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Paving the way for improved neuroblastoma treatments

October 8, 2014

‘Research results such as these help us discover new treatments, specifically designed for children, to ensure they experience the highest possible quality of life.’ Professor Glenn Marshall AM, Head of Translational Research, CCI.

An important discovery in the search for new ways to treat neuroblastoma, one of the most aggressive forms of childhood cancer, has been made by researchers working at the Children’s Cancer Institute (CCI), Sydney, Australia.

While the survival rate of several types of childhood cancer has improved dramatically over the years, the rate for neuroblastoma – the most common solid tumour in infants – remains disappointingly low at around 50%.

Cancer researchers have learnt from previous studies that some children treated for neuroblastoma do much better than others in part because of the genetic make-up of their neuroblastoma cells. One genetic feature strongly linked to a poor prognosis (i.e. the likelihood of a poor outcome) is having multiple copies of a cancer-promoting gene called the N-myc oncogene.

‘Normally humans only have two copies of the N-Myc gene, but children who have neuroblastoma can have 100 to 150 copies,’ explains Dr Tao Liu, Group Leader of CCI’s Histone Modification Group. Children who have this ‘N-Myc amplification’ are much more likely to die of the disease than those who don’t, he adds
Dr Liu and CCI colleagues, working with collaborators at the Harry Perkins Institute of Medical Research and the University of Western Australia, have uncovered the roles of two molecules that appear to control the behaviour of the N-myc oncogene in neuroblastoma cells. The first, a ‘long noncoding RNA’, plays a critical part in neuroblastoma tumour formation, while the second, the NonO oncoprotein, contributes to the severity of the cancer.

Both of these molecules represent potential targets for researchers to design new treatments to aim for. Called targeted therapies, such treatments are specifically designed to inhibit the action of particular molecules in cancer cells, reducing their negative effects.

CCI’s Head of Translational Research, Professor Glenn Marshall AM, says the research has significantly improved our understanding of what leads to neuroblastoma, as well as uncovering new targets for anti-cancer treatments.

‘Side-effects associated with conventional chemotherapy used to treat kids with cancer are a significant clinical problem,’ he explains. ‘Research results such as these help us discover new treatments, specifically designed for children, to ensure they experience the highest possible quality of life.’

This research has been published in the US ‘Journal of the National Cancer Institute’ http://jnci.oxfordjournals.org/content/106/7/dju113.long

Pictured Neuroblastoma cells under the microscope

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