Haller Michael J, Viener Hilla-Lee, Wasserfall Clive, Brusko Todd, Atkinson Mark A, Schatz Desmond A
Department of Pediatrics, University of Florida, Gainesville, FL 32610, USA.
Exp Hematol. 2008 Jun;36(6):710-5. doi: 10.1016/j.exphem.2008.01.009. Epub 2008 Mar 20.
The physical, emotional, and economic costs of type 1 diabetes (T1D) mandate continued efforts to develop effective strategies to prevent or reverse the disease. Herein, we describe the scientific and therapeutic rationale underlying efforts utilizing umbilical cord blood (UCB) as a therapy for ameliorating the progression of this autoimmune disease.
We recently embarked on a pilot study to document the safety and potential efficacy of autologous UCB infusion in subjects with T1D. Under this protocol, patients recently diagnosed with the disease and for whom autologous cord blood is stored, undergo infusion. Studies are performed before infusion and every 3 to 6 months postinfusion for immunologic and metabolic assessment. To date, 15 autologous infusions have been performed.
Preliminary observations suggest that autologous cord blood transfusion is safe and provides some slowing of the loss of endogenous insulin production in children with T1D. Mechanistic studies demonstrate that umbilical cord blood contains highly functional populations of regulatory T cells (Treg) and that increased Treg populations may be found in the peripheral blood of subjects more than 6 months after cord blood infusion. We provide the rationale for cord blood-based therapies, a summary of our initial protocol, and plans for future studies designed to explore the potential of cord blood-derived regulatory T cells to treat T1D.
Prolonged follow-up and additional mechanistic efforts are urgently needed to determine if umbilical cord blood-derived stem cells can be used as part of safe and effective therapies for T1D.
1型糖尿病（T1D）在身体、情感和经济方面的代价促使人们持续努力制定有效的预防或逆转该疾病的策略。在此，我们描述了利用脐带血（UCB）作为改善这种自身免疫性疾病进展的疗法的科学和治疗原理。
我们最近开展了一项试点研究，以记录自体脐带血输注对T1D患者的安全性和潜在疗效。根据该方案，对近期诊断出患有该疾病且储存了自体脐带血的患者进行输注。在输注前以及输注后每3至6个月进行免疫和代谢评估研究。迄今为止，已进行了15次自体输注。
初步观察表明，自体脐带血输血是安全的，并且能在一定程度上减缓T1D儿童内源性胰岛素分泌的丧失。机制研究表明，脐带血含有功能高度活跃的调节性T细胞（Treg）群体，并且在脐带血输注6个月以上的受试者外周血中可能会发现Treg群体增加。我们提供了基于脐带血疗法的原理、初始方案总结以及未来研究计划，旨在探索脐带血来源的调节性T细胞治疗T1D的潜力。
迫切需要进行长期随访和更多的机制研究，以确定脐带血来源的干细胞是否可作为T1D安全有效治疗方法的一部分。
