Neuroblastoma: Chemotherapy & Immunotherapy Improve Outcomes in Clinical Trial

A new international clinical trial offers hope for children and adolescents battling neuroblastoma, an aggressive pediatric cancer. Researchers have found that combining chemotherapy with a specific immunotherapy drug, dinutuximab beta, leads to improved treatment responses in patients with a poorer prognosis. The findings, published in the Journal of Clinical Oncology, represent a significant step forward in addressing a cancer where current treatments often fail or result in relapse.

A Challenging Cancer

Neuroblastoma affects approximately 90 children and adolescents each year in Spain, accounting for roughly 10% of all childhood cancers. A substantial number of these young patients do not respond well to existing therapies, leading to low survival rates and a critical need for innovative treatment strategies.

The BEACON Trial

The study, known as BEACON, is described as the largest international clinical trial to date evaluating this specific treatment combination in patients with neuroblastoma that is resistant to treatment or has returned after initial remission. Led by Dr. Lucas Moreno of Vall d’Hebron, in collaboration with researchers from the University of Birmingham, the trial involved 65 patients between the ages of one and 18 from 29 centers across seven European countries.

Did You Know? The BEACON project involved collaboration between 43 European hospitals, the European Neuroblastoma Group of the International Society of Paediatric Oncology (SIOPEN), and the Innovative Therapies for Children with Cancer (ITCC) consortium.

Patients were divided into two groups: 22 received chemotherapy alone, while 43 received chemotherapy combined with dinutuximab beta. The treatment plan involved six cycles for each patient, with 13 patients initially receiving only chemotherapy later being switched to the combination therapy when initial results were unfavorable.

Promising Results

The research team assessed treatment response by measuring tumor size reduction. The combination of immunotherapy and chemotherapy resulted in tumor reduction in 30.2% of cases, compared to 18.2% in patients treated with chemotherapy alone.

the study analysed progression-free survival – the time patients live without tumor growth or new lesions. Patients receiving both chemotherapy and immunotherapy experienced an average progression-free survival of 11.1 months, significantly longer than the 3.8 months observed in those receiving chemotherapy alone. One year after entering the study, 44% of patients on the combined therapy were still alive with stable disease, compared to 27% of those on chemotherapy alone.

Expert Insight: The observed improvements in both tumor reduction and progression-free survival suggest that combining immunotherapy with standard chemotherapy could represent a meaningful advancement in the treatment of neuroblastoma, particularly for patients facing a difficult prognosis.

The study also indicated that the combination therapy was generally well-tolerated, with side effects being mild, such as fever, allergic reactions, drowsiness, or dizziness.

Looking Ahead

Building on the BEACON trial, researchers have already launched BEACON2. This next phase will compare the benefits of chemotherapy with bevacizumab or dinutuximab beta, as well as the combination of both. Dr. Moreno notes that BEACON2 is designed as a platform to evaluate new drugs and combinations for neuroblastoma, with additional options potentially being added in the coming years.

In parallel, the BEACON-BIO study is underway, analysing patient samples to identify genomic biomarkers and assess liquid biopsies. This research aims to understand why patients respond differently to treatment and to facilitate the development of personalized medicine approaches.

Frequently Asked Questions

What is neuroblastoma?

Neuroblastoma is an aggressive type of pediatric cancer that affects approximately 90 children and adolescents each year in Spain and represents around 10% of all childhood cancers.

What did the BEACON trial involve?

The BEACON trial involved 65 patients with neuroblastoma in relapse or resistant to treatment, divided into two groups: one receiving chemotherapy alone and the other receiving chemotherapy combined with dinutuximab beta.

What were the key findings of the study?

The combination of chemotherapy and immunotherapy led to a higher rate of tumor reduction (30.2%) and longer progression-free survival (11.1 months) compared to chemotherapy alone (18.2% and 3.8 months, respectively).

What impact might these findings have on the future treatment of neuroblastoma?