Extract
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators have revolutionised cystic fibrosis (CF) care, leading to significant improvements in lung function, decreased frequency of pulmonary exacerbations and improved quality of life [1]. The list of variants eligible for CFTR modulators has increased over time, currently covering almost 90% of individuals with CF in Caucasian populations. For rare and poorly characterised CF-causing variants, ex vivo analysis of intestinal organoids may assess CFTR function and guide personalised therapy [2, 3]. We report the case of an individual with CF whose rectal organoids were used to evaluate response to CFTR modulators and to guide therapeutic decision.
Abstract
Ex vivo analysis of intestinal organoids has great translational potential as a precision medicine tool for the prediction of clinical efficacy of CFTR modulators and, for patients with rare variants, may hold the key to these life-changing therapies http://bit.ly/3G1FkDp
Footnotes
Conflict of interest: G. Mitropoulou, Z. Balmpouzis, S. Blanchon, G. Dorta and A. Sauty report no financial relationships pertaining to this work. N. Brandenberg, S. Hoehnel and C. Ceroni have received funding for organoid research from FreeNovation (a funding programme of the Novartis Research Foundation), the Swiss Society for Cystic Fibrosis (CFCH), the Swiss Foundation “Respirer” and EIT Health, a body receiving support from the European Union Horizon 2020 Research and innovation programme. N. Brandenberg and S. Hoehnel have leadership roles in SUN bioscience SA. The Ecole Polytechnique Fédérale de Lausanne has filed for patent protection on technologies enabling organoid assays, and N. Brandenberg and S. Hoehnel are named as inventor on those patents; N. Brandenberg and S. Hoehnel are shareholders in SUN bioscience SA, which is commercialising those patents. A. Koutsokera has received the Grant Pépinière from the University of Lausanne, a Research Grant from the Swiss Lung Association (grant number 2018-16), and a BRIDGE Discovery Grant (40B2-0_194701/1) from the Swiss National Science Foundation (SNFS).
Support statement: This work was supported by CFCH, Swiss Foundation Respirer, FreeNovation and H2020 European Institute of Innovation and Technology. Funding information for this article has been deposited with the Crossref Funder Registry.
- Received July 5, 2022.
- Accepted October 31, 2022.
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