Tigges Marvin, Dräger Sören, Piccini Ilaria, Bieber Katja, Vorobyev Artem, Edelkamp Janin, Bertolini Marta, Ludwig Ralf J
QIMA Life Sciences, QIMA Monasterium GmbH, Münster, Germany.
Department of Dermatology, University Medical Center of the State of Schleswig-Holstein (UKSH), Lübeck, Germany.
Front Immunol. 2025 Mar 17;16:1537428. doi: 10.3389/fimmu.2025.1537428. eCollection 2025.
Pemphigoid diseases constitute a group of organ-specific autoimmune diseases characterized and caused by autoantibodies targeting autoantigens expressed in the skin and mucous membranes. Current therapeutic options are still based on unspecific immunosuppression that is associated with severe adverse events. Biologics, targeting the IL4-pathway or IgE are expected to change the treatment landscape of pemphigoid diseases. However, clinical studies demonstrated that targeting these pathways alone is most likely not sufficient to meet patient and healthcare partitioners expectations. Hence, model systems are needed to identify and validate novel therapeutic targets in pemphigoid diseases. These include pre-clinical animal models, and model systems, hypothesis-driven drug repurposing, as well as exploitation of real-world-data. In this review, we will highlight the medical need for pemphigoid diseases, and in-depth discuss the advantages and disadvantages of the available pemphigoid disease model systems. Ultimately, we discuss how rapid translation can be achieved for the benefit of the patients.
类天疱疮疾病是一组器官特异性自身免疫性疾病,其特征是由针对皮肤和黏膜中表达的自身抗原的自身抗体引起。目前的治疗选择仍然基于与严重不良事件相关的非特异性免疫抑制。靶向白细胞介素4通路或免疫球蛋白E的生物制剂有望改变类天疱疮疾病的治疗格局。然而,临床研究表明,仅靶向这些通路很可能不足以满足患者和医疗保健人员的期望。因此,需要模型系统来识别和验证类天疱疮疾病中的新治疗靶点。这些包括临床前动物模型和模型系统、假设驱动的药物再利用,以及对真实世界数据的利用。在这篇综述中,我们将强调类天疱疮疾病的医疗需求,并深入讨论现有类天疱疮疾病模型系统的优缺点。最终,我们将讨论如何实现快速转化以造福患者。