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新型肾脏药物研发面临慢性肾病

New renal drug development to face chronic renal disease.

作者信息

Moll Solange, Meier Matthias, Formentini Ivan, Pomposiello Silvia, Prunotto Marco

机构信息

University Hospital Geneva, Institute of Clinical Pathology , Geneva , Switzerland.

出版信息

Expert Opin Drug Discov. 2014 Dec;9(12):1471-85. doi: 10.1517/17460441.2014.956075. Epub 2014 Sep 6.

Abstract

INTRODUCTION

For the first time in 2012, chronic kidney diseases were added to WHO disease list because of their impact on morbidity/mortality and their substantial impact on the cost to the health care system. Although representing a clear unmet medical need and a huge business case for the pharmaceutical industry, its clinical treatment still mainly relies on drugs invented in the 1980s used for controlling blood pressure.

AREAS COVERED

In this review, the authors aim to elucidate why renal drug development is feasible today. The article provides a particular focus on the treatments that target the pathways involved in inflammation, fibrosis and the core mechanisms driving the vicious cycle responsible for disease progression and organ function loss.

EXPERT OPINION

Currently, it is plausible to develop effective therapeutics for renal diseases with a plethora of approaches available for their development at a preclinical and clinical level. Furthermore, the relevance of biomarkers and the use of surrogate rare disease indications as proof of mechanism for faster and/or smaller clinical development are now possible; and these developments could revolutionize the way we treat renal disease in the future.

摘要

引言

2012年,慢性肾脏病首次被列入世界卫生组织疾病清单,因其对发病率/死亡率有影响,且对医疗保健系统成本有重大影响。尽管这代表着明显未被满足的医疗需求,对制药行业来说是个巨大商机,但其临床治疗仍主要依赖于20世纪80年代发明的用于控制血压的药物。

涵盖领域

在本综述中,作者旨在阐明为何如今肾脏药物研发是可行的。本文特别关注针对炎症、纤维化以及驱动疾病进展和器官功能丧失恶性循环的核心机制所涉及途径的治疗方法。

专家观点

目前,利用临床前和临床层面大量可用的研发方法来开发针对肾脏疾病的有效治疗药物是可行的。此外,生物标志物的相关性以及使用替代罕见病适应症作为机制证据以实现更快和/或规模更小的临床开发现在已成为可能;这些进展可能会彻底改变我们未来治疗肾脏疾病的方式。

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