Recent Breakthrough Increases Efficacy of Cystic Fibrosis Drugs

In an attempt to gain understanding, Brochiero and her team are trying to gain some insight. By collecting nasal cells from patients (with their consent, of course) and reusing lungs that have been removed from cystic fibrosis patients during transplants, she has been able to study the disease itself and test alternative therapeutic approaches. Eventually, by tissue engineering, the scientists were even able to recreate airway epithelial tissue.

Brochiero explains why the findings of these efforts are important, "patients with cystic fibrosis present genetic defects that cause the abnormal production of a protein called CFTR in cells and an excess of mucus in organs. Current drugs act to correct the defect in the CFTR protein. In a sterile in vitro environment, Kalydeco and Orkambi treatments work well. But in real life, the lungs of sick patients are colonized by bacteria, Pseudomonas aeruginosa in particular. Much evidence has indicated that the bacteria could interfere with the treatment. Thanks to this study, we have been able to determine which substances released by the bacteria could reduce the efficacy of the treatments.