Research Team led by Queen's University developed the First Ever Drug to treat Celtic Gene

A research team led by Queen's University developed the first ever drug to treat Celtic gene in patients suffering from cystic fibrosis (CF). Those patients suffering from cystic fibrosis who took the drug 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 because 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.

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

This is the first drug 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.

VX-770 will open the defective channel in the lung cells of people with Cystic Fibrosis and allow proper lung clearance of bacteria. This is a ground breaking treatment because it treats the basic defect caused by the gene mutation in patients.