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. 2025 Jan 1;74(1):12-21.
doi: 10.2337/db24-0439.

Emerging Concepts and Success Stories in Type 1 Diabetes Research: A Road Map for a Bright Future

Roberto Mallone  1   2   3 , Emily Sims  4   5 , Peter Achenbach  6 , Chantal Mathieu  7 , Alberto Pugliese  8 , Mark Atkinson  9   10 , Sanjoy Dutta  11 , Carmella Evans-Molina  5   12   13 , David Klatzmann  14   15 , Anne Koralova  16 , S Alice Long  17 , Lut Overbergh  7 , Teresa Rodriguez-Calvo  6 , Anette-Gabriele Ziegler  6 , Sylvaine You  1   3
Affiliations

Emerging Concepts and Success Stories in Type 1 Diabetes Research: A Road Map for a Bright Future

Roberto Mallone et al. Diabetes. .

Abstract

Type 1 diabetes treatment stands at a crucial and exciting crossroad since the 2022 U.S. Food and Drug Administration approval of teplizumab to delay disease development. In this article, we discuss four major conceptual and practical issues that emerged as key to further advancement in type 1 diabetes research and therapies. First, collaborative networks leveraging the synergy between the type 1 diabetes research and care community members are key to fostering innovation, know-how, and translation into the clinical arena worldwide. Second, recent clinical trials in presymptomatic stage 2 and recent-onset stage 3 disease have shown the promise, and potential pitfalls, of using immunomodulatory and/or β-cell protective agents to achieve sustained remission or prevention. Third, the increasingly appreciated heterogeneity of clinical, immunological, and metabolic phenotypes and disease trajectories is of critical importance to advance the decision-making process for tailored type 1 diabetes care and therapy. Fourth, the clinical benefits of early diagnosis of β-cell autoimmunity warrant consideration of general population screening for islet autoantibodies, which requires further efforts to address the technical, organizational, and ethical challenges inherent to a sustainable program. Efforts are underway to integrate these four concepts into the future directions of type 1 diabetes research and therapy.

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Conflict of interest statement

Duality of Interest. C.M. serves on the advisory panel for Novo Nordisk, Sanofi, Eli Lilly and Company, Novartis, Boehringer Ingelheim, Roche, Medtronic, Imcyse, Biomea Fusion, and Vertex Pharmaceuticals. Financial compensation for these activities was received by KU Leuven. C.M. is president of the European Association for the Study of Diabetes (EASD). Information on all external support of EASD is available from www.easd.org. C.E.-M. has served on advisory boards related to type 1 diabetes research clinical trial initiatives: Provention Bio, Dompé Pharmaceuticals, Isla Technologies, MaiCell Therapeutics, Avotres, and DiogenX. C.-E.M. has patent 16/291,668, Extracellular Vesicle Ribonucleic Acid (RNA) Cargo as a Biomarker of Hyperglycaemia and Type 1 Diabetes, and has provisional patent 63/285,765, Biomarker for Type 1 Diabetes (PDIA1 as a biomarker of β-cell stress). These activities have not dealt directly with topics covered in this manuscript. D.K. is a scientific advisor for and holds shares in ILTOO Pharma. E.S. has served as a consultant for Sanofi and DRI Healthcare and received compensation for educational lectures from Sanofi, HealthMatters CME, Medscape, and Med Learning Group. S.Y. serves as a consultant for Amarna Therapeutics. R.M. serves on advisory, research and lecture activities for Immunocore, King’s College London, Endotope Biosciences, Human Cell Design, Provention Bio, Novo Nordisk, Sanofi, Lilly, and Amarna Therapeutics. Financial compensation for these activities was received by INSERM, Assistance Publique - Hôpitaux de Paris, and INNODIA iVZW. A.-G.Z. serves on advisory panels and Data Monitoring Committee for Sanofi. No other potential conflicts of interest relevant to this article were reported.

Figures

Figure 1
Figure 1
Opportunities and challenges in studying and optimizing disease modification in type 1 diabetes. Created with BioRender (biorender.com). CGM, continuous glucose monitoring; IPSCs, induced pluripotent stem cells; T1D, type 1 diabetes.
Figure 2
Figure 2
Road map for acceleration of type 1 diabetes research and therapy. Created with BioRender (biorender.com). T1D, type 1 diabetes.
See this image and copyright information in PMC

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