Medulloblastoma is the most common malignant pediatric brain tumor and is associated with significant morbidity and mortality in the pediatric population. Despite the use of multiple therapeutic approaches consisting of surgical resection, craniospinal irradiation, and multiagent chemotherapy, the prognosis of many patients with medulloblastoma remains dismal. Additionally, the high doses of radiation and the chemotherapeutic agents used are associated with significant short- and long-term complications and adverse effects, most notably neurocognitive delay. Hence, there is an urgent need for the development and clinical integration of targeted treatment regimens with greater efficacy and superior safety profiles. Since the adoption of the molecular-based classification of medulloblastoma into wingless (WNT) activated, sonic hedgehog (SHH) activated, group 3, and group 4, research efforts have been directed towards unraveling the genetic, epigenetic, transcriptomic, and proteomic profiles of each subtype. This review aims to delineate the progress that has been made in characterizing the neurodevelopmental and molecular features of each medulloblastoma subtype. It further delves into the implications that these characteristics have on the development of subgroup-specific targeted therapeutic agents. Furthermore, it highlights potential future avenues for combining multiple agents or strategies in order to obtain augmented effects and evade the development of treatment resistance in tumors.

In recent times, a greater emphasis has been placed on framing patient clinical assessments and QOL as both endpoints and prognostic factors for neuro-oncological care. However, there is currently a lack of consensus on the most effective metrics and instruments for use in patients with primary brain tumors. Due to the heterogeneity in clinical characteristics, histological classification, anatomical location, and available treatment modalities for glioma, quantifying patient clinical condition and QOL within this unique population is particularly challenging. In this paper, we comprehensively review the available literature on the use of clinical assessment tools in glioma patients, highlighting the strengths and limitations of prominent instruments. We demonstrate that clinical outcome assessment (COA) instruments provide valuable and complementary insights into the physical, psychological, and spiritual well-being of glioma patients.