31/07/2026
🧬 CF Research News!
💙 A second chance for CF patients who had "no options"
For a small group of people with cystic fibrosis, even the best available treatment, ETI, doesn't work. The reason: they carry a rare "double hit," a second hidden mutation (L467F) sitting on the same gene copy as the common F508del mutation. This combination traps the CFTR protein inside the cell so completely that standard triple therapy can't reach it, leaving these patients with no treatment options at all.
🔬 Researchers tested a next-generation drug combination, vanzacaftor/tezacaftor, in nasal cells donated by six patients with this exact double mutation, alongside lab models designed to mimic the trapped protein. They then followed the real-world case of a 36-year-old patient with severe lung decline who was granted compassionate access to the new therapy.
✨ The results were encouraging: the new combination succeeded where ETI had failed, restoring at least some CFTR function, and the patient who received it clinically improved. It's early-stage evidence, not yet a full clinical trial, but for people who were told their genotype meant no treatment could help them, this is real reason for hope. 💙
📖 Read the full study: https://pubmed.ncbi.nlm.nih.gov/42243036/
📰 Also in this issue:
🌬️ Could your breath reveal whether your CF treatment is working?
Early research suggests analyzing exhaled breath could offer a fast, simple way to track early response to Kaftrio/Trikafta.
📖 Read more: https://pubmed.ncbi.nlm.nih.gov/42386422/
👃 A new treatment for severe nasal polyps in people with cystic fibrosis
For people with CF and severe nasal polyps that haven't responded to surgery or standard treatment, a new study found the biologic drug dupilumab meaningfully improved sinus symptoms and quality of life.
📖 Read more: https://pubmed.ncbi.nlm.nih.gov/41986171/
🦠 Friendly bacteria may help protect CF lungs from infection
Scientists discovered that a naturally occurring bacteria in CF lungs, Prevotella, can help defend lung cells against the harmful CF pathogen Staphylococcus aureus.
📖 Read more: https://pubmed.ncbi.nlm.nih.gov/41198444/
🧫 Does the newest CF treatment work for rare genetic variants too?
Using mini gut models grown from patient cells, researchers found the newest once-daily CF treatment works about as well as current triple therapy, even for people with rare CFTR variants often left out of clinical trials.
📖 Read more: https://pubmed.ncbi.nlm.nih.gov/42303492/
👶 New guidance for babies exposed to CF drugs before birth or through breastfeeding
A new international consensus, endorsed by the European Cystic Fibrosis Society, offers the first clear guidelines for monitoring children exposed to CFTR modulators during pregnancy or breastfeeding.
📖 Read more: https://pubmed.ncbi.nlm.nih.gov/42049593/
🦷 Why dental health matters for lung health in CF
New research links dental problems like tooth decay and gum disease to lung health in adolescents and young adults with CF, a reminder that oral care matters for CF health too.
📖 Read more: https://pubmed.ncbi.nlm.nih.gov/42414103/
💙 Every new study brings us one step closer to better treatments and better lives for everyone with cystic fibrosis. Which of these research updates did you find most interesting?
