Our official English website, www.x-mol.net, welcomes your
feedback! (Note: you will need to create a separate account there.)
Emerging concepts and success stories in type 1 diabetes research: a roadmap for a bright future
Diabetes ( IF 6.2 ) 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
Diabetes ( IF 6.2 ) 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
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.
中文翻译:
1 型糖尿病研究的新兴概念和成功案例:光明未来的路线图
自 2022 年美国食品和药物管理局 (FDA) 批准 teplizumab 以延缓疾病发展以来,1 型糖尿病治疗正处于一个关键且令人兴奋的十字路口。在这篇 Perspective 文章中,我们讨论了四个主要的概念和实际问题,这些问题是进一步推进 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的协作网络是促进创新、专业知识和转化为全球临床领域的关键。其次,最近针对症状前 2 期和近期发病的 3 期疾病的临床试验表明,使用免疫调节和/或 β 细胞保护剂实现持续缓解或预防的前景和潜在陷阱。第三,临床、免疫学和代谢表型和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的决策过程至关重要。第四,早期诊断 β 细胞自身免疫的临床益处值得考虑对一般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固有的技术、组织和伦理挑战。正在努力将这四个概念整合到 1 型糖尿病研究和治疗的未来方向中。
更新日期:2024-10-24
中文翻译:
1 型糖尿病研究的新兴概念和成功案例:光明未来的路线图
自 2022 年美国食品和药物管理局 (FDA) 批准 teplizumab 以延缓疾病发展以来,1 型糖尿病治疗正处于一个关键且令人兴奋的十字路口。在这篇 Perspective 文章中,我们讨论了四个主要的概念和实际问题,这些问题是进一步推进 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的协作网络是促进创新、专业知识和转化为全球临床领域的关键。其次,最近针对症状前 2 期和近期发病的 3 期疾病的临床试验表明,使用免疫调节和/或 β 细胞保护剂实现持续缓解或预防的前景和潜在陷阱。第三,临床、免疫学和代谢表型和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的决策过程至关重要。第四,早期诊断 β 细胞自身免疫的临床益处值得考虑对一般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固有的技术、组织和伦理挑战。正在努力将这四个概念整合到 1 型糖尿病研究和治疗的未来方向中。