16 March 2012

Funding Boost for Cystic Fibrosis Gene Therapy Trial

A pioneering clinical trial that could lead to a potentially life-changing new treatment for cystic fibrosis (CF) has received a £3.1m funding boost from the Medical Research Council (MRC) and the National Institute for Health Research (NIHR).

The research, which has been supported by the Cystic Fibrosis Trust for a decade, had faced an uncertain future due a funding shortfall. Today’s investment by the MRC and NIHR will ensure the next stage of the programme can continue as planned.

The phase two clinical trial will be undertaken by the UK Cystic Fibrosis Gene Therapy Consortium. It will examine whether a promising treatment, developed previously by the Consortium and tested in a small number of patients, can improve the long-term health and wellbeing of people with CF.

The MRC will also fund a further £1.2m to help the Consortium develop a potentially more efficient delivery method for the gene therapy, which could contribute to an even more effective treatment in the future.

Dr Wendy Ewart, Deputy Chief Executive of the MRC, said:

“Gene therapy represents one of the most promising treatment avenues for cystic fibrosis and many other devastating conditions that currently have no effective cure. By investing more than £4m in this exciting research, the MRC reaffirms its commitment to driving the translation of laboratory and clinical science into new treatments for human disease. The investment will also strengthen further our significant collaborative links with the NIHR, which make this research possible.”

Andrew Lansley, Secretary of State for Health, said:

"We want to create a world-class NHS where patients benefit from the latest and best treatments. This funding provided by the Government, via the MRC and the NIHR, will enable pioneering studies demonstrating how science in the lab can be translated into gene therapy for patients with cystic fibrosis. This work is another example of the Government's continued commitment to reducing the burden of genetic disease and follows recent recommendations from the Human Genomics Strategy Group, which I have welcomed, which provides a strategic vision for the adoption of genomic technologies across healthcare to diagnose and treat genetic conditions."

David Willetts, Minister for Universities and Science, said:

"This £3.1 million investment will deliver a groundbreaking clinical trial in cystic fibrosis gene therapy, which is an important and promising area of health research. It is an excellent example of collaborative working between Government, the research base and a leading health charity. In addition, the　£1.2 million investment from the Medical Research Council to support work in more effective delivery methods will help bring improvements in treatment and benefits for patients."

Cystic fibrosis is a common inherited disease that affects around 9,500 people in the UK. It is caused by a single faulty gene that leads to a build-up of thick, sticky mucus in the lungs and digestive system. There is currently no cure and the only available treatments rely on alleviating the symptoms, not the underlying genetic cause. Even today, the average life expectancy is 41 years.

A treatment that successfully delivers a working copy of the defective gene – known as CFTR (cystic fibrosis transmembrane conductance regulator) – directly into the lungs of patients has been developed previously by the UK Cystic Fibrosis Gene Therapy Consortium. The gene is administered in a fine mist, which can be inhaled easily by patients via a nebuliser.

The phase two clinical trial, funded through the MRC-NIHR Efficacy and Mechanism Evaluation programme and conducted by Imperial College London, and the Universities of Edinburgh and Oxford, will evaluate whether delivering this therapy once a month for a year can improve lung function and reduce the amount of mucus, inflammation and infection in CF patients.

The second lab-based study, funded directly by the MRC, and conducted by the University of Oxford, Imperial College London, and the University of Edinburgh 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.

Together the two studies will build upon scientific evidence and could help achieve a lasting cure for cystic fibrosis. They will also inform the development of gene therapies for other conditions.

Notes Contact Carmel Turner

Chief press officer, Medical Research Council

T: 0207 395 2273 / 0207 395 2345 (out of hours: 07818 428 297)

E: press.office@headoffice.mrc.ac.uk

  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. The UK Gene Therapy Consortium was established over ten years ago. It comprises teams of experts from Imperial College London, the University of Edinburgh and the University of Oxford. The patients will be studied at the Royal Brompton Hospital, London, and the Western General Hospital and the Royal Hospital for Sick Children, Edinburgh. http://www.cfgenetherapy.org.uk/
  5. The Cystic Fibrosis Trust is the UK’s only national charity dealing with all aspects of Cystic Fibrosis (CF). It funds research to treat CF and aims to ensure appropriate clinical care and support for people with CF. http://www.cftrust.org.uk/