A novel treatment strategy for cystic fibrosis (CF) that’s based on a cellular structure called the porosome showed promise in cell and animal models of CF caused by the common F508del mutation. In the cell model, this approach was more potent than tezacaftor and ivacaftor, which are CFTR modulators used in approved CF therapies, according to data presented by Porosome Therapeutics, the company developing the approach. Findings were showcased at the April summit of the American Physiological Society in Long Beach, California, in the poster “Cystic fibrosis rescued using a reprogrammed po ..read more

Highly effective CFTR modulators, which target the cause of cystic fibrosis (CF), may ease signs of low iron levels in some patients with the disease, but low iron levels may remain even with treatment with the medications, a study finds. The scientists also emphasized that people with CF who are not on modulators due to lack of a qualifying mutation, intolerance to treatment, or lack of availability are prone to iron deficiency. The study, “Association between CFTR modulators and changes in iron deficiency markers in cystic fibrosis,” was published in the Journal of Cystic Fibrosis by resear ..read more

Biopharma and biotech companies have launched more than 450 clinical trials into potential new treatments for cystic fibrosis (CF) since 2018, according to a report from Novotech, a contract research organization that specializes in helping drug developers to run trials. “There are ample opportunities presented by advancements in technology, personalized medicine, and the emergence of new therapies, all of which hold the promise of improving outcomes,” the report states, adding that “the future for individuals with CF is increasingly hopeful.” CF is caused by mutations in the gene that e ..read more

I’ve had so many surgeries that I’ve lost track. You’d think I’d be used to the routine by now, but I’m not. With each surgery comes more anxiety as my trauma stacks up. I was convinced it’d get easier as I underwent more procedures, but I’ve learned that each one presents unique challenges. Growing up with cystic fibrosis (CF) brings a plethora of health hiccups. But while the CF community often talks about the physical symptoms we face, the emotional impact of our disease doesn’t get as much attention, nor does it resolve overnight. My CF led to a lifesaving double-lung transplant six years ..read more

The CFTR modulator therapy Kalydeco (ivacaftor) can safely be given to babies with cystic fibrosis (CF) as young as 4 weeks old, according to data from a Phase 3 clinical trial. The findings were the basis of the U.S. Food and Drug Administration (FDA)’s decision to expand approval of Kalydeco for children in this age range last year. The therapy was recently recommended for a similar expansion in Europe, where it’s approved for babies 4 months and older. “This is a huge moment in cystic fibrosis,” Paul McNally, MD, co-author of the study at the Royal College of Surgeons in Ireland, said in a ..read more

Treatment with Kaftrio (elexacaftor/tezacaftor/ivacaftor) led to gains in body mass among cystic fibrosis (CF) patients that were largely driven by increases in various types of fat and low-density muscles, a study found. High-density muscle, which is stronger than its lower-density counterparts, didn’t change with the treatment, sold as Trikafta in the U.S. A higher amount of low-density muscle was linked to worse lung function, although patients generally experienced lung function gains regardless. The study, “Impact of CFTR Modulator Therapy on Body Composition as Assessed by Thoracic Comp ..read more

While Kaftrio — sold as Trikafta in North America — can improve lung function in children with cystic fibrosis (CF), treatment effects are unlikely to be as dramatic in real-world practice as those reported in clinical trials. Rather, for eligible children without substantial lung problems, Kaftrio’s use can help to stabilize lung health. But it probably won’t lead to the substantial gains seen in trials that specifically enrolled young patients with poor lung function, scientists in the U.K. report. “The importance of stability and maintenance of normal lung function … is perhaps an importan ..read more

The Cystic Fibrosis Foundation (CFF) on April 30-May 1 will present ResearchCon, a free virtual event for patients, caregivers, scientists, and clinicians that will explore research advancements and the future of cystic fibrosis (CF). The event, created with the CF community, will include keynote addresses, presentations, roundtable discussions, and social lounges. Registration is open. In addition to topics including gene therapy development, life without CFTR modulators, and pain management, this year’s meeting also will cover “popular topics in the CF community” such as clinical research p ..read more

Mutations that cause cystic fibrosis (CF) lead to biochemical changes in lung cells that make it easier for infectious bacteria to stick to the cells, a new study reports. Findings notably suggest that treatment with Trikafta (ivacaftor/tezacaftor/elexacaftor) did not reverse these changes, which may help to explain why the therapy generally isn’t good at fending off infections. The study, “Akt-driven TGF-β and DKK1 Secretion Impairs F508del CF Airway Epithelium Polarity,” was published in the American Journal of Respiratory Cell and Molecular Biology. Recommended Reading March 26, 2024 Ne ..read more

In lab studies, researchers have identified rare mutations in the CFTR gene — which are the cause of cystic fibrosis (CF) — that are not currently eligible for CFTR modulator therapies but might be treatment responsive. The researchers hope the findings will provide guidance to developer Vertex Pharmaceuticals when considering seeking label expansions that would make the treatments eligible for more types of mutations. They also might inform doctors and patients about possibly seeking off-label trial periods of the medications where certain noneligible mutations exist. The study, “In vit ..read more