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

Statement from Professor Alton

on behalf of the UK CF Gene Therapy Consortium

Gene therapy for cystic fibrosis has come a long way since Rosie Barnes, then the Chief Executive of the CF Trust, brought together the UK CF Gene Therapy Consortium over ten years ago. However, not surprisingly, it is very expensive research and takes years to complete. There is no quick fix or magic formula to develop a product which the Consortium hopes will be effective and safe for CF patients with any type of mutation.

We have made good progress, and are very excited about how the programme is developing. The double blind placebo-controlled trial of our Wave 1 product, wrapping up the CF gene in fat globules (liposomes), has now started. This is the biggest study ever to have been carried out, and will tell us whether gene therapy, in its current form, can improve lung disease in CF patients. Results will be available in early 2014. Clearly, it takes time to get answers from the Wave 1 programme and although we have high expectations, it seems unwise to put all our eggs in one basket. We have, therefore, also developed a Wave 2 product, putting the CF gene into a novel virus. This is showing even higher efficiency, and was recently identified by the government's Medical Research Council as their innovative product of the year.

We are hugely grateful to the CF community who, through the CF Trust, have supported the gene therapy programme so enthusiastically and generously to date; nothing would have been achieved without this. However, the CF Trust was not able to support this work beyond the end of February 2012. To fund the Wave 1 trial, the Consortium therefore successfully applied to the Efficacy and Mechanism Evaluation (EME) programme (funded by the MRC and NIHR, with contributions from the CSO in Scotland, NISCHR in Wales and the HSC R&D, Public Health Agency in Northern Ireland). We were also fortunate in obtaining funding for the initial stages of developing the Wave 2 product through the Medical Research Council's Developmental Pathway Funding Scheme (DPFS).

At present there is no route for anyone in the CF community, who may be kind enough to support the gene therapy programme, to provide funding into our work. The Consortium, therefore, re-approached Rosie to ask whether she would be willing occasionally to take time off from her grandchildren, and once again act as our Ambassador in a fund-raising campaign. She has agreed to this, under the banner of Just Gene Therapy, and we are delighted and proud to re-establish this very productive relationship.



The UK CF Gene Therapy Consortium's Strategy Group

The UK CF Gene Therapy Consortium's Strategy Group

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