title: Clinical trial perspective for adult and juvenile Huntington's disease using genetically-engineered mesenchymal stem cells.
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.
authors.affiliation: Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health System, Sacramento, CA, USA.
authors.forename: Peter
authors.initials: P
authors.lastname: Deng
authors.affiliation: Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health System, Sacramento, CA, USA.
authors.forename: Audrey
authors.initials: A
authors.lastname: Torrest
authors.affiliation: Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health System, Sacramento, CA, USA.
authors.forename: Kari
authors.initials: K
authors.lastname: Pollock
authors.affiliation: Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health System, Sacramento, CA, USA.
authors.forename: Heather
authors.initials: H
authors.lastname: Dahlenburg
authors.affiliation: Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health System, Sacramento, CA, USA.
authors.forename: Geralyn
authors.initials: G
authors.lastname: Annett
authors.affiliation: Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health System, Sacramento, CA, USA.
authors.forename: Jan A
authors.initials: JA
authors.lastname: Nolta
authors.affiliation: Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health System, Sacramento, CA, USA.
authors.forename: Kyle D
authors.identifier: 0000-0002-0235-5038
authors.initials: KD
authors.lastname: Fink
keywords: Huntington's disease; brain derived neurotrophic factor; genetic engineering; mesenchymal stem cells; neurodegenerative disorders