1 型糖尿病大挑战:有史以来最大的一笔 5000 万英镑投资和全英国的合作思考可能产生的变革性影响。

IF 3.2 3区 医学 Q2 ENDOCRINOLOGY & METABOLISM Diabetic Medicine Pub Date : 2024-06-04 DOI:10.1111/dme.15376
Elizabeth Robertson, Jessica Sutcliffe, Rachel Connor, Simon R. Heller
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引用次数: 0

摘要

十八个月前,举办 1 型糖尿病大型挑战赛 (T1DGC) 还只是我们战略计划中的一个愿望。我们看到了机会--英国 1 型糖尿病研究人员群体的实力、为实现治愈而进行的令人兴奋的研究进展、英国国家健康与护理研究所开发的临床基础设施、单一的国家医疗服务,以及英国人口的规模和多样性,这些都为试用任何新的创新技术做好了准备。我们所缺少的是能够让我们在英国实现这一潜力的巨额资金。史蒂夫-摩根基金会具有远见、决心和资源,能够为英国 1 型糖尿病领域带来变革性和颠覆性的变化。基金会选定了三个主题:细胞疗法、免疫洞察力和新型胰岛素。本评论将重点介绍细胞疗法主题迄今为止取得的进展。从一开始,史蒂夫和莎莉-摩根就决定必须将合作作为新投资的核心,将英国的 1 型血群体聚集在一起,推动大规模创新,以取代或再生细胞疗法,使我们更接近治愈。史蒂夫-摩根基金会、英国 JDRF 和英国糖尿病协会合作的第一步是邀请国际专家加入科学顾问小组 (SAP),就实现 1 型糖尿病重大挑战主题的最佳方法提供独立建议。Simon Heller 教授是 SAP 的独立主席。马蒂亚斯-海布鲁克教授(Matthias Hebrok)担任β细胞疗法SAP副主席,豪尔赫-费雷尔(Jorge Ferrer)教授、道格-梅尔顿(Doug Melton)教授、洛伦佐-皮埃蒙特(Lorenzo Piemonti)教授、洛里-苏塞尔(Lori Sussel)教授和唐启智教授为其提供支持。这些国际性的独立建议对于确保我们专注于能够实现宏伟挑战目标的研究领域和资助计划至关重要。1型糖尿病患者的意见贯穿了整个大挑战活动,包括确定优先事项、设计研究方案和审查资助申请,由1型糖尿病专家组成的经验丰富的专家小组将监督不断扩大的研究组合,以确保其关注1型糖尿病患者的需求。维多利亚-萨利姆(Victoria Salem)博士、詹姆斯-坎特利(James Cantley)博士和萨拉-理查德森(Sarah Richardson)教授正在合作开展创新项目,开发保护移植β细胞的软植入物、再生1型糖尿病患者体内的β细胞以及了解不同患者的免疫攻击有何不同。他们在 2024 年 4 月英国糖尿病专业会议上的研讨会上发表的《糖尿病医学》(Diabetic Medicine)论文将展示这一合作研究金项目的潜力。2023年底,我们宣布了四项重要的、新的、互补的转化计划,将来自英国和欧洲的跨学科研究团队聚集在一起,为干细胞衍生的胰岛细胞(SC-β)设计利于存活的合成微环境,探索两种增量素受体是否是β细胞精准治疗的分子靶点,开发微颗粒以促进移植后胰岛的功能,以及评估许旺细胞及其分泌产物改善SC-β细胞成熟和功能的潜力。我们计划在明年年初发表一篇论文,展示这些转化成果。我们还没有为研究界提供稳定的细胞,也没有为新兴细胞疗法提供快速的临床试验网络。我们将以多学科思维为核心,颁发高风险、高回报的新基金,通过将目前参与 T1DGC 的数百名研究人员聚集在一起,我们希望能继续加快这一转变的步伐,使我们在为 1 型糖尿病患者开发治疗方案方面取得快速进展的梦想从愿望变为现实。最后,我们首先要感谢史蒂夫-摩根基金会(Steve Morgan Foundation)对英国 1 型糖尿病社区投下的信任票,感谢研究界对这一千载难逢的机会做出的积极响应,感谢在这一激动人心的旅程中加入我们的新兴合作伙伴。研究工作任重而道远,也不能保证一定成功,但我们比 2020 年时想象的要走得更远。
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The type 1 diabetes grand challenge: The potential for transformative impact from the largest ever investment, £50 million and collaborative thinking across the UK

Eighteen months ago, the opportunity to run a type 1 diabetes grand challenge (T1DGC) was just an aspiration in our strategic plans. We could see the opportunity—the strength of the type 1 researcher community in the UK, the exciting research developments in moving towards a cure, the clinical infrastructure developed by the National Institute of Health and Care Research, the single national health service and the size and diversity of the UK population potentially ready to trial whatever new innovations are in the pipeline. The missing piece was significant funds that enable us to deliver on this potential in the UK. The Steve Morgan Foundation had the foresight, determination and resources to enact this transformative and disruptive change in the UK type 1 landscape. Three themes were selected: cell therapies, immune insights and novel insulins. This commentary focuses on progress to date with the cell therapies theme. As the T1DGC progresses, a series of Diabetic Medicine papers will update on new developments.

