Foti Alice, Allia Fabio, Briglia Marilena, Malaguarnera Roberta, Tamburrini Gianpiero, Cecconi Francesco, Pagliarini Vittoria, Nazio Francesca, Graziano Adriana Carol Eleonora
Department of Medicine and Surgery, University of Enna "Kore", 94100 Enna, Italy.
Department of Neuroscience, Sensory Organs, Chest-Pediatric Neurosurgery, Fondazione Policlinico Universitario A. Gemelli IRCCS, 00168 Rome, Italy.
Pharmaceutics. 2025 Jun 4;17(6):736. doi: 10.3390/pharmaceutics17060736.
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. 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. 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. 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.
髓母细胞瘤是一种罕见的肿瘤，几乎占所有儿童胚胎性脑肿瘤病例的三分之二。目前的治疗方法，包括手术、放疗和化疗，常常伴有不良反应，如毒性、耐药性和缺乏特异性。根据多种基于组学的整体和单细胞研究方法，现在已经明确，髓母细胞瘤的每个分子亚组都具有内在的遗传和分子特征，这些特征可以推动确定不同的治疗靶点以及有可能改善诊断的标志物。纳米医学提供了一种有前景的方法，通过精准靶向治疗和融合诊断与治疗的诊疗平台来克服这些挑战。本综述探讨了纳米医学在髓母细胞瘤中的作用。在此，对结合靶向药物递送和同步成像的可能的诊疗纳米平台进行了综述，强调了它们作为个性化医疗工具的潜力。我们使用主要的基于网络的研究平台对文献进行了按时间顺序的分析，重点关注分子靶点以及纳米医学克服传统治疗局限性的潜在应用。基于纳米颗粒的药物递送系统的进展能够选择性地靶向关键分子途径，提高治疗效果，同时将脱靶效应降至最低。此外，基于纳米技术的成像剂，包括磁共振成像造影剂和荧光探针，提高了诊断准确性和治疗监测水平。尽管有这些优势，但仍存在一些重大挑战，包括克服血脑屏障、确保生物相容性以及解决临床转化的监管途径。总之，我们试图确定关于该主题的现有知识，并希望激发未来的研究，以获得用于个性化医疗的新纳米平台。
