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New neuroblastoma treatment combo helps more children with relapsed disease

Tim Gunn
by Tim Gunn | News

10 January 2024

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A Cancer Research UK-funded researcher in the lab.
© Jane Stockdale for Cancer Research UK


Researchers have found a more effective combination of drugs for treating children and young people whose neuroblastoma isn’t responding to standard chemotherapies, thanks to a trial we helped fund.

The findings, published this week in the Journal of Clinical Oncology, are an important step forward in treating neuroblastomas that come back or continue growing after initial drug treatments. Currently, these relapsed and refractory forms of the disease have some of the lowest survival rates for any childhood cancer.  

By adding bevacizumab (Avastin), a treatment that targets the way solid cancers get nutrients and oxygen from blood, to chemotherapy, doctors working on the trial were able to shrink more children’s and young people’s tumours. This can make it possible to give patients further, potentially life-saving, treatments. 

Doctors across the UK are already using the findings to improve how they care for children and young people with relapsed and refractory neuroblastomas. 

“Incremental improvements in treatment can make all the difference for young cancer patients,” said Dr Laura Danielson, our children’s and young people’s research lead. “It’s fantastic to see that the standard of care across the UK has already been updated based on these results, giving children with relapsed neuroblastoma more treatment options.” 

What is neuroblastoma?

Neuroblastoma is a rare type of cancer that begins in developing nerve cells. It mostly affects children under 5.  

Around 100 children under 14 are diagnosed with neuroblastoma every year in the UK. Very rarely it can develop in older children, teenagers and adults.

Neuroblastoma that does not go away with treatment is called refractory neuroblastoma. Relapsed (or recurrent) neuroblastoma is neuroblastoma that has come back after treatment.

A new option for refractory neuroblastoma

The BEACON-Neuroblastoma trial, an international collaboration led by the Cancer Research UK Clinical Trials Unit at the University of Birmingham, is the largest randomised trial ever conducted into relapsed and refractory neuroblastoma. It recruited 160 children and young people from across 11 European countries. 

Birmingham schoolboy Abdullah was one of them. 

Abdullah’s story

Bushra Mir had just qualified as a primary school teacher when her 4-year-old son Abdullah was diagnosed with neuroblastoma in November 2017.  

Doctors gave Abdullah two full courses of chemotherapy, but neither one could stop his tumour growing. After the second, they told Bushra and her husband Yasir Ali Mir about the BEACON trial, which was specifically set up to try and find better treatments for children and young people in Abdullah’s position.

“It was our last hope,” said Bushra.

A picture of Abdullah after surgery to remove his neuroblastoma.
Abdullah in early 2019. This picture was taken shortly after his surgery, which was made possible by the BEACON trial.

“We were desperate because he’d had two lots of chemotherapy that hadn’t shrunk his tumour at all. There were no other options so we signed up thinking that, even if it didn’t benefit Abdullah, it might help someone else.”  

Abdullah was randomised to the group receiving a combination of chemotherapy and bevacizumab, which was designed to stop tumours from creating blood vessels, limiting their ability to bring in the food and oxygen they need to survive and grow. That’s exactly what it did. Within months, the treatment shrunk Abdullah’s neuroblastoma by more than half, meaning he could have a life-saving stem cell transplant. 

Abdullah today.
Abdullah in 2024, 6 years on from his diagnosis.

By January 2019 he was fit enough for surgery to remove what remained of the tumour. He then had 10 weeks of radiotherapy.

Now 10, Abdullah spends his days like his mum: in school, rather than hospital.    

“Abdullah has always been very strong and positive, but he does remember treatment and it was a very difficult time,” said Bushra. “Due to COVID, year 3 was his first full year at school but he’s doing really well. He’s crazy about football and he loves school.”

 

Overall, BEACON-Neuroblastoma showed adding bevacizumab to chemotherapy can shrink more relapsed and refractory neuroblastomas. The proportion of children and young people who responded to treatment increased from 18 in every 100 who received chemotherapy alone to 26 in every 100 given the new drug combination. 

“These are very exciting results that hopefully get us closer to finding treatments for children who develop neuroblastomas,” said Simon Gates, Professor of Biostatistics and Clinical Trials at the University of Birmingham and senior lead author of the BEACON-Neuroblastoma paper. “Currently, the outcomes are really poor for children who get this horrible cancer and so even seemingly small increases in the chance that a patient is going to be able to shrink their tumours is significant.” 

Following BEACON

Drugs that interfere with a tumour’s blood supply were originally created to treat cancer in adults. Thanks to BEACON-Neuroblastoma, we now have evidence that they can be effective for treating childhood cancer too.  

The way the trial was designed, with multiple arms testing different combinations of drugs, also showed a potentially helpful interaction between bevacizumab and a chemotherapy drug called irinotecan. Although adding irinotecan doesn’t make a difference to response rate on its own, it seems to work with bevacizumab to stop neuroblastomas growing or returning for longer. The researchers plan to investigate the biology behind this and work out the optimal way of using the new drug combination in future studies. 

Moreno L, Weston R, Owens C, Valteau-Couanet D, Gambart M, Castel V, et al. Bevacizumab, irinotecan, or topotecan added to temozolomide for children with relapsed and refractory neuroblastoma: Results of the ITCC-SIOPEN BEACON-neuroblastoma trial. J Clin Oncol. 2024; https://doi.org/10.1200/jco.23.00458 

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