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£3.1 million to fund CF gene therapy trial

16 March 2012


Government funding announced today will allow a groundbreaking gene therapy trial for cystic fibrosis (CF) to go ahead. The £3.1million grant from the National Institute for Health Research (NIHR) and the Medical Research Council (MRC) will fund the largest trial of its type yet with 130 adults and children with CF taking part.


The trial, which is due to begin very soon, will be co-ordinated by the UK Cystic Fibrosis Gene Therapy Consortium (GTC), a group of scientists and clinical teams from Royal Brompton & Harefield NHS Foundation Trust, Imperial College London, the universities of Oxford and Edinburgh and NHS Lothian. The group has worked together for the last decade to develop gene therapy for CF, which is the most common fatal inherited disease in the UK, affecting around 9,500 people nationally and over 90,000 worldwide.


The trial participants, of whom more than 50 per cent are patients at Royal Brompton Hospital, will receive the treatment by inhaling molecules of DNA wrapped in fat globules that deliver the replacement gene into the cells in the lung lining. Half the participants will receive the real treatment and half a placebo in a double-blind study. Patients aged 12 and over will receive one dose a month for one year.

Over 30 patients have each received a single dose of the gene therapy in the consortium’s previous studies, looking at how effective the therapy is at replacing the protein encoded by the defective CF gene. By delivering multiple doses over the course of a year, the researchers aim to determine whether the therapy can improve symptoms for CF patients.


Professor Eric Alton, consultant physician at Royal Brompton Hospital and co-ordinator of the GTC, said: “Conventional treatments have extended the life expectancy for people with CF. We’re hoping that this therapy will achieve a step change in the treatment of CF that focuses on the basic defect rather than just addressing the symptoms. This trial will assess if giving gene therapy repeatedly for a year will lead to the patients’ lungs getting better. Eventually we hope gene therapy will push CF patients towards a normal life expectancy and improve their quality of life significantly.”


At present, Royal Brompton’s children’s cystic fibrosis unit is under threat as a result of the proposed removal of children’scardiac surgery services and paediatric intensive care and anaesthesia. If recommendations by a Joint Committee of Primary Care Trusts are implemented, Royal Brompton’s entire paediatric unit will be destabilised with specialist respiratory care for children being untenable as a standalone service. A detrimental impact on Royal Brompton’s adult cystic fibrosis unit is also predicted as 70-80 per cent of adult patients progress from the paediatric service.


Angharad Sutton, 32, who has been a patient at Royal Brompton for 10 years and took park in the earlier single dose pilot study, said: “There is a huge amount of benefit to being a patient at a hospital like Royal Brompton. You are getting your care within an environment of research and everything that is happening to you is being thought about in that wider context.”


The outcome of the gene trial will be known in spring 2014 and regular progress reports will be posted on www.cfgenetherapy.org.uk.


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