Department of Medicine, University of Alabama at Birmingham, 1819 University Boulevard (MCLM 768), Birmingham, AL 35294, USA.
Thorax. 2012 Oct;67(10):882-90. doi: 10.1136/thoraxjnl-2012-202550.
Cystic fibrosis (CF), the most common life-shortening genetic disorder in Caucasians, affects approximately 70 000 individuals worldwide. In 1998, the Cystic Fibrosis Foundation (CFF) launched the CF Therapeutics Development Network (CF-TDN) as a central element of its Therapeutics Development Programme. Designed to accelerate the clinical evaluation of new therapies needed to fulfil the CFF mission to control and cure CF, the CF-TDN has conducted 75 clinical trials since its inception, and has contributed to studies as varied as initial safety and proof of concept trials to pivotal programmes required for regulatory approval. This review highlights recent and significant research efforts of the CF-TDN, including a summary of contributions to studies involving CF transmembrane conductance regulator (CFTR) modulators, airway surface liquid hydrators and mucus modifiers, anti-infectives, anti-inflammatories, and nutritional therapies. Efforts to advance CF biomarkers, necessary to accelerate the therapeutic goals of the network, are also summarised.
囊性纤维化（CF）是白种人最常见的缩短寿命的遗传疾病，全球约有 70000 人受其影响。1998 年，囊性纤维化基金会（CFF）发起了囊性纤维化治疗开发网络（CF-TDN），作为其治疗开发计划的核心要素。该网络旨在加速临床评估新疗法，以实现 CFF 控制和治愈 CF 的使命，自成立以来，已经进行了 75 项临床试验，并为从初步安全性和概念验证试验到监管批准所需的关键项目的研究做出了贡献。这篇综述强调了 CF-TDN 的最新和重要的研究成果，包括对涉及 CF 跨膜电导调节剂（CFTR）调节剂、气道表面液体水合作用和黏液修饰剂、抗感染药物、抗炎药和营养治疗的研究的贡献总结。还总结了推进 CF 生物标志物的努力，这对于加速网络的治疗目标是必要的。
