脐带间充质干细胞治疗肌萎缩侧索硬化的安全性与有效性研究

目的观察人脐带间充质干细胞（UC—MSC）治疗肌萎缩侧索硬化（ALS）的临床效果及不良反应。方法ALS30例，给予UC—MSC静脉滴注联合鞘内注射治疗，每次静脉滴注约3×10^7个细胞，鞘内注射约7×107个细胞，1次为1个疗程。采用ALSFRS评分量表及APPEL评分对患者治疗前及疗程结束后1周、1个月、3个月时神经功能进行评定，对比治疗前及治疗后上述各项指标的变化，并对患者年龄、病程、治疗前病情严重程度（ALSFRS评分）与治疗后改善程度相关性进行分析。结果治疗后1周、1个月、3个月与治疗前相比ALSFRS评分升高（P〈0．05），APPEL评分降低（P〈0．05）。治疗后1个月与治疗后1周相比ALSFRS评分升高（P〈0．05），APPEL评分降低（P〈0．05）。治疗后3个月与治疗后1个月相比，ALSFRS评分升高（P〉0．05），APPEL评分降低（P〉0．05），差异无统计学意义。患者年龄、病程、性别与症状改善程度无明显相关性（P〉0．05）；治疗前神经损伤程度与改善程度呈负相关（P〈0．05）。结论US—MSC鞘内注射联合静脉滴注治疗ALS，在一定程度上可延缓病情进展，未出现严重不良反应。

Safety and Efficacy Analysis on the Treatment of Amyotrophiclateralsclerosis with Umbilical Cord Mesenchymal Stem Cells

Objective To observe the safety and efficacy of umbilical cord mesenchymal stem cells（ UC - MSC） in the treatment of amyotrophiclateralsclerosis（ALS）. Methods 30 cases of patients with ALS, who were treated at department of neurology or cell therapy research center from July 2010 to March 2011, were given UC - MSC by intrathecal injection at a dose of 7×10^7 cells and intravenous injection at a dose of 3 ×10^7 ceils as a course. The nerval function were evaluated with the ALS - FRS and APPEL, scores were given and compared before and 1 week, I month, 3 months after the treatment. The correlations between age, the course of disease, the gender, the degree of nerval inju- ry before treatment（ scores of ALS - FRS）and the degree of improvement were performed using statistical analysis method. Results The results showed that the scores of APPEL were significantly decreased（P 〈 0.05）, the scores of ALS - FRS were significantly increased after treatment （ 1 week, 1 month,3 months, P 〈 0.05 ）. The scores of APPEL at 1 months were significantly decreased compared with 1 week （ P 〈 0.05）. The scores of ALS - FRS at 1 month were significantly increased compared with 1 month（ P 〈 0.05 ）. The scores of ALS - FRS or APPEL at 3 months showed no significant difference compared with 1 month （P 〉 0.05）. The degree of improvement was not related with age or the course of disease or the gender（ P 〉 0.05 ）, but it was negatively correlated with the severity of disease（ r = -0. 541, P 〈 0.05 ）. The observed side effects included febrile, dizziness, back painsand, headache, which disappeared within 1 -2 days. After treatments, the symptoms were soon abated. There were no serious adverse reactions. Conclusion UC - MSC implantation by intrathecal injection and intravenous injection is able to delay disease progression of ALS to some extent. There were no serious adverse reactions.