July 3, 2015

cystic fibrosis gene therapy trial results

Cystic fibrosis (CF) is the most common lethal inherited disease in the UK, affecting around 10,000 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 introducing a normal copy of this gene using gene therapy.

The UK Cystic Fibrosis Gene Therapy Consortium, 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, have been working together to develop a gene therapy since 2001.

The long-awaited results of the Consortium’s first multi-dose clinical trial, involving 130 patients, have been published in The Lancet Respiratory Medicine. Members of the consortium came to the SMC to discuss the trial’s results, how the therapy works, and what the impact might be for patients.

Roundup comments accompanied this briefing.

Speakers:

Prof. Eric Alton, Professor of Gene Therapy and Respiratory Medicine, Imperial College London; coordinator of the UK Cystic Fibrosis Gene Therapy Consortium

Dr Christopher Boyd, Senior Research Fellow, University of Edinburgh

Prof. Jane Davies, Professor of Paediatric Respirology and Experimental Medicine, Imperial College London

Prof. Deborah Gill, Associate Professor of Gene Medicine, University of Oxford

Prof. Uta Griesenbach, Professor of Molecular Medicine, Imperial College London