A groundbreaking 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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Notes to editors:
1. The clinical trial has been awarded by the Efficacy and Mechanism Evaluation programme (www.eme.ac.uk ) researcher-led work stream, which is funded by the Medical Research Council (MRC) and managed by the National Institute for Health Research (NIHR). The trial will be managed by the NIHR Evaluation, Trials and Studies Coordinating Centre (NETSCC) based at the University of Southampton.
2. For almost 100 years the Medical Research Council has improved the health of people in the UK and around the world by supporting the highest quality science. The MRC invests in world-class scientists. It has produced 29 Nobel Prize winners and sustains a flourishing environment for internationally recognised research. The MRC focuses on making an impact and provides the financial muscle and scientific expertise behind medical breakthroughs, including one of the first antibiotics penicillin, the structure of DNA and the lethal link between smoking and cancer. Today MRC funded scientists tackle research into the major health challenges of the 21st century. www.mrc.ac.uk
3. The National Institute for Health Research provides the framework through which the research staff and research infrastructure of the NHS in England is positioned, maintained and managed as a national research facility. The NIHR provides the NHS with the support and infrastructure it needs to conduct first-class research funded by the Government and its partners alongside high-quality patient care, education and training. Its aim is to support outstanding individuals (both leaders and collaborators), working in world class facilities (both NHS and university), conducting leading edge research focused on the needs of patients. www.nihr.ac.uk
4. About Imperial College London
Consistently rated amongst the world's best universities, Imperial College London is a science-based institution with a reputation for excellence in teaching and research that attracts 14,000 students and 6,000 staff of the highest international quality. Innovative research at the College explores the interface between science, medicine, engineering and business, delivering practical solutions that improve quality of life and the environment - underpinned by a dynamic enterprise culture.
Since its foundation in 1907, Imperial's contributions to society have included the discovery of penicillin, the development of holography and the foundations of fibre optics. This commitment to the application of research for the benefit of all continues today, with current focuses including interdisciplinary collaborations to improve global health, tackle climate change, develop sustainable sources of energy and address security challenges.
In 2007, Imperial College London and Imperial College Healthcare NHS Trust formed the UK's first Academic Health Science Centre. This unique partnership aims to improve the quality of life of patients and populations by taking new discoveries and translating them into new therapies as quickly as possible.
5. Oxford University's Medical Sciences Division is one of the largest biomedical research centres in Europe, with over 2,500 people involved in research and more than 2,800 students. The University is rated the best in the world for medicine, and it is home to the UK's top-ranked medical school.
From the genetic and molecular basis of disease to the latest advances in neuroscience, Oxford is at the forefront of medical research. It has one of the largest clinical trial portfolios in the UK and great expertise in taking discoveries from the lab into the clinic. Partnerships with the local NHS Trusts enable patients to benefit from close links between medical research and healthcare delivery.
A great strength of Oxford medicine is its long-standing network of clinical research units in Asia and Africa, enabling world-leading research on the most pressing global health challenges such as malaria, TB, HIV/AIDS and flu. Oxford is also renowned for its large-scale studies which examine the role of factors such as smoking, alcohol and diet on cancer, heart disease and other conditions.
6. Royal Brompton & Harefield NHS Foundation Trust
Royal Brompton & Harefield NHS Foundation Trust is a national and international specialist heart and lung centre based in Chelsea, London and Harefield, Middlesex. The Trust helps patients from all age groups who have heart and lung problems and is the country's largest centre for the treatment of adult congenital heart disease. For details visit www.rbht.nhs.uk
7. University of Edinburgh
Founded in 1583, the University of Edinburgh has for more than 400 years been one of the most influential centres of knowledge in the world.
Located in the Scottish capital, among the great figures who have studied at Edinburgh are naturalist Charles Darwin, philosopher David Hume, telephone inventor Alexander Graham Bell and former British Prime Minister Gordon Brown. Pioneering research linked to Edinburgh includes the discovery of chloroform, the TB vaccination, microchips which power iPod music players and the cloning of Dolly the Sheep. Today Edinburgh is home to nearly 28,000 students.