A groundbreaking gene therapy trial for cystic fibrosis (CF) will begin in March, thanks to a $4.9 million grant from National Institute for Health Research (NIHR) and the Medical Research Council (MRC) through the Efficacy and Mechanism Evaluation program.

One hundred and thirty adults and children with CF will take part in the largest trial of its type yet, coordinated by the UK Cystic Fibrosis Gene Therapy Consortium (GTC), a group of scientists and clinical teams from Imperial College London, the Universities of Oxford and Edinburgh, Royal Brompton & Harefield NHS Foundation Trust and NHS Lothian.

Patients in the trial will receive treatment by inhaling molecules of DNA wrapped in fat globules that deliver the replacement gene into the cells in the lung lining. Half the participants will receive the real treatment and half a placebo in a double-blind study. Patients aged 12 and over at Royal Brompton Hospital, London and Western General and Royal Hospital for Sick Children in Edinburgh, will receive one dose a month for one year. Over 30 patients have each received a single dose of the gene therapy in the Consortium's previous studies, which evaluated efficacy of replacing the protein encoded by the defective CF gene.

”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,” said Professor Eric Alton, GTC Coordinator from Imperial College London. “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.”

A second, lab-based study will investigate a more advanced version of the therapy using a modified virus to carry the replacement gene into the lungs, which could lead to a more efficient delivery mechanism in the future. The MRC, through their Developmental Pathway Funding Scheme (DPFS), will fund this study as well, giving $1.9 million to GTC.

The outcome of the inhalant trial will be known in spring 2014 and regular progress reports will be posted on the Consortium's website.