Study Rationale: Chronic neuroinflammation plays a critical role in the development and progression of Parkinson’s disease (PD). Neuroinflammation damages the brain microenvironment, disrupts the blood-brain barrier and creates a condition that promotes further inflammation. Mesenchymal stem cells, which inhabit bone marrow, possess immune-modulatory properties, are capable of migrating to sites of injury and are able to secrete anti-inflammatory molecules and growth factors that interact with the surrounding cells, restoring tissue health and allowing cells to regenerate. With these properties, mesenchymal stem cells seem good candidates for diminishing neuroinflammation in PD.
Hypothesis: We hypothesize that multiple intravenous infusions of mesenchymal stem cells taken from the bone marrow of a healthy donor will significantly slow the progression of PD when compared to placebo.
Study Design: We aim to recruit 45 individuals with early to moderate PD and assign them to one of three treatment arms. One group will receive three infusions of placebo every three months; a second will receive three infusions of mesenchymal stem cells (107 cells/kg of body weight); a third will receive two infusions of cells and one of placebo. All subjects will receive three infusions, with three month intervals in between. Participants will be monitored for adverse reactions and clinical improvement for a year after the last infusion.
Impact on Diagnosis/Treatment of Parkinson’s Disease: Cell-based therapy is a transformative approach in the treatment of PD. Mesenchymal stem cells can stop the disease process and thus restore health to the brain microenvironment. By ending the destructive inflammatory process, mesenchymal stem cells can re-establish a condition that promotes cell regeneration.
Next Steps for Development: This trial aims to prove that multiple infusions of mesenchymal stem cells are a safe and effective way to change the progression of PD. The results will provide insights into mechanisms of action and will lead to a large, multisite clinical trial to identify the most effective number of interventions.
Trial Phase: Phase IIa