Innovation honour for cystic fibrosis treatment
08 June 2011
A group of scientists from the UK Cystic Fibrosis Gene Therapy Consortium has won two prestigious Medical Futures Innovation Awards for their work to develop a gene therapy for cystic fibrosis.
The scientists were awarded an MRC Translational Research Award and Best Therapeutic Innovation in the respiratory category. The Medical Futures Innovation Awards are a national recognition of clinical and commercial excellence. They aim to help encourage, support and reward new ideas and advancements in healthcare that may improve people's lives.
Cystic fibrosis is caused by a faulty gene that leads to thickened mucus secretions in the lungs and digestive tract. It causes breathing and digestion difficulties, with progressive disability and often early death. The disease affects more than 70,000 people worldwide, with only half of those expected to live past their mid 30s. In the UK alone, more than two million people carry the gene that causes cystic fibrosis and the cost of existing patient treatments ranges from £20,000 to £100,000 per patient per year.
The gene therapy innovation is a novel, ‘friendly’ virus that delivers a normal version of the faulty gene into the lungs of patients. The gene encodes the protein that enables the body to produce normal mucus and digestive juices. A single dose of the virus generates large amounts of this protein and the effect is sustained for at least two years in animal models. The therapy can be given repeatedly for lifelong treatment.
The award-winning team includes Professor Eric Alton, Dr Uta Griesenbach, Dr Jane Davies and Tracy Higgins from Imperial College London; Dr Deborah Gill and Dr Stephen Hyde from the University of Oxford; and Dr Chris Boyd, Dr Alistair Innes and Professor David Porteous from the University of Edinburgh.
John Jeans, MRC chief operating officer and deputy chief executive, who presented the MRC Translational Research Award commented: “This award is an example of many years of investment into discovery science. Using viral vectors in gene therapy presents many challenges, most notably causing immune responses in the body. The consortium’s innovative approach is re-targeting vectors with the aim of developing a long-term treatment for cystic fibrosis. This has huge implications for patients and their families and I congratulate the team on their achievements.”
The consortium will receive a bespoke package of support tailored to the needs of the project with the overall aim of realising its therapeutic potential.
