Siwek Tomasz, Jezierska-Woźniak Katarzyna, Maksymowicz Stanisław, Barczewska Monika, Sowa Mariusz, Badowska Wanda, Maksymowicz Wojciech
Department of Neurology, School of Medicine, University of Warmia and Mazury, Olsztyn, Poland.
Department of Neurology, University Hospital in Olsztyn, Olsztyn, Poland.
Med Sci Monit. 2020 Dec 10;26:e927484. doi: 10.12659/MSM.927484.
BACKGROUND The aim of this study was to investigate repeated intrathecal injection of autologous bone marrow-derived mesenchymal stem cells (BM-D MSCs) to patients for treatment of sporadic amyotrophic lateral sclerosis (ALS). MATERIAL AND METHODS Autologous MSCs were isolated from the patients' bone marrow, plated, expanded, harvested, and passaged. Stem cells from a single bone marrow collection were used for 3 injections per patient, given over a 3-month period. Outcomes were measured with the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R). Participants were observed for a minimum of 6 months before transplantation to assess the natural course of ALS and for the same amount of time after transplantation to compare the rate of disease progression, estimated based on average monthly changes in ALSFRS-R scores. Data from 8 of the 15 participants eligible for the study were analyzed. RESULTS The safety of the MSC injections was confirmed and various effects of the therapy were documented. In patients who had ALS with an inherently slow course, there were no significant changes in the rate of disease progression. In patients who had ALS with an inherently rapid course, slowing of the disease was noted following treatment with MSCs. However, because that subgroup was so small, it was not possible to assess whether the changes were statistically significant. CONCLUSIONS Identifying groups of patients who are not responding or potentially responding negatively to injection of MSCs may help prevent it from being offered to individuals who may not benefit from the therapy. One of the limitations of this treatment method is the amount of time required for long-lasting preparation of bone marrow-derived MSCs for a disease that is rapidly progressive. Therefore, it is worth looking for other allogeneic sources of stromal cells for these types of injections.
背景 本研究旨在探讨向患者反复鞘内注射自体骨髓间充质干细胞（BM-D MSCs）以治疗散发性肌萎缩侧索硬化症（ALS）。
材料与方法 从患者骨髓中分离出自体间充质干细胞，接种、扩增、收获并传代。每次骨髓采集的干细胞用于每位患者3次注射，在3个月内完成。采用修订的肌萎缩侧索硬化功能评定量表（ALSFRS-R）测量结果。在移植前至少观察参与者6个月以评估ALS的自然病程，并在移植后观察相同时间以比较疾病进展速度，根据ALSFRS-R评分的平均每月变化进行估计。对符合研究条件的15名参与者中的8名的数据进行了分析。
结果 证实了间充质干细胞注射的安全性，并记录了该疗法的各种效果。在病程本身较慢的ALS患者中，疾病进展速度没有显著变化。在病程本身较快的ALS患者中，用间充质干细胞治疗后疾病进展减缓。然而，由于该亚组规模太小，无法评估这些变化是否具有统计学意义。
结论 识别对间充质干细胞注射无反应或可能产生负面反应的患者群体，可能有助于避免将其提供给可能无法从该疗法中获益的个体。这种治疗方法的局限性之一是对于快速进展的疾病，长期制备骨髓来源的间充质干细胞所需的时间。因此，值得寻找其他同种异体基质细胞来源用于这类注射。
