Immune checkpoint inhibitors in pediatric central nervous system tumors: biology, clinical experience, and translational pathways to precision immunotherapy - PubMed
8 hours ago
- #immune checkpoint inhibitors
- #precision immunotherapy
- #pediatric CNS tumors
- Immune checkpoint inhibitors (ICI) have limited efficacy in pediatric central nervous system (CNS) tumors due to low tumor mutational burden and immunosuppressive microenvironments.
- ICI monotherapy targeting PD-1/PD-L1 has largely failed in pediatric trials, highlighting the need for biomarker-driven patient selection.
- Some pediatric CNS tumors, like DNA replication-repair-deficient high-grade gliomas, show durable responses to PD-1 blockade due to robust neoantigen burden.
- Potential strategies to improve ICI efficacy include early/neoadjuvant use, non-traditional checkpoint combinations, metabolic/genomic targeting, advanced drug delivery, and gut microbiota modulation.
- Future directions involve overcoming resistance mechanisms, improving toxicity management, and redefining immunotherapy response assessment with advanced imaging and liquid biopsies.