Children with neuroblastoma that does not respond to initial treatment or returns after therapy may benefit from adding antibody treatment to standard chemotherapy, according to new results from the BEACON phase 2 clinical trial. The study was coordinated by an international group of researchers led by the University of Birmingham's Cancer Research UK Clinical Trials Unit and published in the Journal of Clinical Oncology.
The trial focused on children with high-risk neuroblastoma, a rare cancer mainly affecting those under five years old. Researchers tested the addition of a monoclonal antibody called dinutuximab beta (dB) to standard chemotherapy regimens. Results showed that after six cycles, tumors shrank more frequently in patients who received dB alongside chemotherapy compared to those who had chemotherapy alone.
The best objective response rate, which measures how many patients saw their cancer partially or completely disappear, was 18.2% for those receiving only standard treatment. When dB was added, this rate increased to 30.2%. Children in the experimental group also experienced an average of 11 months without disease progression and an overall survival time close to 26 months. In contrast, patients receiving only standard care had about four months progression-free and survived around 17 months on average.
“These are really encouraging results, which will contribute towards developing better treatments for children with neuroblastoma. We are continuing to investigate combining dinutuximab beta with chemotherapy, called chemo-immunotherapy, in the BEACON-2 trial. This trial is now open in many UK centres, and aims to improve the chemo-immunotherapy, so that more children can benefit,” said Professor Juliet Gray from the University of Southampton and University Hospital Southampton.
Professor Amos Burke, Director of the Cancer Research UK Clinical Trials Unit at the University of Birmingham stated: “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. These results are very important and could improve the odds and lived experience for these children. Here at the University of Birmingham we remain focused on trialling tomorrow's treatments for children who need them most.”
Each year in the UK about 100 children between birth and age 14 are diagnosed with neuroblastoma. The disease often begins in immature nerve cells in a child’s abdomen but can spread to other organs such as bones, skin, and liver.
The BEACON trial included 65 patients averaging four years old; 28 had refractory neuroblastoma while 37 had relapsed cases. Alongside tumor response rates and survival times, researchers monitored side effects such as neurotoxicity. About one-third of patients treated with dB experienced mild symptoms like drowsiness versus nine percent in those receiving standard therapy alone. Severe symptoms were rare—occurring in just over two percent of dB-treated patients compared to about four percent among controls.
Previous research from the BEACON consortium found that adding bevacizumab—a different anti-tumor drug—to chemotherapy also led to tumor shrinkage and influenced current UK pediatric oncology practices regarding neuroblastoma treatment strategies. The ongoing BEACON-2 trial is now examining combinations involving both bevacizumab and dinutuximab beta chemoimmunotherapy.
