March 30, 2015

Cystic fibrosis is a genetic disease characterized by the buildup of abnormally thick and sticky mucus that can clog the airways, leading to breathing problems and bacterial lung infections. Although models have been generated to study this disorder in a number of animal species, including mice, pigs and ferrets, species differences are very apparent. Therefore, a more human-relevant approach to studying cystic fibrosis is necessary to better understand the biological mechanisms of the disease and to test new therapeutic approaches.

In an effort to develop a human-relevant alternative to animal models in cystic fibrosis research, scientists have created an in vitro three-dimensional model using cells derived from patients with cystic fibrosis. Skin cells from patients were reprogrammed into induced pluripotent stem cells, which were then differentiated into cells that developed into lung tissue.

These “mini-lungs” mimicked some of the key features of lung disease associated with cystic fibrosis, including problems with transporting chloride across cell membranes. It is this deficiency which affects water transport, ultimately causing mucus to thicken. The researchers also showed that their model could serve as an efficient platform for testing therapeutic drugs, as cells treated with a drug which was previously shown to improve chloride transport problems was also effective in this model.

Dr. Nick Hannon, a researcher on the study, recognizes the significance of the human relevance of this in vitro model and the impact it may have on the use of animals in experimentation. He noted, “This is far more practical, should provide more reliable data and is also more ethical than using large numbers of mice for such research.”

What do you think of these findings? Send your questions and comments to sciencecorner@navs.org. I look forward to hearing from you.

–Dr. Pam Osenkowski, Director of Science Programs

Scientists at the University of Cambridge have successfully created ‘mini-lungs’ using stem cells derived from skin cells of patients with cystic fibrosis, and have shown that these can be used to test potential new drugs for this debilitating lung disease.

For more information see: EurekAlert