Dual Drug Therapy Shows Promise for Aggressive Childhood Brain Cancer | Quick Digest

Dual Drug Therapy Shows Promise for Aggressive Childhood Brain Cancer | Quick Digest
Australian researchers have identified a promising dual drug therapy for aggressive childhood brain cancers, including DIPG. Lab studies and mouse models showed the combination effectively killed cancer cells and slowed tumor growth. The drugs target proteins crucial for cancer cell transcription, offering new hope.

Australian scientists discovered a novel dual drug therapy for childhood brain cancer.

Research focuses on Diffuse Midline Gliomas (DMG), including fatal DIPG.

Combination targets FACT and BET proteins to halt cancer gene transcription.

Lab and mouse studies showed significant tumor cell death and growth reduction.

Treatment also enhanced immune system signals against cancer cells.

Both drugs are in clinical trials for other cancers; DIPG trials are planned.

A collaborative study by researchers at the Children's Cancer Institute and UNSW Sydney in Australia has unveiled a promising new dual drug therapy for aggressive childhood brain cancers, particularly Diffuse Midline Gliomas (DMG), which include the notoriously fatal Diffuse Intrinsic Pontine Glioma (DIPG). Published in *Science Translational Medicine*, the research indicates that combining two specific medicines may be significantly more effective than single-drug approaches for these difficult-to-treat tumors. Conjoint Professor David Ziegler and Conjoint Associate Professor Maria Tsoli led the study, focusing on epigenetic therapies that target two critical proteins, FACT and BET, which are found at high levels in cancer cells. These proteins play a crucial role in the transcription process, essentially switching on thousands of genes that drive cancer growth. The researchers found that using drugs to block both proteins simultaneously successfully shut down this transcription process. In laboratory experiments, the combination therapy effectively killed cancer cells. Further testing in mouse models demonstrated that the dual treatment significantly slowed tumor growth and extended the animals' survival. A key finding was also that the treatment activated signals linked to the immune system, suggesting it could potentially make cancer cells more susceptible to recognition and attack by the body's own defenses, possibly enhancing the effectiveness of future immune-based therapies like CAR T-cell therapy. While the findings are still at the pre-clinical stage, conducted in labs and on animal models, both types of drugs involved are already in development and undergoing clinical trials for other forms of cancer. This expedites the potential path to clinical trials for children with DMG and DIPG, offering a beacon of hope for a disease where children typically survive only about 12 months after diagnosis due to a lack of effective treatments.
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