New wonder drug can heal lungs of Cystic Fibrosis sufferers

A breakthrough drug for a rare form of cystic fibrosis has been shown in trials to transform the lives of the most ill patients. Kalydeco (ivacafto), is the first of its kind and alters a protein on the defective gene.

One patient to have experienced an astonishing return to health is Georgina Petrie, who just weeks ago was confined to a wheelchair.

The 22-year-old, whose lungs had been damaged by infections caused by the mucus that is the hallmark of cystic fibrosis, was struggling to breathe. Her consultants believed that a lung transplant was her only hope.



Back on the bike: Cystic Fibrosis sufferer Georgina Petrie was confined to a wheelchair for 18 months before she started taking Kalydeco and now she is able to ride her bicycle again

Within a week of starting on Kalydeco, she no longer needed an oxygen mask to aid her breathing. A week later, she had started cycling, swimming and taking long walks.

‘It all happened so quickly, I’m finding it hard to take in,’ she says. ‘A few weeks ago I was very poorly and could do nothing.’

CF is caused by any one of several defects in a protein, cystic fibrosis transmembrane conductance regulator (CFTR), which regulates fluid flow within cells and affects components of sweat, digestive fluids, and mucus.

The CFTR mutation in CF patients allows too much salt and water into cells, which results in a build-up of thick, sticky mucus in the body’s tubes and passageways that damages the lungs, digestive system and other organs.



One type of mutation, G551D, is found in about five per cent of cases of CF, and Kalydeco is effective for these patients.



The drug thins the mucus, preventing further damage and allowing the lungs to heal.



Kalydeco (not pictured) is a drug which which could change lives for CF patients



In the past two years, about 15 patients at the Royal Brompton Hospital in London and a handful in Belfast have been given the drug as part of a trial. It has now been licensed for NHS use but NICE is reviewing the cost.

The daily tablet is estimated to cost £200,000 per year per patient. About 600 patients have the G551D form of CF in the UK, but US manufacturers Vertix are already trialling a new drug that will be effective in 50 per cent of all CF cases.

Patients have to stay on the drug permanently for it to continue to work.

Georgina was prescribed the new drug with the support of her doctor at the Royal Brompton. Prior to this she was on a cocktail of 20 drugs, nebulisers, inhalers and oxygen.

Dr Jane Davies, a respiratory consultant at London’s Royal Brompton Hospital, headed the UK trial of the drug.