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干细胞疗法作为肌萎缩侧索硬化症新兴疗法的安全性和有效性:对照临床试验的系统评价

The Safety and Efficacy of Stem Cell Therapy as an Emerging Therapy for ALS: A Systematic Review of Controlled Clinical Trials.

作者信息

Aljabri Ammar, Halawani Alhussain, Bin Lajdam Ghassan, Labban Suhail, Alshehri Samah, Felemban Razaz

机构信息

College of Medicine, King Saud Bin Abdulaziz University for Health Sciences, Jeddah, Saudi Arabia.

King Abdullah International Medical Research Center, Jeddah, Saudi Arabia.

出版信息

Front Neurol. 2021 Dec 1;12:783122. doi: 10.3389/fneur.2021.783122. eCollection 2021.

DOI:10.3389/fneur.2021.783122
PMID:34938264
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8685950/
Abstract

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease with a heterogeneous course that ultimately leads to death. Currently, there is no cure, and new treatments that can slow the progression of the disease are needed. Stem cell (SC) transplantation is an emerging therapy that has shown a lot of potential in recent clinical trials. This review is aimed to examine the results of various clinical trials on this topic, thus assessing the safety and efficacy of SC transplantation as a potential treatment for ALS. We identified 748 studies in our search, of which 134 full-text studies were assessed for eligibility. Six studies met the inclusion criteria and were included in this review. Although some of the included studies showed the positive effect of SC transplantation, other studies found that there was no significant difference compared to the control group. We observed more positive effects with bone marrow mesenchymal stem cells (BM-MSC) treatments than Granulocyte colony-stimulating factor (G-CSF) ones. However, other factors, such as route of administration, number of doses, and number of cells per dose, could also play a role in this discrepancy. Based on this information, we conclude that more properly conducted clinical trials are needed to appreciate the benefit of this treatment.

摘要

肌萎缩侧索硬化症(ALS)是一种神经退行性疾病,病程各异,最终会导致死亡。目前,尚无治愈方法,因此需要能够减缓疾病进展的新疗法。干细胞(SC)移植是一种新兴疗法,在最近的临床试验中已显示出很大潜力。本综述旨在研究关于该主题的各种临床试验结果,从而评估SC移植作为ALS潜在治疗方法的安全性和有效性。我们在检索中确定了748项研究,其中134项全文研究被评估是否符合纳入标准。六项研究符合纳入标准并被纳入本综述。虽然一些纳入研究显示了SC移植的积极效果,但其他研究发现与对照组相比没有显著差异。我们观察到骨髓间充质干细胞(BM-MSC)治疗比粒细胞集落刺激因子(G-CSF)治疗产生的积极效果更多。然而,其他因素,如给药途径、剂量数量和每剂量的细胞数量,也可能在这种差异中起作用。基于这些信息,我们得出结论,需要进行更规范的临床试验来评估这种治疗方法的益处。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a5fb/8685950/7ea4066d72d9/fneur-12-783122-g0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a5fb/8685950/7ea4066d72d9/fneur-12-783122-g0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a5fb/8685950/7ea4066d72d9/fneur-12-783122-g0001.jpg

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Looking backward to move forward: a meta-analysis of stem cell therapy in amyotrophic lateral sclerosis.回顾过去,展望未来:肌萎缩侧索硬化症干细胞治疗的荟萃分析。
NPJ Regen Med. 2021 Apr 1;6(1):20. doi: 10.1038/s41536-021-00131-5.
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Mesenchymal stromal cell therapeutic potency is dependent upon viability, route of delivery, and immune match.间充质基质细胞的治疗效力取决于细胞活力、给药途径和免疫匹配度。
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Long-term survival benefits of intrathecal autologous bone marrow-derived mesenchymal stem cells (Neuronata-R®: lenzumestrocel) treatment in ALS: Propensity-score-matched control, surveillance study.鞘内注射自体骨髓间充质干细胞(Neuronata-R®:伦祖美罗塞尔)治疗肌萎缩侧索硬化症的长期生存获益:倾向评分匹配对照监测研究
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Neurology. 2019 Dec 10;93(24):e2294-e2305. doi: 10.1212/WNL.0000000000008620. Epub 2019 Nov 18.
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