Gene therapy clinical trial offers hope for muscular dystrophy
22 Oct 07
A gene therapy clinical trial begins this week which could offer new hope to muscular dystrophy sufferers. A new treatment called molecular patch therapy has been developed at the University of Oxford that has the potential to give young men born with Duchenne Muscular Dystrophy (DMD) the chance to preserve their muscle function.
DMD, which affects boys, is caused by a single faulty gene, and results in progressive muscle wasting. In a world first, a small group of patients will be injected with a genetic ‘patch’ which it is hoped will extend their lives.
Conventional gene therapy approaches for this disorder have proven problematic, but a pioneering technique developed at Oxford University places a ‘patch’ of DNA over the genetic errors causing DMD. Animal work at Oxford has showed extremely promising results, and now the treatment is to be tested in humans.
DMD affects one in 3,500 young men and is caused by reduced production of dystrophin protein – vital for muscle function. The progression of the condition is so severe that untreated young men lose the ability to walk by their early teens and are only expected to live into their twenties.
The ‘patch’ technique has been developed at Oxford by Dr Matthew Wood in the Department of Physiology, Anatomy and Genetics, and a consortium called MDEX has been responsible for generating and testing multiple molecular patches to find the optimal one to be used in this trial. The consortium is formed by scientists from Imperial College London, Newcastle University, Oxford University, and Royal Holloway University of London. The charities Muscular Dystrophy Campaign (MDC), Parent Project UK (PPUK) and Duchenne Parent Support Group also participate in the Consortium.
The molecular patches were tested in cell cultures, and the patch that restored the highest level of dystrophin protein is now being taken to clinical trial.
The phase one trial is due to start by the end of October at the Hammersmith Hospital, London and St Mary’s Hospital, London. It will recruit nine young men with DMD aged 12–17, who will have the molecular patch administered by injection into a small muscle in the foot.
Subject to the trial’s success, there is already a plan to proceed with another trial to deliver the molecular patch under the skin, so that all muscles in the body could be treated.
Dr Wood said: ‘Beginning the translation of this gene therapy to the clinic is an exciting moment, and the start of learning how to make these new therapies work in patients.’
The Consortium has attracted a £1.6m grant from the Department of Health, which is funding the present study. Recent additional funds from the Medical Research Council (MRC), MDC, PPUK, Big Lottery Fund (BLF), Parent Project Muscular Dystrophy (PPMD) and Muscular Dystrophy Ireland (MDI) will allow the Consortium to take the current studies forward. The molecular patch design is in collaboration with Professor Steve Wilton (Perth, Australia). Trial support and clinical grade molecular patches are being provided by AVI BioPharma (Portland, Oregon, USA).