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Government helps In Fight For Sight
Trial Starts To Find Cure For Childhood Blindness
A Department of Health funded UK team have begun a clinical gene therapy trial to test a revolutionary treatment for blindness in children. The trial, which was given £1million by the Department, is the first of its kind and could have a significant impact on future treatments for eye disease.
The team from University College London Institute of Ophthalmology and Moorfields Eye Hospital, led by Professor Robin Ali, includes leading eye surgeon Mr James Bainbridge and leading retinal specialist Professor Tony Moore.
The trial involves adults and children who have 'inherited childhood blindness' a progressive deterioration in vision caused by an abnormality in a particular gene . This defect prevents normal function of their retina, the light-sensitive layer of cells at the back of the eye. There are currently no effective treatments available for this condition.
Health Minister Lord Hunt said:
"This new trial is a global first of its kind and could mean the ability to restore sight to children with this condition. The UK leads europe in gene therapy, with over 40% of clinical trials, and we are second only to the US. Investment into novel gene therapy clinical trials began with a committment in our 2003 Genetics White Paper and has helped the NHS bring important research such as this from the scientist's bench to the patient's bedside. I am delighted to be supporting such excellent work at the world renowned Moorfields Eye Hospital."
The new technique involves inserting normal copies of the gene into the cells of the retina to help them to function normally. This is achieved by an operation to deliver the normal genes to the retina, using a harmless virus or "vector" to carry the gene into the cells.
The purpose of this study is to find out how safe and effective the new intervention is in patients.
Professor Ali said:
"We have been developing gene therapy for eye disease for almost 15 years but until now we have been evaluating the technology only in the laboratory. Testing it for the first time in patients is very important and exciting and represents a huge step towards establishing gene therapy for the treatment of many different eye conditions."
So far the operation has been performed in young adult patients who developed the condition as children. Mr James Bainbridge, who leads the surgical team said:
"It is very encouraging that we can deliver genes to an extremely fragile site in the eye without complications. It will be many months before we know the outcome of the procedure but we expect the best outcome in younger patients."
Professor Martin Gore, Chairman of the Gene Therapy Advsiory Committee, which gave the ethical approval for this trial, said:
"Gene therapy was originally conceived for the treatment of single gene disorders as in theory, such an approach addresses the very nature of such disorders. However, in practice, over 70% of gene therapy trials have been for cancer. It is heartening to see that Government funding to support single gene disorders has enabled researchers to come forward with an exciting and novel trial proposal which offers hope to patients affected by these very serious diseases. This trial demonstrates that the original ideas behind gene therapy are still very much alive."
Notes to editors:
1. The team is supported by funding from the Department of Health, Sir Jules Thorn Charitable Trust, The Wellcome Trust, The British RP Society, The European Union (EVI Genoret and Clinigene programmes), The Medical Research Council, Foundation Fighting Blindness USA, Fight for Sight
2. 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 2008. 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.
3. 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.
4. Since the Gene Therapy Advisory Committee approved the first UK gene therapy trial in 1993, there have been 112 approved gene therapy clinical trials, involving over 1262 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 & neck, colorectal and liver cancer as well as skin cancer. For details of these trials please see the Gene Therapy Advisory Committee's 13th Annual Report available from this website:http://www.advisorybodies.doh.gov.uk/genetics/gtac
5. UCL Institute of Ophthalmology is one of a number of specialised research centres linked to University College London and is, together with Moorfields Eye Hospital, one of the leading centres for eye research. The Institute scored a 5*A (highest point) in the last Research Assessment Exercise. The Institute is committed to a multi-disciplinary research portfolio that furthers an understanding of the eye and visual system linked with clinical investigations targeted to specific problems in the prevention and treatment of eye disease. The combination of the Institute's research resource with the resources of Moorfields Eye Hospital, which has the largest ophthalmic patient population in the Western World, opens the way for advances at the forefront of vision research.
6.Robin Ali - is Professor of Human Molecular Genetics at UCL Institute of Ophthalmology and Head of Division of Molecular Therapy. James Bainbridge - is a Wellcome Trust Advanced Fellow at UCL Institute of Ophthalmology and Consultant Ophthalmologist at Moorfields Eye Hospital. Tony Moore - is Professor of Ophthalmology at UCL Institute of Ophthalmology and Consultant Ophthalmologist at Moorfields Eye Hospital
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