Open AccessClinical trial perspective for adult and juvenile Huntington′s disease using genetically-engineered mesenchymal stem cells
Author(s) -
Peter Deng,
Audrey Torrest,
Kari Pollock,
Heather Dahlenburg,
Geralyn Annett,
Jan A. Nolta,
Kyle D. Fink
Publication year - 2016
Publication title -
neural regeneration research/neural regeneration research
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.93
H-Index - 38
eISSN - 1876-7958
pISSN - 1673-5374
DOI - 10.4103/1673-5374.182682
Subject(s) - mesenchymal stem cell , medicine , neuroscience , stem cell , neurotrophic factors , spinocerebellar ataxia , huntington's disease , stem cell therapy , disease , tolerability , bioinformatics , pathology , pharmacology , psychology , biology , adverse effect , genetics , receptor
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.