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

作     者：Peter Deng Audrey Torrest Kari Pollock Heather Dahlenburg Geralyn Annett Jan A.Nolta Kyle D.Fink 

作者机构：Stem Cell Program and Institute for Regenerative Cures University of California Davis Health System 

出 版 物：《Neural Regeneration Research》 (中国神经再生研究（英文版）)

年 卷 期：2016年第11卷第5期

页      面：702-705页

核心收录：

学科分类：1002[医学-临床医学] 100204[医学-神经病学] 10[医学] 

基　　金：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) philanthropic donors from the HD community,including the Roberson family and Team KJ 

主　　题：mesenchymal stem cells neurodegenerative disorders, Huntington's disease genetic engineering brain derived neurotrophic factor 

摘      要：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.