As a result of the reduced ability of phagocytes in CGD to clear infections, patients can suffer from troublesome, recurrent infections and inflammation. The projects we fund are finding better ways of treating inflammation in CGD through understanding the way that normal cells clear infection and comparing it with cells carrying the mutations that cause CGD.
Multiple defects in the immune system contributes to the severity of CGD
Dr Andrew Smith and Dr Farooq Rahman
Department of Medicine, University College London.
People with CGD frequently develop inflammatory bowel disease, the severity of which can vary from person to person. This project will study possible alternative immune defects that are associated with CGD and that could affect disease severity. The funding will contribute to an existing program of work on abnormal genes and proteins involved in inflammatory bowel disease in CGD. The aim is to develop novel drugs that will improve the clinical management of CGD.
‘Dr Farooq Rahman and I are really pleased to have received this award from the Trust. Neutrophils normally control microbial infections using a molecule called NADPH oxidase and this is defective in people with CGD due to genetic mutations. This aspect of CGD has been intensely investigated over the years but recently, research has become available which may provide additional information on CGD and highlight alternative immune defects associated with the NADPH oxidase. A major finding is that NADPH oxidase has multiple functions within the immune system and is not just involved in microbial killing. We believe that in CGD the immunological defect is much more diverse than previously thought. We hope to identify these additional abnormalities so that new therapeutic targets can be found and this funding will help us achieve this goal. We hope this work will lead to the development of novel drugs to provide improved clinical management of this chronic disorder’
Dr Andrew Smith
Dr Brian Cobb and Miss Colleen Lewis
Case Western Reserve University School of Medicine, Cleveland, Ohio, USA.
This project will help develop treatments for abscess formation in CGD. Abscesses are the immune system’s way of quarantining harmful microbes within the body. They are very painful and often require surgical drainage because they prevent antibiotics from working and can serve as reservoirs leading to larger systemic infections. In CGD abscesses can be extremely severe because of an imbalance of the molecules that normally destroy infections. The Trust will be supporting a two-year research programme led by Dr Brian Cobb, at a total cost of $115,000, investigating treatments for CGD abscesses. The work will form part of a post-graduate studentship for Colleen Lewis. They explain, “The infectious agents common to CGD patients are coated with a thick outer surface made up of sugar molecules. These have been demonstrated to be the immune system’s trigger to form abscesses. Our work will investigate the underlying biology of immune responses that are mediated by the body’s anti-microbial molecules against the abscess-inducing sugars. We will perform pilot studies using models that mimic CGD in which a known drug will be tested to modulate the immune system’s ability to form abscesses in response to these sugars. This drug may increase antibiotic efficacy and dramatically improve the long-term health of CGD sufferers”.
How does Gamma-Interferon work to improve resistance to infection in CGD?
Professor Jerrold Weiss
Department of Medicine, University of Iowa. £34,000 over one year.
The major clinical problem in CGD is increased susceptibility to certain bacterial infections due to reduced anti-microbial activity of specialized white blood cells. Many CGD patients in the USA receive gamma-interferon on a continual basis and this significantly reduces the frequency of serious bacterial infections. However, how this treatment actually works is not completely understood. This project will help find out more about the anti-microbial pathways affected by gamma interferon so that new and better treatments can be developed to fight infections caused by the rise of multi-drug resistant strains of bacteria. This work will add considerably to our understanding of how gamma interferon works and help increase the armoury of drugs to fight infections in patients who have immune systems that do not work properly.
“I am delighted to receive support from the CGD Research Trust to study how gamma interferon works to improve resistance to infection in CGD. We are hopeful that our studies will provide new insights on the role of gamma interferon in amplification of host defenses against infection that could translate to even more effective treatment in the future. I am particularly happy to have work supported by the CGD Research Trust, a charity that has stimulated efforts to better understand and treat CGD.”
Professor Jerrold Weiss
Find out more about previously funded 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.
© 2001-2005 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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