Safety and Potential Efficacy of Expanded Umbilical Cord-Derived Mesenchymal Stromal Cells in Luminal Ulcerative Colitis Patients.

Hanan Jafar, Dana Alqudah, Reem Rahmeh, Dana Al-Hattab, Khalid Ahmed, Rama Rayyan, Awni Abusneinah, Mohammad Rasheed, Yaser Rayyan, Abdalla Awidi
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Abstract

Inflammatory bowel disease (IBD) is characterized by periods of flare-ups and remission. It is likely to be an autoimmune in origin, presenting persistent therapeutic challenges despite current therapies. This study aims to investigate the potential of umbilical cord mesenchymal stromal cells (UCMSCs) in treating ulcerative colitis (UC). This study is a prospective phase 1 pilot, open-label, single-arm, and single-center study. UCMSCs were cultured under current Good Manufacturing Practice (cGMP) conditions and intravenously administered to six patients with UC. Safety and efficacy were evaluated using the Mayo Score/Disease Activity Index. Among the six enrolled adult patients, five completed long-term follow-ups. All exhibited at diagnosis active UC confirmed through comprehensive assessment methods. Each patient received three injections intravenously 2 weeks apart with a dose of 100 million UCMSC each. No significant short-term or intermediate-term adverse events were detected post-UCMSC administration. Long-term follow-up at 12 and 24 months showed sustained safety and no adverse events. Notably, three out of five patients achieved a Mayo score of 0 for UC, maintained at both 12 and 24 months, indicating a highly significant response (P < 0.001). This study demonstrates the safety and potential efficacy of UCMSCs in active UC. However, larger trials are warranted to validate these preliminary findings and to establish the role of UCMSC therapy as an option for managing UC.

扩增脐带间充质基质细胞对腔隙性溃疡性结肠炎患者的安全性和潜在疗效
炎症性肠病(IBD)的特点是时而发作,时而缓解。它很可能是一种自身免疫性疾病,尽管目前有各种疗法,但在治疗方面仍存在挑战。本研究旨在探讨脐带间充质基质细胞(UCMSCs)治疗溃疡性结肠炎(UC)的潜力。本研究是一项前瞻性的 1 期试验、开放标签、单臂、单中心研究。UCMSCs 在现行《药品生产质量管理规范》(cGMP)条件下培养,并静脉注射给六名 UC 患者。研究采用梅奥评分/疾病活动指数评估其安全性和有效性。在入组的六名成年患者中,有五名完成了长期随访。所有患者在确诊时均表现为通过综合评估方法确认的活动性 UC。每位患者均接受了三次静脉注射,每次剂量为 1 亿 UCMSC,间隔 2 周。UCMSC 用药后未发现明显的短期或中期不良反应。12 个月和 24 个月的长期随访显示,该疗法具有持续安全性,未出现任何不良反应。值得注意的是,五名患者中有三人的 UC 梅奥评分达到了 0 分,并且在 12 个月和 24 个月期间都保持了这一评分,这表明患者的反应非常明显(P < 0.001)。这项研究证明了 UCMSCs 对活动性 UC 的安全性和潜在疗效。不过,还需要进行更大规模的试验来验证这些初步研究结果,并确定 UCMSC 治疗在治疗 UC 中的作用。
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