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In a breakthrough study, researchers have found that a drug currently used to manage excess copper in the body could significantly improve the survival rate for children with high-risk neuroblastoma, a rare and aggressive childhood cancer. The drug, known as TETA (triethylenetetramine), could potentially boost the effectiveness of immunotherapy, raising the survival rate from just 10% to 50% for children with this deadly disease.
Neuroblastoma, which accounts for 15% of childhood cancer deaths, is especially difficult to treat, with children diagnosed with high-risk cases facing only a 1 in 2 chance of survival. The situation is even more dire for those who relapse, with survival dropping to as low as 1 in 10. For these children, one of the few remaining treatment options is Anti-GD2 antibody therapy, a promising immunotherapy that relies on a strong immune system to work effectively.
Lead researcher Associate Professor Orazio Vittorio, from UNSW Sydney’s School of Biomedical Sciences and the Children’s Cancer Institute, explained that the drug TETA works by transferring copper from neuroblastoma tumors to immune cells called neutrophils. These white blood cells help the body fight infections and heal injuries, and by “empowering” them, TETA weakens the tumors and strengthens the immune system.
“It’s like Robin Hood stealing copper from the tumor and giving it back to the immune cells to make them stronger,” said A/Prof. Vittorio. “When we combine TETA with existing immunotherapy for neuroblastoma, we can increase the survival rate for high-risk cases from 10% to 50%.”
TETA is currently used to treat Wilson’s disease, a genetic disorder that causes copper buildup in the body. In this new context, it’s shown promise in animal models, offering an exciting potential for a novel cancer treatment. However, A/Prof. Vittorio cautioned against increasing copper intake through diet or supplements, as this could actually make tumors stronger without the drug’s action to redirect copper.
The research team, which includes scientists from institutions like The University of Western Australia, Curtin University, and the Sydney Children’s Hospital Network, published their findings in Nature Communications. They plan to begin a multi-year clinical trial next year, with the hope of eventually incorporating this copper-chelation therapy into standard treatment regimens for neuroblastoma.
Dr. Jourdin Rouaen, the lead study author from UNSW’s School of Clinical Medicine and the Children’s Cancer Institute, expressed optimism about the future impact of this treatment. “Copper chelation represents a significant step forward from traditional cancer treatments,” said Dr. Rouaen. “It’s non-toxic, has no concerning side effects, and is already approved for use. If successful, this therapy could drastically improve survival rates and the quality of life for children with neuroblastoma.”
The study also highlighted the crucial role of neutrophils in immune function, suggesting that these cells could be a key target for future immunotherapies. By repurposing an existing drug like TETA, the researchers are saving both time and money compared to developing a new drug from scratch—a process that typically takes over a decade and costs billions of dollars.
“The ability to repurpose an existing drug is especially important when trying to improve treatment for a deadly childhood cancer,” Dr. Rouaen said. “We hope that this therapy could soon become a routine part of neuroblastoma immunotherapy.”
As the research moves into clinical trials, families of children with neuroblastoma are hopeful that this innovative treatment could offer a life-saving solution.
