Approved Targeted Treatments for Pediatric Brain Tumors

Advances in molecular and genomic techniques have significantly improved our understanding of pediatric brain tumors and enabled the development of targeted therapies directed at specific genetic alterations. The emergence of these therapies is reshaping the management of pediatric brain tumors, offering the potential for more effective and personalized treatment strategies, with reduced toxicity and improved clinical outcomes. This paradigm shift in pediatric neuro-oncology is still at an early stage but is expected to lead to substantial changes in clinical practice in the coming years. Clinical research has already resulted in the approval of several targeted agents, particularly for tumors driven by alterations in the MAP kinase pathway. These successes underscore the importance of integrating molecular diagnostics into routine care. This review summarizes the clinical development and regulatory approval of targeted therapies in pediatric brain tumors. It also addresses key challenges in the design and conduct of clinical trials in this unique population, including small patient numbers. In addition, several critical questions remain unresolved, such as the optimal duration of therapy, the durability of responses, mechanisms of resistance, and the long-term effects of these treatments in children. Finally, this review discusses issues related to access and equity, particularly the availability of these often-costly therapies in low- and middle-income countries, which remains a significant barrier to their global implementation.