It’s Alive! CF Lung Tissue Grown in Lab

Screening thousands of compounds against a cell line turned up the first drug that gets at the roots of cystic fibrosis by correcting the genetic defect that causes about 2% of cases.

Now researchers say they have developed a better cell line that could be used to find a treatment for the vast majority of cystic fibrosis cases, caused by a different mutation that leads to the sticky mucus, breathing difficulties, and infections that typically kill patients in their 30s.

The group first created pluripotent stem cells from the skin of cystic fibrosis patients and then turned them into an unlimited supply of lung epithelium that expresses both of the major genetic defects identified in cystic fibrosis.

“We’re not talking about a cure for CF, we’re talking about [finding] 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,” lead author Jayaraj Rajagopal, MD, of Harvard, explained in a press release.

Because the same epithelial cells are involved in asthma, lung cancer, and chronic bronchitis as well, the strategy could be “a platform for dissecting human lung disease,” the researchers wrote in Cell Stem Cell.

- C.P.

By MedPage Today Staff