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Potentiators and Correctors in Paediatric Cystic Fibrosis Patients: A Narrative Review

R Dobra1, C Edmondson1, D Hughes1

  • 1The Department of Cystic Fibrosis and Chronic Lung Infection, National Heart and Lung Institute, Imperial College London, Emmanuel Kaye Building, 1B Manresa Road, London, SW36LR, UK.

Paediatric Drugs
|October 18, 2018
PubMed

Insights

New cystic fibrosis treatments target the defective CFTR protein, showing promise in clinical trials. Research is expanding to include younger patients and common mutations, despite high drug costs.

Area of Science:

  • Medical Research
  • Genetics
  • Pharmacology

Background:

  • Cystic fibrosis (CF) is a common inherited disorder in Caucasians, significantly reducing life expectancy.
  • Current treatment advances focus on correcting the underlying defect in the cystic fibrosis transmembrane conductance regulator (CFTR) protein.

Purpose of the Study:

  • To review recent advances in CFTR-targeted therapies for cystic fibrosis.
  • To summarize ongoing clinical trials exploring new and existing therapies.

Main Methods:

  • Review of clinical trial data for CFTR modulators.
  • Analysis of emerging drug pipelines for cystic fibrosis treatment.
  • Examination of therapeutic strategies for early-life intervention.

Main Results:

  • Several CFTR-targeting drugs have shown promising results and are in clinical use.
  • Ongoing trials are evaluating safety and efficacy in pediatric populations.
  • Development is progressing for therapies addressing common CFTR mutations.

Conclusions:

  • Disease-modifying therapies offer significant potential for improving cystic fibrosis outcomes.
  • Early intervention with novel therapies is becoming increasingly relevant.
  • High drug costs present a significant challenge for widespread access to these advanced treatments.

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