Steve Bryson, PhD,  science writer—

Researchers generated functional bile duct cells from human-derived stem cells that can be used to support the development of therapies for liver disease in people with cystic fibrosis (CF), a study reported. “These studies highlight the importance of generating mature cells from stem cells that faithfully mimic the functional…

The microbiome diversity in the lungs of cystic fibrosis (CF) patients increased after treatment with Trikafta, becoming similar to a person without CF, an analysis showed. In addition, small molecule metabolites produced by cells varied widely across patients while on the therapy, with the largest decrease seen in…

For the first time, many features of cystic fibrosis (CF) were modeled accurately in a human Lung Airway Chip lined with cells from CF patients, which included enhanced mucus secretion, inflammation, and microbial infections, a study has shown. This “organ-on-a-chip model” of the disease may support the rapid development…

Scientists using phage therapy — specifically a bacteriophage they dubbed “Muddy” — successfully treated an infection by an antibiotic-resistant strain of Mycobacterium abscessus bacteria in a zebrafish model of cystic fibrosis (CF). For five days, the team treated the zebrafish, which were infected with M. abscessus, a lung-damaging bacteria…

Vertex Pharmaceuticals has signed a letter of intent with the pan-Canadian Pharmaceutical Alliance (pCPA), which is an agreement in principle to extend public reimbursement to Trikafta, a triple-combination therapy for eligible cystic fibrosis (CF) patients. The extension adds to the letter of intent (LOI) with the pCPA for…

Researchers at the Children’s Hospital Colorado Breathing Institute are participating in four new studies to evaluate the long-term impact of Trikafta (elexacaftor, tezacaftor, and ivacaftor) in people with cystic fibrosis (CF) ages 6 and older, its effectiveness in infants and children younger than 6, and to find alternative…

Kaftrio successfully improved lung function and quality of life in three Italian cystic fibrosis (CF) patients who have severe lung disease with an unknown mutation in the CFTR gene in addition to the common F508del mutation, a case series reported. Kaftrio (elexacaftor, tezacaftor, and ivacaftor), sold as Trikafta in the…

Vertex Pharmaceuticals is initiating two Phase 3 clinical trials into the safety and efficacy of a potentially new, next-generation triple combination therapy for cystic fibrosis (CF): VX-121 plus tezacaftor and VX-561 (deutivacaftor). Expected to begin later this year, the development program will compare this once-daily therapy with Vertex’s…

Most primary care providers (PCPs) in Ontario — family physicians, pediatricians, and midwives — voiced a willingness and confidence in providing some level of care to infants testing positive for cystic fibrosis (CF) in newborn screening programs, a Canadian survey reported. A majority favored treating minor illnesses like infections…

Inflammation in the airways of people with cystic fibrosis (CF) regulates the pH of the surface liquid and increases the response to approved CFTR modulators, a cell-based study suggested. Airway inflammation may be a key determinant in response to CF medicines, the researchers noted. “To advance personalized CFTR…