A novel Phase 1 clinical trial at Mass General Brigham is testing the safety and feasibility of an autologous stem cell therapy for Parkinson’s disease (PD). This open-label study uses induced pluripotent stem cells (iPSCs) derived from a patient’s own blood to create dopaminergic neurons, aiming to replace those lost to neurodegeneration. The trial is the first to test this approach in human patients, with researchers monitoring outcomes for at least 12 months.
The study, conducted at Brigham and Women’s Hospital, has already enrolled and treated three participants, with plans to include a total of six.
Dr. Kerry Ressler, Chief Scientific Officer at McLean Hospital, emphasized the significance of the study, stating,
“It is extraordinary to witness that investigators at our institution can bring new treatments to patients through the entire process of laboratory ‘bench to bedside,’ and it inspires many investigators to similarly pursue their scientific and medical insights to reach patients in need.”
Developed at McLean Hospital’s Neuroregeneration Research Institute (NRI), the therapy takes advantage of iPSC technology to convert patient-derived blood cells into midbrain dopamine neurons. By using autologous cells, the approach eliminates the need for immunosuppressive drugs, which are typically required when transplanting foreign cells.
According to Dr. Ole Isacson, founding director of the NRI,
“Seeing this patient-specific dopamine neuron replacement therapy progress from basic research to clinical application is incredibly gratifying. We believe this approach may open up a new treatment paradigm and lead to the development of many additional cell therapies to restore damaged brain systems and replace degenerated brain cells in other diseases.”
Dr. Isacson has contributed significantly to the field of autologous stem cell therapy for PD.
With decades of research supporting the potential benefits of dopaminergic neuron transplantation, the results of this Phase 1 study could help determine whether stem cell therapy offers a viable disease-modifying treatment for Parkinson’s.
