Medulloblastoma: From TP53 Mutations to Molecular Classification and Liquid Biopsy

SIMPLE SUMMARY: Medulloblastoma is a common malignant brain tumor in children. Recent progress includes a paradigm shift from histology-based diagnostics to the molecular and genetic profiling of the tumor for an improved correlation with clinical outcomes. This review aims to introduce the clinical physician as well as the basic scientist to a century of research in this field, including the importance of the cell of origin, animal models, and liquid biopsy. ABSTRACT: A recent paradigm shift in the diagnostics of medulloblastoma allowed the distinction of four major groups defined by genetic data rather than histology. This new molecular classification correlates better with prognosis and will allow for the better clinical management of therapies targeting druggable mutations, but also offer a new combination of monitoring tumor development in real-time and treatment response by sequential liquid biopsy. This review highlights recent developments after a century of milestones in neurosurgery and radio- and chemotherapy, but also controversial theories on the cell of origin, animal models, and the use of liquid biopsy.