Two Steps Forward for Targeting Childhood Brain Cancer

New research on childhood brain cancers has identifies target for treatment, giving hope for patients.

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Researchers at the Hudson Institute of Medical Research have discovered a genetic target for childhood brain cancer and a method to identify patients most likely to benefit from it.

They believe that these findings are a major step toward developing new drugs to treat some of the most difficult-to-treat paediatric cancers.

The study, published in the Journal of Clinical Investigation Insight, identified MCL1, an anti-cell death gene involved in brain cancer cells’ survival.

It also found DNA modifications in a different gene, BCL2L1. This modification predicted a tumour’s response to treatments that targeted MCL1. These modifications are known as methylation, which acts to activate or suppress the activity of specific genes.

In 2024, 760 children aged 0-14 were diagnosed with cancer. Childhood brain cancer, specifically paediatric high-grade gliomas (pHGGs), is one of the most aggressive forms of brain cancer in children.

Gliomas are tumours that start in the brain or spinal cord and grow and disrupt the actions of the affected areas of the brain and spinal cord.

Lead researcher Dr. Shazia Adjumain said childhood gliomas have distinct features that distinguish them from adult gliomas, which in turn differentiates the treatment strategies.

She further stressed the urgent need to develop more effective and less toxic treatments than conventional therapies.

“We showed that blocking MCL1 function with targeted drugs induces significant anti-tumour effects,” Dr. Adjumain said.

“We also identified a unique DNA modification in the BCL2L1 gene that can predict a tumour’s response to MCL1-targeting treatments, offering a strategy to identify patients who would benefit most from these therapies.”

“This work provides important insights into developing tailored treatments for childhood brain cancers,” she said.

A Step Forward in Combatting Childhood Cancer

The study’s supervisor, Professor Ron Firestein, further stressed the importance of the research.

“Despite significant overall improvements in survival rates over the past 50 years, cancer remains the leading disease-related cause of death among Australian children,” he said.

Currently, there are only 12 cancer drugs approved for children in the last 40 years, compared to 500 for adults. The disparity further highlights the need to develop treatments for children.

“Our discovery of the unique BCL2L1 methylation mark as a biomarker enables a precision medicine approach, allowing for the identification of patients most likely to respond to MCL1-targeted drugs,” she said.

“This study bridges basic research and clinical application, paving the way for trials of MCL1-targeted drugs in paediatric cancers, potentially improving survival outcomes for children with these devastating malignancies.”