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Wednesday, February 11, 2009

Cystic Fibrosis May Be Prevented by Spraying Amiloride in Lungs

Category: Respiratory Disease News
Monday, February 09, 2009 at 11:17:21 AM
Following a lab test on mice, scientists have realised that spraying amiloride into the lungs of may help prevent the onset of cystic fibrosis lung disease. The researchers at Department of Paediatrics at Heidelberg University Hospital have demonstrated for the first time that preventative therapy for the lung disease is possible.
Attacking the root cause of the widespread hereditary disease, the mice in the study were given an inhalation treatment with the drug in the first days of life. It was found that no thick mucus forms in the lungs and airway inflammation and chronic lung damage could be prevented. Cystic fibrosis (CF) or mucosviscidosis or is the most common life-shortening genetic disease, caused by defects at a certain locations in the genetic makeup - a mutation in what is called the CFTR ("Cystic Fibrosis Transmembrane Conductance Regulator" gene).
The mutation leads to loss of salt and water and thus dehydration of the surfaces of the mucous membranes in the lungs, intestines, and other organs. Amiloride inhibits "hyperactive" sodium channels and prevents the mucous membranes from drying out Using the mouse model he developed, Dr. Marcus Mall, physician scientist and head of the Cystic Fibrosis Center at the Department of Pediatrics has shown that certain "hyperactive" sodium channels in airway cells are responsible for the increased absorption of salt and water from airway surfaces.
For the study, researchers tested whether inhibiting these hyperactive sodium channels with amiloride could improve hydration of the airway surfaces and prevent lung damage. It was found that in a mouse model, amiloride administered in the first few days of life prevents the typical symptoms of cystic fibrosis and the development of chronic lung disease. However, the treatment brought no improvement if the symptoms were already present Chronic lung damage apparently prevents the amiloride from being effective. "This indicates that the lung damage caused by the disease may be irreversible", stated Dr. Mall. "With amiloride, preventative therapy of cystic fibrosis that attacks the basic defect directly could be possible for the first time. But the children affected would have to be identified at a very early stage", said Dr. Mall. The study has been published in the "American Journal of Respiratory and Critical Care Medicine".
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