McLean Hospital Receives Coveted NIH Grant to Clinically Study Autologous Stem Cell Therapy for Parkinson’s Disease

The National Institutes of Health (NIH) has awarded McLean Hospital’s Neuroregeneration Institute (NRI) a $6 million grant over 5 years to further its groundbreaking research on stem cell-based therapy for Parkinson’s disease (PD).

The NIH developed the Cooperative Research to Enable and Advance Translational Enterprises for Biologics (CREATE Bio) program in 2014. It is managed by the NIH’s National Institute of Neurological Disorders and Stroke (NINDS). The program is designed to accelerate the translation of promising neuroscience discoveries into innovative new treatments. On average, about two research teams per year receive the CREATE Bio award.

About the Study

• $6 million NIH CREATE Bio research grant awarded to McLean Hospital’s Neuroregeneration Institute will help further groundbreaking research on stem cell therapy for Parkinson’s disease
• The research supports the first human studies of Parkinson’s disease treatment that involve replacing a patient’s dopamine neurons with new ones made from the patient’s own cells
• Stem cell-based cell therapy has the promise to cure the movement disorder associated with Parkinson’s

The research leading to this award is built on several decades of innovative work in cell therapy for PD by the NRI’s founding director, Ole Isacson, Dr Med Sci, and his Canadian collaborators. Their work culminated in the 2014 finding that fetal dopamine neurons transplanted into the brain in PD patients remained healthy for over a decade.

This fetal dopamine neuron research was an important foundation for the development of other types of dopamine cell replacement therapies for PD. This includes stem cell therapy.

Stem cell-derived neurons are a much more reasonable and abundant source for cell therapy than fetal cells. In 1998, the NRI team were the first to report how stem cells could be used to create dopamine neurons. In 2002, the team also became the first to show that dopamine neurons made from stem cells could provide functional recovery in PD animal models.

In 2010, Isacson’s team were the first to demonstrate that human-induced pluripotent stem cells (iPS cells) could produce the specific midbrain dopamine neurons needed to improve behavior in PD animal models. At the NRI in 2015, Isacson and Penny Hallett, PhD, became the first to successfully demonstrate the long-term safety and benefits of autologous (derived from the same individual) stem cell therapy in a highly relevant PD animal model.

Using autologous transplantation is particularly advantageous. It circumvents the problem of immunorejection when cells from others are used, thereby making immunosuppression unnecessary.

The NRI team will collaborate with Dana-Farber Cancer Institute and Brigham and Women’s Hospital to conduct clinical studies to test cell therapy with dopamine neurons made from patients’ own iPS cells. Along with Isacson, the NRI’s other principal investigators for this award are Hallett, Teresia Osborn, PhD, and James M. Schumacher, MD.

“This CREATE Bio award is the result of a persistent effort for over a decade, and we’re thrilled to have reached testing of a completely new approach for patients with a severe neurodegenerative disease,” said Isacson.

The NIH reviewers expressed their confidence about the prospects and quality of the proposed studies.

“The NIH review concluded that effective treatments for Parkinson’s disease that do not produce side effects and can maintain functional movement without dyskinesias are urgently needed,” said Chris Boshoff, PhD, CREATE Bio’s Project Officer. “The review panel thought that this team’s proposal has the potential to provide effective, long-lasting autologous iPSC [stem cell] therapy for PD with reduced side effects.

“This study represents a critical first systematic attempt at treating Parkinson’s with autologous neuronal transplants. An iPSC-derived personalized therapy would be a huge advance to clinical practice in PD, and potentially applicable to many other conditions.”