Associate Professor David Ziegler and his team, including the National Coordination role funded by the Children’s Cancer Foundation, are identifying new treatments for patients who had previously run out of options. Our latest PRISM update from AP Ziegler highlights some exciting results.
“The very nature of new, cutting-edge research means you’re never quite sure what the end result will be. Which is why, although every time we start a new clinical trial, we always hope it’s going to have a positive outcome, this isn’t always the case. Trials that don’t produce the outcome we’d like, or expect, are vital in determining next steps. They provide critical information and learnings which help us to move forwards; they help other researchers decide what future paths to take.”
“PRISM, in particular, is a trial which has really exceeded all our expectations and hopes.”
“The key success of the trial is that it identifies new treatments for patients who had previously run out of options. We’re consistently finding new treatment strategies in approximately 70% of patients – which is currently one of the highest success rates in the world. Importantly, we’re also finding that many patients are responding well when started on their recommended treatment program.
“For example, one child taking part in the trial had a tumour in their brain stem. The position of the tumour was particularly difficult and when surgeons tried to operate, even just touching it with forceps led to severe effects on the child’s blood pressure forcing the team to stop.
“The patient was referred to the PRISM trial and we quickly discovered a mutation in the brain cancer that was highly targetable.”
“We started treating the patient with a combination of drugs that specifically targeted this genetic change and we’ve seen that firstly, the child suffered absolutely no side effects and secondly, the tumour started to melt away without the devastating effects of surgery.
“The patient is continuing to take the prescribed medication and comes in every couple of months for a check-up. Other than that, you’d never know they had a brain tumour, which really is quite incredible.
“The National Co-ordination role funded by the Foundation is key to these exciting results because it’s meant we can track patients’ progress at home after treatment.
“We’ve recently enrolled our 300th patient and without the resources provided by the Foundation, we wouldn’t have been able to follow the progress of each child from across Australia.
“Project funding for projects like PRISM is crucial to achieving the pioneering medical advancements we know are within reach. The Foundation’s support for both the PRISM and PaBlo clinical trials has enabled the team here at the Kids Cancer Centre to provide hope to children with cancer when previously there was none.”
“Our plans for the future are to expand on the types of tumours these targeted treatments are used on. We’re considering if this genetic testing should be used for all cancers, not just the most aggressive ones. One day, we hope this kind of targeted testing could become the standard for all childhood cancer patients.
Photo credit: Sydney Children’s Hospitals Foundation