Cystic Fibrosis Research

As a major Adult CF Centre we are actively involved in research into new treatments for CF. From time to time patients may be approached and invited to participate in research projects. This opportunity enables patient’s access to the most up-to-date treatments that are available.
The multidose CF gene therapy trial conducted by the UK CF Gene Therapy Consortium (UKCFGTC) concluded with the last patient treatment in May 2014. This landmark trial was the first in the world to attempt to measure a clinical benefit in lung disease from giving treatment with genetic material. Of 116 patients treated in this trial, 46 were recruited from across Scotland and Northern England, and treated in Edinburgh at the Western General Hospital. The past year has been spent in collating and analysing results, and preparing the main publication of the results. Full results have recently been accepted for publication in The Lancet Respiratory Medicine. Electronic publication is imminent and the paper should appear in the journal in late June or early July 2015.
Recognising the major contribution of time and effort from our patients, a public meeting and webcast was held on Sat 30th May 2015, hosted by the Cystic Fibrosis Trust, to announce the trial results first to the patients. Each patient has also been sent a personal letter from the team thanking them and informing them of whether they received gene therapy or placebo during the trial.
The UKCFGTC are now considering plans for possible follow on studies including investigating whether gene therapy works better in combination with the new small molecule therapies, and also developing the next generation of viral based gene therapy vectors.

Further CF Gene Therapy information is available at:

Last Reviewed: 14/04/2016