DEC 08, 2015 8:00 AM PST

WEBINAR: Advancing PD Cell Therapy: Transplanting Cryopreserved iPSC-derived Neurons

Speaker
  • Senior Research Scientist, Regenerative Medicine RxGen, Inc.
    Biography
      Dr. Wakeman's primary research goals are directed at determining the long-term value of stem cell-based therapeutics for regenerative medicine. His past work using dopamine neurons derived from pluripotent stem cells, both human embryonic stem cells and induced pluripotent stem cells (iPSC), as a cell based strategy for dopamine replacement in animal models of Parkinson's disease has consistently supported therapeutic value moving toward the clinic. Dr. Wakeman recently joined RxGen, Inc., a translational therapeutics and disease modeling company, where he is applying his expertise and experience in regenerative medicine to bridge the translational research gap using primate models of human disease. Dr. Wakeman also holds an Adjunct Assistant Professor position in the Department of Psychiatry at Yale School of Medicine.

    Abstract
    December 8th, 2015 8:00 am PT, 10:00 am CT
     
    Cryopreservation of post-mitotic, induced pluripotent stem cell-derived midbrain lineage dopamine neurons (iPSC-mDA) is a significant advancement for cell therapy in Parkinson’s disease. Here, we demonstrate that cryopreserved iPSC-mDA neurons are reliably thawed with excellent viability and maintain biochemical and physiological signatures indicative of human midbrain dopamine neurons. We also examined the engraftment potential of iPSC-mDA neurons after transplantation into both the rodent brain up to 6-months post-grafting and the nonhuman primate brain up to 3-months post-transplantation. Immunohistochemical analysis demonstrated robust graft survival and maintenance of the midbrain dopaminergic phenotype with extensive fiber innervation into the host. A long-term functional study revealed significant reversal in motor deficits in the 6-OHDA-lesioned rat model of Parkinson’s disease that persisted for up to 6-months post-transplantation. Moreover, we found no evidence of cell proliferation, indicating safety in our initial studies. IND-enabling studies are currently underway to ascertain whether cryopreserved iPSC-mDA neurons are both safe and efficacious at longer time-points in both rodent and nonhuman primate models of Parkinson’s disease. These results indicate considerable promise for the development of pluripotent cell-based therapies in Parkinson’s disease.
     

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