A breakthrough in the development of drugs for the treatment of cystic fibrosis
Last reviewed: 23.04.2024
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Harvard cell biologists from the Massachusetts General Hospital have made a breakthrough in the development of drugs for the treatment of cystic fibrosis, a deadly disease that takes about 500 lives a year in the world.
Scientists reprogrammed the skin cells of patients with cystic fibrosis into induced pluripotent stem cells (iPS), which are similar to embryonic stem cells, and grew out of them a pulmonary epithelium specific for patients with cystic fibrosis of the respiratory tract.
This fabric Harvard scientists can now grow in unlimited quantities. All cells of the epithelium obtained in the laboratory contain a mutation of delta 508, which accounts for about 70 percent of all cases of cystic fibrosis and 90 percent of the disease detected in the United States. In addition, its cells also carry a mutation G551D, which is found in 2% of all patients with cystic fibrosis.
The work was done under the direction of Jayaraj Rajagopal and published in the supplement to the journal Cell Stem Cell.
According to Douglas Melton, one of the directors of the Harvard Stem Cell Institute (Douglas Melton), "the results of this study make it possible to obtain millions of cells for screening drugs, and for the first time, cells of people who are sick can become targets for experimental drugs cystic fibrosis. "
Epithelial tissue, created by Rajagopal and colleagues, also provides cells for the study of respiratory diseases such as asthma, lung cancer and chronic bronchitis. This can speed up the development of treatment methods for these diseases.
Commenting on the achievement of his group, Rajagopal notes, "We are not talking about the treatment of cystic fibrosis, we are only talking about a medicine that can alleviate the main problem of the disease - the defeat of the epithelium of the respiratory system.
Cystic fibrosis, found in the early days of a person's life or in early childhood - the cause of early mortality. Currently, patients can live up to 30 years due to effective treatment of concomitant infections and early medical genetic diagnosis of the disease. But despite these achievements, success in eradicating the underlying cause of cystic fibrosis is not great. A defect of one gene coding for a protein cystic fibrosis transmembrane conductivity regulator causes thickening of the secrets of the glands of external secretion. As a result of the difficulty in the outflow of the secret, changes occur in the respiratory, pancreatic, and gastrointestinal tract, and the cilia of the epithelium are disrupted.
"We have made an excellent cell line so that we can see the effectiveness of the drug with respect to the effects of the G551D mutation, and now we are engaged in screening drugs aimed at the mutation of delta 508," Rajagopal said.
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