Just Gene Therapy has been established by Rosie Barnes in conjunction with Professor Eric Alton and the UK CF Gene Therapy Consortium (see www.cfgenetherapy.org.uk) to raise funds for the continuation of the gene therapy research programme for cystic fibrosis (CF).
There has been considerable research into gene therapy for CF since the faulty gene which causes CF was identified in 1989. It has now reached the stage where a potentially effective product is in a double blind randomised multi-dose trial. Other gene therapy products are almost certain to be needed, to improve efficacy and to benefit more patients.
“I am deighted to say that the Wave 1 trial is proceeding well. We now have approximately 70 CF subjects who have received at least one dose of gene therapy, whilst some are up to the tenth of their allocated twelve monthly doses. The trial has been, as planned, reviewed twice so far by the Safety Committee who had no concerns about progress. We still have room for anyone wishing to participate; please contact either Clare Saunders in London or Maria Dewar in Edinburgh for details. Much as we would like to know about how people are doing on the trial, no analysis of the data is allowed until we reach the end of the study. At present the last patient’s data should become available to us around June next year.
For more information, see the UK CF Gene Therapy Consortium's multi-dose trial page.
The Wave 2 product, which is already in the pipeline, has been demonstrated to be approximately 500 times more effective, so it is essential that this product is developed. Funding has been obtained for the early pre-clinical development; further funding will be needed to take this through to clinical trials.
There are several ways to give to Just Gene Therapy, including donating online. Donations of any size are welcome, from individuals, charitable trusts or companies.
You can also make your own JustGiving page for online sponsorship (ensure you mention Just Gene Therapy).
Just Gene Therapy was formally launched on 13 June 2012 at a musical event at the Stone House, St John’s, South East London. This was followed with talks by Professor Alton in September and November (watch the video in our Events section).
Further events are currently being planned – please check back later for more information.