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

Extract Highly effective cystic fibrosis transmembrane conductance regulator (CFTR) protein-targeting modulator therapies (HEMTs) facilitate strong clinical improvements in a large proportion of people with cystic fibrosis (CF) [1, 2]. More specifically, the European Medicines Agency and US Food and Drug Administration (FDA) approved combination of the CFTR modulators elexacaftor/tezacaftor/ivacaftor (ETI) for people with CF with at least one F508del allele, while the FDA extended eligibility for several rare genotypes [3, 4]. However, 10–15% of those with CF carry CFTR mutations that are unresponsive to HEMTs as monotherapy [1]; furthermore, some suffer from HEMT intolerance, and HEMTs are sometimes not accessible due to practical challenges, such as lack of access due to high costs or legislation and approval challenges. Consequently, the focus in the CF research field has shifted towards filling the unmet clinical need for the people with CF that will not benefit from HEMTs.
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: CF and non-CF bronchiectasis Research letters Source Type: research