From the outset, Steve and Sally Morgan determined that collaboration must be at the heart of new investments, bringing together the UK type 1 community to drive forward innovations at scale to replace or regenerate cell therapies, moving us closer towards a cure. The first step in this partnership between the Steve Morgan Foundation, JDRF UK and Diabetes UK was to invite international experts to join Scientific Advisory Panels (SAPs) to provide independent advice on the best approaches to deliver the type 1 grand challenge themes. Professor Simon Heller is the independent chair of the SAPs. Professor Matthias Hebrok is the vice-chair of the beta cell therapies SAPs supported by Professor Jorge Ferrer, Professor Doug Melton, Professor Lorenzo Piemonti, Professor Lori Sussel and Professor Qizhi Tang. Engaging this international, independent advice has been critical in ensuring we are focusing on areas of research and funding schemes that are going to realise the ambition of the grand challenge. Input from people living with type 1 diabetes has been embedded throughout the grand challenge from setting the priorities, shaping the study designs and reviewing the grant applications, and a type 1 diabetes panel of experts by experience will oversee the growing portfolio of research to ensure it is focused on the needs of people living with type 1.

Working at speed, we appointed three Senior Research Fellows within 6 months of the partnership going live in April 2022. Dr Victoria Salem, Dr James Cantley and Professor Sarah Richardson are working together on innovative programmes to develop soft implants to protect transplanted beta cells, regenerate beta cells in people living with type 1 and understand how immune attacks differ between people with the condition. A subsequent Diabetic Medicine paper from their symposium at the Diabetes UK Professional Conference in April 2024 will showcase the potential of this collaborative fellowship programme.

The imperative to move therapies faster towards clinical care was at the heart of the second funding call focused on collaborative, multidisciplinary translational awards. At the end of 2023, we announced four major, new, complimentary, translational programmes bringing cross-disciplinary teams of researchers together from across the UK and Europe to engineer prosurvival synthetic microenvironments for stem-cell derived islet cells (SC-β), explore if two incretin receptors are molecular targets for precision beta cell therapy, develop microparticles to promote the function of islets following transplantation and assess the potential of Schwann cells and their secretory products to improve SC-β cell maturation and function. A future paper showcasing these translational awards is planned for early next year.

However, we are only at the beginning of this journey. We have yet to deliver stabilized cells for the research community and a rapid clinical trial network for emerging cell therapies. We will be awarding high-risk, high-reward new grants with multidisciplinary thinking at their heart, and by bringing the now hundreds of researchers engaged in the T1DGC together, we hope to continue at pace this transformation, moving our dreams of rapid progress in developing curative options for people living with type 1 from aspirations to much nearer to reality.

To conclude, we are indebted first and foremost to the Steve Morgan Foundation for this vote of confidence in the UK type 1 diabetes community, to the research community, which has really responded to this once-in-a-lifetime opportunity and to emerging partners who are joining us on this exciting journey. There is much to do and no guarantee of success as ever with research, but we are so much farther ahead than we could have imagined back in 2020.

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来源期刊
Diabetic Medicine
Diabetic Medicine 医学-内分泌学与代谢
CiteScore
7.20
自引率
5.70%
发文量
229
审稿时长
3-6 weeks
期刊介绍: Diabetic Medicine, the official journal of Diabetes UK, is published monthly simultaneously, in print and online editions. The journal publishes a range of key information on all clinical aspects of diabetes mellitus, ranging from human genetic studies through clinical physiology and trials to diabetes epidemiology. We do not publish original animal or cell culture studies unless they are part of a study of clinical diabetes involving humans. Categories of publication include research articles, reviews, editorials, commentaries, and correspondence. All material is peer-reviewed. We aim to disseminate knowledge about diabetes research with the goal of improving the management of people with diabetes. The journal therefore seeks to provide a forum for the exchange of ideas between clinicians and researchers worldwide. Topics covered are of importance to all healthcare professionals working with people with diabetes, whether in primary care or specialist services. Surplus generated from the sale of Diabetic Medicine is used by Diabetes UK to know diabetes better and fight diabetes more effectively on behalf of all people affected by and at risk of diabetes as well as their families and carers.”
期刊最新文献
Factors influencing diabetes treatment satisfaction in the INtegrating DEPrEssioN and Diabetes treatmENT randomized clinical trial: A multilevel model analysis. Feasibility of caregiver diabetes distress screening in routine clinical care of youth with type 1 diabetes. Trends in rates of hospitalisation for infection in people with diabetes and the general population. Very-low-calorie diet-based intensive lifestyle intervention for remission of type 2 diabetes: Real-world experience in a South Asian population. 'We're taught green is good': Perspectives on time in range and time in tight range from youth with type 1 diabetes, and parents of youth with type 1 diabetes.
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