Joshua J Crose, Arezou Crose, John T Ransom, Amy L Lightner
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引用次数: 0
Abstract
Background: In this pilot safety study, we hypothesized that a human bone marrow stem cell-derived extracellular vesicle (hBM-MSC EV) investigational product (IP) would be safe and exhibit potential efficacy in amyotrophic lateral sclerosis (ALS) patients.Methods: Ten ALS patients received two 10-ml intravenous infusions of the IP given 1 month apart and evaluated over 3 months.Results: There were no serious adverse events or adverse events related to the IP and 30% of subjects' ALS functional rating scale-revised (ALSFRS-R) scores did not decline.Conclusion: HBM-MSC EVs appear safe in ALS patients. This early investigation suggests a controlled study of EVs for the treatment of ALS is warranted.
背景：在这项安全性试验研究中，我们假设人骨髓干细胞衍生的细胞外囊泡(hBM-MSC EV)研究产品(IP)对肌萎缩性脊髓侧索硬化症(ALS)患者是安全的，并具有潜在疗效：10名ALS患者接受了两次10毫升的IP静脉注射，每次间隔1个月，并在3个月内进行评估：结果：没有发生与 IP 有关的严重不良事件或不良反应，30% 的受试者的 ALS 功能评分表（ALSFRS-R）评分没有下降：结论：HBM-间充质干细胞 EVs 对 ALS 患者似乎是安全的。结论：HBM-间充质干细胞对 ALS 患者似乎是安全的。这项早期调查表明，有必要对 EVs 治疗 ALS 进行对照研究。
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