Background. Given individuals with spinal cord injury (SCI) approaching 2 million, viable options for regenerative repair are desperately needed. Human central nervous system stem cells (HuCNS-SC) are self-renewing, multipotent adult stem cells that engraft, migrate, and differentiate in appropriate regions in multiple animal models of injured brain and spinal cord. Preclinical improved SCI locomotor function provided rationale for the first-in-human SCI clinical trial of HuCNS-SC cells. Evidence of feasibility and long-term safety of cell transplantation into damaged human cord is needed to foster translational progression of cellular therapies. Methods. A first-ever, multisite phase I/IIa trial involving surgical transplantation of 20 million HuCNS-SC cells into the thoracic cord in 12 AIS A or B subjects (traumatic, T2-T11 motor-complete, sensory-incomplete), aged 19 to 53 years, demonstrated safety and preliminary efficacy. Six-year follow-up data were collected (sensory thresholds and neuroimaging augmenting clinical assessments). Findings. The study revealed short- and long-term surgical and medical safety (well-tolerated immunosuppression in population susceptible to infections). Preliminary efficacy measures identified 5/12 with reliable sensory improvements. Unfortunately, without thoracic muscles available for manual muscle examination, thoracic motor changes could not be measured. Lower limb motor scores did not change during the study. Cervical cord imaging revealed, no tumor formation or malformation of the lesion area, and secondary supralesional structural changes similar to SCI control subjects. Interpretation. Short- and long-term safety and feasibility support the consideration of cell transplantation for patients with complete and incomplete SCI. This report is an important step to prepare, foster, and maintain the therapeutic development of cell transplantation for human SCI.

中文翻译：

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受损的脊髓是干细胞移植的合适靶点

背景。鉴于脊髓损伤 (SCI) 的个体接近 200 万，迫切需要可行的再生修复方案。人类中枢神经系统干细胞 (HuCNS-SC) 是自我更新的多能成体干细胞，可在多种脑和脊髓损伤动物模型的适当区域移植、迁移和分化。临床前改善的 SCI 运动功能为 HuCNS-SC 细胞的首次人体 SCI 临床试验提供了依据。需要细胞移植到受损人脐带中的可行性和长期安全性的证据来促进细胞疗法的转化进展。方法。首次多中心 I/IIa 期试验涉及将 2000 万个 HuCNS-SC 细胞手术移植到 12 名 AIS A 或 B 受试者（创伤性、T2-T11 运动完全性、感觉不完整），年龄在 19 至 53 岁之间，证明了安全性和初步疗效。收集了六年的随访数据（感觉阈值和神经影像增强临床评估）。发现。该研究揭示了短期和长期的手术和医疗安全性（对易感染人群的耐受性良好的免疫抑制）。初步功效措施确定 5/12 具有可靠的感官改善。不幸的是，没有可用于手动肌肉检查的胸肌，就无法测量胸肌运动的变化。研究期间下肢运动评分没有变化。宫颈脊髓成像显示，没有肿瘤形成或病变区域的畸形，以及类似于 SCI 对照受试者的继发性病变上结构变化。解释。短期和长期的安全性和可行性支持对完全和不完全 SCI 患者进行细胞移植的考虑。本报告是准备、促进和维持人类 SCI 细胞移植治疗发展的重要一步。