Adding antibody treatment to chemo boosts outcomes for children with rare cancer: Study

Children with a rare form of cancer called neuroblastoma which hasn’t responded to initial treatment or that has relapsed may benefit from adding antibody treatment to usual chemotherapy, according to new results from a clinical trial.

The results of the BEACON phase 2 trial carried out by an international consortium of researchers, coordinated by the University of Birmingham’s Cancer Research UK Clinical Trials Unit. Published in the Journal of Clinical Oncology found that among children who have a high risk form cancer called neuroblastoma, using a monoclonal antibody treatment called dinutuximab beta (dB) alongside typical treatment led to patient’s tumours shrinking after six cycles of treatment.

Children who received dB on top of the usual chemotherapy had significantly improved best objective response rate (ORR), which is a measure that represents the percentage of patients who experience a complete disappearance or partial reduction in cancer cells.

While usual treatment had a rate of 18.2% of patients whose cancer reduced or disappeared, adding dB as well saw a rate improve to 30.2%. In addition, patients had an average time without the cancer progressing of 11 months, and an overall survival time of almost 26 months in the experimental arm, compared to around four (4) months progression-free and 17 months overall survival for usual treatment.

Professor Juliet Gray from the University of Southampton and University Hospital Southampton, and corresponding author of the study said: “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.”

Professor Amos Burke, Director of the Cancer Research UK Clinical Trials Unit at the University of Birmingham said: “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.”

Primarily affects children under five

Neuroblastoma primarily affects children under the age of 5 years and around 100 children between the ages of 0 and 14 years are diagnosed with neuroblastoma each year in the UK, according to Cancer Research UK who co-funded the study along with Imagine for Margo, Solving Kids Cancer UK, and Zoe4life, operated under the Innovative Therapies for Children with Cancer (ITCC) collaborative research group.

This cancer begins in immature nerve cells usually in the abdomen of young children and develops into small tumours that can be experienced as a lump in a child’s abdomen. Neuroblastoma spreads to other parts of the body in around half of children, including the bones, skin and liver.

The BEACON trial recruited 65 patients with an average age of four (4) years old, with 28 patients having refractory (not responding to first line treatment) and 37 patients having relapsing (where the cancer returns) neuroblastoma.

As well as measuring best objective response and survival times, the BEACON consortium also measured neurotoxicity of the treatment arms. For patients in the dB arm, around a third experienced lower grade symptoms such as drowsiness from the treatment compared to 9% in the active arm. There were low levels of more severe grade 3 symptoms in the dB arm (2.3%) and comparable to usual treatment (4.5%).

Previously reported results from the global BEACON consortium found that adding an anti-tumour drug bevacizumab with chemotherapy drugs led to patient’s tumours shrinking. The findings led to changes in how UK paediatric oncologists treat neuroblastoma, and the further trial BEACON-2 is now investigating the difference between a combination of drugs including bevacizumab and dB chemoimmunotherapy.

Reference:

Dinutuximab beta added to temozolomide-based chemotherapy for children with relapsed and refractory neuroblastoma. Results of the ITCC-SIOPEN BEACON Immuno Phase II trial, Journal of Clinical Oncology (2025).