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New Drug Offers Hope For Cystic Fibrosis

June 20, 2011

An international team co-led by scientists at Queen’s University, Belfast, has developed a new drug for Cystic Fibrosis sufferers.

The drug specifically targets the so-called Celtic gene which is common in Ireland.

But the researchers believe the breakthrough will have significant implications for all CF sufferers.

The drug should be available to patients next year. Patients take two tablets a day.

Less than a year ago, scientists were awarded £1.7m to research and develop the drug. Specialists from Europe, America and Australia were involved in the project.

They found significant improvement in lung function, quality of life and a reduction in disease flare ups for those receiving the new treatment.

Stuart Elborn from Queen’s University, Belfast, co-led the team.

“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.”

Dr Judy Bradley, from the University of Ulster said: “This is a ground breaking treatment because it treats the basic defect caused by the gene mutation in patients.

“Correcting the cells with this mutation shows that treatments aimed at the basic mutation can work leading to improvements in lung function and symptoms.”

Dr Damien Downey, from the Belfast Health and Social Care Trust said: “The success of this study illustrates the benefits that come from collaborative work here in Northern Ireland.

“Not only will this breakthrough help patients in Ireland and the UK but it has the potential to change the lives for those with Cystic Fibrosis around the world.”

As a result of the recent work researchers from Queen’s University, University of Ulster and clinicians from Belfast Health and Social Care Trust have been selected to join the European Cystic Fibrosis Society Clinical Trials Network.

This means Cystic Fibrosis researchers in Northern Ireland will be collaborating with their European counterparts to work toward improved treatments for Cystic Fibrosis on a global level. “

The new drug will be submitted for licensing in the Autumn of this year and is expected to be available to patients by as early as next year.

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