Targeted Therapeutic Strategies and Molecular Insights in Medulloblastoma: A Case Report

Figols Ladrón1*, Lafuente Sánchez2

Abstract

Medulloblastoma is the most common malignant pediatric brain tumor, characterized by aggressive growth, early dissemination through the cerebrospinal fluid, and a complex molecular landscape. Recent advances in genomic and transcriptomic profiling have revealed distinct molecular subgroups—WNT, SHH, Group 3, and Group 4—that influence prognosis, treatment response, and potential for targeted interventions. This report presents a case of SHH-subgroup medulloblastoma treated with a combination of surgical resection, craniospinal irradiation, and SHH-targeted therapy, highlighting the role of precision medicine in guiding therapy. The integration of molecularly guided targeted interventions alongside standard-of-care treatment demonstrates potential for improved disease control, preservation of neurocognitive function, and reduced systemic toxicity. These findings emphasize the importance of early molecular characterization to enable individualized treatment strategies in pediatric medulloblastoma.

Keywords

Medulloblastoma; SHH pathway; Targeted therapy; Pediatric brain tumor; Precision medicine; Vismodegib; Molecular profiling.