This trial on neuroblastomas was co-funded by the Enfants sans Cancer 2021 race and was selected as part of our European Fight Kids Cancer grant call.


About Neuroblastomas

A neuroblastoma is a malignant extracerebral tumor that develops from primitive nerve cells called neuroblasts, which normally form the body’s nerve cells. This type of solid tumor is one of the most common in children.

This disease accounts for 10% of solid tumors in children under 15 years old and primarily affects young children, as it is usually diagnosed before the age of five (90% of cases). More than half of the children present with metastatic disease at the time of diagnosis. The exact causes of neuroblastoma are unknown, but it seems that genetic and environmental factors play a role in its onset.

Learn more about this disease.


About Beacon Bio

Despite intensive multimodal therapy, current treatments for high-risk neuroblastomas remain disappointing. Indeed, more than half of the patients relapse. Since the beginning, Imagine for Margo has strongly supported the BEACON program, then BEACON Immuno, to enable these patients to benefit from effective therapeutic combinations. In these two trials, bevacizumab (BEACON) and dinutuximab beta (BEACON IMMUNO) were respectively combined with chemotherapy, with the optimal combination determined in this project.

Although these programs have proven more effective than anything previously available, the results remain limited. For patients who received dinutuximab beta in addition, the response rate to the drug increased from 18.2% to 34.8% (a higher rate with the BEACON trial). However, long-term survival does not seem improved, with a median survival of about three years.

It is therefore necessary to develop more effective combination therapies and to have a deeper understanding of the biology of these tumors and the genetic factors involved.

Objectives of the Project:

  • Identify new potential molecular targets that could lead to new combinations targeting multiple cancer characteristics and addressing its complexity.
  • Analyze and compare relapsing and refractory neuroblastomas to facilitate therapeutic decisions in future clinical trials.
  • Evaluate the prognostic impact of genomic aberrations in RRNB. For example, the identification of ALK, ATRX, or RAS/RAF pathway aberrations has led to the launch of specific clinical trials for these subpopulations. The data generated in this project will help understand resistance and facilitate the development of more powerful combination strategies.

Thus, the research teams are analyzing the genetic and epigenetic profiles of more than 1,000 samples (tumor and blood extracts) collected throughout the BEACON project. Currently, all analysis centers have been recruited, and genomic analyses are underway.

This project involves international collaborations and is expected to have a major impact on the upcoming multi-arm and multi-stage European trial on relapsing and refractory neuroblastoma currently planned by SIOPEN.

Project Summary

  • Promoter: Vall d’Hebron Institute of Research, Spain
  • Principal Investigator: Dr. Lucas Moreno
  • Program Duration: September 2022 – January 2025
  • Funding: Fight Kids Cancer: €499,776, including funding from Imagine for Margo: €300,890
  • Countries Involved: United Kingdom, France, Spain
  • Duration: 2 years