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Published on: July 5, 2022
Type 1 Diabetes TrialNet: A Multifaceted Approach to Bringing Disease-Modifying Therapy to Clinical Use in Type 1
Polly J Bingley1, Diane K Wherrett2, Ann Shultz3
1Diabetes and Metabolism, Bristol Medical School, University of Bristol, Bristol, U.K.
Abstract:
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.
Insights
Bringing disease-modifying therapies for type 1 diabetes (T1D) to patients requires coordinated research efforts. Type 1 Diabetes TrialNet exemplifies this by advancing T1D natural history studies and clinical trials.
Area of Science:
- Immunology
- Endocrinology
- Clinical Trials
Background:
- Type 1 diabetes (T1D) is an autoimmune disease requiring new therapeutic strategies.
- Accelerating the translation of T1D research from discovery to clinical practice is crucial.
Purpose of the Study:
- To describe the Type 1 Diabetes TrialNet initiative as a model for developing and implementing disease-modifying therapies for T1D.
- To highlight TrialNet's contributions to understanding T1D natural history and therapeutic approaches.
Main Methods:
- Longitudinal natural history studies in individuals at risk for or with T1D.
- Clinical trials conducted before and after disease onset.
- Mechanistic and ancillary studies to deepen scientific understanding.
Main Results:
- TrialNet has advanced the understanding of T1D natural history.
- The network facilitates the development and testing of novel T1D interventions.
- Improved efficiency and reduced participant burden in clinical trials.
Conclusions:
- Coordinated, multi-stakeholder efforts are essential for advancing T1D therapies.
- Type 1 Diabetes TrialNet serves as a successful model for translational research in T1D.
- Future plans focus on continued advancement of disease-modifying treatments for T1D.
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