Shenzhen Beike Cell Engineering Research Institute, 2/F, Yuanxing Technology Building, #1 Songpingshan Street, Nanshan District, Shenzhen, Guangdong 518057, China.
J Transl Med. 2013 Aug 27;11:196. doi: 10.1186/1479-5876-11-196.
Autism is a pervasive neurodevelopmental disorder. At present there are no defined mechanisms of pathogenesis and therapy is mostly limited to behavioral interventions. Stem cell transplantation may offer a unique treatment strategy for autism due to immune and neural dysregulation observed in this disease. This non-randomized, open-label, single center phase I/II trial investigated the safety and efficacy of combined transplantation of human cord blood mononuclear cells (CBMNCs) and umbilical cord-derived mesenchymal stem cells (UCMSCs) in treating children with autism.
37 subjects diagnosed with autism were enrolled into this study and divided into three groups: CBMNC group (14 subjects, received CBMNC transplantation and rehabilitation therapy), Combination group (9 subjects, received both CBMNC and UCMSC transplantation and rehabilitation therapy), and Control group (14 subjects, received only rehabilitation therapy). Transplantations included four stem cell infusions through intravenous and intrathecal injections once a week. Treatment safety was evaluated with laboratory examinations and clinical assessment of adverse effects. The Childhood Autism Rating Scale (CARS), Clinical Global Impression (CGI) scale and Aberrant Behavior Checklist (ABC) were adopted to assess the therapeutic efficacy at baseline (pre-treatment) and following treatment.
There were no significant safety issues related to the treatment and no observed severe adverse effects. Statistically significant differences were shown on CARS, ABC scores and CGI evaluation in the two treatment groups compared to the control at 24 weeks post-treatment (p < 0.05).
Transplantation of CBMNCs demonstrated efficacy compared to the control group; however, the combination of CBMNCs and UCMSCs showed larger therapeutic effects than the CBMNC transplantation alone. There were no safety issues noted during infusion and the whole monitoring period.
ClinicalTrials.gov: NCT01343511, Title "Safety and Efficacy of Stem Cell Therapy in Patients with Autism".
自闭症是一种普遍存在的神经发育障碍。目前，自闭症的发病机制尚未明确，治疗方法主要局限于行为干预。干细胞移植可能为自闭症提供一种独特的治疗策略，因为在这种疾病中观察到免疫和神经调节异常。这项非随机、开放标签、单中心的 I/II 期临床试验研究了联合移植人脐血单个核细胞（CBMNC）和脐带间充质干细胞（UCMSC）治疗自闭症儿童的安全性和有效性。
37 名被诊断为自闭症的受试者被纳入本研究，并分为三组：CBMNC 组（14 名受试者，接受 CBMNC 移植和康复治疗）、联合组（9 名受试者，接受 CBMNC 和 UCMSC 联合移植和康复治疗）和对照组（14 名受试者，仅接受康复治疗）。移植包括通过静脉和鞘内注射每周一次进行四次干细胞输注。通过实验室检查和不良事件的临床评估来评估治疗安全性。采用儿童自闭症评定量表（CARS）、临床总体印象量表（CGI）和异常行为检查表（ABC）在基线（治疗前）和治疗后进行疗效评估。
治疗过程中无与治疗相关的严重安全性问题，也未观察到严重不良事件。与对照组相比，治疗 24 周后，两组治疗组的 CARS、ABC 评分和 CGI 评估均有统计学意义（p < 0.05）。
与对照组相比，CBMNC 移植显示出疗效；然而，CBMNC 联合 UCMSC 的联合治疗效果大于单独 CBMNC 移植。输注过程中和整个监测期间均未发现安全性问题。
ClinicalTrials.gov：NCT01343511，标题“自闭症患者干细胞治疗的安全性和有效性”。
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