Neural stem cell-like cells derived from autologous bone mesenchymal stem cells for the treatment of patients with cerebral palsy.
Neural stem cell-like cells derived from autologous bone mesenchymal stem cells for the treatment of patients with cerebral palsy.
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DOI：
10.1186/1479-5876-11-21
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发表时间：
2013-01-26
影响因子：
7.4
通讯作者：
Liu H
中科院分区：
文献类型：
--
作者：
Chen G;Wang Y;Xu Z;Fang F;Xu R;Wang Y;Hu X;Fan L;Liu H
干细胞疗法是一种很有前途的治疗脑瘫的方法，脑瘫是指一类与儿童慢性运动障碍有关的脑部疾病。自体骨髓间充质干细胞可能是一种更好的细胞来源，并已被研究用于治疗脑瘫，因为它们在组织修复和免疫过程的调节功能。为评估自体骨髓间充质干细胞来源的神经干细胞样细胞（NSC-like）作为治疗中重度脑瘫患者的一种新疗法，共60例脑瘫患者参加了这项开放标签、非随机、双盲对照临床研究，随访6个月。移植组30例脑性瘫痪患者接受自体神经干细胞移植（蛛网膜下腔1-2 × 107细胞）及康复治疗，对照组30例仅接受康复治疗。我们记录了治疗后6个月的粗大运动功能测量评分、语言障碍和不良事件。与基线评分相比，移植组的粗大运动功能测量评分在治疗后3个月（评分增加为42.6，95% CI：9.8-75.3，P= 0.011）和6个月（评分增加为58.6，95% CI：25.8-91.4，P= 0.001）显著更高。对照组的粗大运动功能测量评分增加不显著。与两组的基线水平相比，治疗后第1、3和6个月的语言能力增加无统计学意义。60例患者均存活，无一例发生严重不良事件或并发症。我们的研究结果表明，神经干细胞样细胞是安全和有效的治疗脑瘫相关的运动障碍。需要进一步的随机临床试验来确定该手术的功效。
Stem cell therapy is a promising treatment for cerebral palsy, which refers to a category of brain diseases that are associated with chronic motor disability in children. Autologous MSCs may be a better cell source and have been studied for the treatment of cerebral palsy because of their functions in tissue repair and the regulation of immunological processes. To assess neural stem cell–like (NSC-like) cells derived from autologous marrow mesenchymal stem cells as a novel treatment for patients with moderate-to-severe cerebral palsy, a total of 60 cerebral palsy patients were enrolled in this open-label, non-randomised, observer-blinded controlled clinical study with a 6-months follow-up. For the transplantation group, a total of 30 cerebral palsy patients received an autologous NSC-like cells transplantation (1-2 × 107 cells into the subarachnoid cavity) and rehabilitation treatments whereas 30 patients in the control group only received rehabilitation treatment. We recorded the gross motor function measurement scores, language quotients, and adverse events up to 6 months post-treatment. The gross motor function measurement scores in the transplantation group were significantly higher at month 3 (the score increase was 42.6, 95% CI: 9.8–75.3, P=.011) and month 6 (the score increase was 58.6, 95% CI: 25.8–91.4, P=.001) post-treatment compared with the baseline scores. The increase in the Gross Motor Function Measurement scores in the control group was not significant. The increases in the language quotients at months 1, 3, and 6 post-treatment were not statistically significant when compared with the baseline quotients in both groups. All the 60 patients survived, and none of the patients experienced serious adverse events or complications. Our results indicated that NSC-like cells are safe and effective for the treatment of motor deficits related to cerebral palsy. Further randomised clinical trials are necessary to establish the efficacy of this procedure.
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