Organoid-guided synergistic treatment of minimal function CFTR mutations with CFTR modulators, roflumilast and simvastatin: a personalised approach

Sacha Spelier; Karin de Winter-de Groot; Natascha Keijzer-Nieuwenhuijze; Yves Liem; Kors van der Ent; Jeffrey Beekman; Lieke S Kamphuis

doi:10.1183/13993003.00770-2023

Organoid-guided synergistic treatment of minimal function CFTR mutations with CFTR modulators, roflumilast and simvastatin: a personalised approach

Eur Respir J. 2024 Jan 25;63(1):2300770. doi: 10.1183/13993003.00770-2023. Print 2024 Jan.

Authors

Sacha Spelier^{1

2}, Karin de Winter-de Groot¹, Natascha Keijzer-Nieuwenhuijze^{1

2}, Yves Liem³, Kors van der Ent¹, Jeffrey Beekman^{1

2

4}, Lieke S Kamphuis^{5

4}

Affiliations

¹ Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.
² Regenerative Medicine Utrecht, University Medical Center, Utrecht University, Utrecht, The Netherlands.
³ Department of Clinical Pharmacy, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, The Netherlands.
⁴ J. Beekman and L.S. Kamphuis contributed equally to this article as lead authors and supervised the work.
⁵ Department of Respiratory Medicine, Erasmus University Medical Center, Rotterdam, The Netherlands l.kamphuis@erasmusmc.nl.

Abstract

This study describes how preclinical research has guided a successful personalised clinical treatment regimen in a person with minimal function CFTR, upon a synergistic treatment regimen consisting of CFTR modulators, simvastatin and roflumilast https://bit.ly/3rDTHZL

Publication types

Letter

MeSH terms

Aminopyridines*
Benzamides*
Cyclopropanes
Cystic Fibrosis Transmembrane Conductance Regulator* / genetics
Humans
Mutation
Organoids
Simvastatin* / pharmacology
Simvastatin* / therapeutic use

Substances

Cystic Fibrosis Transmembrane Conductance Regulator
Roflumilast
Simvastatin
CFTR protein, human
Aminopyridines
Benzamides
Cyclopropanes