Division of Pulmonary and Critical Care Medicine, University of Arkansas for Medical Sciences, Little Rock, AR 72205, USA.
Ther Adv Respir Dis. 2010 Jun;4(3):177-85. doi: 10.1177/1753465810371107.
The life expectancy of people with cystic fibrosis (CF), a lethal inherited disease, has been greatly extended by advances in therapy. Currently, there are a number of potential drugs for treatment of CF lung disease in clinical trials. These therapies are targeted at all points in the pathogenesis of lung disease, from gene transfer to drugs that treat mucus, infection and inflammation in the airways. An exciting development is that of modulation of the abnormal protein that causes CF, the cystic fibrosis transmembrane conductance regulator (CFTR), where drugs are targeted at specific defects in CFTR transcription, processing or functioning. Inhaled therapies are being developed to augment airway surface liquid height, either by modulating the abnormal ion channel function in the airway epithelial cell or by rehydrating with osmotic agents. Anti-inflammatory therapy is also of great interest in CF and there are several candidate drugs in clinical trials. A number of antibacterial agents formulated for inhalation are at various stages of study or newly approved, which should improve options for chronic management of airway infection. Hopefully, many of these potential therapies will come to market and will further extend the life expectancy of people with CF.
囊性纤维化（CF）是一种致命的遗传性疾病，患者的预期寿命通过治疗手段的进步得到了极大延长。目前，有许多针对 CF 肺部疾病的潜在治疗药物正在临床试验中。这些疗法针对肺部疾病发病机制的各个环节，从基因转移到治疗气道黏液、感染和炎症的药物。一个令人兴奋的进展是针对导致 CF 的异常蛋白——囊性纤维化跨膜电导调节因子（CFTR）的调节，药物靶向 CFTR 转录、加工或功能的特定缺陷。正在开发吸入疗法来增加气道表面液体高度，方法是调节气道上皮细胞中异常的离子通道功能或用渗透压剂进行再水化。在 CF 中，抗炎治疗也非常有意义，有几种候选药物正在临床试验中。许多为吸入而配制的抗菌剂处于不同的研究阶段或刚获得批准，这将改善慢性气道感染管理的选择。希望这些潜在疗法中的许多都能推向市场，并进一步延长 CF 患者的预期寿命。
