Advances in the treatment of cystic fibrosis

Cystic fibrosis is an autosomal recessive disease, characterised by pancreatic insufficiency, abnormal viscous mucus secretions and chronic respiratory tract infections. Treatment is with pancreatin preparations to improve intestinal digestion of food, but the use of high-potency products requires care. Postural drainage and other physiotherapeutic measures are essential to the relief of respiratory obstruction. A new approach to the respiratory problems of cystic fibrosis is dornase alpha, a mucolytic enzyme given by inhalation. Gene therapy may eventually provide the definitive answer to treatment and is already on the therapeutic horizon, but many practical problems remain to be solved.