GivenGain

III. Neuroblastoma Project

Neuroblastoma (NB) is the most common extracranial solid tumor in children, accounting for 7 to 8% of all childhood malignancies and 15% of all cancer-related deaths in this population. It is the most frequently diagnosed cancer during infancy, with a median age at diagnosis of approximately 19 months. While 90% of patients are under 5 years old, NB is very rare after the age of 10. Metastatic disease is present in approximately 50% of cases.

Children with very low-risk NB according to current classifications have an expected long-term survival rate of 99 to 100%, whereas patients in the high-risk subgroup have a long-term survival rate of less than 50%. This is due to the spread of the disease to various organs (metastases), which can lead to a fatal outcome despite intensive multimodal therapy, including surgery, high-dose chemotherapy with autologous bone marrow transplant, radiation therapy, and immunotherapy. New therapies for the high-risk subgroup are therefore urgently needed. One of the hallmarks of this aggressive form is the amplification of a gene called MYCN. This gene acts like an accelerator, driving cancer cells to multiply uncontrollably. Because these cells are capable of adapting and resisting treatment, it is highly unlikely that a single drug will be enough to eradicate the disease.

The goal of our research is to better understand the inner workings of these high-risk neuroblastoma cells to discover what makes them so aggressive. By understanding how their various mechanisms communicate with one another, we will be able to propose new treatments or improve current protocols to provide more hope for children and their families.