2014 March 12
N30 Pharma begins oral dosing in N91115 Phase 1 trial for treatment of cystic fibrosis. N30 Pharmaceuticals announced today that it has begun oral dosing of N91115 in a Phase 1 clinical trial. N91115, a novel inhibitor of S-nitrosoglutathione reductase (GSNOR), is being developed for the treatment of cystic fibrosis (CF). In preclinical studies, N91115 has been shown to rescue or “correct” F508del-CFTR, the most commonly occurring mutation in CF. More …
2014 February 1
The disease-fighting qualities of seaweed compounds are being investigated through trials for new inhalation therapies for cystic fibrosis sufferers. As part of a four-year programme worth £5.4M, awarded by the Norwegian Research Council, researchers including experts from Swansea University, Cardiff University and AlgiPharma AS are employing novel approaches to synthesise and test alginate molecules (components of seaweed) to design a new generation of drug compounds capable of improving the effectiveness of antibiotics. More …
2014 January 14
New Study Shows Bacteria Genetically Evolves to Live in Cystic Fibrosis Patients’ Lungs & Evade Antibiotic Treatments. According to the latest research conducted by scientists from the University of Calgary and the University of Ottawa, the most notorious pathogen that affects cystic fibrosis patients, Pseudomonas aeruginosa, has evolved genetically over time to cause disease in CF patients. In addition, most P. aeruginosa infections are hard to manage, since most antibiotic treatments are less effective in cystic fibrosis patients. More …
2014 January 11
Cornell University biological engineers think they might know why a common pathogen, Pseudomonas aeruginosa, which can lay dormant in healthy individuals, becomes virulent in the lungs of cystic fibrosis patients. They have shown that P. aeruginosa virulence is “turned on” when it feeds on a particular fermentation product called 2,3 butanediol, demonstrating a direct metabolic relationship between fermenting bacteria and P. aeruginosa. This understanding could lead to more effective treatments for cystic fibrosis patients; rather than the use of antibiotics, disrupting P. aeruginosa’s flow of preferred food could be key to preventing cystic fibrosis-related infections in the lungs.
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2013 December 29
Scientists at the European Molecular Biology Laboratory (EMBL) in Heidelberg and Regensburg University, both in Germany, and the University of Lisboa, in Portugal, have discovered a promising potential drug target for cystic fibrosis. Their work, published online in Cell on 12 September 2013, also uncovers a large set of genes not previously linked to the disease, demonstrating how a new screening technique can help identify new drug targets. More …
2013 December 16
Novel potentiator shows more activity in pre-clinical studies than market leader Kalydeco® Start of Phase 1 expected before end 2014. Galapagos NV announced today that it nominated a pre-clinical candidate potentiator for clinical development in its cystic fibrosis (CF) program. More …
2013 December 11
How quickly do hands turn pruney in the water? Slowed wrinkling could mean cystic fibrosis patients are responding to Kalydeco® , a case report suggested. More …
2013 December 5
Using stem-cell technology, Dutch researchers from the Hubrecht Institute and University Medical Center (UMC) in Utrecht have genetically repaired cultured ‘miniature intestines’ or organoids of two cystic fibrosis (CF) patients in the laboratory. “It represents a key step towards stem cell therapy using genetically repaired cells for diseases caused by a single defective gene.” More …