| Literature DB >> 34348870 |
Eva Furstova1, Tereza Dousova2, Jakub Beranek3, Malgorzata Libik4, Libor Fila5, Martin Modrak6, Ondrej Cinek2, Milan Macek4, Pavel Drevinek7.
Abstract
Superior efficacy of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) over tezacaftor/ivacaftor (TEZ/IVA) in people with cystic fibrosis (CF) and Phe508del/Phe508del genotype was shown in clinical trials. We utilized intestinal organoid approach to compare in vitro responses to these 2 CFTR modulator drug combinations and to check potential inter-individual variability in therapeutic response to the triple combination. Organoids from 17 subjects with Phe508del/Phe508del were screened with forskolin induced swelling assay. Significantly larger swelling, when exposed to ELX/TEZ/IVA as compared to TEZ/IVA, was observed in 16 of them. However, 1 sample showed no additional effect of ELX. The finding of unique CFTR variants in this sample indicates that genetic traits other than CF-causing CFTR mutation are worth exploring as they may have an impact on the definitive modulator drug response.Entities:
Keywords: CFTR sequence; Cystic fibrosis; Elexacaftor; Intestinal organoids; Ivacaftor; Tezacaftor
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Year: 2021 PMID： 34348870 DOI： 10.1016/j.jcf.2021.07.006
Source DB: PubMed Journal: J Cyst Fibros ISSN： 1569-1993 Impact factor: 5.482