Computer-designed molecules point to new therapy for cystic fibrosis

By developing software that uses 3-D models of proteins involved in cystic fibrosis, a team of scientists at Duke University has identified several new molecules that may ease the symptoms of the disease. Computer algorithms created by the team predict how well a given molecular structure will block a basic protein-protein interaction known to occur in cystic fibrosis. To test the predictions, the scientists synthesized the molecules and measured how well they attached to one of the proteins in that interaction. The team then placed the best molecule into human cells with the cystic fibrosis mutation in a laboratory dish and found that their new drug blocked the protein-protein interaction and increased the cells' ability to balance salt and water levels.The team has made its software freely available, so the computer-design approach could quicken the pace at which molecules and resulting cystic fibrosis therapies are developed.