MRC/NIHR EME Programme to Fund Clinical Trial

Friday, March 16th 2012

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MRC_NIHR_Logo.jpgThe Consortium's Multi-Dose clinical 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.

Patients will receive the treatment by inhaling aerosolised molecules of the plasmid pGM169 (developed by Oxford Gene Medicine) complexed with the lipid GL67A. 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 already received a single dose of the formulation and by delivering multiple doses over the course of a year, we aim to determine whether the therapy can improve symptoms for CF patients.

No one has ever done a gene therapy study like this in cystic fibrosis before. It's a worldwide first in terms of the length of the study, the number of patients involved and the number of doses of gene therapy. By giving the therapy over a whole year, we will have the best chance yet of seeing an improvement in patients.

Dr Deborah Gill, University of Oxford.

We want to create a world-class NHS where patients benefit from the latest and best treatments. This funding provided by the Government, via the MRC and the NIHR, will enable pioneering studies demonstrating how science in the lab can be translated into gene therapy for patients with cystic fibrosis. This work is another example of the Government's continued commitment to reducing the burden of genetic disease and follows recent recommendations from the Human Genomics Strategy Group, which I have welcomed, which provides a strategic vision for the adoption of genomic technologies across healthcare to diagnose and treat genetic conditions.

Andrew Lansley, Secretary of State for Health.

This £3.1 million investment will deliver a groundbreaking clinical trial in cystic fibrosis gene therapy, which is an important and promising area of health research. It is an excellent example of collaborative working between Government, the research base and a leading health charity.

David Willets, Minister for Universities and Science.


Large scale lentivirus production in suspension culture.


Purifying mRNA from tissue samples.


A frozen vial of GL67A (left) and a frozen vial of pGM169 plasmid DNA (right)


Proposed 3D model of the CFTR protein.