DIPG discovery offers hope for a devastating disease

A revolutionary drug combination discovered by scientists from the Kids Cancer Centre (KCC) at Sydney Children’s Hospital, Randwick and the Children’s Cancer Institute (CCI) has shown astonishing results; eradicating cancer cells of the currently incurable Diffuse Intrinsic Pontine Glioma (DIPG), a cancer that tragically takes the life of around 20 Australian children each year.  

DIPG is one of the most devastating childhood cancers, with children typically dying from the disease within 12 months of diagnosis,” A/Prof David Ziegler.

Associate Professor David Ziegler, Paediatric Oncologist and Lead Researcher, has shown for the first time that the polyamine pathway (amino groups that are found in human cells) is critical to the growth of DIPG cells, leading to development of a drug combination treatment that has already proven to be the most effective treatment ever tested in laboratory models of this disease.

Our laboratory discovery led us to combining two drugs together;  difluoromethylornithine (DFMO), an anti-cancer drug developed several decades ago, and AMXT 1501, a new investigational agent. On its own, DFMO was unable to stop the growth of DIPG tumours, but when combined with AMXT 1501, we were able to block  the transport of polyamines into the DIPG cancer cell, which led to a significantly increased survival and minimal side effects in laboratory models,”  A/Prof David Ziegler.

Clinical trials of the drug combination in DIPG are planned to begin this year in a global study led by the Kid’s Cancer Centre at Sydney Children’s Hospital, Randwick.  

A/Prof David Ziegler led the development of Australia’s first research program into DIPG, which uses tumour cells donated by the parents of children who have passed away from the disease. Children like Liliana, who was the first to donate DIPG cells, have helped make this breakthrough possible.  

We made the difficult decision to donate Liliana’s tumour because we wanted to make a difference, there were no treatments to save Liliana from this devastating disease, but if her cancer cells help advance research so there be new treatments for children in the future, this will be a lasting memory of our little girl,” Rachel, Liliana’s mum.