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Cash boost for key leukaemia research

Written by Lautaro Vargas

Wednesday, 12 September 2007

A young, Cambridge-based researcher described as both a brilliant and outstanding talent has received a major grant to progress his pioneering work on a rare disease in children that can lead to a particularly devastating form of leukaemia. The Association for International Cancer Research (AICR) has provided 37-year old Dr Wojciech Niedzwiedz of Poland its annual fellowship of £820,000 – its biggest single award of the year – to study Fanconi Anaemia (FA), an inherited genetic condition in which children develop acute myeloid leukaemia. Unless a suitable bone marrow transplant can be made, many young patients die. As well as providing a deeper understanding of this disease, Dr Niedzwiedz’s work at the MRC Laboratories of Molecular Biology could eventually lead to the development of life-saving treatments for a great number of patients affected by other fatal diseases. “Cancer is caused by damage to the genes that control cell division and genome integrity,” said Dr Niedzwiedz. “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.” FA is a recessive disorder, which means that if both parents carry a defect – mutation – in the FA gene, each of their children has a 25 per cent chance of inheriting the defective gene from both parents, which is when the disease would occur. (article continues after advertisement) Although considered primarily a blood disease, it can affect many systems of the body and all patients have a 15,000 times greater risk of eventually developing acute myeloid leukemia than people without Fanconi anaemia. Less than a thousand cases are recorded worldwide for the disease, which is characterised by short stature, skeletal anomalies and bone marrow failure. The majority of these young patients go on to develop serious leukemia or other cancers before reaching adulthood. FA patients are also 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. Dr Mark Matfield, AICR’s scientific adviser, said: “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 a new gene therapy to prevent the disease from happening.” AICR is a totally independent charity based in St Andrews in Scotland. It funds what it considers to be the best researchers and the most valuable studies, wherever they are in the world, an innovative approach to funding research that has enabled it to contribute significantly to furthering the world’s understanding of cancer.

 

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