Research innovation wins grant awards for Oxford researchers

23 March 2011

Oxford University researchers have won three grants totalling £4.4m for the development of innovative medical technologies and treatments.

The three grants were awarded by the Health Innovation Challenge Fund (HICF), a translational research award scheme funded equally by the Wellcome Trust and the Department of Health.

Research to develop a potential treatment for a form of incurable blindness, improved tests to determine different types of leukaemia and the development of drugs to treat the most lethal form of muscular dystrophy will be funded.

The awards are among the first to be made by the HICF initiative, which was established in 2009 to address gaps in funding for innovative medical research and help turn scientific breakthroughs into potential medical treatments.

A £2.5 million HICF award to Dr Matthew Wood of the Department of Physiology, Anatomy and Genetics and Somerville College will fund research into Duchenne muscular dystrophy (DMD), the most deadly form of the disease. It is caused by damage to the dystrophin gene and affects one in every 3,500 newborn boys.

Dr Wood and colleagues at the MDEX Consortium - a UK-based translational medicine network of neuromuscular disease experts working to turn scientific findings into drugs and other forms of healthcare - have successfully trialled a treatment using antisense oligonucleotides (AOs) – DNA-based drugs that can repair the gene and restore functional dystrophin to the muscles of DMD patients.

Currently the AO drug is only moderately effective in skeletal muscle and does not work on heart muscle.

Led by Dr Wood, the group is now developing a new generation of AO drugs which will dramatically improve the delivery and restoration of dystrophin by all muscle. The treatment will be tested in a clinical trial on nine DMD patients, starting in 2013.

Dr Wood said: ‘Being selected as one of the first recipients of an HICF award is a fantastic opportunity to expand our world-leading science and to develop and test a ground-breaking new treatment for Duchenne muscular dystrophy, a devastating disease where new treatments are desperately needed.’

Professor Robert MacLaren, from Oxford’s Department of Ophthalmology, has won a £1.2 million award to investigate a new treatment for choroideraemia, an incurable blindness and form of retinitis pigmentosa – a disease causing damage to the retina.

The problem develops in childhood and often runs in families with night blindness the most likely initial symptom. The disease mostly occurs in men who become totally blind, usually in their forties.

Professor MacLaren and Professor Miguel Seabra from Imperial College London, an expert in the molecular biology of choroideraemia, have developed an experimental genetic treatment, which has been tested successfully in Oxford University’s Nuffield Laboratory of Ophthalmology. They plan four more years of research on a potential gene therapy treatment.‘

To be involved in developing a potential treatment for an incurable form of blindness is very exciting indeed,’ Professor MacLaren said. ‘I am delighted to see the Oxford Eye Hospital emerge as a world leader for a planned clinical trial.’

Leukaemia is a form of cancer that affects blood cells and arises in the bone marrow or lymphoid organs. There are several types of leukaemia, depending on which cells are affected.

To be effective, treatment must be tailored to the individual. However, current genetic tests used to guide therapy are not sufficiently precise. This means that some patients suffering from leukaemia may not respond to treatment or may suffer adverse side-effects.

Dr Samantha Knight, Dr Jenny Taylor, Dr Anna Schuh and Professor Chris Holmes from the Wellcome Trust Centre for Human Genetics and the National Institute for Health Research Oxford Biomedical Research Centre (a partnership between the University of Oxford and Oxford Radcliffe Hospitals) have developed specialised approaches to test the genetic make-up of blood cells from patients with B-cell chronic lymphocytic leukaemia (CLL).

The £730,000 award from the HICF will enable these approaches to be validated in a three year study using patient samples from the UKCLL National Cancer Research Network trials.

The more precise detection of relevant genetic alterations will allow doctors to provide the most suitable treatment for patients, reducing mortality and NHS care costs, and minimising side-effects of treatment.

Dr Knight said: 'The HICF has provided us with a unique opportunity to develop an approach that will modernise the genetic testing of leukaemias within the NHS. It will enable doctors to provide individually tailored patient treatments, minimising side-effects and mortality and reducing NHS costs.

For more information contact

Dr Matthew Wood at matthew.wood@dpag.ox.ac.uk / 07815 900476
Dr Samantha Knight sknight@well.ox.ac.uk / 01865 287511

Notes for editors

  • The Nuffield Laboratory of Ophthalmology is a University Department established in 1942 and relocated to the John Radcliffe Hospital West Wing site in 2008. It is linked to the Oxford Eye Hospital, part of the Division of Clinical Neurosciences, Trauma and Specialist Surgery at the ORH Trust.
  • The Health Innovation Challenge Fund (HICF) is a parallel funding partnership between the Wellcome Trust and the Department of Health. The funders are collaborating to stimulate the creation of innovative healthcare products, technologies and interventions, and facilitate their development for the benefit of patients in the NHS and beyond. The HICF operates a succession of thematic calls for proposals, each selected to focus on unmet needs in healthcare relevant to the NHS, and will support innovative developments that are within three to five years of launch or adoption. www.hicfund.org.uk
  • The Wellcome Trust is a global charitable foundation dedicated to achieving extraordinary improvements in human and animal health. It supports the brightest minds in biomedical research and the medical humanities. The Trust’s breadth of support includes public engagement, education and the application of research to improve health. It is independent of both political and commercial interests.www.wellcome.ac.uk
  • The Department of Health (DH) works to improve the health and well-being of people in England. The Department sets overall policy on all health issues and is responsible for the provision of health services through the National Health Service. www.dh.gov.uk