Phase II Trial of Bone Marrow-Derived Mesenchymal Stem Cells as a Disease-Modifying Therapy for Parkinson’s Disease

Study Rationale: 
Chronic brain inflammation plays a critical role in the development and progression of Parkinson’s disease. This inflammation damages the brain, disrupts the blood-brain barrier, and creates a pro-inflammatory condition. Mesenchymal stem cells can modulate immune response and are capable of migrating to sites of injury. Such stem cells are able to recognize and react to the environment by secreting several molecules, including anti-inflammatory molecules and growth factors that interact with the surrounding cells, that can allow damaged brain cells to regenerate.

Hypothesis:
Our hypothesis is that multiple intravenous infusions of mesenchymal stem cells from the bone marrow of a healthy donor will significantly slow the progression of Parkinson’s disease when compared to placebo over a year.

Study Design:
We aim to recruit 45 patients with early to moderate Parkinson’s disease and assign them to one of three treatment arms. Group one will receive 3 infusions of placebo every 3 months; group two will receive 2 infusions of 10 X 10⁶ MSC/Kg every 3 months and 1 placebo infusion; and group three will receive 3 infusions of 10 X 10⁶ MSC/Kg every 3 months. All subjects will receive 3 infusions, with 3 months intervals in between. They 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 has the potential to be a transformative approach in the treatment of Parkinson’s disease. By ending the destructive inflammatory process, mesenchymal stem cells may re-establish an equilibrium that promotes cell regeneration. This could slow or stop the disease process and restore health to the brain.

Next Steps for Development:
The team expects this trial intends to provide evidence on the safety, effectiveness, and mechanism of action of multiple infusions of mesenchymal stem cells as a treatment to change the progression of Parkinson’s disease. If results are positive, the investigators expect to progress to a large multi-site clinical trial to identify the most effective number of interventions.

A portion of funding for this project is provided through the generous support of the Demoucelle Parkinson Charity and the Stop Parkinson Walk.