Abstract
Embryonal tumors with multilayered rosettes (ETMR) are relatively rare but highly aggressive intracranial tumors that mainly occur in children under four years of age. Despite high-intensity and multi-modal treatment, the five-year overall survival rate of patients with ETMR remains <30%. Therefore, it is necessary to improve understanding of the molecular biological changes in ETMR. The present review presents an overview of the recent molecular and biological characteristics of ETMR in children, the current recommended treatments, and research into potential targeted strategies based on these findings. ETMR are molecularly characterized by distinct DNA methylation signatures and dysregulated expression of oncogenic miRNAs. Despite increased knowledge of the novel molecular characteristics of ETMR in children, treatment outcomes have only marginally improved. Thus, there is an urgent need to translate these new insights in ETMR biology into more effective treatment.