Department of Pharmaceutics, Ernest Mario School of Pharmacy, Rutgers, The State University of New Jersey , Piscataway, NJ , USA.
J Drug Target. 2013 Dec;21(10):914-25. doi: 10.3109/1061186X.2013.829078. Epub 2013 Aug 27.
Cystic fibrosis (CF) is an autosomal recessive monogenetic disease that afflicts nearly 70,000 patients worldwide. The mutation results in the accumulation of viscous mucus in multiple organs especially in the lungs, liver and pancreas. High associated morbidity and mortality is caused by CF due to the lack of effective therapies. It is widely accepted that morbidity and mortality caused by CF is primarily due to the respiratory manifestations of the disease. Consequently, several approaches were recently developed for treatment of lung complications of CF. However, the lack of effective methods for delivery and especially targeted delivery of therapeutics specifically to lung tissues and cells limits the efficiency of the therapy. Local pulmonary delivery of therapeutics has two major advantages over systemic application. First, it enhances the accumulation of therapeutics specifically in the lungs and therefore increases the efficiency of the treatment. Second, local lung delivery substantially prevents the penetration of the delivered drug into the systemic circulation limiting adverse side effects of the treatment on other organs and tissues. This review is focused on different approaches to the treatment of respiratory manifestations of CF as well as on methods of pulmonary delivery of therapeutics.
囊性纤维化(CF)是一种常染色体隐性单基因疾病,影响全球近 70,000 名患者。该突变导致粘性粘液在多个器官中积累,特别是在肺部、肝脏和胰腺中。由于缺乏有效的治疗方法,CF 导致高发病率和死亡率。人们普遍认为,CF 引起的发病率和死亡率主要是由于该疾病的呼吸道表现。因此,最近开发了几种治疗 CF 肺部并发症的方法。然而,由于缺乏有效的方法将治疗药物递送至肺部组织和细胞,特别是靶向递送至这些部位,限制了治疗的效率。与全身应用相比,治疗药物的局部肺部给药有两个主要优势。首先,它增强了治疗药物在肺部的积聚,从而提高了治疗效率。其次,局部肺部给药可大大防止所给药物渗透到全身循环中,限制了治疗对其他器官和组织的不良副作用。本文综述了治疗 CF 呼吸道表现的不同方法以及治疗药物的肺部给药方法。