Author: Aimee Cunningham / Source: Science News
For years, scientists have struggled to find a therapy that works for most cystic fibrosis sufferers.
Now, two new triple-drug approaches, still undergoing testing, are offering hope.Cystic fibrosis is caused by mutations in a gene called CFTR. These mutations mean the body either makes defective versions of a protein, also called CFTR, or none of the protein at all. The new therapies work to partly fix underlying problems with one type of defective protein.
Two triple-drug approaches, taken for four weeks, each significantly improved the lung function of people with the most common cystic fibrosis–causing mutation. About 90 percent of people with the disease have this mutation. Plus, the drugs were safe, with tolerable side effects, researchers report online October 18 in two studies in the New England Journal of Medicine.
With continued testing and approval, a triple-drug combination could potentially provide an effective treatment for the first time for the vast majority of cystic fibrosis patients.
The triple-drug approach produced “a really robust improvement in pulmonary function,” says Hartmut Grasemann, a pediatrician at the Hospital for Sick Children in Toronto who was not part of the research. And it “works for almost all patients with cystic fibrosis, which is quite amazing.
”In cystic fibrosis, which affects about…
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