Institute: Children’s Cancer Institute
Recipient: Prof Michelle Haber
Funding: $200,000 from March 2016 to March 2017
The ‘DFMO Clinical Trial’ is an exciting international collaboration offering hope to children with hard-to-treat relapsed neuroblastoma.

The clinical trial, developed from research conducted at the Children's Cancer Institute, will run across 16 hospitals in Australia and The United States.

The research is being led in Australia by Dr David Ziegler, a researcher at the Children’s Cancer Institute and Paediatric Oncologist at Sydney Children’s Hospital Randwick.
This trial offers a new treatment approach with an existing drug called DFMO, which hasn’t been used to treat cancer before, but is already being safely used to treat other diseases.

DFMO acts by blocking the production of a type of molecule (polyamines) that is required for cells to grow and proliferate. 

Researchers at CCIA have shown that neuroblastoma cells are extremely sensitive to the action of DFMO and that when DFMO is used in combination with other drugs, it blocks the growth and progression of tumour cells. This is because the molecules DFMO targets play a critical role in the development of neuroblastoma – hence they represent an “Achilles heel”.

Currently, a child with high-risk neuroblastoma who relapses after initial treatment has less than a 20% chance of survival.

The Phase I clinical trial that TKCP supports, involves children with relapsed high-risk neuroblastoma and aims to establish the maximum tolerated dose of DFMO in combination with other drugs. This can hopefully lead the way to the development of a more effective treatment of relapsed high risk neuroblastoma.

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