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Univeristy of Edinburgh & NHS Lothian Clinical Research Facility (CRF) Newsletter - June 2011

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The Edinburgh & NHS Lothian Clinical Research Facility Newsletter for June 2011 contains information on the new gene therapy facilities at the University of Edinburgh & NHS Lothian CRF.

The newsletter also contains an article on the UKCFGTC winning two Medical Futures Innovation Awards. For more information on the awards please visit the MFIA 2011 page of our website.

Message to Run-in Volunteers and their Families - March 2011

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You may already be aware of an announcement made by the CF Trust on their website (http://www.cftrust.org.uk/) regarding our Gene Therapy programme. As a volunteer or parent of a child who has helped us with the Run-in study, we wish to provide you with some further information and expand on this.

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 CF 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 you know, the Run-In study was conceived to allow us to design the best multi-dose trial, choosing optimal outcome measures and most suitable patients. We will very shortly be contacting those of you still waiting to hear whether, based on your Run-In data, you are in the optimal group to go forward.  In addition to this, we have also conducted a single-dose safety trial (Pilot study) of this product, 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 contacting everyone regarding a September start date for the multi-dose trial, for which everything is now in place. However, related to the economic downturn, the CF Trust has experienced a  severe reduction in income which we have recently become aware of. This has resulted in their recent decision to take stock of their current financial situation and to ask us to revise our programme. Both the CF Trust and the Consortium intend the multi-dose trial to continue, and we are working closely together on this, but as you will realise, this inevitably will lead to some delay. The focus of the trial will remain as before, looking to produce improvements in lung disease. If successful, the Wave 1 product would then move rapidly into further trials as previously planned. 

We will be in a much clearer position within the next few weeks and will keep closely in touch with you.

We wish to take this opportunity to thank you very much for your participation in the gene therapy research programme; without volunteers such as you and your children, we would have been completely unable to make any of this progress.

Current Status of Run-in Patients

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The upcoming multi-dose clinical trial will determine if gene therapy can improve CF lung disease. This is a very important proof-of-principle study and it is, therefore, important to select the most appropriate patients to answer this question. The multi-dose trial will have the best chance of success if we select patients that (a) have healthy enough lungs to ensure that the gene is delivered to most of the airways, but (b) also have sufficient lung disease to allow us to measure an improvement after gene therapy. 

Based on the results of the Run-in study we are able to assign patients to one of three groups below. You will have been contacted by the consortium to let you know which group you are in.

Group 1: patients asked to continue on the Run-in with a view to participate in the Multi-dose trial

These patients have healthy enough lungs to allow efficient delivery of the gene, but also have sufficient lung disease to allow us to assess improvement after gene delivery.

These patients have largely been seen for a visit five and will be contacted in the furture regarding further Run-in visits.

 

Group 2: Patients not suitable for progression into the Multi-dose trial

 

There are two reasons why patients may not be suitable for the multi-dose trial:

Patients who are extremely well and have very healthy lungs are not suitable because although we would be able to deliver the gene to the airways of these patients efficiently, we would not be able to measure an improvement in lung disease after gene therapy, because the lungs are already very healthy.

Patients who have quite advanced lung disease are not suitable because the comparatively advanced lung disease in these patients would make it very difficult to deliver the gene and, therefore, reduce the chance of seeing an improvement in lung disease severity. In addition, we are concerned that the treatment may be less well tolerated in patients with severe lung disease, because gene delivery may cause a transient drop in lung function.

These patients have been invited to attend a visit five to exit the Run-in study.

Group 3: Patients for whom we cannot make a decision yet, but hope to do so shortly

There are two reasons why we may not be able to make a decision yet:

Patients who are quite well and have only limited lung disease. Although we would be able to deliver the gene to the airways of these patients, we may not be able to measure an improvement in lung disease after gene therapy, because the lungs are fairly healthy. Patients may have abnormal results in some, but not all of the tests we plan to include in the multi-dose trial.  Further detailed analysis of our data will help us decide if patients in this group may be suitable for taking part in the trial.

Patients whose average FEV1 has been lower than 50% during the Run-in study. We anticipate that we would be able to deliver the gene to the airways of these patients, and would also be able to measure an improvement after gene therapy. However, we are concerned that the treatment may be less well tolerated because gene delivery may cause a transient drop in lung function. Over the next few months we will be looking at ways to prevent the transient decrease in lung function after gene therapy. If this is successful, patients in this group may be suitable for inclusion in the multi-dose trial.

Once decisions have been made, these patients will be invited for a visit five either to continue in the run-in or to exit the study.^Top

Last updated: 25 August, 2011 16:32

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