One of the Trust's key aims is to fund work that will result in a cure for CGD. Gene therapy is a potential new treatment for inherited diseases such as CGD. We rely on our genes functioning correctly, so when a gene is missing or not functioning properly, a genetic disease will result. Gene therapy is a way of putting the correct genetic information back into a cell so that it follows a normal healthy set of instructions. This will mean that CGD affected individuals will be able to fight against infection.
What is Involved in Gene Therapy?
The key to gene therapy is delivering the right gene to the cells that
need it. For CGD the cells that need correcting are called stem cells.
These cells are present in bone marrow and blood and are able to
generate all the different cell types that are present in blood.
For gene therapy, some bone marrow or blood is taken from the patient
and mixed together with the normal healthy gene spliced to a harmless
virus (termed vector). The virus carrying the corrective gene then
penetrates the patient's stem cells replacing the defective gene with
the healthy gene. These corrected cells are then grown up in an incubator.
Once sufficient numbers have been obtained the corrected cells are
then re-injected into the patient's blood stream. Gradually they are
absorbed back into the patient's blood marrow where they carry on their
work of manufacturing blood cells - cells that are now capable of fighting
infections.
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.
Key Areas of Research for Gene Therapy
The Trust funds key areas of research that will improve gene therapy
procedures for the cure of CGD. These include: -
Increasing the efficiency of gene therapy so that an optimum amount
of the normal healthy gene is delivered to the right cells in a manner
that is safe and effective over the long-term. This is dependent on
the design of the gene therapy tool or 'vector'.
Establishing treatments that allow the gene corrected cells to engraft
successfully in the patient. This is needed to make space in the patient's
bone marrow for the new corrected cells to grow. This part of the work
is dependent on the use of bone marrow transplantation techniques and
chemotherapy.
News on the Research Front
Jeans for Genes 10 th Anniversary Award
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.
The Start of 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. 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'. 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.
Visit the 'Gene Therapy Advisory Committee' website.
Approval for gene therapy clinical trials for CGD at other sites in Europe has also been given and patients are now being recruited into the trials. The Trust eagerly awaits the results of this groundbreaking work.
Sponsorship of the British Society for Gene Therapy
This year the CGD Research Trust helped sponsor the First Annual Conference
of the British Society for Gene Therapy at Keble College in Oxford.
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. This stimulating meeting
brought together many eminent scientists and clinicians that are
in the process of developing gene therapy protocols for the treatment
of numerous diseases.
At the meeting Professor Thrasher gave a key note lecture on 'Gene Therapy Trials for Monogenic Disorders' that gave insight into the exciting advances made in gene therapy for CGD.
Visit the British Society for Gene Therapy website at www.bsgt.org.
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.
© 2001-2005 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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