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Our project? To cure kids' cancer for good

MGMT cancer gene therapy trial

MGMT cancer gene therapy trial

MGMT Cancer Gene Therapy Trial

Brain tumours are the second most common childhood cancer. These tumours can be extremely difficult to treat. Only 20 percent of children diagnosed with a brain tumour will survive five years after their treatment. This is a figure that has not improved over the last twenty five years.

Current treatments do not have a great enough impact on these types of tumours. However, the Cancer Gene Therapy Program at the Children’s Hospital at Westmead is initiating a Phase I clinical trial to test the safety and feasibility of a gene therapy strategy to better treat these children.

In this trial, bone marrow stem cells will be genetically modified with a DNA repair protein (called MGMT) to make them drug resistant. This strategy may then allow for increased doses of chemotherapy to be delivered to a child to better target the brain tumour, having eliminated the harmful side effects (for the bone marrow) which limit the dose of chemotherapy a child is typically able to receive.

The Cancer Gene Therapy Program at The Children’s Hospital at Westmead has entered an extremely exciting phase of Australian firsts as it approaches trial initiation. The MGMT Cancer Gene Therapy Trial will be the first in Australia to target bone marrow with gene therapy in an attempt to protect the cells to allow for an increase in chemotherapy dosage.

This is only the second trial in Australia to target this cell type for gene modification in children. The first trial was also been conducted at The Children’s Hospital at Westmead. This trial will also be the first in Australia, targeting the bone marrow, in which all of the critical reagents, including the gene therapy vector and the small molecule drug being used to enhance the chemotherapy, have been manufactured in Australia.


Research overviewMGMT Muscle Stem Cell Project Cancer Gene Therapy Trial Col Reynolds Scholarship Tumour Bank Anti-cytoskeleton Drug Discovery Program Childhood Cancer Cytoskeleton Consortium (OCF-C4)

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