Factors impeding the discovery of an intervention-based treatment for type 1 diabetes
Factors impeding the discovery of an intervention-based treatment for type 1 diabetes
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DOI：
10.1111/cei.12656
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发表时间：
2016-01-01
影响因子：
4.6
通讯作者：
Atkinson, M. A.
中科院分区：
文献类型：
--
作者：
von Herrath, M. G.;Korsgren, O.;Atkinson, M. A.
1 型糖尿病 (T1D) 是影响儿童和成人的最常见和最严重的慢性疾病之一。这种疾病的病因仍不清楚，迄今为止还没有“真正”的治愈方法。事实上，将葡萄糖代谢控制到最佳程度的外源性胰岛素替代疗法仍然是当前的护理标准。然而，尽管最近出现了一系列旨在加强疾病管理的真正令人印象深刻的改进（例如胰岛素类似物、连续血糖监测、胰岛素泵），但绝大多数患者仍然难以达到建议的目标 HbA1C 水平（< 7.0%）。由于疾病管理不理想，太多 T1D 患者出现疾病相关并发症的风险增加，例如肾病、神经病、视网膜病以及低血糖。因此，迫切需要新的治疗方式为 T1D 患者带来“真正的”治愈（疾病预防/疾病逆转）。在这里，我们考虑共同构成 T1D 研究的主要障碍的问题，以确定预防和/或治愈该疾病的方法。我们首先讨论人类 T1D 胰腺研究中产生的新见解；研究结果可能至少部分解释了为什么以前寻求疾病预防/逆转的干预措施没有产生足够的益处。然后我们寻求可以优化未来临床试验结果的建议。最后，我们关注对全球 T1D 研究界的建议；我们认为这些信息有可能提高我们找到难以捉摸的 T1D“治愈方法”的机会。
Type 1 diabetes (T1D) is one of the most common and severe chronic diseases affecting both children and adults. The aetiology of the disease remains unknown, and thus far no 'true' cure for those affected is available. Indeed, exogenous insulin replacement therapy to manage glucose metabolism to the best degree possible remains the current standard of care. However, despite a recent array of truly impressive improvements designed to enhance disease management (e.g. insulin analogues, continuous glucose monitoring, insulin pumps), it is still difficult for the vast majority of patients to reach recommended target HbA1C levels (< 7.0%). As a result of suboptimal disease management, far too many patients with T1D have an increased risk for disease-associated complications such as nephropathy, neuropathy and retinopathy, as well as hypoglycaemia. New treatment modalities are therefore needed urgently to bring a 'true' cure (disease prevention/disease reversal) to patients with T1D. Here we consider issues that collectively pose a major stumbling block in T1D research with respect to identifying a means to prevent and/or cure the disease. We begin this Perspective by discussing new insights emanating from studies of the pancreas in human T1D; findings which may, at least in part, explain why previous interventions seeking disease prevention/reversal have yielded insufficient benefit. We then turn to suggestions that could optimise the outcome of future clinical trials. Finally, we direct attention to recommendations for the global T1D research community; messages we deem to have the potential to improve our chances of finding the elusive T1D 'cure'.