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Treatment of cystic fibrosis during embryonic development reduces disease in later stages

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Friday, May 17, 2019

Several studies suggest that the pathogenesis generated by cystic fibrosis begins in the early stages of development, so a team of researchers at the University of Iowa wanted to test whether the application of treatment during pregnancy would have any significant effect on the disease. A new article published by the group in Science Translational Medicine shows that the drug ivacaftor partially protects model ferret embryos from cystic fibrosis. The treated individuals showed higher survival and growth rates and a significant improvement in symptomatology was observed.

Cystic fibrosis is an inherited pathology of the mucus and sweat glands that mainly affects the lungs, pancreas, liver and intestines. Affected individuals have mutations in the CFTR protein, which regulate the flow of chlorine ions through the cell membrane. Due to these mutations, the cells that cover certain organs produce thicker and stickier mucus than normal, which hinders their function. Severe respiratory problems are generated and there is an excessive growth of bacteria, which causes numerous lung infections.

The authors of this study created a ferret line with a mutation in the CFTR gene that can be treated with ivacaftor. The researchers selected this animal model because it reproduces characteristics of cystic fibrosis characteristic of human infants and has a more modest size when compared to the alternative model in pigs. Prenatal treatment of ferrets with ivacaftor improved pancreatic function, glucose tolerance, and showed a reduction in mucus accumulation and in the number of bacterial infections in the lungs.

These results focus on early treatment of cystic fibrosis and provide a preclinical model that may help to understand the relationship between CFTR protein function and disease progression.

Access to original article: In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model of cystic fibrosis. Science Translational Medicine.

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