Cystic fibrosis breakthrough has doctors buzzing

(CBS) "It has the potential to change the lives for those with cystic fibrosis."

That's what Dr. Damien Downey, physician at Belfast Health and Social Care Trust in Northern Ireland, said of a new drug some people are calling a miracle treatment.

The drug, called VX-770, targets the so-called "Celtic gene" that's found in many people who suffer from the disease. The G551D gene bears the nickname because of its prevalence in Ireland, where cystic fibrosis is the most common, potentially lethal-hereditary disease.

But the researchers behind VX-770's early experiments say the drug can even help cystic fibrosis sufferers without the Celtic gene, because it opens a defective channel in the lungs, helping the body expel bacteria. People who took VX-770 saw significant improvement in lung function and quality of life, in addition to a reduction in disease flare ups.

"This is a ground breaking treatment because it treats the basic defect caused by the gene mutation in patients," Dr Judy Bradley, a respiratory disease specialist from the University of Ulster in Belfast, said in a written statement.

The drug is at least a year away from hitting the market, but the early results are so positive that Vertex Pharmaceuticals announced it would start giving the medicine to patients who are in "critical medical need," according to a statement on the Cystic Fibrosis Foundation website.

And that's why scientists are giddy over the treatment implications.

"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," co-atuhor Dr. Stuart Elborn, director for the Centre for Infection and Immunity at Queen's University, said in the statement.

Cystic fibrosis causes the body to produce abnormally thick mucus that builds up in the lungs and pancreas. It may lead to life-threatening lung infections and digestive problems. On average, people with cystic fibrosis live only 35 years.

The Cystic Fibrosis Foundation has more on the disease.