Repurposing therapies for the personalised treatment of cystic fibrosis

Introduction: Cystic Fibrosis (CF) is an inherited, lethal and expensive rare disease affecting more than 85,000 people worldwide. CF is caused by more than 2000 loss-of-function mutations in the gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR). Personalized interventions on individual CF patients probably constitute the future of CF management. Areas covered: The molecular genetics exploration of CF has led in the last years to the development of mutation-specific therapies that aim at directly targeting the mutant CFTR to reverse disease phenotype. Here, we will summarize the strategies of drug repositioning, i.e. the use of ‘old drugs’ for ‘new purposes’ in the perspective of a personalized approach to CF treatment. Expert opinion: Rare diseases like CF may well enter the era of precision medicine. A personalized/repositioning medicine in CF could help to provide the right drug to the right patient and potentially reduce the costs of developing disease-modifying drugs.