Gene could be important drug target for children's brain tumour

In collaboration with Adfero

Scientists have discovered a gene that could lead to more effective treatments for a form of childhood brain cancer called paediatric high grade glioma.

The study, which was part-funded by Cancer Research UK, also identified a number of other genetic differences between the adult and childhood forms of the disease, which can both be hard to treat.

Researchers at The Institute of Cancer Research (ICR), the University of Nottingham and St Jude Children's Research Hospital in the US scanned the genomes of 78 newly-diagnosed children and compared their tumour samples with those taken from adult gliomas.

They discovered that a gene called PDGFRA, located on chromosome 4q12, was often repeated several times in paediatric gliomas, but not in adult tumour samples.

Paediatric gliomas also often had extra copies of chromosome 1q that were not present in adult gliomas.

The study, which is published in the Journal of Clinical Oncology, is the first to show underlying differences in the genetics of paediatric and adult forms of the disease.

Dr Joanna Owens, Cancer Research UK's senior science information officer, said: "This research shows that there are important genetic differences between glioma in children and adults, providing clues to help us find better ways to treat the disease. The researchers have also unearthed a gene that looks to be involved in the development of glioma, which might prove to be a target for life-saving drugs in the future."

Dr Chris Jones, leader of the Paediatric Molecular Pathology Team at the ICR, revealed: "We found significant differences between the genomes of adult and young people's gliomas.

"This is an important finding because it means studies on adult gliomas cannot simply be applied to younger patients, and it has particular implications for drug trials."

Professor Richard Grundy, from the Children's Brain Tumour Research Centre at the University of Nottingham, added: "This cancer gene is unusually active in paediatric high grade gliomas and is likely to be an important drug target."

The researchers also tracked the activity of genes in 53 of the tumour samples and found that, even in paediatric gliomas that did not have several copies of PDGFRA, associated genes were still switched on.

They concluded that this biological pathway - which helps control cell growth, cell division and survival - may therefore play an important role in the development of paediatric high grade glioma.


  • Paugh, B. et al (2010). Integrated Molecular Genetic Profiling of Pediatric High-Grade Gliomas Reveals Key Differences With the Adult Disease Journal of Clinical Oncology DOI: 10.1200/JCO.2009.26.7252