Paediatric Respiratory Medicine

Major trial on new treatment for Cystic Fibrosis show promising results


A major trial on 1,108 patients, published in the New England Journal of Medicine, showed a combination of drugs could bypass the genetic errors that cause cystic fibrosis (CF) and improve lung function.

Patients at Nottingham University Hospitals took part in the trial of a new treatment for CF patients which has shown a significant improvement in patients’ symptoms. The trial was lead by Professor Alan Smyth from The University of Nottingham School of Medicine and supported by the Children’s Research Team at NUH.

One in every 2,500 babies in the UK has cystic fibrosis and the condition can shorten lives, as mucus clogs and damages lungs leaving individuals prone to infection.

“As this leading edge of science continues to be explored and better understood, we are hopeful that a future of personalised medicines is increasingly within reach.” Janet Allen, Director of Research, Cystic Fibrosis Trust.

In the research trial the combination of drugs – lumacaftor and ivacaftor – were designed to repair the genetic defect that leads mucus accumulating in the lungs. The trial showed that those patients given the combination for 24 weeks had better lung function and had fewer chest infections.

There are however, many types of error in the DNA that can cause in cystic fibrosis. The treatment combination should work on around half of patients, while one of the drugs on its own corrects a small proportion of errors. New treatments are still required for the remaining patients.

Janet Allen, the director of research at the Cystic Fibrosis Trust charity, said: “These results open up a new front in the fight against cystic fibrosis and this combination therapy looks set to be an important additional treatment option that could improve the lives of many.”


Find out more about Paediatric Respiratory Medicine research at The University of Nottingham School of Medicine.

Read more research news from Nottingham University Hospitals.

Posted on Thursday 9th July 2015

Paediatric Respiratory Medicine Research Group

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