Wonnacott Keith
US Food and Drug Administration, Center for Biologics Evaluation and Research, Rockville 20852, MD.
Am J Ther. 2005 Nov-Dec;12(6):600-4. doi: 10.1097/01.mjt.0000178765.60234.39.
Over the past 12 years, the US Food and Drug Administration (FDA) has reviewed more than 40 investigational new drug applications for the use of allogeneic pancreatic islets to treat type 1 diabetes mellitus. Recent advances in islet cell isolation, transplantation, and immunosuppressive maintenance have led to multiple centers reporting promising results in the treatment of type 1 diabetes with allogeneic islet cells. The FDA held an advisory committee meeting on October 9-10, 2003, to explore the potential for licensing allogeneic islets as a therapy for severe type 1 diabetes. This article highlights the manufacturing challenges, discussed by the FDA advisory committee, that remain to be resolved before allogeneic islets can be approved for treatment of type 1 diabetes. This article also briefly addresses the challenges facing the clinical trial design of studies that could be used to support licensure.
在过去12年里，美国食品药品监督管理局（FDA）已审查了40多项关于使用异体胰岛治疗1型糖尿病的研究性新药申请。胰岛细胞分离、移植及免疫抑制维持方面的最新进展，已使多个中心报告了在使用异体胰岛细胞治疗1型糖尿病方面取得的有前景的结果。FDA于2003年10月9日至10日召开了一次咨询委员会会议，以探讨将异体胰岛作为严重1型糖尿病治疗方法进行许可的可能性。本文重点介绍了FDA咨询委员会讨论的、在异体胰岛被批准用于治疗1型糖尿病之前仍有待解决的生产挑战。本文还简要阐述了可用于支持许可的研究的临床试验设计所面临的挑战。
