Phase 1 study examines the safety and feasibility of stem cell treatment for Parkinson's disease

Phase 1 study examines the safety and feasibility of stem cell treatment for Parkinson's disease

A recently launched Phase 1 clinical trial at Mass General Brigham is studying the safety and feasibility of a breakthrough treatment approach for Parkinson's disease that involves reprogramming a patient's stem cells to replace dopamine cells in the brain damaged by the disease. The first autologous stem cell transplant study, based on research and technologies invented and validated preclinically at McLean Hospital's Neurorgeneration Research Institute (NRI), has enrolled and treated three patients at Brigham and Women's Hospital.

A total of six participants will be included in the Phase 1 study, where patients will be followed for 12 months and beyond to determine the safety of the procedure and monitoring for improvements in Parkinson's disease. After the first 6 patients transplanted in the Phase 1 study, researchers hope to expand and recruit more patients in the Phase 2A study.

This novel therapeutic approach to treating Parkinson's disease involves the use of stem cells derived from a patient's own blood that had been converted into induced pluripotent stem cells (IPSCs). These cells are then reprogrammed to turn into specific midbrain dopaminergic neurons ready for transplantation. The autologous transplant approach of using a person's own cells circumvents the need for immunosuppressive treatments that are necessary when cells from other donors are used.

Cell replacement for Parkinson's disease replaces the dopamine neurons lost through degeneration and can restore dopaminergic function in the brain, providing a completely new treatment modality compared to currently available treatments. The founding director of the NRI, Ole Isacson, Dr. Med Sci, who is also a professor of neurology (neuroscience) at Harvard Medical School and Mass General Brigham, has pioneered cell therapy for Parkinson's disease for the past 30 years and provided the foundation for this clinical trial.

“To see this transformative new patient experience with cell-based replacement of their own dopamine neurons come to fruition – from the basic science breakthroughs in our laboratory to being fully translated into a clinical application for the patient's suffering from Parkinson's disease – is very gratifying,” said Isacson. “We believe this approach may open 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.”

Under Isacson's leadership, the NRI at McLean has advanced autologous cell-based recovery in Parkinson's disease with a landmark preclinical publication using stem cells and the first preclinical demonstration of effective human IPS cell-derived dopamine neuron use in 2015, the NRI team of Penny and Penny, the NRI team led by ISACON and Penny Hall, PHD, led, developed and patented in the NRI team. Associate Professor of Psychiatry at Harvard Medical School provided the first evidence of long-term safety and benefits of autologous stem cell therapy in a highly relevant Parkinson's disease non-human primate animal model.

The NRI received official approval from the U.S. Food and Drug Administration (IND) on August 23, 2023 for a Phase 1 clinical trial to test this unique autologous dopamine neuronal cell therapy.

Following this FDA approval for the phase 1 clinical trial, the NRI’s innovative preclinical work was translated into the clinic with the first patient treated on September 9, 2024. This collaboration includes NRI investigators James Schumacher, MD, and Oliver Cooper, PhD, and colleagues in the Neurology (Michael Hayes, MD) and Neurosurgery (John Rolston, MD, PhD, principal investigator of the Phase 1 trial) departments at Brigham and Women’s Hospital. Isacson is not directly involved in the clinical trial as he is the innovator patent holder of the technology and also co-founder of Oryon Cell Therapies, which has the license for this technology. The effort is being led by Hallett and colleagues within the General Brigham Healthcare System and its medical school-affiliated institutions at Harvard.

It is extraordinary to observe that our institution's investigators can bring new treatments to patients through the entire bench-to-bed laboratory process, and it inspires many investigators to similarly pursue their scientific and medical discoveries to reach patients in need. “

Kerry Ressler MD, PhD, Chief Scientific Officer, McLean Hospital

The Phase 1 clinical trial will be the first such study testing blood-derived autologous iPSC-derived dopamine neurons in patients with Parkinson's disease and will be funded by the National Institute of Health's National Institute of Neurological Disorders and Stroke (NINDS). The NINDS awarded the highly competitive collaborative research to enable and advance Translational Biologics Enterprises (Create BIO) for this work in 2020.

People looking for more information on the trial can email: [email protected]

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