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.”
1. For media enquiries and to arrange interviews please contact the
Department of Health news desk on tel: 020 7210 5221
2. 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
3. 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.
4. 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.
5. 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:advisorybodies.doh/genetics/gtac
6. 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.
7.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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