Trial begins to find childhood blindness cure

A clinical gene therapy trial has begun on order to test a revolutionary treatment for blindness in children.

The trial, which has received £1 million funding from the Department of Health, is the first of its kind and could have a significant impact on future treatments for eye disease.

The trial is being carried out by a team from University College London Institute of Opthalmology and Moorfields Eye Hospital, led by Professor Robin Ali. The team includes leading eye surgeon 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.

The new technique involves inserting normal copies of the gene into the cells of the retina to help them 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.

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 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 can expect the best outcome in younger patients."

Gene therapy is the deliberate introduction of genetic material into human somatic cells for therapeutic, preventative or diagnostic purposes.

The therapies study 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.

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."

(KMcA/SP)