DEPARTMENT OF HEALTH
News Release (2007/0099) issued by The Government News Network on 1
May 2007
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
[ENDS]