Recent studies have made significant strides in understanding and treating pediatric brain tumors, particularly high-grade gliomas and medulloblastomas. A phase 1 trial evaluated a peptide vaccine targeting the cytomegalovirus (CMV) antigen pp65 in children and young adults with recurrent high-grade glioma and medulloblastoma, demonstrating safety and immunogenicity in a cohort of 36 individuals (ref: Thompson doi.org/10.1038/s43018-025-00998-z/). Another pivotal study from the Children's Oncology Group reported that high-dose methotrexate improved complete response rates in young children with high-risk embryonal brain tumors, with a notable 63% complete response rate compared to 30% without methotrexate (ref: Mazewski doi.org/10.1093/neuonc/). Furthermore, the addition of nimotuzumab to chemoradiation therapy for newly diagnosed pediatric diffuse intrinsic pontine glioma (DIPG) showed promising outcomes, although it did not reach statistical significance compared to historical data (ref: Liu doi.org/10.1016/j.ijrobp.2025.05.063/). These findings underscore the ongoing exploration of novel therapeutic strategies to enhance treatment efficacy and patient survival in pediatric brain tumors. In addition to treatment advancements, studies have also focused on the epidemiological burden of central nervous system tumors. A comprehensive analysis revealed that malignant CNS tumors accounted for a staggering 364,223 years of life lost in the United States, with glioblastoma being the leading contributor (ref: Gerstl doi.org/10.1093/neuonc/). Moreover, research into the metastatic potential of medulloblastomas has led to innovative approaches, such as using targeting peptide-engineered extracellular vesicles to deliver LOXL1-AS1-siRNAs, aiming to suppress metastasis while minimizing neurocognitive deficits associated with traditional treatments (ref: Do doi.org/10.1186/s12951-025-03554-0/). Collectively, these studies highlight the multifaceted approach required to tackle pediatric brain tumors, from novel immunotherapies to understanding the broader implications of tumor burden on public health.