The Cystic Fibrosis Trust is the UK’s only national charity dealing with all aspects of Cystic Fibrosis (CF). It funds research to treat and cure CF and aims to ensure appropriate clinical care and support for people with Cystic Fibrosis. The CF Trust invests over £4 million each year in gene therapy research.

In the early part of this decade, the Cystic Fibrosis Trust brought together the UK’s leading CF gene therapy teams into a single working group – the UK CF Gene Therapy Consortium. There are now over 80 scientists and clinicians in Edinburgh, London and Oxford dedicated to gene therapy for Cystic Fibrosis. It is the largest group studying CF gene therapy in the world.

The UK CF Gene Therapy Consortium has developed a product for CF gene therapy. Detailed trials to ensure it is safe are now being carried out.

200 people with CF in Edinburgh and London have already been recruited to take part in a multi-dose clinical trial and will be monitored for a year to measure vital functions. This is called the Run-in.

In February 2009, a pilot trial for CF gene therapy began. 27 young adults with Cystic Fibrosis in London are being given a single dose of the gene therapy product. This will provide important information on the correct level of dose and possible toxic effects. The pilot study is estimated to last eight months and is due to end in September 2009.

Once the results of the pilot study have been analysed, a further round of safety tests is necessary. At the same time, up to 100 of the people recruited for the Run-in will be chosen to take part in the multi-dose clinical trial. Doctors and scientists are looking for the most suitable candidates from the original 200 patients to take part.

If all is well in the pilot trial and as long as the safety studies and the product are passed by the regulatory authority, these patients, all over 12, will be given either the gene therapy product or a placebo in a double blind trial for a year. We estimate this will begin in 2011.

This is the first time anywhere in the world that CF gene therapy has been studied in this way.

It is estimated that the multi-dose clinical trial will cost at least £6½ million. A more precise figure will be known following the pilot trial.

If successful, a major multi-centre trial (potentially including Europe and the USA) would take place. This would be the definitive study for safety and efficiency.

If this trial was successful, a major pharmaceutical company would then develop the treatment into a product for the market and everyday clinical use. This is a best case scenario for CF gene therapy.

Wave 2 gene therapy

The 80 dedicated scientists and clinicians who make up the Consortium have been working on the Wave 1 product, as described above. Although this is their focus, there have also been some interesting developments on a Wave 2 product, which uses a viral carrier, and which looks able to be given repeatedly (although a virus is good at getting to the lungs, they cannot normally be re-used as the body becomes immune). This product development is also being rigorously pursued.