Medulloblastoma: Molecular Targets and Innovative Theranostic Approaches

Background/Objectives: Medulloblastoma is a rare tumor that represents almost two-thirds of all embryonal pediatric brain tumor cases. Current treatments, including surgery, radiation, and chemotherapy, are often associated with adverse effects, such as toxicity, resistance, and lack of specificity. According to multiple bulk and single-cell omics-based approaches, it is now clear that each molecular subgroup of medulloblastoma possesses intrinsic genetic and molecular features that could drive the definition of distinct therapeutic targets, and of markers that have the potential to improve diagnosis. Nanomedicine offers a promising approach to overcome these challenges through precision-targeted therapies and theranostic platforms that merge diagnosis and treatment. This review explores the role of nanomedicine in medulloblastoma. Here, possible theranostic nanoplatforms combining targeted drug delivery and simultaneous imaging are reviewed, highlighting their potential as tools for personalized medicine. Methods: We performed a chronological analysis of the literature by using the major web-based research platforms, focusing on molecular targets, and the potential application of nanomedicine to overcome conventional treatment limitations. Results: Advances in nanoparticle-based drug delivery systems enable selective targeting of key molecular pathways, improving therapeutic efficacy while minimizing off-target effects. Additionally, nanotechnology-based imaging agents, including MRI contrast agents and fluorescent probes, improve diagnostic accuracy and treatment monitoring. Despite these advantages, some significant challenges remain, including overcoming the blood-brain barrier, ensuring biocompatibility, and addressing regulatory pathways for clinical translation. Conclusions: In conclusion, we sought to identify the current knowledge on the topic and hope to inspire future research to obtain new nanoplatforms for personalized medicine.