New drug to treat cystic fibrosis patients

A research team led by Queen's University has developed the first ever drug to treat 'Celtic gene' in patients suffering from cystic fibrosis.

Those who were given the new treatment, showed significant improvement in the lung function, quality of life and a reduction in disease flare-ups.

The drug (VX-770) is a significant breakthrough not only for those with the 'Celtic gene', known as G551D, but also for all other cystic fibrosis sufferers as it indicates that the basic defect in cystic fibrosis can be treated.

This is the first drug aimed at the basic defect in cystic fibrosis to show an effect.

It may or may not improve the life expectancy, but the improvements in the breathing tests and the reduction in flare-ups suggested the survival will be better.

“The development of this drug is significant because it is the first to show that treating the underlying cause of cystic fibrosis may have profound effects on the disease, even among people who have been living with it for decades.

The remarkable reductions in sweat chloride observed in this study support the idea that VX-770 improves protein function thereby addressing the fundamental defect that leads to CF,” said Stuart Elborn, Centre Director in the Centre for Infection and Immunity at Queen's University.

“This drug opens the defective channel in the lung cells of people with cystic fibrosis and allows proper lung clearance of bacteria. This is a ground breaking treatment because it treats the basic defect caused by the gene mutation in patients,” added Dr Judy Bradley, from the University of Ulster.

The drug is expected to be available to patients by as early next year.