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Medical News Briefs

PTC 124 Shows Promising Phase 2 Results

PTC Therapeutics, Inc. recently announced the findings from two Phase 2 trials of PTC124 for the treatment of CF.  PTC124 targets “nonsense mutations,” which are changes in the CF gene that prematurely stop the production of the full-length CFTR protein.  The stunted protein cannot form the channel needed for chloride secretion and regulation of sodium absorption.  Preliminary results showed that PTC124 can restore CFTR function in airway cells and reduce blood neutrophil counts, which are a hallmark of the CF inflammation/infection cycle.   Improved pulmonary function and body weight were also observed.  Plans are underway to begin a Phase 3 trial.

Gilead Takes Antibiotic To Phase 3

Gilead Sciences Inc. released promising results from a new antibiotic treatment for people with CF. A Phase 3 trial, carried out in collaboration with the CF Foundation, involved 247 volunteers with Pseudomonas aeruginosa infections who were on chronic inhaled tobramycin. Researchers found that volunteers receiving aztreonam lysine for inhalation could do without additional inhaled or intravenous antibiotic treatments for significantly longer than those who received the placebo treatment. Another Phase 3 trial focusing on volunteers with CF who do not use inhaled tobramycin regularly is nearing completion.

Azithromycin Follow-up Trial Underway

Cystic Fibrosis Foundation Therapeutics, Inc. is sponsoring a follow-up clinical trial to study the effects of azithromycin on children ages 6 to 18 years with CF who are not infected with P. aeruginosa.  Three previous trials showed improvements in lung function and a decrease in pulmonary exacerbations with azithromycin use.  This evidence has led to widespread use of this therapy for patients chronically infected with P. aeruginosa.  This new trial aims to determine whether azithromycin could provide benefits to CF patients who are not infected with P. aeruginosa, as it does for those infected with the bacterium.