Cystic fibrosis transmembrane conductance regulator modulators: Present and future in cystic fibrosis treatment. A review

Arch Argent Pediatr. 2019 Apr 1;117(2):e131-e136. doi: 10.5546/aap.2019.eng.e131.
[Article in English, Spanish]

Abstract
in English, Spanish

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are the present and future of drug management for patients with cystic fibrosis. The objective of this article is to review this therapeutic option. Scientific articles were reviewed by searching the MedLine database, which is available through the Cystic Fibrosis Foundation's official website, from 2009 to 2018, in English. Twelve articles about the current status of research in CFTR modulators were selected without restrictions regarding the type of study. To date, the United States Food and Drug Administration has approved three modulators: ivacaftor, lumacaftor + ivacaftor, and tezacaftor + ivacaftor, while other 11 drugs are being studied in different investigation phases. CFTR modulator therapy is a developing reality aimed at the highest goal of personalized medicine and promises to improve the quality of life of cystic fibrosis patients.

Los moduladores cystic fibrosis transmembrane conductance regulator (CFTR) representan el presente y el futuro del manejo farmacológico para los pacientes con fibrosis quística. El objetivo de esta publicación es realizar una revisión de esta opción terapéutica. Se revisaron artículos científicos consultando las bases de datos MedLine, información disponible a través de la página oficial Cystic Fibrosis Foundation, desde 2009 hasta 2018, en el idioma inglés. Sin restricciones respecto al tipo de estudio, se seleccionaron 12 artículos que incluyeron información sobre el estado actual de la investigación sobre moduladores CFTR. Actualmente, están aprobados por la Food and Drug Administration tres moduladores: ivacaftor, lumacaftor + ivacaftor y tezacaftor + ivacaftor, y hay otros 11 en diferentes fases de estudio. La terapia con moduladores CFTR es una realidad en desarrollo que apunta al máximo objetivo de la medicina personalizada y que promete mejorar la calidad de vida de pacientes con fibrosis quística.

Keywords: Cystic fibrosis; Cystic fibrosis transmembrane conductance regulator; Mutation; Therapy.

Publication types

  • Review

MeSH terms

  • Aminophenols / administration & dosage
  • Aminopyridines / administration & dosage
  • Benzodioxoles / administration & dosage
  • Cystic Fibrosis / drug therapy*
  • Cystic Fibrosis / physiopathology
  • Cystic Fibrosis Transmembrane Conductance Regulator / drug effects*
  • Cystic Fibrosis Transmembrane Conductance Regulator / metabolism
  • Drug Combinations
  • Humans
  • Indoles / administration & dosage
  • Precision Medicine
  • Quality of Life*
  • Quinolones / administration & dosage

Substances

  • Aminophenols
  • Aminopyridines
  • Benzodioxoles
  • CFTR protein, human
  • Drug Combinations
  • Indoles
  • Quinolones
  • lumacaftor, ivacaftor drug combination
  • tezacaftor, ivacaftor drug combination
  • Cystic Fibrosis Transmembrane Conductance Regulator