Radical treatment gives new hope to CF patients

CYSTIC fibrosis sufferers have been given hope by a radical new treatment that could save thousands of people with the lung disease from an early death.

Scientists have found combining three drugs has a major positive impact on the lung function of most people with the condition.

Researchers had earlier achieved success in combining two agents, which help slow down lung damage in around 40% of CF patients.

Now they have shown adding a third element produces similar benefits in up to 90% of patients – including those who only have one copy of a faulty gene that causes the disease, called F508del.

Drugs company Vertex, the makers of Orkambi, are behind the research and claim their results are ‘clear and compelling, indicating significant potential benefit’.

CF is a genetically inherited disease that affects the lungs and digestive system. Ireland has the highest rate of CF per head of population in the world – one in 19 people here carry a version of the altered gene that causes the condition. Around 1,200 Irish people are living with the condition, with approximately 50 new cases being diagnosed each year.

Genetic fault means sufferers’ lungs produce too much mucus, eventually leading to scarring, and patients often need a lung transplant to prolong their lives.

Professor Jane Davies, honorary consultant in paediatric medicine at the Royal Brompton Hospital in London explained how the new ‘triple therapies’ involved combining the existing ‘double therapy’ medicine, called Orkambi, with a third agent.

She said: ‘The trial data show that even if you’ve only got one F508del mutation, these triple combinations work effectively so you get significant clinical improvement.’

Should these ‘very, very early’ results hold up in further trials, and the method prove safe, the tripletherapies could be used in ‘somewhere between 70% and 90% of CF patients globally’, she said – a ‘transformative’ prospect.

However, she cautioned that they would not clear up irreversible damage done by long-term CF.