Lübeck Institute of Experimental Dermatology, University of Lübeck, 23562 Lübeck, Germany.
Acta Derm Venereol. 2020 Feb 12;100(5):adv00055. doi: 10.2340/00015555-3400.
Pemphigoid diseases are organ-specific autoimmune diseases of the skin and/or mucous membranes. They are caused by autoantibodies targeting adhesion molecules located at the dermal-epidermal junction. While the diagnostics of pemphigoid diseases and insights into their pathogenesis have improved significantly, the development of novel treatments that are effective and safe remains an unmet medical need. However, numerous pre-clinical studies and early clinical trials have recently been launched. This review summarizes some pathways leading to drug development in pemphigoid diseases, namely: (i) hypothesis-driven drug development; (ii) omics-based drug development; (iii) drug repurposing; (iv) screening-based drug development; and (v) drug development based on careful clinical observations. Ultimately, it is hoped that this will lead to personalized and curative treatments.
天疱疮病是一种皮肤和/或黏膜的器官特异性自身免疫性疾病。它们是由靶向位于表皮-真皮交界处的黏附分子的自身抗体引起的。虽然天疱疮病的诊断和对其发病机制的认识有了显著提高，但开发有效和安全的新型治疗方法仍然是未满足的医疗需求。然而，最近已经开展了许多临床前研究和早期临床试验。这篇综述总结了一些导致天疱疮病药物开发的途径，即：（i）假设驱动的药物开发；（ii）基于组学的药物开发；（iii）药物再利用；（iv）基于筛选的药物开发；和（v）基于仔细临床观察的药物开发。最终，人们希望这将导致个性化和治愈性的治疗。
