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Cystic fibrosis drug offers hope for children with the disease

Cystic fibrosis drug offers hope for children with the disease

Jane Davies
Professor Jane Davies

7 July 2017


Orkambi, a combination drug for cystic fibrosis, has been found to improve the lung function of children suffering from the disease.


An international study, which included researchers from Royal Brompton Hospital, showed that the drug Orkambi can improve lung damage caused by cystic fibrosis in under two weeks and could add years to the lives of people with the disease.


Cystic fibrosis (CF) is caused by mutations in the gene which controls the movement of salt and water in and out of cells. This leads to a build-up of mucus in the lungs and digestive system. 


Orkambi – a combination of lumacaftor and ivacaftor – is one of the first therapies to target the most common form of mutation, rather than the symptoms. About half of the people with cystic fibrosis in the UK could benefit from the treatment.


The phase three trial involved 204 patients aged 6 to 11 years old, with half the patients receiving Orkambi for six months, and the other half a placebo. The results were recently published in The Lancet Respiratory Medicine


Researchers found that the drug could halt or even reverse lung damage in children under 12 years. It has also been found to slow the loss of lung function in older patients.


The study showed that the children’s airways were significantly healthier based on a sensitive test of gas mixing in the lungs. There was also a 20 per cent reduction in the amount of chloride in their sweat – a common symptom of the disease.


Jane Davies, honorary consultant in paediatric respiratory medicine at the Trust, who helped coordinate the trial at Royal Brompton Hospital said: “This is the first time the lung clearance index has been used in this type of clinical trial, which was only possible thanks to the co-ordinated efforts of European and North American groups. It shows that even young children with relatively early stage CF can benefit from new treatments, but that trial design is really key.”


The UK is one of the countries with the highest prevalence of cystic fibrosis in Europe, with around 10,500 people suffering from the genetic condition.  Whilst the prognosis for people with the disease has vastly improved over the years - a child born today is likely to have a mean life expectancy of over 40-50 years - experts stress that early treatment in children is critical for their long-term outlook.


Orkambi has been approved for use in other countries but is not yet available on the NHS because of its £104,000 a year per patient price tag. 


The Cystic Fibrosis Trust is campaigning to have the drug reconsidered. Nick Medhurst, head of policy at the charity, said: “Orkambi has been licensed for use in those aged over 12 in the UK for over two years, but people are still not receiving it.” 


Seminar on new ways of detecting and treating infection in respiratory disease


Professor Jane Davies will be speaking about her clinical research on detecting and treating infection in cystic fibrosis patients at a free seminar on Tuesday 25 July. 


The event takes place at Royal Brompton Hospital and all are welcome.


Find out more information and register your interest







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