Moni Sivakumar S, Al Basheer Asmaa
Department of Pharmaceutics, College of Pharmacy, Jazan University, Jazan 45142, Saudi Arabia.
Saudi Pharm J. 2022 Dec;30(12):1736-1747. doi: 10.1016/j.jsps.2022.10.002. Epub 2022 Oct 8.
Cystic fibrosis (CF) is a genetic disease that affects the exocrine glands and is caused by cystic fibrosis transmembrane conductance regulator gene (CFTR) mutations. Lung disease is the leading cause of morbidity in patients. Target-specific treatment of CF has been achieved using monoclonal antibodies (mAbs). The purpose of this article is to discuss the possibility of treating CF with mAbs through their significant target specificity. We searched electronic databases in Web of Science, PubMed, EMBASE, Scopus, and Google Scholar from 1984 to 2021. We discussed the critical role of targeted therapy in cystic fibrosis, as it will be more effective at suppressing the molecular networks. After conducting a critical review of the available literature, we concluded that it is critical to understand the fundamental molecular mechanisms underlying CF prior to incorporating biologics into the therapy regimen. Omalizumab, Mepolizumab, Benralizumab, Dupilumab and KB001-A have been successfully screened for asthma-complicated CF, and their efficacies have been well reported. Despite the availability of effective targeted biologics, treating CF has remained a difficult task, particularly when it comes to reduction of secondary inflammatory mediators. This review emphasizes the overall views on CF, the immunological mechanism of CF, and the prospective therapeutic use of mAbs as potential targeted biologics for enhancing the overall status of human health.
囊性纤维化(CF)是一种影响外分泌腺的遗传性疾病,由囊性纤维化跨膜传导调节因子基因(CFTR)突变引起。肺部疾病是患者发病的主要原因。使用单克隆抗体(mAb)已实现对CF的靶向特异性治疗。本文旨在探讨通过mAb显著的靶向特异性来治疗CF的可能性。我们检索了1984年至2021年期间Web of Science、PubMed、EMBASE、Scopus和谷歌学术等电子数据库。我们讨论了靶向治疗在囊性纤维化中的关键作用,因为它在抑制分子网络方面将更有效。在对现有文献进行批判性综述后,我们得出结论,在将生物制剂纳入治疗方案之前,了解CF潜在的基本分子机制至关重要。奥马珠单抗、美泊利单抗、贝那利珠单抗、度普利尤单抗和KB001-A已成功筛选用于哮喘合并CF的治疗,且其疗效已有充分报道。尽管有有效的靶向生物制剂,但治疗CF仍然是一项艰巨的任务,尤其是在减少继发性炎症介质方面。本综述强调了对CF的总体看法、CF的免疫机制以及mAb作为潜在靶向生物制剂在改善人类健康总体状况方面的前瞻性治疗用途。