The era of CFTR modulators: improvements made and remaining challenges

Cuevas-Ocaña, Sara; Laselva, Onofrio; Avolio, Julie; Nenna, Raffaella

Abstract

This last decade has created historical moments for CF, primarily driven by the development of CFTR modulators. First for patients with gating mutations who benefited from Kalydeco, then for those patients with one F508del copy who could benefit from Orkambi, and most recently, patients with at least one F508del copy who can benefit from Trikafta. Despite heterogeneity in patient response, the majority of CF patients will be greatly impacted by using a CFTR modulator therapy, thus changing the trajectory of their life. Furthermore, it remains to be determined whether the next generation of modulators will be effective for individuals bearing rare mutations that are Orkambi resistant. However, it should not be forgotten that there still remains 10% of the CF population who do not have a targeted CFTR modulator treatment. In addition, even with these novel drug therapies, managing infections will continue to be a challenge, thus the CF community will need to adapt the standards for an improving, but ageing CF population.

Citation

Cuevas-Ocaña, S., Laselva, O., Avolio, J., & Nenna, R. (2020). The era of CFTR modulators: improvements made and remaining challenges. Breathe, 16(2), Article 200016. https://doi.org/10.1183/20734735.0016-2020

• Journal Article Type: Article
• Acceptance Date: Mar 29, 2020
• Online Publication Date: Sep 25, 2020
• Publication Date: Sep 25, 2020
• Deposit Date: Jun 16, 2023
• Publicly Available Date: Jun 19, 2023
• Journal: Breathe
• Print ISSN: 1810-6838
• Electronic ISSN: 2073-4735
• Publisher: European Respiratory Society
• Peer Reviewed: Peer Reviewed
• Volume: 16
• Issue: 2
• Article Number: 200016
• DOI: https://doi.org/10.1183/20734735.0016-2020
• Public URL: https://nottingham-repository.worktribe.com/output/21925980
• Publisher URL: https://breathe.ersjournals.com/content/16/2/200016