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Our project? To cure kids' cancer for good

Gene therapy trial

MGMT cancer gene therapy trial

The ‘Gene Therapy Trial’ is a world-first and offers hope to children with brain tumours.

  • Brain tumours account for more deaths than any other childhood cancer.
  • Half of all Australian children diagnosed with a brain tumour will not survive.
  • Some forms of childhood brain cancer have no treatment options and no survival.


Brain tumours are the most common solid tumours in children. They can be difficult to treat because often the cancer grows among healthy brain tissue, meaning it can’t be removed with damaging vital functions like breathing, swallowing and movement.

Often, the level of chemotherapy needed to affect the child's brain tumour you would cause significant damage to a child’s bone marrow and immune system.

The culmination of 13 years of dedication by The Kids' Cancer Project and The Children's Hospital at Westmead working together, the Gene Therapy Trial is a completely new way of treating childhood brain tumours.

The trial, for the first time, is altering a child's bone marrow by giving them an extra gene to protect them from chemotherapy.

What this means is that the high doses of chemotherapy should not have the same debilitating impact on the child's immune system. They can therefore be given higher doses of chemotherapy, with the hope that there will be a greater impact on their tumour.

The clinical trial, now in its third year, has been praised by some of the world’s foremost experts into childhood cancer.

“Brain tumours are hard to treat because the drugs can’t get into the brain properly. This trial is all about introducing a gene into the bone marrow cells so that they won’t be damaged by the chemotherapy.”

Dr Geoff McCowage
Children’s Hospital at Westmead

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Children's Cancer Institute AustraliaQueensland Children's Medical Research Institute University of Queensland - Institute for Molecular Bioscience The Children's Hospital at WestmeadTelethon Kids' Institute The University of NSW - MedicineCentre for Cancer Biology Sydney Children's Hospital FoundationNationwide Childrens Garvan InstituteCancer Council Victoria