Little toddler boy wearing IV tube looking out window in hospital. Little toddler boy wearing IV tube looking out window in hospital.

Why copper could be the key to treating a rare childhood cancer

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Kate Burke
Kate Burke,

A drug used to manage copper levels could improve the odds for children battling neuroblastoma, new research in animal models suggests.

The survival rate for children with a rare but deadly cancer could one day be improved by adding an existing drug – which is currently used to manage excess copper in the body – to their treatment.

The drug can be used to weaken tumours and strengthen the fighting capacity of immune cells, increasing the success rate of a cancer treatment for high-risk neuroblastoma from 10% to 50%, new research in mice led by UNSW Sydney shows. 

The findings, published in Nature Communications on Thursday night, offer hope for those with neuroblastoma, which accounts for 15% of childhood cancer deaths. 

Neuroblastoma is a cancer that develops from immature nerve cells, known as neuroblasts, found in several areas of the body, most commonly around the adrenal glands. It is most common in infants and children under five years of age.

Despite aggressive treatments, children diagnosed with high-risk neuroblastoma have a one-in-two chance of surviving the disease. This drops to one-in-10 for children who relapse.

For those children, their last chance was anti-GD2 antibody therapy, said lead researcher and senior report author Associate Professor Orazio Vittorio, from UNSW’s School of Biomedical Sciences and the Children’s Cancer Institute.

“It is one of the most important therapies developed, but it only works if there is a strong immune system,” A/Prof. Vittorio said. 

That’s where the drug TETA (triethylenetetramine), marketed as Cuprior, comes in.

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For enquiries about this story and interview requests, please contact Kate Burke, News & Content Coordinator, UNSW Medicine & Health.

Tel: +61 2 9348 2538
Email: kate.burke@unsw.edu.au


Children who relapse with high-risk neuroblastoma have a one-in-10 chance of surviving, but it's hoped this new therapy could change that. Adobe

'Like Robin Hood’: Helping the immune system fight back

TETA is copper chelating agent which is currently used to treat Wilson’s disease – a genetic disorder resulting in excess copper build up. The drug binds to and removes the metal from the body. 

Researchers previously discovered they could use TETA to weaken neuroblastoma tumours, by removing the copper which they ‘feed’ on. Now they have shown in mice that they can transfer this copper to neutrophils, a type of white blood cell that helps the body fight infection and heal injuries.

“It has the double effect of weakening the tumour and empowering the immune system to be strong and fight back,” said A/Prof. Vittorio. 

“When we combine it with the immunotherapy currently used for neuroblastoma, we can increase the survival rate for high risk cases from 10% to 50%.”

A/Prof. Vittorio compares the drug’s effect to the legend of Robin Hood.

“The tumour is like this greedy rich guy who basically takes the money, or in this case copper, from the immune cells, making them very weak. Then the drug, our Robin Hood, comes in, steals the copper and gives it back to the immune cells, so they can become stronger and fight back.

“It re-establishes that equilibrium that was compromised by the presence of the tumour,” he added.

A/Prof. Vittorio stressed that increasing copper intake via dietary changes or supplements would not have the same effect. 

“Never ever increase the level of copper you are taking if you have a tumour, because without this drug, all the extra copper available will reach the tumour and make it stronger.”

The researchers, from a dozen institutions including Children’s Cancers Institute, The University of Western Australia, Curtin University and the Sydney Children’s Hospital Network, plan to start a multi-year clinical trial next year. 

More research key to evolving cancer treatment

Lead study author Dr Jourdin Rouaen, from UNSW’s School of Clinical Medicine and the Children’s Cancer Institute, hopes the combined therapy could one day improve the survival rate and quality of life for children with neuroblastoma.  

"Copper chelation therapy represents a significant evolution from traditional cancer treatments. It’s non-toxic, has demonstrated no concerning side effects, and is already approved for use,” Dr Rouaen said.

The therapy also has the potential to work for other tumours, with the team working with Australian and US experts to study the impact of copper chelation on mesothelioma, caused by exposure to asbestos. They are also collaborating with US researchers to support a clinical trial testing the therapy for breast cancer patients. 

Dr Rouaen said the findings to date shone a light on the important role that could be played by often overlooked neutrophils.

“They are drastically understudied. We're challenging the paradigm of how useful these neutrophils can be, and we want to see more research into how these can be harnessed for immunotherapies in the future.” 

The benefit of repurposing existing drugs

It is hoped that the drug, if found effective in clinical trials, could become a routine part of immunotherapy treatment for neuroblastoma in the next few years.  

“It typically takes between eight to 13 years of research to develop a new drug for cancer treatment and costs an average of $US4.7 billion,” said Dr Rouaen. 

“By repurposing an existing drug we’re saving a great deal of money and time, which is particularly crucial when we’re looking to improve treatment for this deadly childhood cancer.”  

It’s news that will offer hope to those affected by neuroblastoma, even if the treatment could still be some years away. 

The multi-year study was funded by a Cure Cancer Australia grant and National Health and Medical Research Council Ideas Grant. 

“We are proud to have supported this pioneering research from its earliest stages,” said Cure Cancer Australia Chief Executive Officer Nikki Kinloch.

“This breakthrough demonstrates the incredible potential of supporting emerging scientists who bring fresh perspectives and innovative approaches to cancer research."