Institute: Children’s Cancer Institute
Recipient: Associate Professor David Ziegler
Funding: $200,000 March 2016 to March 2017
An exciting international collaboration offering hope to children with hard-to-treat cancer.
Phase one of this clinical trial involves children with relapsed high-risk neuroblastoma. Currently, these patients have less than 20 per cent chance of survival.

The trial, which is running across 16 hospitals in Australia and the USA, offers a new treatment approach with difluoromethylornithine (DFMO) which is safely used to treat other diseases.

The pre-existing drug acts by blocking the production of polyamines that are required for cells to grow and proliferate. Neuroblastoma cells are extremely sensitive to the action of DFMO, when the drug is used in combination with chemotherapy, it blocks the growth and progression of tumour cells.

This study aims to establish the maximum tolerated dose of DFMO in combination with other drugs and will lead the way to the development of more effective treatments.
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