Last month, we received the encouraging news that the board of the EME Programme has made an "in principle" decision to fund the Gene Therapy Consortium (GTC) to conduct the Phase 2b clinical trial into gene therapy for Cystic Fibrosis. Discussions between the EME Programme and GTC are continuing, but we expect a formal decision early in 2012.

Jump to our latest questions and answers

What next?
As long as the EME Programme decision is finalised in the New Year, enough money will have been raised to make the gene therapy trial happen, which is a fantastic achievement. From April 2011 through to the end of 2014 we will pay the Gene Therapy Consortium nearly £5m to cover their work so far, preparations for the phase 2 trial, and manufacture of the drugs to be used in the trial. We have updated our Q&A below regarding next steps.

Extraordinary efforts
It's not only your capacity to raise the £1.6m which is incredible; it's the way in which you have done it too. James Peckham cycled between the three gene therapy research centres -Imperial College London, Oxford's John Radcliffe Hospital and Edinburgh University Hospital - to deliver a CF Rose of Hope. Amanda Chalmers has raised nearly £3,000 by spending a week dressed as a reindeer while receiving hospital treatment for Cystic Fibrosis. And supporters in Northern Ireland raised £13,000 by auctioning, among other things, a calf and a shovel!

Into 2012
Our goal continues to be to fund research which has the potential for the most significant impact on Cystic Fibrosis. Alongside gene therapy research we are currently funding a broad range of work. This includes research to significantly improve access to lung transplants for people with CF, a study to ensure babies diagnosed with CF remain free of lung damage to give them as good a start as possible, and important research into how the exciting new generation of CFTR drugs can be improved even further - more details are available on our website. Over the next few years, we will be working hard with partners in the UK and across the world to increase the amount of CF research being carried out so that new treatments and better understanding of the root causes of CF can be found. The programme will be ambitious and designed to have the highest possible impact on CF.

Questions and Answers

1. How much income has been raised or pledged to date?
We have had a tremendous response from the CF community to the Gene Therapy Appeal, with hundreds of events taking place across the country. In addition, we have secured significant pledges from individuals to help us reach our target. To date, we have raised £1.6m.

2. Did the Gene Therapy Consortium (GTC) receive the whole grant?
The Efficacy and Mechanism Evaluation (EME) programme has decided to fund this clinical study in principle, subject to some changes being made in the application, as a result of the scientific peer review. The GTC and EME programme will now go into a period of discussion to determine the details of the study and the size of the grant. Applications are managed through a robust, peer-reviewed process, which ensures scientific rigour and value to the public purse; this is all part of the standard process for EME programme-funded studies. The peer review is designed to add value and further strengthen the study going forward.

3. How long is it likely to be before any clarifications or changes are agreed between the GTC and the EME programme?
It is difficult to put a clear time line to this; the project team will need to discuss and implement the required changes. Once this has been done, a contract has to be drawn up and agreed between the funder and those responsible for the research. Once this contract has been signed the GTC can start the research, subject to the necessary trial governance being in place.

4. If all the changes requested are made, will the trial then go ahead?
The trial has the biggest chance of going ahead once the necessary trial governance has been put been in place and a contract has been signed between relevant parties. We will update our website as more information becomes available.

5. The CF Trust has funded this gene therapy research to date; why can't the Trust make the decision on whether or not to start the trial?
This piece of gene therapy research is the work of the UK Gene Therapy Consortium which is made up of Imperial College London, Edinburgh and Oxford Universities.

As with any clinical trials the Consortium must fulfil certain criteria and follow special protocols as set out by the health authorities. We do not have the authority to say when and how this trial will happen and so we work closely with the UK GTC to help them with funding the project.

6. What is the difference between the EME, NIHR and MRC? Where did the Gene Therapy Consortium apply for funding?
The Gene Therapy Consortium has applied for an EME Programme grant (which is funded by the MRC and NIHR) hence the confusion around different acronyms.

For your information, here is a little information on the bodies we refer to:

EME - Efficacy and Mechanism Evaluation programme
The EME programme sits between, and works closely with, the Medical Research Council's Experimental Medicine schemes (which support the early development of novel therapies, interventions, and diagnostics) and the established NIHR Health Technology Assessment (HTA) programme (which produces research information about the effectiveness, costs and broader impact of health technologies for those who use, manage and provide care in the NHS). In this way, clinical studies for new treatments or diagnostics at all stages of development or evaluation have potential funding. www.eme.ac.uk/

NIHR - National Institute for Health Research
www.nihr.ac.uk

MRC - Medical Research Council
www.mrc.ac.uk

7. If we get more than £1.6m, what will we do with the additional funds?
As long as the EME Programme is finalised in the New Year, enough money will have been raised to make the gene therapy trial happen, which is a fantastic achievement. From April 2011 and through to 2014 we will pay the Gene Therapy Consortium nearly £5m to cover their work so far, preparations for the trial, and manufacture of the drugs to be used in the trial.

8. If I still wish to support gene therapy past the end of the Appeal, how do I go about doing this?
We believe that gene therapy offers a real potential new therapy in CF, which is why we are encouraging grant applications from scientists working in this area, including the UK CF Gene Therapy Consortium. At the moment, we have no active grant requests in this area. Any funds that are donated specifically for gene therapy research will be ring-fenced for this work and will be allocated by our Research Advisory Committee, which ensures that we support research which has the highest potential benefit for people with CF. The CF Trust is committed to continue funding key elements of the clinical trial through to 2014.

9. What research is the CF Trust funding, now and in the future?
Our goal continues to be to fund research which has the potential for the most significant impact on CF. Alongside gene therapy research, we are currently funding a broad range of work. This includes research to significantly improve access to lung transplants for people with CF, a study to ensure babies diagnosed with CF remain free of lung damage to give them as good a start as possible, and important research into how the exciting new generation of CFTR drugs can be improved even further. Find out more about our research. Over the next few years, we will be working hard with partners in the UK and across the world to increase the amount of CF research being carried out so that new treatments and better understanding of the root causes of CF can be found.

10. I always thought that the CF Trust wanted to be the sole funder of the GTC to ensure a swift move to clinic of the final drug - has this changed?
We are very proud of our association with the Gene Therapy Consortium. Over the past ten years, we have spent in excess of £30m to get to this stage; this is a fantastic achievement and a real tribute to our supporters. We remain committed to seeing a viable, clinically-successful gene therapy product available for use by people with CF in the UK, which is why we hope that a successful outcome of the forthcoming clinical trial will also attract the interest of pharmaceutical companies, who will be in a far stronger position to bring a clinical product forward for use in the UK which can cost in excess of £100m.

11. What else are you spending fundraised money on, apart from GT or research?
Over the coming weeks, we will be introducing our new strategy for the next four years. We will be focussing on making real progress in four key areas: increasing length of life; improving quality of life, increasing the number of clinicians and researchers working in CF, and; improving awareness and understanding of CF. This is an ambitious programme of work which includes providing support for people with CF in education and employment, improving access to psychosocial support and campaigning for expansion in services. We also want to build a world class ambitious research programme linked to key research happening in other countries to ensure that between us we can fight CF.