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Cystic Fibrosis Gene Therapy Programme Gets Green Light with Public Funding

Published: Friday, March 16, 2012
Last Updated: Thursday, March 15, 2012
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Gene therapy trial for CF to begin in March.

A gene therapy trial for cystic fibrosis (CF) will begin in March, thanks to a new grant from a government funding body.

One hundred and thirty adults and children with CF will take part in the largest trial of its type yet, coordinated by the UK Cystic Fibrosis Gene Therapy Consortium (GTC).

The trial will be funded by a £3.1 million grant from the National Institute for Health Research (NIHR) and the Medical Research Council (MRC) through the Efficacy and Mechanism Evaluation programme.

The MRC, through their Developmental Pathway Funding Scheme (DPFS), will also fund a £1.2m study by the GTC aiming to develop a potentially more efficient delivery method for the gene therapy, which could contribute to an even more effective treatment in the future.

The GTC is a group of scientists and clinical teams from Imperial College London, the Universities of Oxford and Edinburgh, Royal Brompton & Harefield NHS Foundation Trust and NHS Lothian who have worked together for the last decade to develop gene therapy for CF.

CF is the commonest lethal inherited disease in the UK, affecting around 9,500 people nationally and over 90,000 worldwide. Patients' lungs become filled with thick sticky mucus and they are vulnerable to recurrent chest infections, which eventually destroy the lungs.

The cause of CF, mutations in a gene located on chromosome 7, was identified in 1989, opening the door to replacing this faulty gene using gene therapy.

Patients 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 at Royal Brompton Hospital, London and Western General and Royal Hospital for Sick Children in Edinburgh, 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.

The second, lab-based study will investigate a more advanced version of the therapy using a modified virus to carry the replacement gene into the lungs, which could in future lead to a more efficient delivery mechanism.

Professor Eric Alton, the GTC Coordinator, from Imperial College London and consultant physician at Royal Brompton Hospital 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.

"We would like to thank the NIHR and MRC for their confidence in us, and also all of those whose support has reinforced our determination to carry out this programme for the benefit of CF patients. We would also like to thank the Cystic Fibrosis Trust and the many families and supporters for funding our programme up to this stage."

The outcome of the trial will be known in spring 2014 and regular progress reports will be posted on the Consortium's website (

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