Optic pathway glioma: current treatment approaches and ongoing clinical trials—a review article

Optic pathway glioma (OPG) is a rare pediatric low-grade glioma, frequently associated with neurofibromatosis type 1 (NF-1), that presents unique therapeutic challenges due to its anatomical location and its potential to impair vision, endocrine function, and developmental trajectories. Current clinical management prioritizes a multidisciplinary, patient-specific approach aimed at tumor control while preserving long-term quality of life. Strategies vary based on clinical presentation, ranging from observation in asymptomatic cases to chemotherapy for progressive or symptomatic tumors. Surgical and radiation options are limited due to potential risks and complications. In recent years, advances in molecular characterization have guided the development of targeted therapies, particularly MEK inhibitors, which demonstrate encouraging efficacy and reduced toxicity profiles. In parallel, investigational therapies including immunotherapy and precision medicine-based approaches are under clinical evaluation. This review synthesizes current standard practices and recent progress in targeted treatment development. Actively recruiting clinical trials are also cataloged to inform both clinicians and families about available therapeutic options. To conduct this review, we searched PubMed, ClinicalTrials.gov, and Scopus for English-language articles published between 2010 and 2025 using terms such as “optic pathway glioma,” “NF1,” “MEK inhibitors,” and “targeted therapy.” Studies were selected based on relevance to treatment strategies, trial design, and clinical applicability. Data extraction focused on trial outcomes, therapeutic mechanisms, and patient-centered considerations. Through integrating clinical evidence and ongoing research, this review aims to provide a comprehensive update on current and emerging treatment strategies for OPG and to support informed, evidence-based care planning.