Keck Medicine of USC researchers are testing an experimental approach to Parkinson’s disease that implants lab-grown, dopamine-producing cells into a movement-control region of the brain. The early-stage Phase 1 REPLACE trial involves up to 12 people with moderate to moderate-severe Parkinson’s disease, and the U.S. Food and Drug Administration has granted the study fast-track designation.
Parkinson’s disease is a progressive neurological condition linked to the loss of dopamine-producing brain cells, which can contribute to tremors, muscle stiffness and slowed movement. More than one million people in the United States are living with Parkinson’s disease, and about 90,000 new diagnoses are made each year. While existing treatments can ease symptoms, no therapy has been proven to slow the disease itself.
Researchers at Keck Medicine of USC are now testing whether replacing lost dopamine-producing cells could help restore dopamine signaling in the brain. The study is evaluating induced pluripotent stem cells (iPSCs)—stem cells created by reprogramming adult cells such as skin or blood cells—that are prepared in the lab to become dopamine-producing brain cells.
“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 Keck Medicine neurosurgeon and principal investigator. Xenos Mason, MD, a Keck Medicine neurologist and co-principal investigator who specializes in Parkinson’s disease, said the team believes the iPSCs “can reliably mature into dopamine-producing brain cells” and may help “jump-start” dopamine production.
During the procedure, surgeons create a small opening in the skull and use magnetic resonance imaging (MRI) guidance to implant the cells into the basal ganglia, a brain region involved in movement control. After surgery, participants are monitored closely for 12 to 15 months for changes in symptoms and for potential side effects, including dyskinesia—excess movements—or infection. Follow-up is expected to continue for up to five years.
Keck Medicine of USC is one of three U.S. sites participating in the Phase 1 REPLACE clinical trial. The therapy being studied, called RNDP-001, is produced by Kenai Therapeutics. The FDA has granted the trial fast-track designation, a regulatory status intended to help speed development and review of treatments for serious conditions.
Keck Medicine said the announcement of the study is informational and is not a call for participants.