Abstract: 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.

Key words: Umbilical cord mesenchymal stem cells, Hereditary spastic paraplegia, Effectiveness