Coming soon: A treatment for cystic fibrosis — a fatal lung disease, says an international team of scientists led by an Indian-origin researcher.
Prof Jayaraj Rajagopal at Massachusetts General Hospital and colleagues say that they have taken a critical step in making possible the discovery in the relatively near future of a drug to control cystic fibrosis.
Beginning with skin cells of patients with CF, they first created induced pluripotent stem (iPS) cells, and then used those cells to create human disease-specific functioning lung epithelium, the tissue that lines the airways and is the site of the most lethal aspect of CF, where the genes cause irreversible lung disease and inexorable respiratory failure.
That tissue, which researchers now can grow in unlimited quantities in the laboratory, contains the delta-508 mutation, the gene responsible for about 70 per cent of all CF cases and 90 per cent of the ones in the US.
The tissue also contains the G551D mutation, a gene that is involved in about 2 per cent of CF cases and the one cause of the disease for which there is now a drug.
Mr Doug Melton, a team member, said: “This work makes it possible to produce millions of cells for drug screening, and for the first time human patients’ cells can be used as the target,” the Cell Stem Cell journal reported.
The epithelial tissue created by the scientists also provides researchers with the same cells that are involved in a number of common lung conditions, including asthma, lung cancer, and chronic bronchitis, and may hasten the development of new insights and treatments into those conditions as well.
“We’re not talking about a cure for CF; we’re talking about a drug that hits the major problem in the disease. This is the enabling technology that will allow that to happen in a matter of years,” said Prof Rajagopal.