从胰岛素替代到治疗 1 型糖尿病的潜在自身免疫性疾病。

IF 2.6 3区 医学 Q3 ENDOCRINOLOGY & METABOLISM
Jantje Weiskorn, Thomas Danne
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引用次数: 0

摘要

目前,1 型糖尿病的治疗模式已从胰岛素替代疗法转变为治疗潜在的自身免疫性疾病。Teplizumab是一种人源化单克隆抗CD3抗体,是美国食品及药物管理局批准的第一种治疗临床前2型糖尿病的药物。目前,针对 TNF alpha 的单克隆抗体 Golimumab、酪氨酸激酶抑制剂 baricitinib 或抗 CD40 配体的单克隆抗体 frexalimab 等药物的研究仍在进行中。已用于其他适应症的药物,如钙通道阻滞剂维拉帕米、用于实体器官移植的抗体制剂抗胸腺细胞球蛋白(ATG)、用于治疗 2 型糖尿病和肥胖症的胰高血糖素样肽-1 激动剂,或抗病毒药物pleconaril 和利巴韦林等,在保护β细胞功能方面已显示出积极的效果。目前正在开发新的疗法,以阻止自身免疫并恢复第一至第三阶段的β细胞,通过诱导性多能干细胞取代β细胞功能,已显示出对长期1型糖尿病患者的血糖控制和胰岛素独立性,尽管同时伴有免疫抑制。开发临床试验网络(如 INNODIA)或研究平台(如 EDENT1FI)以在早期阶段阻止 1 型糖尿病为目标的多中心跨国计划将有助于研究这些新策略。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Moving from Insulin Substitution to the Treatment of the Underlying Autoimmune Disease in Type 1 Diabetes.

Currently, a paradigm change occurs in type 1 diabetes from insulin substitution to the treatment of the underlying autoimmune disease. Teplizumab, a humanized monoclonal anti-CD3 antibody, is the first FDA-approved disease-modifying treatment of preclinical stage 2 diabetes. Research of drugs like golimumab, a monoclonal antibody specific for TNF alpha, baricitinib, a tyrosine kinase inhibitor, or frexalimab, a monoclonal antibody against the CD40 ligand, is still ongoing. Repurposing drugs that have been used in other indications like the calcium channel blocker verapamil, antithymocyte globulin (ATG), an antibody preparation used in solid organ transplantation, glucagon-like peptide-1 agonists utilized in type 2 diabetes and obesity, or the antiviral drugs pleconaril and ribavirin have shown positive effects in preserving beta-cell function. While new therapies to halt autoimmunity and restore beta cells in stages one to three are being developed, replacing beta-cell function via inducible pluripotent stem cells have shown glucose control and insulin independence in long-standing type 1 diabetes, albeit with concomitant immunosuppression. Multicenter multinational initiatives developing a clinical trial network like INNODIA or a research platform with the goal of stopping type 1 diabetes in its early stages like EDENT1FI will be instrumental to study these new strategies.

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来源期刊
Hormone Research in Paediatrics
Hormone Research in Paediatrics ENDOCRINOLOGY & METABOLISM-PEDIATRICS
CiteScore
4.90
自引率
6.20%
发文量
88
审稿时长
4-8 weeks
期刊介绍: The mission of ''Hormone Research in Paediatrics'' is to improve the care of children with endocrine disorders by promoting basic and clinical knowledge. The journal facilitates the dissemination of information through original papers, mini reviews, clinical guidelines and papers on novel insights from clinical practice. Periodic editorials from outstanding paediatric endocrinologists address the main published novelties by critically reviewing the major strengths and weaknesses of the studies.
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