脐带间充质干细胞在遗传性痉挛性截瘫中的应用

目的观察脐带间充质干细胞（UC-MSC）治疗遗传性痉挛性截瘫（HSP）的临床疗效及安全性。方法2010年9月及2011年4月，分别给予一HSP家系父子2人行UC-MSC鞘内注射治疗，两个疗程，每次1×106 cells/Kg，每周1次，4次为1个疗程。采用改良的Ashworth肌张力分级标准（MAS）、国际合作共济失调评分量表（ICARS）及日常生活量表（ADL），对患者治疗前后神经功能、日常生活能力进行评定。结果第一疗程结束1个月与治疗前比较，2人MAS分级、ICARS及ADL评分均降低，两人行走站立稳定性及言语流利程度较治疗前改善；第二疗程结束后1个月与该疗程治疗前比较，2人ICARS及ADL评分降低，儿子肌张力进一步降低，父亲双上肢共济失调减轻。2人治疗后均未见明显不良反应发生。末次治疗结束后随访20个月，父、子俩分别于第二疗程治疗结束7个月及8个月后，症状继续加重。结论 UC-MSC鞘内注射治疗是安全的，可在一定时间内一定程度上减轻患者临床症状，提高患者生活质量，延缓疾病进展，但疗效不能持久。

Umbilical Cord Mesenchymal Stem Cells in the Treatment of Hereditary Spastic Paraplegia

Objective To observe the safety and effectiveness of umbilical cord mesenchymal stem cells（UC-MSC） in the treatment of hereditary spastic paraplegia（HSP） from a familial father and son. Methods In September 2010 and April 2011, a familial father and son with HSP were given UC-MSCs by intrathecal injection for 2 courses, at a dose of 1x106/kg, once a week, 4 times as a course. Modified Ashworth Scale（MAS）, International Cooperative Ataxia Rating Scale（ICARS） and Activity of Daily Living Scale （ADL） were used to evaluate patients＇ neural function and quality of daily life before and after cell therapy. Results Comparing their MAS grading, ICARS and ADL scores one month after the first course therapy with those before therapy, it is found that MAS grading, ICARS and ADL scores were decreased, their stability of walking and standing, as well as verbal fluency were improved. Comparing their ICARS and ADL scores one month after the second course therapy with those before the second course therapy, it is found that their ICARS and ADL scores were decreased, son ’s muscular tension was further reduced, and father’s ataxia was relieved. No side effect was observed in both of them. A follow-up of 20 months after the second course therapy showed that the father and son ’s symptoms progressed respectively at the time of 7 months and 8 months after all therapy. Conclusion Intrathecal injection of UC-MSCs is safe and can ameliorate clinical symptoms, improve life quality of HSP patients to some extent within a certain period, and slow down disease progression, but efficacy can not permanently maintain.