Cash for UK gene therapy trials

Gene therapy trials for haemophilia, muscular dystrophy and childhood blindness are to receive £3 million funding, the UK Department of Health has announced. A further £1 million has been set aside for research into the long-term safety of some gene therapy techniques. The funding is part of a £50 million government initiative to incorporate new genetic technologies into healthcare, outlined in the Genetics White Paper published last June.

Health Secretary John Reid said he was delighted to announce the successful bidders for the gene therapy research funds. 'Investment saves lives - that is why it is vital that we fund research into the latest cutting edge treatments such as gene therapy so that Britain remains at the forefront of medical research', he said. Gene therapy, a technique still in its infancy, aims to treat disease symptoms by replacing a faulty or missing gene with a working copy, or by switching off a harmful gene.

Gene therapy trials for Duchenne muscular dystrophy (DMD) - a severe inherited muscle weakness disorder - will receive £1.6 million of the new funds. Jenny Versnel, Head of Research at the Muscular Dystrophy Campaign (MDC), which successfully applied for the money, welcomed the announcement: 'It is hoped that the severity of Duchenne muscular dystrophy could be reduced to a much milder form, significantly improving the quality and length of life', she said. DMD is caused by a missing or faulty version of a unusually large gene which makes a crucial muscle protein called dystrophin. 'We are looking at developing a molecular patch - a tiny bit of genetic material which sticks over parts of the gene and restores some of the order needed so it can produce some dystrophin', explained Joe Korner of the MDC. The research will be carried out by a team at Imperial College in London, lead by Professor Francesco Muntoni.

A further £900,000 will go to researchers at the Institute of Child Health and the Institute of Ophthalmology, to fund a trial aimed at treating childhood blindness. The Oxford University-based company BioMedica will receive £500,000, for a trial to treat the blood-clotting disorder Haemophilia A. Money for research into gene therapy safety issues will go to groups at the University of Glasgow, the Royal Free and University College London Medical School, Kings College London and University College London.

Since the Gene Therapy Advisory Committee (GTAC) approved the first gene therapy trial in 1993, there have been 90 clinical trials in the UK, involving over 700 patients. Over 70 per cent of these trials tested new cancer treatments, with others testing gene therapy treatments for cystic fibrosis, Hurler's syndrome, HIV (human immunodeficiency virus) and heart disease patients. GTAC has just published its tenth annual report for 2003, which includes details of all 13 proposed gene therapy trials approved by the committee last year.