Applications were submitted from ten research teams during the bid-for-funding process which opened during Children’s Cancer Awareness Month in September. The charity’s recently appointed international Scientific Advisory Board, comprising three of the world’s preeminent neuroblastoma specialists independently reviewed all research proposals.

Two projects were considered to be outstanding by the Neuroblastoma Australia Scientific Advisory Board and have been recommended for grants of $125,000 each for 2020. 

The winning projects

One of the two winning research projects will use an existing drug to treat a group of high-risk children with an extremely rare TERT oncogene rearrangement in their neuroblastoma, who currently have a less that 40% chance of survival.  This research will be undertaken by Associate Professor Tao Liu at the Children’s Cancer Institute.

The second successful project, led by Dr Alla Dolnikov also at the Children’s Cancer Institute, is researching a new way to make CAR T therapy more effective in treating neuroblastoma tumours by suppressing the MYC oncogene. Currently CAR T therapy which is seen as a promising way of using the body’s own immune system to tackle cancer cells has not worked on neuroblastoma tumours.  But the award-winning research looks like there could be a way of changing this.

High-risk neuroblastoma cases with TERT oncogene rearrangement

Photo of Associate Professor Tao LiuNeuroblastoma is the most common solid tumour in early childhood and accounts for 15% of all childhood cancer death. Approximately a quarter of high-risk neuroblastoma is caused by TERT oncogene rearrangement.

Children with this subtype of neuroblastoma have less than 40% survival rate and are typically treated with surgery and toxic chemotherapy, meaning those that do survive often suffer life-long side effects.

The funding from Neuroblastoma Australia will allow our team at Children’s Cancer Institute to identify the specific drivers of TERT oncogene rearrangement in high-risk neuroblastoma and demonstrate novel therapies that specifically target these drivers. Dr Tao LiuWe hope that, if successful, these novel therapies can be taken into clinical trials to see the first targeted therapy for patients with neuroblastoma due to TERT oncogene rearrangement, leading to better survival rates and better quality of life for children with this devastating childhood cancer.


Using MYC inhibitors to potentiate CART cell therapy for neuroblastoma

Current therapies for high-risk neuroblastoma are ineffective and leave devastating side effects due to the toxicity of the treatment. There is an urgent need for novel therapeutic approaches to more effectively treat these children.
A promising new approach is Chimeric Antigen Receptor (CAR) T-cell therapy, currently used in some types of leukaemia, which programs a child’s immune cells (known as T cells) to destroy the cancer cells.
“This strategy has traditionally proved to be ineffective in children with neuroblastoma as the MYC oncogene (a gene that leads to poor patient survival), which is activated in high-risk neuroblastoma, acts to suppress the immune cells,” explained Dr Dolnikov.
Dr Alla Dolnikov and her team at Children’s Cancer Institute have identified a drug that acts as an inhibitor to the MYC oncogene, opening up the potential of the immune cells to fight the tumour.

This is an exciting discovery that that will hopefully lead to the development of a novel therapy using this drug that will mean that we can apply CAR-T cell therapy more effectively in children with high-risk neuroblastoma. It will provide a more effective and less toxic way to treat this devastating childhood cancer. Dr Dolnikov

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