Cystic fibrosis–Ten promising therapeutic approaches in the current era of care

Abstract

Introduction: Cystic fibrosis (CF) is a genetic disease affecting multiple organ systems. Research and innovations in novel therapeutic agents and health care delivery have resulted in dramatic improvements in quality of life and survival for people with CF. Despite this, significant disease burden persists for many and this is compounded by disparities in treatment access and care which globally necessitates further work to improve outcomes. Because of the advent of numerous therapies which include gene-targeted modulators in parallel with specialized care delivery models, innovative efforts continue. Areas covered: In this review, we discuss the available data on investigational agents in clinical development and currently available treatments for CF. We also evaluate approaches to care delivery, consider treatment gaps, and propose future directions for advancement. Expert opinion: Since the discovery of the CF gene, CFTR modulators have provided a hallmark of success, even though it was thought not previously possible. This has led to reinvigorated efforts and innovations in treatment approaches and care delivery. Numerous challenges remain because of genetic and phenotypic heterogeneity, access issues, and therapeutic costs, but the collaborative approach between stakeholders for continued innovation fuels optimism. Abbreviations: CF cystic fibrosis; CFF Cystic Fibrosis Foundation (USA); CFTR cystic fibrosis transmembrane regulator; CRISPR clustered regularly interspaced short palindromic repeats; COX cyclo oxygenase; FDA US Food and Drug Administration; FEV1% forced expiratory volume in one second % predicted; F508del deletion of phenylalanine (F) in the 508th position (most common mutation); G551D substitution of the amino acid glycine by aspartate at position 551 in the nucleotide binding domain-1 of the CFTR gene; LMIC low- and middle-income country; LTB4 leukotriene B4; MDT multi-disciplinary care team; NO nitric oxide; NSAIDs non-steroidal anti-inflammatory drugs; SLPI secretory leukocyte protease inhibitor.