Cancer Gene Therapy

Group leaders: Dr Geoff McCowage and Dr Belinda Kramer

Our links with children being treated for cancer in our hospitals ensure our research is based on real life questions that come directly from the cancer ward. Doctors in the hospital’s clinical oncology service have reported significant problems with toxic side effects of chemotherapy on the bone marrow of children with brain tumours. The Cancer Gene Therapy Project uses gene therapy to protect the bone marrow of these children.

The project, initiated by Dr Geoffrey McCowage in the Oncology Unit, uses a DNA repair protein, Methyl-Guanine-Methyl Transferase (MGMT), to provide protection of the bone marrow. The project brings together staff of the Children’s Cancer Research Unit’s Cancer Gene Therapy Group, the Oncology Unit and the Gene Therapy Research Unit, under the umbrella of Sydney Cell and Gene Therapy on the Westmead Campus.

The Phase I MGMT Cancer Gene Therapy trial opened in June 2012 and is registered on the Australian and New Zealand Clinical Trail Registry.

Specialised infrastructure in the form of state-of-art cleanrooms, and well-established quality systems underpinning their operations, allow for the manipulation of patient cells using gene transfer protocols for this trial.

Aside from our clinical trial work, research activities are currently focused on the development of new vectors for gene transfer targeting haematopoietic stem cells. This work includes scale up of vector manufacture to clinical batch size, as well as function, safety and stability testing of proposed vectors for future clinical trials use.