Spinal cord injury (SCI) is a devastating condition with limited pharmacological treatment options to restore function. Regenerative approaches have recently attracted interest as an adjuvant to current standard of care. Adipose tissue-derived (AD) mesenchymal stem cells (MSCs) represent a readily accessible cell source with high proliferative capacity. The CELLTOP study, an ongoing multidisciplinary phase 1 clinical trial conducted at Mayo Clinic (ClinicalTrials.gov Identifier: NCT03308565), is investigating the safety and efficacy of intrathecal autologous AD-MSCs in patients with blunt, traumatic SCI. In this initial report, we describe the outcome of the first treated patient, a 53-year-old survivor of a surfing accident who sustained a high cervical American Spinal Injury Association Impairment Scale grade A SCI with subsequent neurologic improvement that plateaued within 6 months following injury. Although he improved to an American Spinal Injury Association grade C impairement classification, the individual continued to be wheelchair bound and severely debilitated. After study enrollment, an adipose tissue biopsy was performed and MSCs were isolated, expanded, and cryopreserved. Per protocol, the patient received an intrathecal injection of 100 million autologous AD-MSCs infused after a standard lumbar puncture at the L3-4 level 11 months after the injury. The patient tolerated the procedure well and did not experience any severe adverse events. Clinical signs of efficacy were observed at 3, 6, 12, and 18 months following the injection in both motor and sensory scores based on International Standards for Neurological Classification of Spinal Cord Injury. Thus, in this treated individual with SCI, intrathecal administration of AD-MSCs was feasible and safe and suggested meaningful signs of improved, rather than stabilized, neurologic status warranting further clinical evaluation.

中文翻译：

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CELLTOP临床试验：自体脂肪组织衍生的间充质干细胞在治疗创伤性脊髓损伤所致麻痹的1期试验中首次报告。

脊髓损伤（SCI）是一种破坏性疾病，只能通过有限的药物治疗选择来恢复功能。最近，再生疗法作为当前护理标准的佐剂引起了人们的兴趣。脂肪组织来源的（AD）间充质干细胞（MSC）代表了具有高增殖能力的易于获取的细胞来源。CELLTOP研究是一项由Mayo Clinic进行的正在进行的多学科1期临床试验（ClinicalTrials.gov标识符：NCT03308565），旨在研究鞘内自体AD-MSC在钝性，创伤性SCI患者中的安全性和有效性。在这份初次报告中，我们描述了第一位接受治疗的患者的结果，一位53岁的冲浪事故幸存者，患有高颈椎病的美国脊髓损伤协会损害量表A SCI，其神经系统改善在受伤后6个月内达到稳定。尽管他升格为美国脊椎损伤协会的C级损伤分类，但此人仍然受到轮椅束缚并严重虚弱。研究入组后，进行脂肪组织活检，并分离，扩增和冷冻保存MSC。根据方案，患者在受伤后11个月，在L3-4级进行标准腰椎穿刺后，接受鞘内注射1亿个自体AD-MSC。病人对手术耐受良好，没有发生任何严重的不良事件。在3、6、12观察到疗效的临床体征 注射后18个月，根据国际脊髓损伤神经分类标准，在运动和感觉评分上均达到标准。因此，在这个患有SCI的受治疗个体中，鞘内注射AD-MSC是可行和安全的，并提出了改善而不是稳定的神经系统状态的有意义的迹象，值得进一步的临床评估。