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Daijiworld Media Network – New Delhi
New Delhi, Jan 17: Australian researchers have identified a potential new treatment strategy for aggressive childhood brain cancers by combining two existing medicines, a laboratory study has found.
The research, published in Science Translational Medicine, shows that using the two drugs together is significantly more effective than administering either treatment alone. The study was conducted by scientists from the Children’s Cancer Institute in collaboration with the University of New South Wales (UNSW).
The team focused on diffuse midline gliomas (DMG), a group of notoriously hard-to-treat brain tumours that includes diffuse intrinsic pontine glioma (DIPG), a rare and almost always fatal cancer in children. Most children diagnosed with DIPG survive for only around a year.
According to conjoint associate professor Maria Tsoli of UNSW, the failure of single-drug therapies to eliminate aggressive brain cancers prompted the researchers to explore a combination-based approach.
UNSW conjoint professor David Ziegler explained that one of the greatest challenges in treating DMG is the simultaneous activation of thousands of genes that fuel tumour growth, making it extremely difficult to shut the cancer down with a single targeted therapy.
The study found that abnormal gene regulation drives the uncontrolled growth of DMG cells. Researchers identified a combination of drugs that can effectively halt the transcription process—the mechanism by which genes are switched on—thereby silencing thousands of cancer-promoting genes at once.
The therapy targets two key transcription-related proteins, FACT and BET, which are present at unusually high levels in cancer cells. While drugs that inhibit these proteins already exist, they have shown only limited success when used individually. However, when applied together, the drugs caused cancer cells to die in laboratory experiments.
Further testing in mice demonstrated that the combined treatment slowed tumour progression and significantly extended survival.
In addition, the researchers observed that the therapy triggered immune-related signalling pathways, potentially making cancer cells more visible to the body’s immune defences. This finding suggests that pairing the drug combination with immunotherapies such as CAR T-cell treatment could further enhance its effectiveness.
The researchers noted that both classes of drugs involved in the study are already in development and undergoing clinical trials, raising hope that this combination approach could move more quickly toward testing in patients.
