Precision Therapies for Children with Cystic Fibrosis

Combination therapy with a protein-corrective agent and a membrane-enhancement agent rapidly improved lung function.

Cystic fibrosis (CF) is caused by multiple genetic mutations of the cystic fibrosis transmembrane conductance regulator (CFTR). These mutations result in absent or impaired folding and trafficking of the CFTR protein or impaired function of the protein at the cell membrane. Now, two industry-funded, international, randomized, placebo-controlled, phase III trials were conducted to evaluate the efficacy of a new protein-corrective agent (tezacaftor) with a membrane-enhancement agent (ivacaftor).

Taylor-Cousar and colleagues conducted a parallel-group study to compare tezacaftor plus ivacaftor (T-I) versus placebo for 24 weeks in 504 CF patients (>12 years of age; mean age, 26–27 years) who were homozygous for the CFTR Phe508del mutation. Force…

Reviewing Author

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)

Precision Therapies for Children with Cystic Fibrosis

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Reviewing Author