HBS-101, a novel midkine inhibitor, reduced optic nerve proliferation, normalized RNFL thickness, and decreased the expression of tumor (Neu4, Gpr17) and TME (Ccl2, Ccl3, Ccl4, Ccl5) genes, supporting its continued development as a targeted therapy for pediatric NF1-associated glioma. This evidence concerns the gene GPR17 and central nervous system cancer.