A team of Australian researchers conducted lab studies testing a novel method for treating childhood brain cancer. By combining two medicines, the researchers discovered that the dual treatment approach might be more effective than using each medicine individually. The study, detailed in the journal Science Translational Medicine, focused on treating difficult-to-treat brain tumors known as diffuse midline gliomas (DMG).
The team, comprising experts from the Children’s Cancer Institute and University of New South Wales, specifically targeted diffuse intrinsic pontine glioma (DIPG), a rare and often fatal childhood brain cancer subtype. Typically, children diagnosed with DIPG have a survival rate of around 12 months. Conjoint Associate Professor Maria Tsoli from UNSW emphasized the challenge of eradicating aggressive brain cancers with a single drug treatment, prompting the exploration of combined therapies for improved efficacy.
UNSW Conjoint Professor David Ziegler highlighted the complexity of these tumors, where numerous genes are simultaneously activated, fueling cancer progression. The researchers’ investigation aimed to identify a treatment strategy that could effectively silence the abnormal gene activity driving DMG cell growth. By targeting two key proteins, FACT and BET, associated with transcription in cancer cells, the study uncovered a promising drug combination capable of simultaneously deactivating thousands of genes.
The study’s findings revealed that while existing drugs targeting FACT and BET individually only slowed cancer growth marginally, their combined use resulted in the death of cancer cells in laboratory settings. Furthermore, experiments on mice demonstrated a reduction in tumor growth rate and prolonged survival. Additionally, the treatment triggered immune system-related signals, potentially enhancing the immune system’s ability to recognize and attack cancer cells. The researchers suggested that incorporating immune-based therapies like CAR T-cell therapy could further enhance treatment outcomes.
The team indicated that both types of drugs are currently undergoing clinical trials for patient use, indicating promising prospects for future treatment approaches.
