Professor Michelle Haber’s and Professor Murray Norris’s team at Children’s Cancer Institute discovered that a drug called DFMO was much more effective at killing neuroblastoma cells with the addition of a polyamine uptake inhibitor called AMXT 1501 (developed by Aminex Therapeutics). Should their promising pre-clinical research be successful, potentially new treatments can be developed for this devastating disease and improve survival rates for children with hard-to-treat relapsed neuroblastomas.

Exciting breakthrough with a combination drug therapy

Neuroblastoma Australia is supporting research into the drug DFMO conducted by Children’s Cancer Institute (CCI) . DFMO has been in clinical trial for a number of years and has shown it can affect neuroblastoma tumours.

The Team at CCI found that if DFMO were combined with a polyamine inhibitor, the drug becomes far more powerful in killing neuroblastoma cells. Polyamines are molecules which fuel the rapid growth of neuroblastoma cells.  As a result of their world-class research, a new trial testing this combination will be started in 2022. There is a strong rationale that this could ultimately lead to the development of potentially new treatments for this devastating disease and improve survival rates for children with hard-to-treat relapsed neuroblastomas.

As this research has shown such promising data to date, organisations in Europe and the US are also keen to support a clinical trial. It is expected that a trial involving US, Australia and Europe could be started in 2022 which will help accelerate this research.

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Project Update

As of June 2023, adult clinical trials are underway in Australia. We're waiting for updates regarding formulations for children. 

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