Capitis Research Institute, 11189 Sorrento Valley Road, Suite 105, San Diego, CA 92121, USA.
Direct Biologics, LLC, 5301 Southwest Parkway, Building 1 Suite 415, Austin, TX 78735, USA.
Neurodegener Dis Manag. 2024;14(3-4):111-117. doi: 10.1080/17582024.2024.2344396. Epub 2024 May 28.
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. Ten ALS patients received two 10-ml intravenous infusions of the IP given 1 month apart and evaluated over 3 months. 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. HBM-MSC EVs appear safe in ALS patients. This early investigation suggests a controlled study of EVs for the treatment of ALS is warranted.
在这项初步安全性研究中，我们假设一种人骨髓间充质干细胞衍生的细胞外囊泡（hBM-MSC EV）的研究性产品（IP）在肌萎缩侧索硬化症（ALS）患者中是安全的，并具有潜在的疗效。10 名 ALS 患者接受了两次 10ml 的静脉输注，两次输注间隔 1 个月，在 3 个月内进行评估。没有严重不良事件或与 IP 相关的不良事件，30%的受试者的肌萎缩侧索硬化症功能评定量表修订版（ALSFRS-R）评分没有下降。hBM-MSC EV 在 ALS 患者中似乎是安全的。这项早期研究表明，有必要对 EV 治疗 ALS 进行对照研究。
