New treatments for neuroblastoma

Dr Karen MacKenzie and her team have researched molecular changes in neuroblastoma cancer cells which will lead to more targeted treatments for children with this cancer. By investigating the relationship between levels of specific molecules in cancer cells, tumour growth and the effectiveness of chemotherapy, treatment will become more effective even for aggressive and advanced stage neuroblastoma. Since similar molecular changes are also observed in other cancer types, the findings also have the potential to benefit a broad spectrum of cancer patients.

The need

Neuroblastoma is a cancer that occurs in children and starts in immature nerve cells. It accounts for about 15% of deaths related to childhood cancer, and the outlook for patients in the high-risk category is particularly poor, with less than a 40% chance of long-term survival. 

Previous research has shown that neuroblastoma tumours that progress to advanced stages activate an enzyme called telomerase and that high levels of a certain protein in telomerase in cancer cells could predict the outcome for children with neuroblastoma. Translating these findings into therapy that specifically targets these molecular changes will greatly benefit children with neuroblastoma.

HighlightsNeuroblastoma is a cancer that occurs in children and starts in immature nerve cells

  • The researchers demonstrated the relationship between high levels of a certain protein in telomerase and cancer outcomes for neuroblastoma patients, using tumour samples from 197 neuroblastoma patients. 
  • The laboratory-based experiments showed that reducing this protein to levels similar to those in normal cells slowed cancer growth and also made chemotherapy treatment more effective.
  • The researchers gained an understanding of how suppressing this protein impairs cancer growth.
  • Based on these findings, the researchers developed a new system to block this protein in laboratory experiments.


Despite attempts to improve outcomes for high-risk patients over the past decades, disease recurrence and the harmful side effects of chemotherapy remain a challenge in treating neuroblastoma patients.

The evidence gained through this research project will enable the application of therapies that target specific molecular changes. This will make chemotherapy treatment more effective even for aggressive cancers, enabling lower drug doses and reduced side effects. This will greatly benefit children with advanced stage neuroblastoma who do not respond well to current therapies and are at a high risk of dying from their cancer.

Since telomerase and its protein component play key roles in a range of cancer types, the findings also have a potential benefit to people affected by different cancers.

Dr Karen MacKenzie,
Children’s Cancer Institute
Australia for Medical Research
Dr Jamie Fletcher,
Children’s Cancer Institute
Australia for Medical Research

Professor Michelle Haber
Professor Murray Norris
Professor Glenn Marshall





Cancer Council NSW funding: $352,524 (2011-2014)