Department of Industrial and Physical Pharmacy, College of Pharmacy, Purdue University, 575 Stadium Mall Drive, West Lafayette, IN 47907, USA.
Expert Opin Drug Deliv. 2011 Apr;8(4):451-66. doi: 10.1517/17425247.2011.561310. Epub 2011 Mar 5.
Cystic fibrosis (CF) is a multisystem genetic disorder, which usually results in significant respiratory dysfunction. At present there is no cure for CF, but advances in pharmacotherapy have gradually increased the life expectancy of CF patients. As many drugs used in the therapy of CF are delivered by inhalation, the demand for effective and convenient inhalational CF drug formulations will grow as CF patients live longer. Knowledge of the current limitations in inhalational CF drug delivery is critical in identifying new opportunities and designing rational delivery strategies.
This review discusses current and emerging therapeutic agents for CF therapy, selected physiological challenges to effective inhalational medication delivery, and various approaches to overcoming these challenges. The reader will find an integrated view of the known inhalational drug delivery challenges and the rationales for recent investigational inhalational drug formulations.
An ideal drug/gene delivery system to CF airways should overcome the tenacious sputum, which presents physical, chemical and biological barriers to effective transport of therapeutic agents to the targets and various cellular challenges.
囊性纤维化(CF)是一种多系统遗传性疾病,通常会导致严重的呼吸功能障碍。目前,CF 尚无治愈方法,但药物治疗的进步逐渐延长了 CF 患者的预期寿命。由于 CF 治疗中使用的许多药物都是通过吸入给药的,因此随着 CF 患者寿命的延长,对有效且方便的吸入 CF 药物制剂的需求将会增加。了解吸入 CF 药物输送的当前局限性对于发现新的机会和设计合理的输送策略至关重要。
本文综述了 CF 治疗的当前和新兴治疗药物、有效吸入药物输送面临的选定生理挑战以及克服这些挑战的各种方法。读者将全面了解已知的吸入式药物输送挑战以及最近的研究性吸入式药物制剂的基本原理。
理想的 CF 气道药物/基因传递系统应克服粘稠的痰液,因为痰液对治疗剂向靶标和各种细胞挑战的有效输送构成了物理、化学和生物学障碍。