Gene Therapy Could Now Restore Blindness

British scientists have completed the first major clinical trial involving human beings for treating blindness using gene therapy.

A team of researchers and scientists at the University College of London Institute of Ophthalmology and Moorfields Eye hospital in London have conducted the trial on a human patient, Robert Johnson.

Mr. Johnson who was born with a degenerative disorder which meant that he could only see outlines during the day and in poor light and in the evening nothing at all had copies of healthy genes inserted into the cells of the retina in his eyes to help them to function normally. Mr. Johnson suffered from a condition called Leber’s Congenital Amaurosis (LCA), which is a type of inherited retinal degeneration.

Despite the optimism surrounding this groundbreaking new technique scientists are quick to counsel caution saying that though it could lead onto treatment of age related “macular degeneration” which affects in excess of 400,000 people in the UK it is more likely that the procedure will only work on children and young adult’s because at this stage scientists believe older People’s eyesight may have deteriorated too much.

The research team conducting the trial is led by Professor Robin Ali and Professor Ali said “we have been developing gene therapy eye disease for almost 15 years up until now we have been evaluating the technology only in the laboratory. Testing it for the first time in human patients is very important and exciting, and represents a huge step towards establishing gene therapy for the treatment of many different conditions.”

The team from Moorfields Eye Hospital also included leading retinal specialist Professor Tony Moore who went home to add “Some indications of the results off the trial may be available within several months. However, the subjects will need to be followed up to assess the long-term effect of treatment. It will be many months before we have the full picture. We anticipate the best outcome in younger patients, as we will be treating the disease in the early stages of its development.”

Professor Moore’s assisting search, Robert Maclaren said he thought Mr. Johnson’s operation, which apparently is the first of eleven planned trials, had gone “extremely well.”

Elsewhere opinion was similarly positive. At the Royal National Institute for the Blind, the campaigns manager, Barbara MacLaughlin stated “we welcome this first clinical trial and research which may result in treatment for this currently untreatable cause of blindness. We would be interested to learn more about developments in how the research might provide ways to combat other diseases that cause degeneration to the retina.”

On behalf of the British Society for Gene Therapy, Professor Leonard Seymour, President, said: “the retina is a really good place for Gene therapy because it can be accessed by direct injection, overcoming the problem off Gene delivery. When gene delivery is efficient, the whole power of Gene therapy is unleashed.”

Previous work using animal models have demonstrated that this gene therapy can improve and preserve vision but this was the first time anywhere in the world is therapy have been tried on human patients.