Patricia Inácio, PhD, science writer —

Scientists at Boston University’s Center for Regenerative Medicine have made two stem cell discoveries that could help doctors treat cystic fibrosis (CF) without having to resort to transplants. They prevented stem cells from creating cells for a lot of organs at once and producing only the lung cells they need…

Vertex Pharmaceuticals recently announced positive results of two Phase 3 clinical trials, the EVOLVE and EXPAND studies, evaluating the effects of a combination of tezacaftor (VX-661) and ivacaftor (sold under the brand name Kalydeco) in cystic fibrosis (CF). The results showed that the new combined therapy significantly improved…

Kalydeco (ivacaftor, VX-770) improves bicarbonate secretion in cystic fibrosis (CF) patients with a certain gene mutation, according to a study. Bicarbonate is essential to digestion and the flow of several secretions in the body, including those of the intestine and pancreas. The report, “Impact of CFTR…

Scientists at the University of California-Riverside have developed a novel way to help target therapeutics for cystic fibrosis (CF) and other diseases. Their study, “Inhibition of nonsense-mediated RNA decay by ER stress,” appeared in the journal RNA. The information encoded in human DNA is first converted to RNA, and…

Rare mutations in the CFTR gene — which is defective in patients with cystic fibrosis (CF) — may occur in specific ethnic groups, suggests a new study that urges wider screening of mutations in patients who test negative for most previously identified mutations linked to CF. The study, “A rare CFTR mutation…

A mouse model that reproduces the human symptoms of cystic fibrosis (CF) and chronic obstructive pulmonary disease (COPD) gives scientists a new way to study, and perhaps develop treatments for, the diseases, according to new research. Researchers at Kumamoto University in Japan developed the model, then tested it to see how…

Although patients with cystic fibrosis (CF) often have low levels of the antioxidant glutathione (GSH), the clinical benefits of GSH as an inhaled therapy appear to depend on the inflammatory status of their airways. The study, “Increasing sputum levels of gamma-glutamyltransferase may identify cystic fibrosis patients who do not…

In a small randomized clinical trial, researchers at Karolinska Institute in Sweden showed that vitamin D supplements may improve lung function in cystic fibrosis (CF) patients. A daily intake of high levels of vitamin D are required, however, for the effects to be noticed. The study, “Clinical…

Measuring antibodies in the blood and administering lung-function tests, either alone or in combination, significantly increase health-care specialists’ ability to detect the serious bacteria Pseudomonas aeruginosa in children with cystic fibrosis (CF). The study, “Assessment of serology and spirometry and the combination of both to complement microbiological isolation…

Scientists have identified the three-dimensional structure of the protein responsible for cystic fibrosis (CF), a development that will help them understand how mutations of the protein lead to the disease. They also found an area in the protein where many disease-causing mutations are located, and a region within that area…