Researchers find new way to target childhood cancer
November 18, 2019
An Australian-led international research effort has broken fresh ground in the race to find more effective treatments for the childhood cancer neuroblastoma.
An Australian-led international research effort has broken fresh ground in the race to find more effective treatments for the childhood cancer neuroblastoma, by uncovering a new and as-yet unexploited target in cancer cells that therapeutic drugs can be aimed at.
Neuroblastoma kills more children under the age of 5 than any other cancer. One of the factors that makes neuroblastoma ‘high risk’ in a child is high levels of a molecule known as the MYCN oncogene. But despite knowing the importance of MYCN, scientists have not found a drug capable of targeting it and cancelling out its effects. Instead, the focus has been on finding molecules that work hand-in-hand with MYCN, that may prove easier to target.
The new research, led by scientists at Children’s Cancer Institute in collaboration with scientists in the US, Europe and China, and published this month in Nature Communications*, has identified one such target molecule. By analysing hundreds of tumour samples of children with neuroblastoma, the researchers found that high levels of a long non-coding RNA (called ‘lncNB1’) is a marker of poor prognosis; in other words, children whose tumours have high levels of lncNB1 tend to do worse than other children with neuroblastoma. Excitingly, the scientists were not only able to show that lncNB1 actively promotes the growth and survival of neuroblastoma cells, but also that ‘knocking down’ or inhibiting the expression of this molecule causes neuroblastoma cells to die, and tumours to stop growing and regress in mice with the disease.
“This research opens the door to developing new targeted therapies that we hope can proceed to clinical trial in children with high-risk neuroblastoma,” said Associate Professor Tao Liu, Group Leader at Children’s Cancer Institute. “Targeted therapies are designed to specifically target cancer cells and not healthy cells, and so are much safer as well as more effective than conventional chemotherapy. They represent a significant step forward and are the way of the future.”
In the last few decades, a number of protein-coding genes have been shown to work closely with MYCN to promote the aggressive growth of tumours. However, very little is known about the role of long non-coding RNA molecules in MYCN-driven cancers. As well as uncovering the role of lncNB1, the current study also found that several other long non-coding RNAs are over-expressed in MYC-driven neuroblastoma cells, raising the possibility that more therapeutic targets could be identified in the near future.
“Continued dedicated research will lead to the development of improved treatments for children with cancer,” said A/Professor Liu. “Ultimately, this means we will not only be able to cure more children, but also give survivors a higher quality of life.”
Children’s Cancer Institute:
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About Children’s Cancer Institute
Originally founded by two fathers of children with cancer in 1976, Children’s Cancer Institute is the only independent medical research institute in Australia wholly dedicated to research into the causes, prevention and cure of childhood cancer. Forty years on, our vision is to save the lives of all children with cancer and improve their long-term health, through research. The Institute has grown to now employ nearly 300 researchers, operational staff and students, and has established a national and international reputation for scientific excellence.
Our focus is on translational research, and we have an integrated team of laboratory researchers and clinician scientists who work together in partnership to discover new treatments which can be progressed from the lab bench to the beds of children on wards in our hospitals as quickly as possible. These new treatments are specifically targeting childhood cancers, so we can develop safer and more effective drugs and drug combinations that will minimise side-effects and ultimately give children with cancer the best chance of a cure with the highest possible quality of life.