A £3.1 million grant from the UK National Institute for Health Research (NIHR) and the Medical Research Council (MRC) will fund a gene therapy study in which 130 children and adults with cystic fibrosis will receive either an inhaled placebo or inhaled DNA delivered using lipids. The UK Cystic Fibrosis Gene Therapy Consortium (GTC) is coordinating the trial. The GTC includes teams from Imperial College London, the Universities of Oxford and Edinburgh, Royal Brompton & Harefield NHS Foundation Trust and NHS Lothian.
A second study, funded by £1.2m from the MRC through its Developmental Pathway Funding Scheme (DPFS), will provide data for development of a lentiviral gene therapy product, also delivered by inhalation. The project won the MRC Best Translational Research Innovation at the 2011 Medical Futures Innovation awards.
GTC Coordinator Eric Alton of Imperial College London and Royal Brompton Hospital commented, “Conventional treatments have extended the life expectancy for people with CF. We’re hoping that this therapy will achieve a step change in the treatment of CF that focuses on the basic defect rather than just addressing the symptoms. This trial will assess if giving gene therapy repeatedly for a year will lead to the patients’ lungs getting better. Eventually we hope gene therapy will push CF patients towards a normal life expectancy and improve their quality of life significantly.”
“We would like to thank the NIHR and MRC for their confidence in us, and also all of those whose support has reinforced our determination to carry out this programme for the benefit of CF patients. We would also like to thank the Cystic Fibrosis Trust and the many families and supporters for funding our programme up to this stage,” Alton continued.
The Consortium plans to provide regular updates on its website and expects trial results in 2014.
Read the GTC press release.
Watch video on GTC’s gene therapy for CF below:
