Statement from the UK Cystic Fibrosis Consortium with regards to future funding from the CF Trust
The CF Trust has been fully committed to supporting the UK CF Gene Therapy Consortium for the last 10 years in its mission to develop clinically beneficial gene therapy for cystic fibrosis. The Trust has generously funded the preclinical development which allowed us to identify the best, currently-available gene therapy product to take forward. Their funding has also allowed us to conduct a number of clinical studies, which we believe have been genuinely world-leading, not only for gene therapy, but also for other future CF research.
As part of our clinical programme, the Run-In study has allowed us to design the best Multi-dose gene therapy trial, choosing optimal outcome measures and most suitable patients. We have also conducted a Single-dose safety trial of this product (Pilot study), which is nearing completion, and which has allowed us to choose the dose for the Multi-dose trial. Based on the above we had been anticipating a September 2011 start date for the Multi-dose Trial, for which everything is now in place. However, related to the economic downturn, the CF Trust in keeping with other charities, has experienced a reduction in income. This has resulted in their recent decision to take stock of their current financial situation and to ask us to revise our programme to fit with the reduced available funding.
Both the CF Trust and the Consortium intend the Multi-dose Trial to continue, and we are working closely together on this. The focus of the trial will remain as before, looking to produce improvements in lung disease. If successful the product would then move rapidly into large scale trials, in partnership with a pharmaceutical company as previously planned.
We anticipate a short delay related to the above. Subsequently this extensively peer-reviewed, audited and ground-breaking programme of work will deliver the world's largest trial of gene therapy for CF, undertaken by the continued synergy between the UK CF Gene Therapy Consortium and the CF Trust.