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

Research output: Contribution to journalReview article

24 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)702-705
Number of pages4
JournalNeural Regeneration Research
Volume11
Issue number5
DOIs
StatePublished - May 1 2016

Keywords

  • Brain derived neurotrophic factor
  • Genetic engineering
  • Huntington’s disease
  • Mesenchymal stem cells
  • Neurodegenerative disorders

ASJC Scopus subject areas

  • Developmental Neuroscience

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