Unsuspected characteristics of new cystic fibrosis drugs found, offering potential paths to more effective therapies
A large phase 3 clinical trial for cystic fibrosis patients has concluded, showing that a combination of two new cystic fibrosis drugs modestly improved lung function and offered better health outcomes for some patients. Now, scientists have shown that one of these drugs counteracts the intended beneficial molecular effect of the other.
Abstract Kairen Griffiths, Cystic Fibrosis Nurse Specialist, NHS Grampian, discusses her role caring for people with cystic fibrosis and why she feels lucky to have worked in this field for over two decades. PMID: 31714832 [PubMed - in process]
ConclusionsTherapeutic drug monitoring-guided meropenem may be a sound new therapeutic option in eradicating multidrug resistant Acinetobacter and offer a novel therapeutic option in the field of personalized medicine.
Vertex Pharmaceuticals Inc's drugs for lung condition cystic fibrosis will now be available to patients across the UK after the company reached a pricing deal with Wales on Wednesday.
ConclusionCFTR could inhibit cell autophagy by enhancing PI3K/AKT/mTOR signaling pathway, thereby playing a protective role in LPS-induced ALI in mice.
Cystic fibrosis (CF) is the most common severe autosomal recessive genetic disease in Caucasians caused by variants in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR), a chloride channel expressed in epithelial cells throughout the body . To date, over 2,000 variants in the CFTR gene have been identified world-wide. The international CF genetics research community has contributed these data to the CFTR2 database (http://www.cftr2.org), which provides information on different CFTR variants and their disease liability by a phenotype-driven approach .
ConclusionNebulized NaHCO3 inhalation appears to be a safe and well tolerated potential therapeutic agent in the management of CF. Nebulized NaHCO3 inhalation temporarily elevates airway liquid pH and reduces sputum viscosity and viscoelasticity.
Authors: Ruszel N, Kubisa B, Lisowski P, Piotrowska M, Kubisa MJ, Brykczyński M, Wojtyś M, Pieróg J, Czarnecka M, Wójcik J, Wójcik N, Sielicki P, Bielewicz M, Grodzki T PMID: 31708987 [PubMed]
Publication date: Available online 11 November 2019Source: The Lancet Respiratory MedicineAuthor(s): Daniel J Wolter, Frankline M Onchiri, Julia Emerson, Mimi R Precit, Michael Lee, Sharon McNamara, Laura Nay, Marcella Blackledge, Ahmet Uluer, David M Orenstein, Michelle Mann, Wynton Hoover, Ronald L Gibson, Jane L Burns, Lucas R Hoffman, Daniel J Wolter, Frankline M Onchiri, Julia Emerson, Mimi R Precit, Michael LeeSummaryBackgroundStaphylococcus aureus is the bacterium cultured most often from respiratory secretions of people with cystic fibrosis. Both meticillin-susceptible S aureus and meticillin-resistant S aureus (MR...
Publication date: Available online 11 November 2019Source: The Lancet Respiratory MedicineAuthor(s): Steve Cunningham
(eLife) Scientists have revealed how common respiratory bugs that cause serious infections in people with cystic fibrosis interact together, according to a new study in eLife.