PDF(Pubmed)
Abstract：
BACKGROUND: Amyotrophic lateral sclerosis (ALS) is an incurable disease. There are vigorous attempts to develop treatments to reduce the effects of this disease, and among these treatments is the transplantation of stem cells. This study aimed to retrospectively evaluate a mesenchymal stem cell (MSC) therapy cohort as a promising novel treatment modality by estimating some additional new parameters, such as immunological and biochemical factors.
METHODS: This study was designed as an open-label, one-arm cohort retrospective study to evaluate potential diagnostic biomarkers of repeated infusions of autologous-bone marrow-derived mesenchymal stem cells (BM-MSCs) in 15 confirmed patients with ALS, administered at a dose of 1 × 106 cells/kg BW with a one-month interval, in equal amounts in both an intravenous (IV) and intrathecal (IT) capacity simultaneously, via various biochemical (iron (Fe), ferritin, total-iron-binding capacity (TIBC), transferrin, and creatine kinase (CK)) and immunological parameters (tumor necrosis factor-alpha (TNF-α), neurofilament light chain (NFL), and glial-cell-derived neurotrophic factor (GDNF) levels, evaluated during the three-month follow-up period in serum and cerebrospinal fluid (CSF).
RESULTS: Our study indicated that, in the case of immunological biomarkers, TNF-α levels in the CSF showed a significant decrease at month three after transplantation compared with levels at month zero, and the p-value was p < 0.01. No statistically significant changes were observed for other immunological as well as biochemical parameters and a p-value of p > 0.05.
CONCLUSIONS: These results can indicate the potential benefit of stem cell transfusion in patients with ALS and suggest some diagnostic biomarkers. Several studies are required to approve these results.
METHODS: This study was designed as an open-label, one-arm cohort retrospective study to evaluate potential diagnostic biomarkers of repeated infusions of autologous-bone marrow-derived mesenchymal stem cells (BM-MSCs) in 15 confirmed patients with ALS, administered at a dose of 1 × 106 cells/kg BW with a one-month interval, in equal amounts in both an intravenous (IV) and intrathecal (IT) capacity simultaneously, via various biochemical (iron (Fe), ferritin, total-iron-binding capacity (TIBC), transferrin, and creatine kinase (CK)) and immunological parameters (tumor necrosis factor-alpha (TNF-α), neurofilament light chain (NFL), and glial-cell-derived neurotrophic factor (GDNF) levels, evaluated during the three-month follow-up period in serum and cerebrospinal fluid (CSF).
RESULTS: Our study indicated that, in the case of immunological biomarkers, TNF-α levels in the CSF showed a significant decrease at month three after transplantation compared with levels at month zero, and the p-value was p < 0.01. No statistically significant changes were observed for other immunological as well as biochemical parameters and a p-value of p > 0.05.
CONCLUSIONS: These results can indicate the potential benefit of stem cell transfusion in patients with ALS and suggest some diagnostic biomarkers. Several studies are required to approve these results.
摘要：
背景：肌萎缩侧索硬化症（ALS）是一种无法治愈的疾病。有积极的尝试来开发治疗以减少这种疾病的影响，这些治疗方法之一是干细胞移植。本研究旨在回顾性评估间充质干细胞（MSC）治疗队列作为一个有前途的新的治疗方式，通过估计一些额外的新参数。如免疫和生化因素。
方法：本研究设计为开放标签，单臂队列回顾性研究，以评估15例确诊的ALS患者重复输注自体骨髓间充质干细胞（BM-MSCs）的潜在诊断生物标志物，以1×106细胞/kg体重的剂量给药，间隔一个月，同时在静脉(IV)和鞘内(IT)容量中等量，通过各种生化(铁(Fe)，铁蛋白,总铁结合能力（TIBC），转铁蛋白,和肌酸激酶（CK））和免疫学参数（肿瘤坏死因子-α（TNF-α），神经丝轻链(NFL),和神经胶质细胞源性神经营养因子（GDNF）水平，在三个月的随访期间评估血清和脑脊液（CSF）。
结果：我们的研究表明，在免疫学生物标志物的情况下，脑脊液中的TNF-α水平在移植后第3个月与第0个月相比显着降低，p值为p<0.01。对于其他免疫学和生化参数没有观察到统计学上显著的变化,并且p值p>0.05。
结论：这些结果可以表明干细胞输注对ALS患者的潜在益处，并提出一些诊断性生物标志物。需要一些研究来批准这些结果。
方法：本研究设计为开放标签，单臂队列回顾性研究，以评估15例确诊的ALS患者重复输注自体骨髓间充质干细胞（BM-MSCs）的潜在诊断生物标志物，以1×106细胞/kg体重的剂量给药，间隔一个月，同时在静脉(IV)和鞘内(IT)容量中等量，通过各种生化(铁(Fe)，铁蛋白,总铁结合能力（TIBC），转铁蛋白,和肌酸激酶（CK））和免疫学参数（肿瘤坏死因子-α（TNF-α），神经丝轻链(NFL),和神经胶质细胞源性神经营养因子（GDNF）水平，在三个月的随访期间评估血清和脑脊液（CSF）。
结果：我们的研究表明，在免疫学生物标志物的情况下，脑脊液中的TNF-α水平在移植后第3个月与第0个月相比显着降低，p值为p<0.01。对于其他免疫学和生化参数没有观察到统计学上显著的变化,并且p值p>0.05。
结论：这些结果可以表明干细胞输注对ALS患者的潜在益处，并提出一些诊断性生物标志物。需要一些研究来批准这些结果。
