Translational stem cell therapy for amyotrophic lateral sclerosis
Translational stem cell therapy for amyotrophic lateral sclerosis
复制标题
DOI：
10.1038/nrneurol.2011.191
复制
发表时间：
2012-03-01
影响因子：
38.1
通讯作者：
Feldman, Eva L.
中科院分区：
文献类型：
--
作者：
Boulis, Nicholas M.;Federici, Thais;Feldman, Eva L.
肌萎缩侧索硬化症（ALS）是一种以运动神经元缺失为特征的致命性神经退行性疾病，目前急需有效的治疗方法。2009年，FDA批准了将神经干细胞直接椎管内移植到ALS患者体内的第一个I期安全性试验，目前正在进行中。干细胞技术代表了治疗ALS的一种有前途的方法，但在将有前途的实验性ALS疗法转化为患者时必须解决几个问题。本文重点介绍了支持使用干细胞治疗ALS的关键研究，并讨论了I期试验的基本原理和方法。该试验的完成可能为ALS和其他神经退行性疾病的干细胞治疗的持续进展铺平道路。
Effective treatments are urgently needed for amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease characterized by the loss of motor neurons. In 2009, the FDA approved the first phase I safety trial of direct intraspinal transplantation of neural stem cells into patients with ALS, which is currently in progress. Stem cell technologies represent a promising approach for treating ALS, but several issues must be addressed when translating promising experimental ALS therapies to patients. This article highlights the key research that supports the use of stem cells as a therapy for ALS, and discusses the rationale behind and approach to the phase I trial. Completion of the trial could pave the way for continued advances in stem cell therapy for ALS and other neurodegenerative diseases.