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药物研发面临的挑战及应对新发和耐药真菌病原体的策略。

Drug development challenges and strategies to address emerging and resistant fungal pathogens.

机构信息

a Department of Medicine, Joan and Sanford I Weill Medical College of Cornell University , New York , NY , USA.

b Weill Cornell Medical Center , Transplantation-Oncology Infectious Diseases Program , New York , NY , USA.

出版信息

Expert Rev Anti Infect Ther. 2017 Jun;15(6):577-584. doi: 10.1080/14787210.2017.1328279. Epub 2017 May 15.

DOI:10.1080/14787210.2017.1328279
PMID:28480775
Abstract

Invasive fungal infections represent an expanding threat to public health. The recent emergence of Candida auris, which is often resistant to existing antifungal agents and is associated with a high mortality rate, underscores the urgent need for novel drug development strategies. Areas covered: In this paper, we examine both challenges and opportunities associated with antifungal drug development and explore potential avenues to accelerate the development pipeline, including data sharing, surrogate endpoints, and the role of historical controls in clinical trials. Expert commentary: We review important lessons learned from the study of other rare diseases, including mitochondrial storage diseases and certain forms of cancer that may inform strategies to develop new antifungal agents while highlighting promising new compounds such as SCY-078 for the treatment of invasive fungal infections.

摘要

侵袭性真菌感染对公共健康构成了日益严重的威胁。最近出现的耳念珠菌(Candida auris)通常对现有抗真菌药物具有耐药性,并且死亡率很高,这突显了迫切需要开发新的药物治疗策略。

涵盖领域

本文研究了抗真菌药物开发所面临的挑战和机遇,并探讨了加速开发管道的潜在途径,包括数据共享、替代终点以及历史对照在临床试验中的作用。

专家评论

我们回顾了从研究其他罕见疾病(包括线粒体贮积病和某些形式的癌症)中吸取的重要经验教训,这些经验教训可能为开发新的抗真菌药物提供策略,并强调了一些有前途的新型化合物,如治疗侵袭性真菌感染的 SCY-078。

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