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Drug research and development » Pre-clinical development » Pre-clinical validation of CFTR therapies

Pre-clinical validation of CFTR therapies

A microbiologist testing medicine in a laboratoryPrior to validation in clinical trials, CFTR-modulating therapies and other therapies need to be validated in pre-clinical models.1 This is to ensure that only the best candidates are investigated in patients.1 The possible models include ex vivo in native tissue from CF patients and cellular models.1

Current animal murine models have limited significance. This partly due to murine and human CFTR channels exhibiting different characteristics and are regulated in a different manner. In addition, mouse models lacking functional CFTR do not develop the characteristic manifestations of CF as observed in humans.1

For CFTR-repairing molecules, efficacy testing on human bronchial epithelial (HBE) cells is considered to be the ‘gold standard’. While this was successfully used for validation of the VX-770 modulator and correlated well with its efficacy in clinical trials, validation with additional compounds is required to demonstrate that efficacy in HBE cells correlates with clinical efficacy in vivo.1

  1. Bell SC, De Boeck K, Amaral MD. New pharmacological approaches for cystic fibrosis: promises, progress, pitfalls. Pharmacol Ther 2015;145:19-34.