GD2: hopes and challenges for the treatment of pediatric patients with tumors of the central nervous system



Summary

GD2 ganglioside, highly expressed in several pediatric central nervous system (CNS) tumors like neuroblastoma and medulloblastoma, presents a promising therapeutic target. GD2-targeted therapies, including monoclonal antibodies and CAR-T cell therapy, have shown potential in improving outcomes for these aggressive cancers. However, challenges remain. These include on-target off-tumor toxicity to normal tissues expressing GD2, limited penetration of therapies across the blood-brain barrier, and the development of resistance mechanisms within tumors. Overcoming these hurdles through innovative delivery strategies, combination therapies, and refined targeting approaches is crucial to realizing the full potential of GD2-directed treatments for pediatric CNS tumors.

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