Department of Neurology, University of Massachusetts Medical School, Worcester, MA USA.
Neurol Res. 2021 Aug;43(8):616-632. doi: 10.1080/01616412.2021.1893564. Epub 2021 Feb 25.
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease of upper and lower motor neurons with high burden on society. Despite tremendous efforts over the last several decades, there is still no definite cure for ALS. Up to now, only two disease-modifying agents, riluzole and edaravone, are approved by U.S. Food and Drug Administration (FDA) for ALS treatment, which only modestly improves survival and disease progression. Major challenging issues to find an effective therapy are heterogeneity in the pathogenesis and genetic variability of ALS. As such, stem cell therapy has been recently a focus of both preclinical and clinical investigations of ALS. This is because stem cells have multifaceted features that can potentially target multiple pathogenic mechanisms in ALS even though its underlying mechanisms are not completely elucidated. Here, we will have an overview of stem cell therapy in ALS, including their therapeutic mechanisms, the results of recent clinical trials as well as ongoing clinical trials. In addition, we will further discuss complications and limitations of stem cell therapy in ALS. The determination of whether stem cells offer a viable treatment strategy for ALS rests on well-designed and appropriately powered future clinical trials. Randomized, double-blinded, and sham-controlled studies would be valuable.
肌萎缩侧索硬化症（ALS）是一种影响上下运动神经元的进行性神经退行性疾病，给社会带来了沉重负担。尽管在过去几十年中付出了巨大努力，但目前仍没有针对 ALS 的明确治愈方法。迄今为止，仅有两种疾病修饰剂——利鲁唑和依达拉奉——获得美国食品和药物管理局（FDA）批准用于 ALS 治疗，仅能适度改善生存和疾病进展。找到有效治疗方法的主要挑战问题是 ALS 的发病机制和遗传变异性存在异质性。因此，干细胞治疗最近成为 ALS 的临床前和临床研究的重点。这是因为干细胞具有多方面的特征，即使其潜在机制尚未完全阐明，也有可能针对 ALS 的多种致病机制。 在这里，我们将概述 ALS 中的干细胞治疗，包括其治疗机制、最近临床试验的结果以及正在进行的临床试验。此外，我们还将进一步讨论 ALS 中干细胞治疗的并发症和局限性。干细胞是否为 ALS 提供可行的治疗策略，取决于精心设计和适当强度的未来临床试验。随机、双盲和假对照研究将具有价值。
