Singapore, April 30, 2008: Researchers from the Moorfields Eye Hospital/UCL Institute of Ophthalmology, National Institute for Health Research (NIHR), Biomedical Research Center in United Kingdom have announced results from the world's first clinical trial to treat a type of inherited blindness by successfully administering gene therapy to the human retina.

 

The results, published in the New England Journal of Medicine, show that the experimental treatment is safe and can improve sight. The findings are a landmark for gene therapy technology and are an important milestone for translational research in the UK.

 

The trial, which received an additional £1 million from the Department of Health's Genetic White Paper funding, involved young patients with a condition called Leber's congenital amaurosis (LCA), a type of inherited retinal degeneration which causes progressive deterioration in vision and blindness in teenagers. The trial has shown that the sight of a patient with LCA has made a major improvement.

 

This is the first major outcome from the 12 Biomedical Research Centres which were set up by the National Institute for Health Research (NIHR) in April 2007. These centres work in major areas of ill-health and clinical need to speed up the translation of fundamental science into benefits for patients. Together they will receive more than £485 million over five years.

 

Ms Dawn Primarolo, Minister for Public Health, Government of United Kingdom said, "This is a major achievement for British science and the NHS and shows we truly are at the forefront of innovation. The success of this research has huge implications for sufferers of this condition, as well as for a much larger group of inherited retinal diseases which affect 1 in 3,000 people.

Prof Sally C. Davies, Director of Research and Development at the Department of Health said, "This is fantastic work by the research team and shows how this country is now leading some of the most exciting health research in the world today. I am particularly pleased that the Department of Health has contributed more than one million pounds towards this important development. ”

He further said, "The Moorfields Eye Hospital/UCL Institute of Ophthalmology NIHR Biomedical Research Centre will continue to provide support, ensuring the full potential of the findings are realised and applied to treat other eye diseases. This is precisely the type of translational research that we have established our Biomedical Research Centers and Units to conduct, and will speed up the development of improved treatments for NHS patients."