Repurposing of ivacaftor shows potential to treat ROR1 expressing high-grade serous ovarian cancer.

ABSTRACT:

Background

Drug repurposing has emerged as an effective strategy to accelerate drug discovery. Using the pipeline established from a large collaborative drug repurposing project focused on high-grade serous ovarian cancer (HGSOC), we identified ivacaftor, an FDA-approved cystic fibrosis medication, as a drug candidate predicted to interact with the receptor tyrosine kinase-like orphan receptor 1 (ROR1) which we have previously demonstrated as a therapeutic target in ovarian cancer.

Objectives

This study aimed to provide preclinical evidence supporting the potential repurposing of ivacaftor for HGSOC treatment.

Design

Ivacaftor was tested in 2D and 3D preclinical models as well as patient-derived organoid models in vitro.

Methods

Dose-response analysis was under

SUBMITTER: Liu D 

PROVIDER: S-EPMC12759121 | biostudies-literature | 2026

REPOSITORIES: biostudies-literature