A global clinical trial suggests that combining an antibody medication with regular chemotherapy could help more children with difficult-to-treat neuroblastoma react to treatment and live longer lives. 

A new worldwide clinical trial provides tempered hope for children with a rare and severe cancer that has returned or failed to respond to initial treatment. According to the researchers, adding an antibody treatment to regular chemotherapy helped more children with high-risk neuroblastoma see their tumours shrink, which extended the time many lived without their cancer worsening. 

The findings are from the BEACON phase 2 trial, which was led by the University of Birmingham's Cancer Research UK Clinical Trials Unit and published in the Journal of Clinical Oncology. The trial looked at children with neuroblastoma that either didn't get better with the first treatment or came back after earlier treatment—cases where there aren't many other options and the outcomes are often poor. 

Neuroblastoma is a cancer that arises from immature nerve cells, typically in the abdomen, and primarily affects very young children. In around half of instances, the disease spreads to other regions of the body, including the bones, skin, and liver. According to Cancer Research UK, approximately 100 children aged 0 to 14 are diagnosed with the illness each year in the UK, with the majority being under the age of five. 

The BEACON experiment enrolled 65 children, with an average age of four. Of these, 28 had disease that had not responded to initial treatment, and 37 had recurrent malignancies. The researchers compared normal treatment to chemotherapy combined with a monoclonal antibody known as dinutuximab beta, or dB. 

Monoclonal antibodies are proteins synthesised in a laboratory that recognise and bind to specific cancer cell targets. Simply said, they help the immune system detect cancer cells more quickly, allowing them to be attacked, whereas chemotherapy kills fast-growing cells directly. Chemo-immunotherapy is a method that combines both to make treatment more effective than chemotherapy alone. 

The experiment found that children who received dinutuximab beta plus chemotherapy had a considerably higher best objective response rate, or ORR. This metric represents the percentage of patients whose tumours shrank or disappeared entirely. While 18.2% of children responded to normal treatment alone, the percentage increased to 30.2% when the antibody medication was added. 

There were also improvements in how long children survived without their malignancy worsening. The average period until illness development in the combination treatment group was 11 months, compared to around four months in the conventional therapy group. Overall survival in the experimental group was over 26 months, compared to approximately 17 months with usual treatment. 

"These are really encouraging results, which will contribute towards developing better treatments for children with neuroblastoma," said the study's corresponding author, Professor Juliet Grey of the University of Southampton and University Hospital Southampton. She said that researchers are currently investigating enhancements to this strategy in the BEACON-2 experiment, which is taking place at numerous UK sites. "This trial aims to improve the chemo-immunotherapy so that more children can benefit." 

Safety was also carefully maintained. Approximately one-third of children receiving dinutuximab beta showed milder neurological adverse effects, such as sleepiness, compared to 9% in the conventional treatment group. More severe neurological symptoms were infrequent and appeared at similar low rates in both groups. 

Professor Amos Burke, Director of the Cancer Research UK Clinical Trials Unit at the University of Birmingham, said the findings might have a significant impact on families with few options. "Neuroblastoma that comes back or doesn't respond to first-line treatment comes with poor outcomes currently for the children who sadly have this disease," he informed me. "These results are very important and could improve the odds and lived experience for these children." 

A previous study from the BEACON consortium had influenced UK practice by demonstrating the benefits of adding another targeted medication, bevacizumab, to chemotherapy. The current BEACON-2 study is now looking into whether combining bevacizumab with dinutuximab beta will enhance outcomes even further, paving the way for more effective, personalised treatments for young patients.