Related Experiment Video

Updated: Aug 11, 2025

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
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Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study

Published on: April 11, 2018

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Future therapies for cystic fibrosis

Lucy Allen1, Lorna Allen1, Siobhan B Carr2,3

  • 1Cystic Fibrosis Trust, London, UK.

Nature Communications
|February 8, 2023
PubMed

Insights

New cystic fibrosis (CF) drugs offer hope but don't work for everyone. Research is exploring new treatments and addressing global access to improve care for all individuals with CF.

Area of Science:

  • Pulmonary Medicine
  • Genetics
  • Pharmacology

Background:

  • Cystic Fibrosis (CF) is a single-gene disorder caused by various genetic variants affecting the cystic fibrosis transmembrane conductance regulator (CFTR) protein.
  • Small molecule, mutation-specific CFTR modulator drugs represent a significant advancement, restoring protein function for some patients.
  • Despite progress, current therapies are not universally effective or accessible, highlighting the need for continued research.

Purpose of the Study:

  • To review the current landscape of CFTR modulator therapies.
  • To identify unmet needs and challenges in CF treatment, including accessibility and efficacy for diverse genetic variants.
  • To outline future research priorities for developing novel and improved CF therapies.

Main Methods:

  • Literature review of current CFTR modulator therapies and ongoing clinical trials.
  • Analysis of the impact of CFTR modulators on clinical manifestations and health inequalities.
  • Discussion of challenges and opportunities in designing and implementing future CF clinical trials.

Main Results:

  • CFTR modulators have transformed care for eligible patients but do not address all genetic variants or disease manifestations.
  • Global disparities in access to modulator therapies exacerbate health inequalities.
  • Alternative therapeutic approaches are in development, with clinical trials facing design and implementation challenges.

Conclusions:

  • While CFTR modulators are a breakthrough, a significant need for alternative and broadly accessible treatments remains.
  • Future research must focus on developing therapies for non-responsive mutations and addressing global access issues.
  • Leveraging existing CF infrastructure is crucial for advancing future research and clinical trials.

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