Future therapies for cystic fibrosis

Lucy Allen, Lorna Allen, Siobhan B. Carr, Gwyneth Davies, Damian Downey, Marie Egan, Julian T. Forton, Robert Gray, Charles Haworth, Alexander Horsley, Alan R. Smyth, Kevin W. Southern and Jane C. Davies ()
Additional contact information
Lucy Allen: Cystic Fibrosis Trust
Lorna Allen: Cystic Fibrosis Trust
Siobhan B. Carr: Royal Brompton & Harefield Hospital, Guy’s & St Thomas’ Trust
Gwyneth Davies: University College London
Damian Downey: Queen’s University Belfast
Marie Egan: Yale University
Julian T. Forton: Noah’s Ark Children’s Hospital for Wales
Robert Gray: Centre for Inflammation Research, University of Edinburgh
Charles Haworth: Royal Papworth Hospital and Department of Medicine
Alexander Horsley: University of Manchester
Alan R. Smyth: School of Medicine, University of Nottingham
Kevin W. Southern: Department of Women’s and Children’s Health, University of Liverpool
Jane C. Davies: Royal Brompton & Harefield Hospital, Guy’s & St Thomas’ Trust

Nature Communications, 2023, vol. 14, issue 1, 1-13

Abstract: Abstract We are currently witnessing transformative change for people with cystic fibrosis with the introduction of small molecule, mutation-specific drugs capable of restoring function of the defective protein, cystic fibrosis transmembrane conductance regulator (CFTR). However, despite being a single gene disorder, there are multiple cystic fibrosis-causing genetic variants; mutation-specific drugs are not suitable for all genetic variants and also do not correct all the multisystem clinical manifestations of the disease. For many, there will remain a need for improved treatments. Those patients with gene variants responsive to CFTR modulators may have found these therapies to be transformational; research is now focusing on safely reducing the burden of symptom-directed treatment. However, modulators are not available in all parts of the globe, an issue which is further widening existing health inequalities. For patients who are not suitable for- or do not have access to- modulator drugs, alternative approaches are progressing through the trials pipeline. There will be challenges encountered in design and implementation of these trials, for which the established global CF infrastructure is a major advantage. Here, the Cystic Fibrosis National Research Strategy Group of the UK NIHR Respiratory Translational Research Collaboration looks to the future of cystic fibrosis therapies and consider priorities for future research and development.

Date: 2023
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DOI: 10.1038/s41467-023-36244-2

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