Division of Endocrinology, University of Alberta, Edmonton, Alberta, Canada.
Division of Endocrinology, University of California, San Diego, CA, USA.
Diabet Med. 2019 Mar;36(3):297-307. doi: 10.1111/dme.13846. Epub 2018 Nov 27.
Many people with Type 1 diabetes struggle with the burden of self-management and are unable to achieve optimal glycaemic control without risk of hypoglycaemia. Future therapies with the potential to reduce the risk for short- and long-term complications while simultaneously reducing the burden of diabetes are therefore attractive. β-cell replacement is one strategy which might achieve this. Islet transplantation is limited by organ supply and the risks of long-term immunosuppression. Encapsulated stem-cell-derived β cells have the potential to address both of these issues and phase I/II clinical trials of encapsulated pancreatic progenitors have begun. A significant risk associated with the translation of stem-cell science to the clinical management of Type 1 diabetes is an underestimation of the complexity of the process and a mismatch between the hype and the expectations of both people with Type 1 diabetes and the public. We provide an update on progress in clinical trials of encapsulated stem-cell-derived β cells and propose a road map for the design and conduct of future trials to facilitate the translation of this exciting science to clinical care.
许多 1 型糖尿病患者在自我管理方面面临着沉重的负担，如果不冒着发生低血糖的风险，他们无法实现最佳的血糖控制。因此，具有降低短期和长期并发症风险同时减轻糖尿病负担的未来治疗方法很有吸引力。β 细胞替代是一种可能实现这一目标的策略。胰岛移植受到器官供应和长期免疫抑制风险的限制。包被的干细胞衍生β细胞有可能解决这两个问题，并且已经开始了包被的胰腺祖细胞的 I/II 期临床试验。将干细胞科学转化为 1 型糖尿病的临床管理的一个显著风险是低估了这个过程的复杂性，以及 1 型糖尿病患者和公众的期望之间存在不匹配。我们提供了关于包被的干细胞衍生β细胞的临床试验进展的最新信息，并提出了未来试验的设计和实施路线图，以促进这一令人兴奋的科学转化为临床护理。
