Parkinson's disease is a progressive neurodegenerative disorder impacting over one million individuals in the United States, with approximately 90,000 new diagnoses annually. While current treatments manage symptoms, there is no established cure or therapy proven to slow the disease's progression.
Disease Mechanism
Parkinson's disease is characterized by reduced dopamine release in the brain. Dopamine is a critical neurotransmitter for movement, memory, and mood. Symptoms such as tremors, stiffness, and slow movement are attributed to the progressive loss of dopamine-producing brain cells, which disrupts the brain's ability to regulate movement.
Clinical Trial Details
Keck Medicine of USC is conducting an early-phase clinical trial to evaluate the safety and effectiveness of implanting specialized stem cells into the brain. These cells are programmed to replace damaged brain cells and produce dopamine.
Dr. Brian Lee, a neurosurgeon with Keck Medicine and principal investigator, stated that:
If the brain can resume normal dopamine production, Parkinson's disease progression may slow, and motor function could be restored.
The therapy utilizes induced pluripotent stem cells (iPSCs), which are adult cells reprogrammed to an undifferentiated state, capable of developing into various cell types, including dopamine-producing brain cells.
Dr. Xenos Mason, a neurologist specializing in Parkinson's disease and co-principal investigator, noted that:
iPSCs are believed to reliably mature into dopamine-producing brain cells, potentially restarting the brain's dopamine production.
Surgical Procedure and Monitoring
During the procedure, a small hole is drilled in the patient's skull to access the brain. Stem cells are then precisely implanted into the basal ganglia, a brain region controlling movement, guided by magnetic resonance imaging (MRI).
Post-surgery, patients are monitored for 12-15 months for changes in Parkinson's symptoms and potential side effects, such as dyskinesia or infection. Patient monitoring will continue for up to five years.
Trial Scope and Objectives
Keck Medicine is one of three organizations in the United States participating in this multisite clinical trial. The study involves a total of 12 participants diagnosed with moderate to moderate-severe Parkinson's disease.
The ultimate goal of the research is to develop a technique that can repair patients' motor function and enhance their quality of life. This announcement serves to share information about Keck Medicine's research involvement and is not a solicitation for participants.