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STRICTLY EMBARGOED UNTIL:
FRIDAY, 2ND OCTOBER 2009 – 22:00 HRS (UK TIME)
London-based experts develop ground-breaking treatment for genetic disease
Experts at Royal Brompton Hospital in London have made a major breakthrough in the treatment of thalassaemia – one of the most common genetic diseases worldwide. Every year around 60,000 children are born with the condition, and in the year 2000, around 50 per cent of thalassaemia (major) patients in the UK died by the age of 35. By using a new scanning technique, a team from Royal Brompton and Imperial College London has shown the potential to dramatically cut the mortality rate for the disease by 71 per cent.
Thalassaemia causes defects in haemoglobin production which in turn results in anaemia. In its most serious form it can cause organ damage, restricted growth, liver disease, heart failure and ultimately death. Those with the conditionneed extensive medical care which includes frequent blood transfusions. But a major side effect of these transfusions is a build-up of potentially life-threatening iron levels in the heart. This can lead to serious, and in many patients, fatal, heart failure.
Results from an international study published in Circulation[1] today, clearly indicate that by using cardiovascular magnetic resonance (CMR) scanning , experts can measure iron levels more accurately than current methods (biopsies or blood tests), and as a result can better predict the development of heart failure in thalassaemia patients. Those affected can then be closely monitored and treated for heart failure at the earliest indication. This breakthrough will increase life expectancy and greatly improve well-being for thalassaemia patients. CMR is non-invasive and has no known side-effects.
Leading the study, Professor Dudley Pennell, director of the NIHR cardiovascular Biomedical Research Unit and the CMR unit at Royal Brompton Hospital and from the National Heart and Lung Institute at Imperial College London, said: “This is a major breakthrough for thalassaemia patients. It gives real hope to sufferers around the world and will completely change the way the disease is managed.
“The study clearly shows that by using CMR we can more accurately predict future heart problems in patients with thalassaemia major. This information can then be used to provide better treatment options - reducing deaths from heart failure and offering patients a much better quality of life.”
Patient Amit Ghelani, who took part in the study, added: “I was getting my iron levels checked in the usual way through blood tests. But after being referred to Royal Brompton my heart iron levels were checked using CMR. For me, it meant that I got the treatment that I needed for heart failure immediately, which essentially saved my life.”
Professor Dame Sally Davies, Director General of Research and Development at the Department of Health said: “This is precisely the sort of practical research aimed at helping patients the National Institute for Health Research is keen to fund. It will help people not just in England but around the world.”
A total of 652 patients with thalassaemia major were included in the study. They received clinical care at 21 centres throughout the UK. The patients were scanned at the CMR unit at Royal Brompton Hospital between 1999 and 2006. Of these patients, 319 were male and 333 female with an average age of 27 years at their first scan.
Ends/
For further information and to interview Professor Pennell, please contact:
Shima Islam, Head of media relations
Royal Brompton & Harefield NHS Foundation Trust
Tel: 020 7351 8672 (Brompton)
Tel: 01895 828 877 (Harefield)
Mobile: 07866 536 345
Email: S.Islam@rbht.nhs.uk
Notes to editors:
· Thalassaemia is most common in people of Mediterranean, Middle Eastern, Southern Asian and African descent. There are approximately 800 sufferers in the UK.
· The research paper ‘Cardiac T2* Magnetic Resonance for Prediction of Cardiac Complications in Thalassaemia Major’ will be published in Circulation on Friday, 2 October at 22:00 (UK time).
· 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. http://www.nihr.ac.uk/
· 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 13,000 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 health in the UK and globally, tackle climate change and develop clean and sustainable sources of energy.
Website: www.imperial.ac.uk
[1] Circulation – Journal of the American Heart Association.
