Neural Regeneration Research ›› 2016, Vol. 11 ›› Issue (5): 702-705.doi: 10.4103/1673-5374.182682

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Clinical trial perspective for adult and juvenile Huntington’s disease using genetically-engineered mesenchymal stem cells

Peter Deng, Audrey Torrest, Kari Pollock, Heather Dahlenburg, Geralyn Annett, Jan A. Nolta, Kyle D. Fink*   

  1. Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health System, Sacramento, CA, USA
  • Received:2016-04-15 Online:2016-05-20 Published:2016-05-20
  • Contact: Kyle D. Fink, Ph.D., kdfink@ucdavis.edu.
  • Supported by:

    Support for this project was provided by a NIH NIGMS Predoctoral Fellowship T32GM099608 (Deng), NIH NRSA Postdoctoral Fellowship F32NS090722 (Fink), a NIH Director’s transformative award 1R01GM099688 (Nolta), A Stewart’s and Dake Family Gift (Fink), California Institute for Regenerative Medicine (CIRM) DR2-05415 (Wheelock/Nolta), and philanthropic donors from the HD community, including the Roberson family and Team KJ.

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Abstract:

"Progress to date from our group and others indicate that using genetically-engineered mesenchymal stem cells (MSC) to secrete brain-derived neurotrophic factor (BDNF) supports our plan to submit an Investigational New Drug application to the Food and Drug Administration for the future planned Phase 1 safety and tolerability trial of MSC/BDNF in patients with Huntington’s disease (HD). There are also potential applications of this approach beyond HD. Our biological delivery system for BDNF sets the precedent for adult stem cell therapy in the brain and could potentially be modified for other neurodegenerative disorders such as amyotrophic lateral sclerosis (ALS), spinocerebellar ataxia (SCA), Alzheimer’s disease, and some forms of Parkinson’s disease. The MSC/BDNF product could also be considered for studies of regeneration in traumatic brain injury, spinal cord and peripheral nerve injury. This work also provides a platform for our future gene editing studies, since we will again use MSCs to deliver the needed molecules into the central nervous system."

Key words: mesenchymal stem cells, neurodegenerative disorders, Huntington’s disease, genetic engineering, brain derived neurotrophic factor