Australian scientists have identified a critical molecular ‘feedback loop’ that helps initiate and cure  neuroblastoma. It is a cancer of the nervous system in children that is developed in embryonal nerve cells. The research team was able to identify an experimental drug, which was currently in clinical trials for adult cancer, with the potential to interrupt the loop and halt tumour progression. This feedback loop massively accelerates cancer development.

The authors showed in laboratory models of neuroblastoma that the drug could block the very start of this embryonal cancer, paving the way to possible prevention strategies in the future.

What is Neuroblastoma?

• Neuroblastoma, a cancer that develops from immature nerve cells found in several areas of the body, is the most common ‘solid tumour’ of early childhood, and is generally diagnosed when the disease is advanced.
• Around half of all children with neuroblastoma have aggressive tumours, and fewer than half of these patients survive, even after intensive treatment. A potential new drug on the horizon is very good news indeed.
• The new study, from the Institute, found that the drug – known as CBL01371 – used in combination with traditional DNA- damaging chemotherapy agents was much more effective than either drug alone because CBL0137 created a ‘synthetic lethal’ state – by preventing the cancer cells from repairing DNA damage induced by chemotherapy, and so ensuring cell death.
• The MYCN gene – already known to be a key driver of neuroblastoma – and a molecule known as FACT, a DNA modifying agent which is the target of CBL0137.

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