A £3.1 million award from the Medical Research Council (MRC) and the National Institute for Health Research (NIHR) will enable a UK consortium to move ahead with efficacy testing of its inhaled gene therapy for cystic fibrosis.
A Phase II clinical trial of the treatment developed by the UK Cystic Fibrosis (CF) Gene Therapy Consortium will start this spring. The MRC has awarded a further £1.2 million for a second study looking at a more advanced version of the therapy that uses a modified virus to carry the replacement gene into the lungs.
Around 130 patients at the Royal Brompton Hospital in London and the Western General Hospital and Royal Hospital for Sick Children in Edinburgh will take part in the Phase II trial of the Consortium’s therapy, whereby a working copy of the defective cystic-fibrosis transmembrane conductance regulator gene is delivered directly into the lungs of patients via a nebuliser.
The trial, to be funded through the MRC-NIHR Efficacy and Mechanism Evaluation (EME) programme and conducted by Imperial College London along with the Universities of Edinburgh and Oxford, will evaluate whether delivering the therapy once a month for a year can improve lung function and reduce the amount of mucus, inflammation and infection in CF patients.
The outcomes should be known by spring 2014.
The second, MRC-funded study, aimed at developing a more efficient delivery mechanism for the gene therapy, will be conducted by the University of Oxford, Imperial College London and the University of Edinburgh.
Experts from these three institutions set up the UK Cystic Fibrosis Gene Therapy Consortium more than 10 years ago. Over that period, the Consortium’s work has been supported by national charity the Cystic Fibrosis Trust.
As the MRC noted, though, the continued progress of the Consortium’s gene therapy programme was in doubt due to a funding shortfall.
Cystic fibrosis affects some 9,500 people in the UK and the only available treatments are geared to alleviating symptoms rather than tackling the underlying genetic cause. The average life expectancy for CF patients is 41 years.