Researchers find targeting rare form of cystic fibrosis earlier prevents organ damage in ferrets

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A team of researchers from the University of Iowa, University Medical Center Utrecht and Vertex Pharmaceuticals Incorporated, reports that earlier administration of a drug already given to patients with a rare form of cystic fibrosis can prevent organ damage—at least in ferrets. In their paper published in the journal Science Translational Medicine, the group describes their study of the impact of the drug ivacaftor on ferret models and what they found. Thomas Ferkol with the Washington University School of Medicine has written a Focus piece on the work done by the team in the same issue.

Cystic fibrosis is a genetic disorder that notoriously leads to lung problems, but it also damages other organs such as the kidney, liver, pancreas and intestines. Prior research has shown that people with cystic fibrosis have problems with production of a protein called CFTR—the protein controls the behavior of charged atoms in cells that make fluids such as sweat, tears, saliva, mucus and digestive material. Prior research has shown that approximately 10 

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