The ‘Gene Therapy Trial’ is a world-first and offers hope to children with brain tumours.
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 without damaging vital functions like breathing, swallowing and movement.
Unfortunately, the level of chemotherapy needed to treat a child's brain tumour can also cause significant side effects in the bone marrow and immune system.
The trial, funded by The Kids’ Cancer Project and conducted by Dr Geoff McCowage and Dr Belinda Kramer at The Children’s Hospital at Westmead, is a Phase I trial: it tests the safety and feasibility of altering a child's bone marrow to make it resistant to chemotherapy.
This unique strategy has the potential to lead to a more effective treatment as it could allow higher doses of chemotherapy and a greater impact on their tumour without the harmful side effects on the bone marrow.
“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” says Dr Geoff McCowage.