A new drug candidate has been found to hold promise for cystic fibrosis patients.
Dr David Sheppard from the University of Bristol said that the early results with VX-770 suggest that drug therapies, which target defects at the root of the disease, have the potential to greatly improve the quality of life of CF patients.
AdvertisementCurrently, there is no cure for CF caused due to a defective gene that causes ducts and tubes in the body to become blocked by thick, sticky mucus. This mucus affects the lungs, pancreas, the intestines, the liver and the reproductive organs. One of the most recognisable symptoms of CF is 'salty sweat', caused by the failure of the sweat ducts to reabsorb salt
The defective gene disables or destroys a protein known as CFTR.
The research team led by Sheppard is investigating how new drugs restore function to defective CFTR proteins.
The new drug therapy (VX-770) was developed by Vertex Pharmaceuticals and will tackle the 'functional' defect.
The researchers have tested the drug on patients in the US who carry a genetic defect known as G551D.
The early results were encouraging. Patients who received 150mg twice a day saw the concentration of salt in their sweat decrease by almost 50 per cent and lung function improve by 10 per cent.
The study was presented at the BA Festival of Science in Liverpool.
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