NYSCF Innovator’s Parkinson’s Disease Breakthroughs
NYSCF – Robertson Stem Cell Investigator Dr. Malin Parmar, a Professor at Lund University in Sweden, published a flurry of papers this summer all describing exciting research advances utilizing stem cell technology to understand and potentially treat Parkinson’s disease.
Human induced pluripotent stem (iPS) cells are a promising source for cell-based therapy to treat Parkinson’s, a disease in which dopaminergic neurons, a type of brain cell, progressively degenerate. The first paper, published in Nature, describes the successful survival and function of human iPS cell-derived dopaminergic neurons in a monkey model of Parkinson’s disease. Further, the treated monkeys showed increased spontaneous movement and no tumor formation after two years of monitoring. This pre-clinical study shows that this technique is clinically applicable for the treatment of Parkinson’s disease patients and may lead to human clinical trials in the near future.
Dr. Parmar and her team also published a paper in Nature Protocols, describing a detailed, multi-step protocol for deriving the specific type of dopaminergic neurons from human iPS cells needed for successful transplantation into animal models and for laboratory study. The researchers also describe a method of cryopreservation in anticipation of shipping cells between research centers. This protocol can be performed under good manufacturing practice (GMP) conditions, and is designed for future use in deriving cells for implantation into humans.
In addition to the Nature Protocols paper, the researchers published in Stem Cell Reports, describing and validating a method of cell sorting to ensure a consistent and homogenous population of iPS cell-derived neurons for transplantation.
These papers pave the way for imminent clinical trials for iPS cell-based cell therapy for Parkinson’s disease in humans.