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Foundations team up to fund stem cell therapy for Parkinson's

Date (2016-06-01)

The National Stem Cell Foundation (NSCF) and the Michael J. Fox Foundation for Parkinson's Research (MJFF) have announced a partnership to fund development of a cutting-edge therapy to replace the dopamine neurons that degenerate in Parkinson's disease, the second most common brain disease after Alzheimer's.

The foundations will collectively grant $625,000 to Ole Isacson, M.D., director of the Neuroregeneration Research Institute at McLean Hospital and principal faculty at Harvard Stem Cell Institute, to support pre-clinical work exploring implantation of dopamine neurons created from engineered stem cells. The treatment aims to alleviate or prevent Parkinson's motor symptoms of tremor, rigidity and slowness.

Dopamine neuron loss causes Parkinson's motor symptoms and may play a role in some non-motor symptoms. Currently available treatments introduce replacement dopamine, but these medications lose efficacy over time and can cause debilitating side effects such as involuntary movements (dyskinesia). Replacing the dopamine neurons might provide a regulated stream of dopamine that would alleviate motor symptoms.

The NSCF/MJFF-funded study will further explore implantation of dopamine neurons made from induced pluripotent stem cells (iPSCs) in the brains of non-human primates. In 2015, Dr. Isacson published that implanted iPSC-derived dopamine neurons survived and that the therapy was associated with motor improvement.

"The restoration of the dopamine supply system would be a significant step forward in our treatment of Parkinson's disease," said Dr. Isacson. "This project brings us closer to realizing such a therapy, though there is still much work to be done."

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