Advancing Targeted Therapy for Childhood Brain Cancer
New research on childhood brain cancers has identified a target for treatment, offering hope for patients.
They believe that these findings are a significant step toward developing new drugs to treat some of the most difficult-to-treat pediatric cancers.
Lead researcher Dr. Shazia Adjumain stated that childhood gliomas have distinct features that set them apart from adult gliomas, leading to different treatment strategies.
She emphasized the urgent need for more effective and less toxic treatments compared to conventional therapies.
“We have demonstrated that blocking MCL1 function with targeted drugs can have significant anti-tumor effects,” Dr. Adjumain explained.
“We have also identified a unique DNA modification in the BCL2L1 gene that can predict how a tumor will respond to MCL1-targeting treatments, providing a method for identifying patients who would benefit most from these therapies.”
“This research offers valuable insights into developing personalized treatments for childhood brain cancers,” she added.
A Step Forward in Combating Childhood Cancer
The study’s supervisor, Professor Ron Firestein, further underscored the importance of the research.
“Despite notable improvements in survival rates over the past 50 years, cancer remains the leading cause of disease-related death among Australian children,” he noted.
Currently, only 12 cancer drugs have been approved for children in the last four decades, compared to 500 for adults, highlighting the necessity for developing treatments specifically for children.
“Our discovery of the unique BCL2L1 methylation mark as a biomarker allows for a precision medicine approach, enabling the identification of patients most likely to respond to MCL1-targeted drugs,” she stated.
“This study bridges the gap between basic research and clinical application, setting the stage for trials of MCL1-targeted drugs in pediatric cancers and potentially improving survival rates for children with these devastating malignancies.”
