Pioneering cancer charity AICR (Association for International Cancer Research) has awarded its biggest grant of the year to a brilliant young Polish scientist, who has been working in Cambridge for the last five years to help him discover more about a rare disease in children that can lead to a particularly aggressive form of leukaemia.
Pioneering cancer charity AICR (Association for International Cancer Research) has awarded its biggest grant of the year to a brilliant young Polish scientist, who has been working in Cambridge for the last five years to help him discover more about a rare disease in children that can lead to a particularly aggressive form of leukaemia.
Dr Wojciech Niedzwiedz, who is just 37 and is based at the MRC Laboratories of Molecular Biology, at the University of Cambridge, has been given AICR's annual fellowship, worth £820,000, to study Fanconi Anaemia (FA), an inherited genetic condition in which children develop acute myeloid leukaemia. Many young patients die unless a suitable bone marrow transplant can be made.
The disease is named after the Swiss paediatrician who originally described this disorder. Dr Guido Fanconi died 30 years ago.
It is a recessive disorder: if both parents carry a defect (mutation) in the same FA gene, each of their children has a 25% chance of inheriting the defective gene from both parents.
FA occurs equally in boys and girls. Although considered primarily a blood disease, it can affect many systems of the body. All patients have a 15,000 times greater risk of eventually developing acute myeloid leukemia than people without Fanconi anemia.
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FA patients are extremely likely to develop head and neck, gynaecological and/or gastrointestinal squamous cell carcinomas, again at a much earlier age than in the general population.
Patients who have had a successful bone marrow transplant and, thus, are cured of the blood problem associated with FA still must have regular examinations to watch for signs of cancer.
There are less than a thousand cases recorded worldwide and the children come from all ethnic backgrounds. FA occurs when they tragically inherit a damaged gene from both parents.
The disease is characterised by short stature, skeletal anomalies and bone marrow failure. But the majority of these young patients go on to develop serious leukemia or other cancers before reaching adulthood.
According to Dr Niedzwiedz, patients inherit a damaged version of one of the FANC genes. He explains: “Cancer is caused by damage to the genes that control cell division and genome integrity.Genes are made of DNA and all cells have mechanisms to repair damage to their DNA. The FANC genes code for proteins that are involved in DNA repair.
“My AICR - funded research project is aimed at investigating exactly how the FANC proteins carry out this type of DNA damage repair and the consequences of their failure to work. In addition, this work will also impact on our understanding of the role of DNA repair processes in fixing genetic damage that ultimately may lead to cancer. ”
This work will also provide a more detailed understanding of a devastating human disease and may lead to the development of life-saving treatments for great number of patients, not only those affected by Fanconi anemia.
Dr Mark Matfield, AICR's scientific adviser says: “ AICR is committed to supporting the brightest and best cancer researchers and it is particularly important to be able to help them at this crucial stage in their career. We believe that Dr Niedzwiedz is an outstanding scientist and we are excited by his work and the prospect it holds, for helping thousands of children worldwide who will suffer from this debilitating disease in the future, by leading to new gene therapy to prevent the disease from happening.”
AICR (Association for International Cancer Research) is a totally independent charity based in St Andrews in Scotland. It has no commercial ties, no links with any particular research institutions and no commitment to follow any particular line of research.
The charity funds what it considers to be the best researchers and the most valuable studies, wherever they are in the world. This innovative approach to funding research has enabled AICR to contribute significantly to furthering man's understanding of cancer.
For further information, please contact the University of Cambridge Office of Communications on 01223 332300
