Just Gene Therapy is a not for profit organisation established to raise funds for gene therapy for cystic fibrosis.

Gene therapy

Initially focusing on the lung, the cause of the vast majority of deaths in people with CF, gene therapy aims to introduce a correct copy of the CF gene into the appropriate cells of the person's lungs enabling them to work as they should.

Initial evidence that this is possible is extremely encouraging. However, the research is very expensive and cannot be rushed. At every stage, safety checks are of paramount importance and the regulations that control genetic research are understandably and appropriately demanding.

The UK CF Gene Therapy Consortium (www.cfgenetherapy.org.uk) is a team of world renowned scientists from Imperial College, London and the universities of Oxford and Edinburgh, who have worked in this area for many years. Ten years ago, the three independent teams started to work together rather than as competitors and are now an integrated and focused team with the clear goal of developing an effective gene therapy for those with cystic fibrosis.

It is believed that if the healthy (non CF) gene is delivered into the appropriate cells and then activated instead of the faulty copy, a correction of the basic defect is likely to result. However, a major problem has been the delivery of the gene to cells that are heavily defended against 'intruders' by the body's defence mechanism in the form of the immune system, exacerbated in those with CF by copious amounts of glutinous mucus and, as the disease progresses, by lung damage.

There are two prongs of attack currently being progressed by the UK CF Gene Therapy Consortium. The first – Wave 1 – uses a fatty substance called a liposome as the delivery system. A pilot study on 30 patients with CF has given some very encouraging results. There has also been an intensive 'run-in' study where almost 200 people with CF were regularly and thoroughly subjected to a range of tests, the results of which will be invaluable in assessing the results of the gene therapy itself.

A larger, longer, multi-dose trial with around 130 patients is now underway. This is a 12 month double blind randomised trial where half the patients receive gene therapy and half receive a placebo, with neither participants nor investigators knowing which, in order to rule out the placebo effect and avoid bias. It is the largest clinical trial for a gene therapy product of this nature anywhere in the world. This trial is being funded by a grant from the Efficacy and Mechanism Evaluation (funded by the Medical Research Council and the National Institute for Health Research, with contributions from the CSO in Scotland, NISCHR in Wales and the HSC R&D, Public Health Agency in Northern Ireland).

The scientists recognise that a virus has the potential to be an even better delivery system for gene therapy, if obstacles to their effectiveness could be overcome, such as the ability to deliver repeated doses without the body developing immunity. Such a virus has been identified, produced by a Japanese company called DNAVEC, and appears very promising indeed. It is potentially far more effective than the Wave 1 product. This represents the second prong, or Wave 2, which although at an earlier stage in research terms, would be the most effective way of correcting the genetic defect, and is likely to benefit patients who are already at a more severe stage of cystic fibrosis. Just Gene Therapy will concentrate on funding the Wave 2 research but will consider any additional requests for funding for any financial shortfall in the Wave 1 trial.

The only obstacle to the progression of this internationally renowned second stage of research using a viral approach is that of funding.




CF patient about to receive gene therapy product

A patient about to receive gene therapy product.

Gene therapy liposome product

A vial of lipid vector (left) and a vial of the plasmid DNA (centre), which are combined to create the Wave 1 product.

Making the Wave 1 product

Making the Wave 1 product: a pharmacist combines the lipid and the plasmid.

© Just Gene Therapy 2012-14 | Just Gene Therapy is raising money on behalf of Imperial College, an exempt charity*.