When it comes to treating cystic fibrosis, the current standard of aggressive antibiotic treatments may not always be the best answer, a decade-long study led by researchers at the University of Michigan has found. Traditionally, bacteria-blasting antibiotics are used to suppress infection in CF patients' lungs to the lowest level possible, but maintaining a diversity of bacterial communities may help some patients stay healthy longer, says the study's senior author, John J. LiPuma, M.D. The findings appear in the Proceedings of the National Academy of Sciences. "The conventional wisdom has been that as patients with CF age and become sicker, as their lung disease progresses, more and more bacteria move in, " says LiPuma, the James L. Wilson, M.D., Research Professor of Pediatrics and Communicable Diseases at the U-M Medical School.
A recent study led by Gergely Lukacs, a professor at McGill University's Faculty of Medicine, Department of Physiology, and published in Cell, has shown that restoring normal function to the mutant gene product responsible for cystic fibrosis (CF) requires correcting two distinct structural defects. This finding could point to more effective therapeutic strategies for CF in the future. CF, a fatal genetic disease that affects about 60, 000 people worldwide, is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR), a membrane protein involved in ion and water transport across the cell surface. As such, CF is characterized by impaired chloride secretion causing the accumulation of viscous mucous that may cause multiple organ dysfunctions, including recurrent lung infection.
Kalydeco has been approved by the Food And Drug Administration (FDA) to treat a vicious type of Cystic Fibrosis (CF). CF is a deadly recessive disease which targets the lungs, but can also harm the liver, pancreas, and intestine. It occurs from the unusual transport of chloride and sodium across the epithelium, causing mucus buildup in the lungs, and thick secretions. In turn, many respiratory problems occur in patients with CF. Diabetes is also common among patients with CF and it is most common among Caucasians. This specific type of CF, which Kalydeco has been approved for, targets children 6 years of age and older who possess the G551D mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene. Experts believe that of the 30, 000 people who are affected in the United States by Cystic Fibrosis, an estimated 1, 200 of them possess the G551D mutation.
The lung clearance index (LCI) is a sensitive non-invasive marker of early lung disease in young children with cystic fibrosis (CF), according to a new study from Australian researchers. "We found that LCI is elevated early in children with CF, especially in the presence of airway inflammation and Pseudomonas aeruginosa, " said Yvonne Belessis, MBBS, MPH, PhD, respiratory staff specialist at the Sydney Children's Hospital. "LCI may not only be a marker of early CF lung disease, but may be useful as an objective outcome measure in future studies of young children with CF." The findings were published online ahead of print publication in the American Thoracic Society's American Journal of Respiratory and Critical Care Medicine. LCI was determined after multiple breath washout (MBW) testing in 47 presymptomatic/minimally symptomatic infants and young children with CF (mean age 1.
According to a new Australian study published online before he print publication in the American Thoracic Society's American Journal of Respiratory and Critical Care Medicine, the lung clearance index (LCI) is a sensitive, non-invasive marker of early lung disease in young children with cystic fibrosis (CF). Yvonne Belessis, MBBS, MPH, PhD, respiratory staff specialist at Sydney Children's Hospital declared: "We found that LCI is elevated early in children with CF, especially in the presence of airway inflammation and Pseudomonas aeruginosa. LCI may not only be a marker of early CF lung disease, but may be useful as an objective outcome measure in future studies of young children with CF." The researchers identified LCI following a multiple breath washout (MBW) conducted in 47 presymptomatic/minimally symptomatic infants and infants with CF with an average age of 1.