Pathogenesis of Craniopharyngiomas and Targeted Approaches to Therapy

Craniopharyngiomas (CPs) are rare brain tumors, categorized into 2 distinct types: adamantinomatous craniopharyngiomas (ACs) and papillary craniopharyngiomas (PCs). Their proximity to critical brain structures and high recurrence rates pose therapeutic challenges. Advances in cancer genomics have uncovered key molecular drivers, including BRAF V600E mutations in papillary and CTNNB1 mutations in ACs. These discoveries have prompted updates in tumor classification and the development of novel targeted therapies. B-Raf proto-oncogene, serine/threonine kinase (BRAF) and mitogen activated protein kinase (MEK) inhibitors have shown remarkable efficacy in treating PCs, while systemic therapeutic options for ACs remain limited. This article highlights the pathogenesis, molecular profiles, and emerging targeted therapies for these challenging tumors.