1 study plans to enroll 12 people and will follow participants for up to five years.

Keck Medicine of USC is participating in an early phase clinical trial evaluating the safety and effectiveness of implanting specialized stem cells into the brain for people with Parkinson’s disease. The goal is to replace dopamine-producing brain cells that are progressively lost in the disease.

Parkinson’s disease affects more than one million people in the United States, with about 90,000 new cases diagnosed each year, according to the release. While current treatments can help manage symptoms, Keck Medicine notes there is no cure or therapy proven to slow disease progression.

“If the brain can once again produce normal levels of dopamine, Parkinson’s disease may be slowed down and motor function restored,” said Brian Lee, MD, PhD, a neurosurgeon with Keck Medicine and principal investigator of the study.

What’s being tested
The investigational approach uses induced pluripotent stem cells (iPSCs), which are adult cells such as skin or blood cells that have been reprogrammed into a stem-like state capable of developing into many cell types. Keck Medicine says the cells are programmed to replace damaged brain cells and produce dopamine, a neurotransmitter involved in movement, memory, mood, and other functions.

“We believe that these iPSCs can reliably mature into dopamine-producing brain cells, and offer the best chance of jump-starting the brain’s dopamine production,” said Xenos Mason, MD, a neurologist who specializes in Parkinson’s disease and other movement disorders with Keck Medicine and co-principal investigator of the study.

Procedure and follow-up
During the procedure, the surgeon drills a small hole in the skull to access the brain and implants the stem cells into the basal ganglia, a region involved in movement control, using magnetic resonance imaging (MRI) guidance.

After surgery, participants will be monitored for 12 to 15 months for changes in Parkinson’s symptoms and for potential side effects, including dyskinesia and infection. Keck Medicine says follow-up will continue for up to five years.

“Our ultimate goal is to pioneer a technique that can repair patients’ motor function and offer them a better quality of life,” said Lee.

Trial scope and regulatory notes
Keck Medicine is one of three U.S. organizations participating in the multisite study, which includes a total of 12 participants with moderate to moderate-severe Parkinson’s disease. The release notes the announcement is not soliciting participants.

The stem cell product, RNDP-001, is manufactured by Kenai Therapeutics. The FDA has granted the Phase 1 REPLACE trial Fast Track designation, which is intended to speed development and review.

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