International DFMO clinical trial

Recipient: Associate Professor David Ziegler
Institute: Children’s Cancer Institute
Funding: $448,142 June 2014 to July 2018

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.