Targeted therapies in optic pathway gliomas

Area of Science:

• Neuro-oncology
• Molecular targeted therapy
• Ophthalmology

Background:

• Optic pathway gliomas (OPGs) are challenging pediatric brain tumors.
• Current management often involves chemotherapy and surgery with significant side effects.
• There is a need for more effective and less toxic treatment options.

Purpose of the Study:

• To systematically review evidence on targeted therapies for OPGs.
• To evaluate the molecular rationale, clinical effectiveness, and safety of these agents.
• To assess their relevance in both NF1-associated and sporadic OPGs.

Main Methods:

• Systematic literature review adhering to PRISMA guidelines.
• Searched PubMed, Web of Science, and Scopus databases up to April 2025.
• Included preclinical and clinical studies, assessing efficacy, molecular targets, and adverse events.

Main Results:

• 13 studies (11 clinical, 2 preclinical) were included.
• Targeted agents comprised MEK, mTOR, anti-VEGF, and BRAF inhibitors.
• MEK inhibitors, particularly selumetinib, demonstrated promising progression-free survival and favorable safety in NF1-OPGs.

Conclusions:

• Targeted therapies represent a paradigm shift in OPG management.
• Biomarker-driven strategies enhance disease control and visual preservation.
• Challenges include resistance, long-term safety, and optimal therapy duration.