London scientists aiming to develop safe gene therapy to treat rheumatoid arthritis
Scientists funded by the Arthritis Research Campaign are hoping to develop a safe form of gene therapy to treat severe rheumatoid arthritis.
Researchers at the Bone and Joint Research Unit at Barts and the London, Queen Mary School of Medicine and Dentistry, have been awarded funding of £230,500 over three years to try to develop a form of anti-TNF therapy that could be delivered to patients as DNA via a single injection.
Principle investigator Dr David Gould said the research was in its very early laboratory stages, but if all went as planned there was a real potential that the research could to lead to new drugs.
Anti-TNF therapy, pioneered by arc scientists in the 1990s, has been available to patients with severe rheumatoid arthritis since 2000 and can have dramatic results.
But their high cost of around £10,000 per patient mean that they have been subject to rationing and postcode prescribing.
"To be successful, anti-TNF delivered as gene therapy would need to be effective in the long term for a single injection, and the therapeutic effect would have to be as good as anti-TNF biologics,” explained Dr Gould. He is confident, however. “The treatment will be considerably cheaper than existing therapies that target anti-TNF, so it could be used throughout the world,” he added.
Mr Gould's project builds on research performed with arc Professor of Rheumatology Yuti Chernajovsky, who has pioneered several novel gene and cell therapies of arthritis during the past ten years.
Gene therapy is an innovative scientific area that aims, through the introduction of genetic material into patient cells, to correct the lack of expression of a defective gene, or to produce a protein that might have therapeutic value. However, despite hundreds of gene therapy trials around the world (most commonly for cancer) and much excitement in the scientific world about clinical potential, lack of strong effects and isolated adverse effects in trials have tempered enthusiasm.
In experiments on model systems Dr Gould and his team will use plasmid DNA as the means of delivering therapy. Plasmid DNA is currently used for vaccination and also in early stage clinical trials for cardiovascular conditions. It is also the safest method of delivering DNA as the body does not react against it.
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