Lonodelestat (also known as POL6014), Santhera Pharmaceuticals’ investigational therapy to treat chronic lung inflammation, was safe and effective at blocking the activity of a pro-inflammatory biomarker in patients with cystic fibrosis (CF), according to data from a Phase 1b trial. “We are very much encouraged by the…
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The University of Portsmouth, U.K., is partnering with Josh Llewellyn-Jones, a record-breaking endurance athlete who has cystic fibrosis (CF), to drive a scientific PhD research program that aspires to improve the lives of people with the disease. Llewellyn-Jones defied the odds by living past his 30s and…
The United States Adult CF Association (USACFA) is accepting applications to award a total of $15,000 in scholarships to four people with cystic fibrosis (CF)pursuing degrees in the creative arts and other fields. Two of the scholarships — worth $5,000 each — are part of the USACFA’s…
Female adolescents with cystic fibrosis (CF) should be offered contraceptive counseling and informed about pregnancy risks, a long-term study shows. Adding a gynecologist to the medical team following female patients with CF could improve their quality of care, the researchers suggested. The study, “Reproductive Health Counseling and Contraceptive…
A rare and disease-causing mutation for cystic fibrosis (CF), called the F1099L mutation and characterized by a protein maturation defect, was identified in a recent study. Treatment with lumacaftor was also seen to effectively rescue function of the defective CFTR protein that results from this mutation, found in two infants…
Work targeting new therapies against antibiotic-resistant infections, led by West Virginia University (WVU) researchers, received a $1.4 million grant from a branch of the U.S. Department of Defense (DOD). The grant, awarded by the Defense Threat Reduction Agency, will be used to identify the best combinations of antibodies and…
The Cystic Fibrosis Foundation (CFF) has given Beyond Air up to $2.17 million to test its portable inhaled nitric oxide therapy for people with cystic fibrosis (CF) who have chronic, treatment-resistant lung infections caused by non-tuberculous mycobacteria (NTM). This grant award will support a pilot clinical trial to determine…
Researchers have developed a patient-derived model of cystic fibrosis (CF) to better understand whether these patients are more susceptible to COVID-19 infection and its associated serious complications. The project is being led by Ruobing “Ruby” Wang, MD, who cares for CF patients in the division of pulmonary medicine…
ELX-02 restored the levels and function of CFTR — the defective protein in CF — in “mini-organs,” or organoids, derived from patients carrying the most common CF-causing nonsense mutation, a study shows. “ELX-02 increased CFTR activity in a dose-dependent fashion,” with these functional increases being “similar to those obtained…
The National Institute for Health Research (NIHR) awarded a grant worth more than £1.5 million (about $2.1 million) to support a U.K. clinical trial investigating ways of safely lowering the treatment burden of cystic fibrosis (CF), a top priority for patients and their healthcare teams. The randomized and open-label…
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