Recent Update in Pharmacological Agents for Optic Pathway Glioma

ABSTRACT

Optic pathway gliomas (OPGs) are insidious, debilitating low-grade tumors. They can affect the optic nerve, optic chiasm, and optic tracts and can be sporadic or associated with neurofibromatosis type 1 (NF1). The location of OPGs within the optic pathway typically precludes complete resection or optimal radiation dose. Treatment is unnecessary for sporadic and NF1-related OPGs that do not cause visual impairments. Chemotherapy is the mainstay of treatment for patients with progressive disease. However, outcomes following standard treatments have been mixed, and standardized outcome measurements are lacking. In recent years, newer molecularly targeted therapies such as anti-vascular endothelial growth factor (VEGF) monoclonal antibody, mitogen-activated protein kinase (MAPK) inhibitor, and mammalian target of rapamycin (mTOR) inhibitor, represent a promising treatment modality.