Cystic fibrosis gene therapy trial 2012

Government funding announced 16 March 2012 will allow a groundbreaking gene therapy trial for cystic fibrosis to go ahead. The £3.1million grant from the National Institute for Health Research (NIHR) and the Medical Research Council (MRC) will fund the largest trial of its type yet with 130 adults and children with cystic fibrosis taking part.
The trial, which is due to begin very soon, will be co-ordinated by the UK Cystic Fibrosis Gene Therapy Consortium (GTC), a group of scientists and clinical teams from Royal Brompton & Harefield NHS Foundation Trust, Imperial College London, the universities of Oxford and Edinburgh and NHS Lothian. The group has worked together for the last decade to develop gene therapy for cystic fibrosis , which is the most common fatal inherited disease in the UK, affecting around 9,500 people nationally and over 90,000 worldwide.

The trial participants, of whom more than 50 per cent are patients at Royal Brompton Hospital, 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 will receive one dose a month for one year.