Therapeutic interventions in amyotrophic lateral sclerosis (ALS) are still far from satisfying. Immune modulating procedures raise hopes for slowing the disease progression. Stem cell therapies are believed to possess the ability to regulate innate and adaptive immune response and inflammation processes. Hence, three intrathecal administrations of autologous bone marrow-derived lineage-negative (Lin^–) cells were performed every six weeks in 40 sporadic ALS patients. The concentrations of inflammatory-related proteins and expression profiles of selected miRNA in the cerebrospinal fluid (CSF) and plasma at different timepoints post-transplantation were quantified by multiplex Luminex and qRT-PCR. The global gene expression in nucleated blood cells was assessed using the gene microarray technique. According to the ALS Functional Rating Scale (FRSr), the study population was divided into responders (group I, n = 17) and non-responders (group II, n = 23). A thorough analysis of the pro-inflammatory expression profiles, regulated miRNA pathways, and global gene expression profiles at the RNA level revealed the local and systemic effects of Lin^– cell therapy on the immune system of patients with ALS. The autologous application of Lin^– cells in CSF modulates immune processes and might prevent the progression of neurodegeneration. However, further in-depth studies are necessary to confirm the findings, and prolonged intervention is needed to maintain therapeutic effects.

中文翻译：

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在ALS患者的免疫途径中反复应用自体骨髓来源的谱系-阴性干细胞/祖细胞-焦点。

肌萎缩性侧索硬化症（ALS）的治疗干预措施仍远未令人满意。免疫调节程序为减缓疾病进程带来了希望。据信，干细胞疗法具有调节先天性和适应性免疫反应以及炎症过程的能力。因此，自体骨髓衍生的谱系阴性（Lin ^–40例散发性ALS患者每六周进行一次）细胞检查。通过多重Luminex和qRT-PCR定量分析移植后不同时间在脑脊液（CSF）和血浆中炎症相关蛋白的浓度以及所选miRNA的表达谱。使用基因微阵列技术评估了有核血细胞中的整体基因表达。根据ALS功能评定量表（FRSr），将研究人群分为应答者（I组，n = 17）和无应答者（II组，n = 23）。在RNA水平上对促炎表达谱，调控的miRNA通路和全局基因表达谱进行了全面分析，揭示了Lin ^–细胞疗法对ALS患者的免疫系统的影响。林的自体应用^-细胞CSF调节免疫过程，并可能防止神经退行性疾病的进展。但是，需要进一步的深入研究来证实这一发现，并且需要长时间的干预以保持治疗效果。