Inês Martins, PhD, managing science editor —

Canadian patients with cystic fibrosis (CF) will have to wait for access to Orkambi (lumacaftor/ivacaftor), whose submission will be reconsidered at a future meeting by the Canadian Drug Expert Committee (CDEC) of the Canadian Agency for Drugs and Technologies in Health (CADTH). CADTH is the nonprofit Canadian organization that makes recommendations for a…

Suppressing a specific type of immune cell called Th17 may aid cystic fibrosis (CF) patients with Pseudomonas aeruginosa infection, one of the most common infections in CF, as suggested by findings of a recent study in mice. The study, “Antiinflammatory effects of bromodomain and extraterminal domain inhibition in cystic…

Interleukin-22 is known to protect against respiratory pathogens through its ability to mediate mucosal immunity and tissue regeneration. But new research into its possible use against Pseudomonas aeruginosa, a main cystic fibrosis pathogen, found that IL-22 disappointed — with scientists reporting IL-22 failed to protect a mice model from such infection and even seem to…

Researchers are working on a way to deliver high doses of a common anti-inflammatory — ibuprofen — directly into the lungs of cystic fibrosis (CF) patients as a possible therapy. More than 20 years ago, scientists found that ibuprofen was able to slow lung function decline in CF patients when given at high…

To date, 2,007 mutations have been identified in the CFTR gene, but their contribution for cystic fibrosis (CF) development has not been defined for the majority of these mutations. Now, researchers at Children’s Hospital Los Angeles and the Genetic Disease Screening Program of the California Department of Public Health have…

Vertex Pharmaceuticals recently revealed new clinical data on real-world long-term outcomes in cystic fibrosis (CF) patients treated with Kalydeco (ivacaftor). The company also presented data from a Phase 3 study evaluating Orkambi (lumacaftor/ivacaftor) for children with CF ages 6-11 years. The results were available June 11-14 at the 39th European Cystic Fibrosis Society (ECFS)…

Raptor Pharmaceutical recently announced new clinical data from a Phase 3 trial evaluating the efficacy of Quinsair (levofloxacin) versus tobramycin in cystic fibrosis (CF) patients with chronic pulmonary infections caused by Pseudomonas aeruginosa. Study results were presented at the 39th European Cystic Fibrosis Conference in Basel, Switzerland. “Patients with cystic fibrosis have few…

Healthcare workers with cystic fibrosis (CF) are more likely to contract methicillin-resistant staphylococcus aureus (MRSA) infections than CF patients not in healthcare scenarios, according to the study “Methicillin-resistant Staphylococcus aureus acquisition in healthcare workers with cystic fibrosis: a retrospective cross-sectional study,” published in BMC Pulmonary Medicine. MRSA infection is caused…

Researchers partially restored the function of the CFTR protein in lung cells isolated from cystic fibrosis (CF) patients by blocking a particular protein involved in its translation. The findings were published in the journal PLOS Biology, in the study “Ribosomal Stalk Protein Silencing Partially Corrects the ΔF508-CFTR Functional…

Nivalis Therapeutics announced the dosing of a first patient in a Phase 2 clinical trial evaluating the efficacy and safety of its investigational drug N91115, in combination with ivacaftor (Kalydeco), in people with cystic fibrosis (CF). CF is a life-shortening genetic disease, characterized by mutations in the CFTR gene that lead to defective chloride…