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治疗斑秃的新药发现的创新策略:针对免疫系统。

Innovative strategies for the discovery of new drugs against alopecia areata: taking aim at the immune system.

机构信息

Department of Dermatology, The Second Affiliated Hospital of Guangdong Medical University, Zhanjiang, China.

Guangdong Medical University, Zhanjiang, China.

出版信息

Expert Opin Drug Discov. 2024 Nov;19(11):1321-1338. doi: 10.1080/17460441.2024.2409660. Epub 2024 Oct 3.

Abstract

INTRODUCTION

The autoimmune hair loss condition alopecia areata (AA) exacts a substantial psychological and socioeconomic toll on patients. Biotechnology companies, dermatology clinics, and research institutions are dedicated to understanding AA pathogenesis and developing new therapeutic approaches. Despite recent efforts, many knowledge gaps persist, and multiple treatment development avenues remain unexplored.

AREAS COVERED

This review summarizes key AA disease mechanisms, current therapeutic methods, and emerging treatments, including Janus Kinase (JAK) inhibitors. The authors determine that innovative drug discovery strategies for AA are still needed due to continued unmet medical needs and the limited efficacy of current and emerging therapeutics. For prospective AA treatment developers, the authors identify the pre-clinical disease models available, their advantages, and limitations. Further, they outline treatment development opportunities that remain largely unmapped.

EXPERT OPINION

While recent advancements in AA therapeutics are promising, challenges remain, including the lack of consistent treatment efficacy, long-term use and safety issues, drug costs, and patient compliance. Future drug development research should focus on patient stratification utilizing robust biomarkers of AA disease activity and improved quantification of treatment response. Investigating superior modes of drug application and developing combination therapies may further improve outcomes. Spirited innovation will be needed to advance more effective treatments for AA.

摘要

简介

自身免疫性脱发疾病斑秃(AA)给患者带来了巨大的心理和社会经济负担。生物技术公司、皮肤科诊所和研究机构致力于了解 AA 的发病机制并开发新的治疗方法。尽管最近进行了许多努力,但仍存在许多知识空白,并且有多种治疗开发途径尚未探索。

涵盖领域

本文综述了 AA 的关键疾病机制、当前的治疗方法和新兴治疗方法,包括 Janus 激酶(JAK)抑制剂。作者确定,由于持续存在的未满足的医疗需求和当前及新兴疗法的疗效有限,仍然需要创新的 AA 药物发现策略。对于有前景的 AA 治疗开发人员,作者确定了可用的临床前疾病模型及其优缺点。此外,他们还概述了仍在很大程度上未开发的治疗开发机会。

专家意见

尽管 AA 治疗的最新进展令人鼓舞,但仍存在挑战,包括缺乏一致的治疗效果、长期使用和安全性问题、药物成本和患者依从性。未来的药物开发研究应侧重于利用 AA 疾病活动的强大生物标志物和改善治疗反应的定量来进行患者分层。探索更优的药物应用模式和开发联合疗法可能会进一步改善结果。需要有创新性的精神来推进更有效的 AA 治疗方法。

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