Diabetes and Metabolism, Bristol Medical School, University of Bristol, Bristol, U.K.
Department of Paediatrics, The Hospital for Sick Children, University of Toronto, Toronto, Canada.
Diabetes Care. 2018 Apr;41(4):653-661. doi: 10.2337/dc17-0806.
What will it take to bring disease-modifying therapy to clinical use in type 1 diabetes? Coordinated efforts of investigators involved in discovery, translational, and clinical research operating in partnership with funders and industry and in sync with regulatory agencies are needed. This Perspective describes one such effort, Type 1 Diabetes TrialNet, a National Institutes of Health-funded and JDRF-supported international clinical trials network that emerged from the Diabetes Prevention Trial-Type 1 (DPT-1). Through longitudinal natural history studies, as well as trials before and after clinical onset of disease combined with mechanistic and ancillary investigations to enhance scientific understanding and translation to clinical use, TrialNet is working to bring disease-modifying therapies to individuals with type 1 diabetes. Moreover, TrialNet uses its expertise and experience in clinical studies to increase efficiencies in the conduct of trials and to reduce the burden of participation on individuals and families. Herein, we highlight key contributions made by TrialNet toward a revised understanding of the natural history of disease and approaches to alter disease course and outline the consortium's plans for the future.
要将疾病修饰疗法应用于 1 型糖尿病的临床实践，需要做哪些工作？这需要参与发现、转化和临床研究的研究人员与资助者和行业合作，并与监管机构保持同步，共同努力。本文描述了这样一种努力，即 1 型糖尿病 TrialNet，这是一个由美国国立卫生研究院资助、JDRF 支持的国际临床试验网络，它是从糖尿病预防试验 1 型（DPT-1）发展而来的。通过纵向的自然史研究，以及疾病发病前和发病后的临床试验，结合机制和辅助研究来增强科学理解并转化为临床应用，TrialNet 正在努力为 1 型糖尿病患者带来疾病修饰疗法。此外，TrialNet 利用其在临床研究方面的专业知识和经验，提高试验的效率，并减轻个人和家庭参与试验的负担。本文重点介绍了 TrialNet 在修订疾病自然史认识和改变疾病进程方法方面所做的重要贡献，并概述了该联盟的未来计划。
