Stempeutics Research Private Ltd, Bangalore, India.
Cytotherapy. 2009;11(7):897-911. doi: 10.3109/14653240903253857.
Spinal cord injury (SCI) is a medically untreatable condition for which stem cells have created hope in the last few years. Earlier pre-clinical reports have shown that transplantation of bone marrow (BM) mesenchymal stromal cells (MSC) in SCI-simulated models can produce encouraging results. In a clinical pilot study, we investigated the growth kinetics of BM MSC from SCI patients, their safety and functional improvement post-transplantation.
Thirty patients with clinically complete SCI at cervical or thoracic levels were recruited and divided into two groups based on the duration of injury. Patients with <6 months of post-SCI were recruited into group 1 and patients with >6 months of post-SCI were included into group 2. Autologous BM was harvested from the iliac crest of SCI patients under local anesthesia and BM MSC were isolated and expanded ex vivo. BM MSC were tested for quality control, characterized for cell surface markers and transplanted back to the patient via lumbar puncture at a dose of 1 x 10(6) cells/kg body weight.
At the time of writing, three patients had completed 3 years of follow-up post-BM MSC administration, 10 patients 2 years follow-up and 10 patients 1 year follow-up. Five patients have been lost to follow-up. None of the patients have reported any adverse events associated with BM MSC transplantation.
The results indicate that our protocol is safe with no serious adverse events following transplantation in SCI patients. The number of patients recruited and the uncontrolled nature of the trial do not permit demonstration of the effectiveness of the treatment involved. However, the results encourage further trials with higher doses and different routes of administration in order to demonstrate the recovery/efficacy if any, in SCI patients.
在过去的几年中，干细胞为脊髓损伤（SCI）这种医学上无法治疗的疾病带来了希望。早期的临床前报告表明，在 SCI 模拟模型中移植骨髓（BM）间充质基质细胞（MSC）可以产生令人鼓舞的结果。在一项临床试点研究中，我们研究了来自 SCI 患者的 BM MSC 的生长动力学、移植后的安全性和功能改善。
招募了 30 名颈或胸段临床完全性 SCI 患者，并根据损伤后时间将其分为两组。损伤后<6 个月的患者纳入第 1 组，损伤后>6 个月的患者纳入第 2 组。在局部麻醉下从 SCI 患者的髂嵴采集自体 BM，分离并体外扩增 BM MSC。对 BM MSC 进行质量控制检测，鉴定其细胞表面标志物，并通过腰椎穿刺以 1x10(6)个细胞/kg 体重的剂量回输至患者体内。
截至撰写本文时，3 名患者已完成 BM MSC 给药后 3 年的随访，10 名患者完成 2 年的随访，10 名患者完成 1 年的随访。5 名患者失访。没有患者报告与 BM MSC 移植相关的任何不良事件。
结果表明，我们的方案在 SCI 患者中是安全的，移植后没有严重的不良事件。招募的患者数量和试验的非控制性不允许证明所涉及治疗的有效性。然而，这些结果鼓励进行更高剂量和不同给药途径的进一步试验，以证明任何 SCI 患者的恢复/疗效。
