Emerging concepts and success stories in type 1 diabetes research: a roadmap for a bright future

IF 6.2 1区 医学 Q1 ENDOCRINOLOGY & METABOLISM Diabetes Pub Date : 2024-10-24 DOI:10.2337/db24-0439
Roberto Mallone, Emily Sims, Peter Achenbach, Chantal Mathieu, Alberto Pugliese, Mark Atkinson, Sanjoy Dutta, Carmella Evans-Molina, David Klatzmann, Anne Koralova, S. Alice Long, Lut Overbergh, Teresa Rodriguez-Calvo, Anette-Gabriele Ziegler, Sylvaine You
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Abstract

Type 1 diabetes treatment stands at a crucial and exciting crossroad since the 2022 U.S. Food and Drug Administration (FDA) approval of teplizumab to delay disease development. In this Perspective article, we discuss four major conceptual and practical issues that emerged as key to further advance 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 beta-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 beta-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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1 型糖尿病研究的新概念和成功案例:光明未来的路线图
自 2022 年美国食品和药物管理局 (FDA) 批准替普利珠单抗用于延缓疾病发展以来,1 型糖尿病的治疗就站在了一个关键且令人兴奋的十字路口。在这篇 "视角 "文章中,我们将讨论四个主要的概念和实践问题,它们是进一步推动 1 型糖尿病研究和治疗的关键。首先,利用 1 型糖尿病研究和护理社区成员之间的协同作用建立合作网络是促进创新、专有技术并将其转化为全球临床应用的关键。其次,最近对无症状的 2 期和近期发病的 3 期疾病进行的临床试验表明,使用免疫调节和/或β细胞保护剂来实现持续缓解或预防,既有希望,也有潜在的隐患。第三,临床、免疫学和代谢表型及疾病轨迹的异质性日益受到重视,这对于推进量身定制的 1 型糖尿病护理和治疗决策过程至关重要。第四,早期诊断β细胞自身免疫的临床益处值得考虑对普通人群进行胰岛自身抗体筛查,这需要进一步努力解决可持续计划所固有的技术、组织和伦理挑战。我们正在努力将这四个概念融入 1 型糖尿病研究和治疗的未来方向。
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来源期刊
Diabetes
Diabetes 医学-内分泌学与代谢
CiteScore
12.50
自引率
2.60%
发文量
1968
审稿时长
1 months
期刊介绍: Diabetes is a scientific journal that publishes original research exploring the physiological and pathophysiological aspects of diabetes mellitus. We encourage submissions of manuscripts pertaining to laboratory, animal, or human research, covering a wide range of topics. Our primary focus is on investigative reports investigating various aspects such as the development and progression of diabetes, along with its associated complications. We also welcome studies delving into normal and pathological pancreatic islet function and intermediary metabolism, as well as exploring the mechanisms of drug and hormone action from a pharmacological perspective. Additionally, we encourage submissions that delve into the biochemical and molecular aspects of both normal and abnormal biological processes. However, it is important to note that we do not publish studies relating to diabetes education or the application of accepted therapeutic and diagnostic approaches to patients with diabetes mellitus. Our aim is to provide a platform for research that contributes to advancing our understanding of the underlying mechanisms and processes of diabetes.
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