Clinical Trials for CGD
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 six years. New vectors have been designed that improve the amount of correction that can be achieved in cells and these vectors have been tested in the laboratory and in models that mimic CGD. Protocols have also been established for the safe treatment of patients with low doses of chemotherapy. These great advances have led to the approval of gene therapy clinical trials for CGD by the UK regulatory authority 'Gene Therapy Advisory Committee'. At present gene therapy for CGD is assessed on a case-by-case basis. This committee advises on the ethical acceptability of proposals for gene therapy research on humans taking account of the scientific merits and the potential benefits and risks, and provides advice to UK health Ministers on developments in gene therapy research.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.
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 also developed ways of assessing where into the patients' chromosomes the gene vector has inserted. These techniques will be used to monitor all patients who undergo gene therapy and will benefit a range of primary immunodeficiency disorders.
The Gene Therapy Laboratory at the ICH
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.
Collaboration with the British Society for Gene Therapy
The CGD Research Trust works in close collaboration with the British Society for Gene Therapy. This new society aims to enhance UK research and development in gene and cell-based therapies by facilitating education, communication and sharing of knowledge and expertise.
At this year's meeting in London, speakers from two CGD Research Trust funded laboratories gave keynote lectures. Professor Thrasher, from Great Ormond Street Hospital, presented work on 'Gene Therapy for Immune Deficiencies' and Dr Manuel Grez, the promising results of the adult CGD gene therapy trials taking place in Frankfurt.
Visit the British Society for Gene Therapy website at www.bsgt.org.
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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