Doctors Implant Dopamine-Producing Stem Cells in Parkinson's Patients in Groundbreaking Trial
Doctors are implanting lab-grown stem cells into Parkinson's patients' brains in a groundbreaking clinical trial to restore dopamine production. The FDA has granted the Phase 1 REPLACE trial fast-track designation, testing iPSC-derived dopamine-producing cells in 12 patients with moderate to severe Parkinson's. Researchers will monitor participants for up to five years to assess motor function restoration and potential side effects.
In a bold new trial, doctors are implanting lab-grown stem cells into the brain to reboot dopamine production in Parkinson's disease. Researchers at Keck Medicine of USC are testing a new approach that aims to address dopamine loss directly in an early phase clinical trial, implanting specially engineered stem cells designed to replace damaged neurons and generate dopamine. The U.S. Food & Drug Administration has granted the clinical trial, Phase 1 REPLACE™, fast-track designation, which is intended to speed up the development and review process.
The treatment uses a newer type of lab-created stem cell known as induced pluripotent stem cells (iPSCs). Unlike embryonic stem cells, iPSCs are made by taking adult cells, such as those from skin or blood, and reprogramming them back into a versatile state. In this form, they can develop into many different kinds of cells in the body. Researchers believe these iPSCs can reliably mature into dopamine-producing brain cells and offer the best chance of jump-starting the brain's dopamine production.
To deliver the cells, neurosurgeons create a small opening in the skull to reach the brain. Using magnetic resonance imaging (MRI) for guidance, they carefully place the stem cells into the basal ganglia, the region responsible for coordinating movement. After the operation, participants are closely observed for 12-15 months to track changes in symptoms and watch for potential side effects, including dyskinesia—excess movements—or infection. Researchers plan to continue following patients and monitoring their condition for up to five years.
The multisite clinical trial includes 12 people with moderate to moderate-severe Parkinson's disease. Keck Medicine is one of three sites in the United States taking part in the study. The stem cell therapy, known as RNDP-001, is produced by Kenai Therapeutics, a biotechnology company focused on developing treatments for neurological disorders.
Parkinson's disease is a long-term neurological condition that gradually worsens over time. More than one million people in the United States are living with the disorder, and about 90,000 new cases are diagnosed each year. Current medications and therapies can ease symptoms, but no treatment has been proven to stop or slow the disease itself. The condition is closely linked to a drop in dopamine levels in the brain, a chemical messenger that plays a critical role in controlling movement, as well as supporting memory, mood, and other essential functions. As dopamine-producing brain cells progressively die off, the brain loses its ability to properly regulate movement, leading to hallmark symptoms including tremors, muscle stiffness, and slowed motion.
The ultimate goal is to pioneer a technique that can repair patients' motor function and offer them a better quality of life. If the brain can once again produce normal levels of dopamine, Parkinson's disease may be slowed down and motor function restored.