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New advances in cystic fibrosis treatments

Harvard researchers believe they are getting close to developing a new medication to treat cystic fibrosis. This fatal lung disease claims about 500 lives each year, with 1,000 new cases diagnosed annually.

Reproducing cells

Using skin cells from cystic fibrosis patients, researchers were able to grow their own diseased samples. The research started two years ago in mice, but now the diseased tissue can be grown in a dish and used for testing, which researchers say will speed up the time it takes to develop a new medication.

“When we talk about research and advances, donors and patients ask: ‘When? How soon?’ And we usually hesitate to answer. But we now have every single piece we need for the final push. So I have every hope that we’ll have a therapy in a matter of years,” said Jayaraj Rajagopal, a Harvard Medical School assistant professor of Medicine.

He adds, “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.”

More about cystic fibrosis

Cystic fibrosis, which used to claim its victims in infancy or early childhood, has evolved into a killer of those in their 30s because treatments of the infections that characterize the disease have improved. But despite those advances, there has been little progress in treating the underlying condition that affects the vast majority of patients: a defect in a single gene that interferes with the fluid balance in the surface layers of the airways and leads to a thickening of mucus, difficulty breathing and repeated infections and hospitalizations.

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