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.
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。