Dr Alla Dolnikov from the Children’s Cancer Institute, is one of the two winners of our 2020 research grants. We asked Dr Dolnikov to outline her research and how it aims to help children with high risk neuroblastoma. 

Using MYC inhibitors to potentiate CART cell therapy for neuroblastoma

Neuroblastoma is the most common solid tumour in early childhood. In about 50% of cases, the cancer will spread aggressively to different parts of the body, resulting in a poor prognosis. Even with the best treatments, a significant proportion of high-risk neuroblastoma patients still die of their disease, and neuroblastoma accounts for approximately 15% of cancer deaths in children. Hence, there is an urgent need for new novel approaches to treatment.

A promising new treatment approach is Chimeric Antigen Receptor (CAR) T-cell therapy, which programs a child’s immune cells (known as T cells) to destroy the cancer cells. This novel therapy has generated breakthrough responses in adult and children with some types of leukaemia and lymphomas.

This therapy has traditionally proved to be ineffective for neuroblastoma as the MYC oncogene, which is activated in high-risk neuroblastoma, acts to suppress CAR-T cells and reduce any effect they can have on the tumour. But Dr Alla Dolnikov and her team 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 project aims to expand the application of CART-cell therapy for children with high risk neuroblastoma.  It is expected that using CART-cells in combination with the identified MYC inhibitors will generate high response rates in neuroblastoma patients, similar to those observed in CART-cell therapy for leukaemias and lymphomas.  The project if successful will form the foundation for a clinical trial to assess the safety and efficacy of administration of CART-cells in combination with MYC inhibitors in neuroblastoma. The data generated using funding from Neuroblastoma Australia will provide the basis for additional funding needed to perform the appropriate validations necessary for regulatory approval and proceed with a clinical trial. 

Dr Dolnikov’s links with the Sydney Children’s and Westmead Hospital will facilitate the translation of this research into the clinic. We hope that this study will pave the way for novel immune cell therapies targeting a range of other childhood and adult cancers improving the cure rates of these cancers.    

More information

• Media release of funding announcement
• PeterMac explanation of CART-T
• Visit our research projects page
• Visit our grant recipients page 
• Find out more about current research projects

Would you like to help us fund more vital research?    

There were eight other wonderful research projects submitted during our funding process. We only had enough to fund the top two. We will be putting our efforts towards raising funds to help support at least another two projects but we can't do this without your help.

Visit our how to help page for ways that you can help us to raise funds for this children's cancer or simply donate to our appeals.

Every little bit helps.