New research into childhood brain cancer is identifying treatment targets and giving patients hope.
They believe these findings are key steps in developing new drugs to treat some of the most challenging and difficult-to-treat pediatric cancers.
Dr Shazia, the prime minister’s researcher, said childhood gliomas have distinct features that distinguish them from adult gliomas, and to distinguish between treatment strategies.
She further emphasized the urgent need to develop treatments that are more effective and less toxic than traditional treatments.
“It has been shown that blocking MCL1 function with targeted drugs induces a significant antitumor effect,” Dr. Adjumain said.
“We also provided a strategy to identify unique DNA modifications of the BCL2L1 gene that can predict tumor response to MCL1 targeting treatments and identify patients who will benefit most from these treatments.”
“This work provides important insight into the development of tailored treatments for pediatric brain cancer,” she said.
A step in the fight against childhood cancer
Professor Ron Finastein, the supervisor of the study, further emphasized the importance of the research.
“Even though there has been a significant improvement in overall survival rates over the past 50 years, cancer continues to be a major cause of disease-related death in Australian children,” he said.
Currently, there are only 12 cancer drugs approved for children in the past 40 years, compared to 500 adults. The disparity further emphasizes the need to develop treatment for children.
“The discovery of the unique BCL2L1 methylation mark as a biomarker enables a precision medical approach and allows for the identification of patients most likely to respond to MCL1 target drugs,” she said.
“This study bridges basic research and clinical applications, paving the way for testing MCL1-targeted drugs in pediatric cancer and may improve survival outcomes in children with these devastating malignant tumors.”