Emerging Cystic Fibrosis Transmembrane Conductance Regulator Modulators as New Drugs for Cystic Fibrosis: A Portrait of in Vitro Pharmacology and Clinical Translation,ACS Pharmacology & Translational Science

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Emerging Cystic Fibrosis Transmembrane Conductance Regulator Modulators as New Drugs for Cystic Fibrosis: A Portrait of in Vitro Pharmacology and Clinical Translation
ACS Pharmacology & Translational Science ( IF 4.9 ) Pub Date : 2019-10-24 , DOI: 10.1021/acsptsci.9b00060
Drishti P. Ghelani ₁ , Elena K. Schneider-Futschik ₁

Affiliation

Pharmacological correction of the defective ion channel with cystic fibrosis transmembrane conductance regulator (CFTR) has become an attractive approach to therapy directed at the root cause of the life-limiting disease cystic fibrosis (CF). CFTR defects range from absence, misfolding, and resulting degradation to functional defects of the CFTR protein. The discovery and development of the CFTR potentiator ivacaftor was a major break-through in CF therapy and has triggered an enormous incentive for seeking effective modulators such as lumacaftor, tezacaftor or elexacaftor for all patients with CF. A number of emerging CFTR modulators are currently in the development pipeline, and rescue levels of CFTR protein approach a cure for cystic fibrosis. In this review, we identify and characterize all preclinical and clinical emerging CFTR modulators and discuss the in vitro pharmacology, looking at CFTR protein expression and chloride transport and the translation to the clinic. The new emerging CFTR modulators could offer new therapeutic solutions for CF patients.

中文翻译：

新型囊性纤维化跨膜电导调节剂作为囊性纤维化的新药：体外药理学和临床翻译的肖像

用囊性纤维化跨膜电导调节剂（CFTR）对有缺陷的离子通道进行药理校正已成为针对生命有限疾病囊性纤维化（CF）根本原因的一种有吸引力的治疗方法。CFTR缺陷的范围从不存在，错误折叠和导致的降解到CFTR蛋白的功能缺陷。CFTR增效剂依伐卡托的发现和开发是CF治疗的一项重大突破，并激发了巨大的动力，希望为所有CF患者寻求有效的调节剂，例如lumacaftor，tezacaftor或elexacaftor。当前正在开发中的许多新兴CFTR调节剂中，CFTR蛋白的抢救水平已接近治愈囊性纤维化的方法。在这篇评论中，在体外药理学中，研究CFTR蛋白的表达和氯离子的转运以及到临床的翻译。新兴的CFTR调节剂可以为CF患者提供新的治疗方案。

更新日期：2019-10-25

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