Patients to benefit from latest developments in genetics
UK Health Secretary, John Reid today announced the successful bidders for £4million of funding to support research into gene therapy in the U.K.
The funding is part of a wider £50 million strategy to harness the potential of advances in genetics to the benefit of NHS patients, annouced in last year's Genetics White Paper.
Gene therapy is a new, innovative treatment which holds great promise for treating many common diseases, such as cancer and heart disease, as well as offering hope to those with inherited conditions such as Duchenne muscular dystrophy.
Many inherited diseases are the result of a missing or damaged gene. Rather than trying to treat the symptoms, gene therapy aims to correct the underlying problem by introducing healthy copies of these genes, or other beneficial genetic material into patient's cells.
Of the funding allocated today, £3million will fund, for the first time, research into clinical trials for three single gene disorders - haemophilia, muscular dystrophy and childhood blindness. A further £1 million will be used to support research into the long-term safety of some techniques used in gene therapy.
Health Secretary, John Reid said:
"I am delighted to announce the successful bidders for £4million of government funding to support gene therapy research. Investment saves lives - that is why it is vital that we fund research into the latest cutting edge treatments such as gene therapy so that Britain remains at the forefront of medical research.
NHS patients are uniquely placed to reap those benefits. The underlying principle of the NHS - healthcare free to all, on the basis of need - means that in the future patients who may benefit from this potentially life-saving medical technology will do so without the threat of being unable to afford treatment.
I would like to particularly congratulate the Muscular Dystrophy Campaign for bringing together patients, parents, and scientists to address real needs and develop a novel treatment strategy for Duchenne muscular dystrophy. Their successful application for £1.6 million pays tribute to this outstanding collaborative effort, and should result in the first ever UK gene therapy trial for DMD."
Head of Research at the Muscular Dystrophy Campaign, Jenny Versnel said:
"This injection of funding by the Department of Health to develop the technique for use in humans is extremely encouraging. It is hoped that the severity of Duchenne muscular dystrophy could be reduced to a much milder form, significantly improving quality and length of life."
Professor Norman Nevin, Chair of the Gene Therapy Advisory Committee, said:
"The time is right for strategic public funding of gene therapy research and safety studies, and I'm delighted that the Government will fund the proposed work of these excellent teams. Gene therapy has unparalleled potential to develop treatments for patients with genetic disease from cancer and heart disease, through to rarer inherited disorders such as muscular dystrophy, childhood blindness and haemophilia. This welcome funding will help to bridge the gap between laboratory and clinic for pioneering and innovative studies".
Related links Gene Therapy Advisory Committee
http://www.dh.gov.uk/ExternalLink?EXTERNAL_LINK=http://www.advisorybodies.doh.gov.uk/genetics/gtac.
Notes to editor
1. The June 2003 White Paper 'Our Inheritance, Our Future: Realising the potential of genetics in the NHS', sets out the Government's commitments to developing genetics knowledge, skills and provisions within the NHS by investing more than £50 million between 2003 and 2006. This funding, over £10 million of which is for gene therapy, will harness the potential of advances in genetics for the benefit of NHS patients.
2. Gene therapy is the deliberate introduction of genetic material into human somatic cells (that is, not into egg or sperm cells), for therapeutic, preventative, or diagnostic purposes.
3. Since the Gene Therapy Advisory Committee approved the first UK gene therapy trial in 1993, there have been 90 approved gene therapy clinical trials, involving over 700 patients. These gene therapy studies target inherited disorders such as Cystic Fibrosis, infectious diseases such as HIV infection, and vascular (heart) disease. Over 70% of trials are for the treatment of cancer, including breast, ovarian, cervical, pancreatic, prostate, bladder, head and neck, colorectal and liver cancer as well as skin cancer. For details of these trials please see the Gene Therapy Advisory Committee's website, which can be accessed by clicking on the link above.
4. £3million will be spent to support gene therapy research into single gene disorders by the following three teams:
£1,600,000 to the Muscular Dystrophy Campaign, headed by Professor Francesco Muntoni (Imperial College London) for research on Duchenne muscular dystrophy (DMD). DMD is a rare disorder with onset in early childhood. Progressive disability due to muscle breakdown is typically followed by death in the early twenties. There are no effective treatments. This will be the first UK gene therapy trial aimed at treating DMD.
£900, 000 to Dr Robin Ali (Institute of Ophthalmology and Institute of Child Health, University College London) for gene therapy research on one form of inherited retinal disease, childhood blindness. There are no effective treatments. This will be the first UK gene therapy trial aimed at treating eye disorders.
£500,000 to Dr Kyriacos Mitrophanous (Oxford BioMedica) for research on Haemophilia A. Haemophilia is a blood condition in which an essential blood-clotting factor is either partly or completely missing. If left untreated internal bleeding into joints, muscles and soft tissues can cause acute pain and severe joint damage. Haemophilia A is due to a mutation in the gene that produces blood-clotting Factor VIII. This will be the first UK gene therapy trial aimed at treating Haemophilia.
Around £1million will be spent to support research into the long-term safety of the use of gene therapy techniques. This has been awarded to four teams:
£450,000 to Dr Linda Scobie (University of Glasgow) for research on the safety of retroviral vectors. This work addresses some of the recommendations for research looking at the safety of gene therapy made by the joint March 2003 working group of the Gene Therapy Advisory Committee and the Committee on Safety of Medicines.
£200,000 to Dr Amit Nathwani (The Royal Free and University College London Medical School) for research on the safety and efficacy of adeno-associated vectors for the purpose of genetic diseases affecting the liver, such as Haemophilia B. Haemophilia B is due to a mutation in the gene that produces blood-clotting Factor IX.
£200,000 to Professor John Fabre (Rayne Institute, Kings College London) for research on liver disease gene therapy, using hydrodynamic gene delivery. This is a new and promising technique for the administration of gene therapy products. It involves delivering the gene therapy drug under conditions of rapid infusion of large fluid volumes.
£125,000 to Professor Mary Collins (UCL) for research on engineering retroviral vectors. This work addresses some of the recommendations for work looking at the safety of gene therapy made by the joint March 2003 working group of the Gene Therapy Advisory Committee and the Committee on Safety of Medicines.
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