Purpose of review: Improved knowledge of the molecular mechanisms underlying cystic fibrosis has led to a wide spectrum of new treatment approaches. This review addresses recent therapeutic strategies that either target the underlying defect or early steps in cystic fibrosis pathophysiology.
Recent findings: While gene therapy does not appear to be a therapeutic option in the near future, cystic fibrosis transmembrane regulator pharmacotherapy is currently being developed as an alternative to reduce cystic fibrosis transmembrane regulator degradation or improve its function. Two drugs that increase chloride secretion via an alternative chloride channel, Moli1901 and denufosol, have been shown to be safe in clinical studies that also suggested clinical efficacy. Osmotic therapy may be an alternative approach to increase airway surface liquid and is being studied as an early intervention strategy.
Summary: The spectrum of treatments for cystic fibrosis lung disease is rapidly increasing. While clinical efficacy for most of the compounds still has to be proven in large clinical trials, there is considerable hope that cystic fibrosis therapy will move from addressing the downstream sequelae of the cystic fibrosis transmembrane regulator defect to a more causal approach in the near future.