Breakthrough cystic fibrosis drug hailed by family of Kesgrave’s Kerry Thorpe

Cystic Fibrosis sufferer, Kerry Thorpe from Kesgrave is pictured with fiance Josh Nelli who gave up work to become her full time carer (Image: Archant)

Mr Nelli, who has helped raise thousands of pounds for the Kerry Alex Thorpe Trust, praised an international trial which has found that new treatment could possibly cut chest infections for half of people living with CF.

CF is a genetic disease in which thick mucus clogs and damages the lungs, leaving people vulnerable to infection. But the new treatment has been shown to modify the genetic defect that causes around half of cases.

Professor Stuart Elborn, who led the European part of the trial at Queen’s University, Belfast, described the drug as a “breakthrough”.He said young patients’ lives were changed to the extent they could start families, go to work and come off lung transplant lists after receiving the drug.

Mr Nelli said: “It’s always positive to read of possible new ways and treatments to make cystic fibrosis patients’ lives more tolerable and comfortable. Having seen first hand with Kerry how debilitating the condition can be, I think it’s fantastic that these treatments are being discovered, research and actively worked upon.

“Like with many conditions, if funding was unlimited, then cures potentially do exist, which is why it’s important to support charities as you never know what newfound cures could be waiting round the corner.”

Lung function was improved and hospital visits reduced in the trial on 1,108 patients, according to results of the clinical trial published in the New England Journal of Medicine.

The US drugs watchdog, the Food and Drug Administration, is expected to make a decision on whether to approve the drug, called Orkambi, by July 5.

An application for its approval in Europe is being considered by European Medicines Agency, with a decision expected in the autumn.

Although the trial cannot show that children will live for longer but by reducing the disease’s severity, researchers hope that the treatment will increase life expectancy.

Janet Allen, director of research and care at the Cystic Fibrosis Trust, said: “These results open up a new front in the fight against cystic fibrosis.”

Kerry, from Kesgrave, bravely battled cystic fibrosis, raising awareness of the condition and the desperate need for people to sign the organ donation register, before passing away on March 21 last year aged 23.