New neuroblastoma treatment combo helps more children with relapsed disease

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.” 

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. 

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.