The Start of Gene Therapy Clinical Trials
Excellent progress has been made over the last five years in developing gene therapy for CGD. This has been made possible with the help of targeted funding from the CGD Research Trust. Over £1.5 million has been committed to this area over the last seven years. In 2004 the CGD Research Trust took the decision to ring fence funds to develop gene therapy approaches for treatment of CGD. This was an important strategic decision for CGD RT and was taken at a time when the potential of gene therapy approaches was uncertain, although evidence from gene therapy treatments for related diseases, notably Severe Combined Immune Deficiency, was encouraging. The Trust decided to support a strategic collaboration between leading research teams at the Institute of Child Health and in Frankfurt, providing resources for work in each centre and for important collaborative overlap.Evidence of Therapeutic Value
The clinical trials of gene therapy for CGD are showing early evidence of therapeutic value. As with all clinical trials, results of this study have informed about the strengths and weaknesses of gene therapy for CGD in humans, and plans are already underway for additional clinical trials using approaches designed to overcome issues observed in the early studies. Research efforts are now focussed on introducing normal 'corrective' gene copies into patients' bone marrow cells in a way that gives lifelong expression and correction of the disorder, and simultaneously does not disturb the structure of surrounding genetic material in the bone marrow cells to avoid unwanted side effects. While pre-clinical animal experiments are crucial to establish basic concepts, it is only by performing studies in humans that we can begin to build a safe and effective treatment approach that should have real and important clinical benefits. Within the next couple of years, as a result of this work, we can expect to see new gene delivery tools reach the clinic and more people with CGD being treated using gene therapy. This is the start of a new branch of medicine for the treatment of inherited disorders such as CGD.
This project will contribute to the development of gene therapy procedures for many genetic disorders.
Funding of clinical grade vector to treat CGD patients
Gene therapy clinical trials for CGD are taking place at Great Ormond Street Hospital in the UK, and in centres in Germany and Switzerland. To date, five people with CGD have been treated by this pioneering and life-saving therapy using reagents developed and produced by funds from the CGD Research Trust. The results are promising and indicate that gene therapy is a realistic therapeutic option for CGD patients in the future. Further improvements and refinements in the technology are now underway, to maximise the effectiveness and safety of the system. This will hopefully lead to the wider application and availability of gene therapy for CGD patients.
Jeans for Genes 10th Anniversary Award in 2005
The Trust has awarded £680,000 over three years to support a collaborative gene therapy project at the Institute of Child Health/Great Ormond Street Hospital and at the Georg-Speyer-Haus Molecular Virology Unit in Frankfurt. This flagship project for Jeans for Genes headed by Professor Thrasher and Dr Manuel Grez respectively at the two centres will focus exclusively on improving gene therapy for CGD. The grant will fund a senior scientist and a research technician at each site and complements the gene therapy clinical trials for CGD that are on-going in London and Frankfurt. Over the course of three years work will be done to improve the design of the tools (termed vectors) that are used to put back the correct form of the gene that causes CGD into patient's bone marrow cells. This project will make gene therapy for CGD safer and more effective.
This project will contribute to the development of gene therapy procedures for many genetic disorders. Dr Giorgia Santilli (pictured top left) who works on the project says: 'What we are trying to do is work out ways of delivering the healthy gene for X-CGD in a safe and eficient way.'
Development of new techniques for monitoring gene therapy patients
After gene therapy it is essential to monitor patients in a careful and detailed manner, so that we can see how well the immune system has recovered and also to ensure that no side effects have occurred after the treatment. A CGD Research Trust funded project developed sophisticated techniques to look at how certain cells of the immune system recover and to determine how well the bone marrow has been corrected after gene therapy. The project funded facilities for the storage and cataloguing of patients' samples from all patients treated by gene therapy. The project also developed ways of mapping of where the replacement gene has inserted into the patients' chromosomes. Monitoring these integration sites and linking them to the design of vectors has provided invaluable information to improve the safety profile of future generations of gene therapy reagents.
These techniques will be used to monitor all patients who undergo gene therapy and will benefit many other people who are affected by genetic conditions treated by gene therapy.
The Gene Therapy Laboratory at the Institute of Child Health, London
The Gene Therapy Laboratory at ICH was established in 2000 and was funded with help from the CGD Research Trust. This laboratory provides an ultra-clean environment for the manipulation of patients' cells for gene therapy.
Read about Currently Funded Gene Therapy Projects
IMPORTANT NOTE :
The information contained on this website is intended only as a guideline, not as a substitute for medical advice. Always consult your doctor if you or your child has any CGD symptoms or concerns.
© 2001-2007 The Chronic Granulomatous Disorder (CGD) Research Trust
Registered Charity No. 1003425 email:cgd@cgdrt.co.uk
The CGD Research Trust is a member of the Association of Medical Research Charities (AMRC), the Genetic Interest Group (GiG) and an associate member of the International Patient Organisation of Primary Immunodeficiencies (IPOPI)
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