Cystic fibrosis: Priorities and progress for future therapies

Significant improvement in the survival of patients with CF has been achieved in the last decades. The improved clinical status of the patients is mainly the result of a better understanding of the natural course of infection and inflammation in CF that has led to the implementation of strategies that increase the life expectancy and quality of life of the patients. These strategies include prompt diagnosis, timely and aggressive nutritional support, augmentation of MCC and improved mucous drainage, initiation of antimicrobial and anti-inflammatory therapy as soon as possible, early treatment of acute exacerbations, implementation of effective hygienic measures in and outside CF centers and prompt identification and treatment of CF-related complications. Treatment at a specialized CF center by a multidisciplinary dedicated team, including frequent visits, and periodic routine tests are essential to detect and treat early changes. Adherence to these therapies is challenging. Maintaining patients in optimal status will allow them to benefit from future treatments designed to correct or modify the basic genetic defect associated with CFTR by gene replacement therapy or pharmacological interventions currently under development. These new therapies are expected to further increase life expectancy of the patients.