Lunn J Simon, Sakowski Stacey A, Feldman Eva L
Department of Neurology, University of Michigan, Ann Arbor, Michigan, USA.
Stem Cells. 2014 May;32(5):1099-109. doi: 10.1002/stem.1628.
Amyotrophic lateral sclerosis (ALS) is a lethal disease involving the loss of motor neurons. Although the mechanisms responsible for motor neuron degeneration in ALS remain elusive, the development of stem cell-based therapies for the treatment of ALS has gained widespread support. Here, we review the types of stem cells being considered for therapeutic applications in ALS, and emphasize recent preclinical advances that provide supportive rationale for clinical translation. We also discuss early trials from around the world translating cellular therapies to ALS patients, and offer important considerations for future clinical trial design. Although clinical translation is still in its infancy, and additional insight into the mechanisms underlying therapeutic efficacy and the establishment of long-term safety are required, these studies represent an important first step toward the development of effective cellular therapies for the treatment of ALS.
肌萎缩侧索硬化症（ALS）是一种导致运动神经元丧失的致命疾病。尽管ALS中运动神经元变性的机制仍不清楚，但基于干细胞的疗法用于治疗ALS已获得广泛支持。在此，我们综述了被考虑用于ALS治疗应用的干细胞类型，并强调了最近的临床前进展，这些进展为临床转化提供了支持依据。我们还讨论了世界各地将细胞疗法应用于ALS患者的早期试验，并为未来的临床试验设计提供了重要考虑因素。尽管临床转化仍处于起步阶段，并且需要对治疗效果的潜在机制有更多了解以及确立长期安全性，但这些研究是朝着开发有效的细胞疗法治疗ALS迈出的重要第一步。
