Cystic fibrosis gene therapy programme gets green light with public funding

A groundbreaking gene therapy trial for cystic fibrosis (CF) will begin in March, thanks to a new grant from a government funding body. 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).

The trial will be funded by a £3.1-million grant from the National Institute for Health Research (NIHR) and the Medical Research Council (MRC) through the Efficacy and Mechanism Evaluation programme.

The MRC, through its Developmental Pathway Funding Scheme (DPFS), will also fund a £1.2m-study by the GTC aiming to develop a potentially more efficient delivery method for the gene therapy, which could contribute to an even more effective treatment in the future.

The GTC is 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 who have worked together for the last decade to develop gene therapy for CF.

CF is the commonest lethal inherited disease in the UK, affecting around 9,500 people nationally and over 90,000 worldwide. Patients' lungs become filled with thick sticky mucus and they are vulnerable to recurrent chest infections, which eventually destroy the lungs. The cause of CF, mutations in a gene located on chromosome 7, was identified in 1989, opening the door to replacing this faulty gene using gene therapy.

Patients will receive the 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.