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A breakthrough has been made in the development of drugs to treat cystic fibrosis
Last reviewed: 01.07.2025
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Harvard cell biologists at Massachusetts General Hospital have made a breakthrough in developing drugs to treat cystic fibrosis, a deadly disease that claims about 500 lives worldwide each year.
Scientists reprogrammed skin cells from cystic fibrosis patients into induced pluripotent stem (iPS) cells, which are similar to embryonic stem cells, and grew lung epithelium specific to patients with cystic fibrosis-related airway disease.
Harvard scientists can now grow this tissue in unlimited quantities. All the cells of the epithelium obtained in the laboratory contain the delta 508 mutation, which causes about 70 percent of all cases of cystic fibrosis and 90 percent of the disease diagnosed in the United States. In addition, its cells also carry the G551D mutation, which is found in 2 percent of all patients with cystic fibrosis.
The work was supervised by Jayaraj Rajagopal and published in a supplement to the journal Cell Stem Cell.
According to Douglas Melton, one of the directors of the Harvard Stem cell Institute, "the results of this study make it possible to obtain millions of cells for drug screening, and for the first time, cells from people with cystic fibrosis can be used as targets for experimental drugs."
The epithelial tissue created by Rajagopal and colleagues also provides cells for research into respiratory diseases such as asthma, lung cancer and chronic bronchitis. This could speed up the development of treatments for these diseases as well.
Commenting on his group's achievement, Rajagopal notes, "We are not talking about a cure for cystic fibrosis, we are talking only about a drug that can alleviate the main problem of the disease - damage to the epithelium of the respiratory organs.
Cystic fibrosis, detected in the first days of a person's life or in early childhood, is the cause of early mortality. Currently, patients can live up to 30 years thanks to effective treatment of concomitant infections and early medical and genetic diagnosis of the disease. But despite these achievements, success in eradicating the original cause of cystic fibrosis is small. A defect in one gene encoding the protein cystic fibrosis transmembrane conductivity regulator causes thickening of the secretions of the glands of external secretion. As a result of the difficulty in the outflow of secretion, changes occur in the respiratory organs, pancreas and gastrointestinal tract, and the work of the cilia of the epithelium is disrupted.
"We've made a great cell line to see if a drug is effective against the effects of the G551D mutation, and we're now looking to screen drugs that target the delta 508 mutation," Rajagopal says.
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