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Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
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Modifying disease in cystic fibrosis: current and future therapies on the horizon

Thida Ong1, Bonnie W Ramsey

  • 1Department of Pediatrics, Seattle Children's Hospital and the University of Washington School of Medicine, Seattle, Washington, USA.

Current Opinion in Pulmonary Medicine
|September 20, 2013
PubMed
Abstract

Insights

New cystic fibrosis (CF) therapies targeting early disease steps show promise. Modulators like ivacaftor and gene therapy offer hope for modifying CF

Area of Science:

  • Pulmonology and Respiratory Medicine
  • Pharmacology and Drug Development
  • Genetics and Genetic Diseases

Background:

  • Cystic Fibrosis (CF) is a genetic disorder affecting multiple organs, primarily the lungs.
  • Traditional treatments focus on symptom management; emerging therapies target underlying pathophysiology.
  • Understanding CF transmembrane conductance regulator (CFTR) protein function is key to developing targeted treatments.

Purpose of the Study:

  • To review recent clinical trials and emerging therapies for cystic fibrosis.
  • To highlight treatments targeting proximal mechanisms in CF pathophysiology.
  • To discuss strategies for modulating early disease progression.

Main Methods:

  • Review of recent clinical trial data for CF therapies.
  • Analysis of approved and investigational CFTR modulators.
  • Examination of genotype-independent modulation strategies.

Main Results:

  • Approval of ivacaftor (Kalydeco), a CFTR protein modulator, signifies progress.
  • Ongoing development of novel CFTR modulators for various mutation classes.
  • Gene replacement therapy trials are progressing slowly.
  • Genotype-independent approaches like airway surface liquid modulation are being explored.

Conclusions:

  • A wide range of therapies targeting early CF stages offers significant hope for altering disease progression.
  • Development of novel biomarkers for early disease detection is crucial.
  • Concurrent advancement in drug development across multiple targets is essential for maximum therapeutic impact.

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