Page URL:

Cash for UK gene therapy trials

29 March 2004
By BioNews
Appeared in BioNews 251

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.

Hope for vital cures in £3m genetic research
The Observer |  28 March 2004
Tenth annual report of the gene therapy advisory committee published
Medical News Today |  26 March 2004
Winners of £4 million for gene therapy
Medical News Today |  29 March 2004
27 June 2011 - by Dr Nadeem Shaikh 
A group of US scientists has successfully used a virus to deliver genes into monkey retinas that could potentially restore damaged photoreceptor cells. The findings could lead to gene therapies being developed to treat blindness and other eye conditions in humans...
3 April 2006 - by BioNews 
A team of scientists based in Frankfurt, Germany, has become the world's first to treat a rare and 'incurable' hereditary immune disease in adults, using gene therapy. Gene therapy has been used successfully in the past, but this has generally been in the treatment of children. Over the past two...
30 March 2006 - by BioNews 
US researchers have begun a clinical trial to test a new gene therapy treatment for Duchenne muscular dystrophy (DMD), an inherited muscle-wasting disorder. The team, based at the Columbus Children's Hospital in Ohio, will test the safety and effectiveness of gene injections in six affected boys aged 8-12 years...
26 August 2004 - by BioNews 
GPs in England are invited to apply for funding to become experts in genetics, the government announced recently. Money is available for ten GPs to take part in a training scheme that aims to make sure genetic knowledge and treatment are widely available to NHS patients. Applications for the funds...
26 July 2004 - by BioNews 
Researchers have overcome one of the major hurdles in using gene therapy to treat muscle diseases: delivery of the gene to muscles throughout the body. The study, to be published in the journal Nature Medicine next month, is further progress towards treating diseases such as Duchenne muscular dystrophy (DMD), which...
27 June 2003 - by BioNews 
The UK government unveiled its plans for genetics in the National Health Service (NHS) last week, in the form of a white paper entitled 'Our inheritance, our future - realising the potential of genetics in the NHS'. The document lays out new measures for strengthening existing genetics services, and for incorporating...
to add a Comment.

By posting a comment you agree to abide by the BioNews terms and conditions

Syndicate this story - click here to enquire about using this story.