ArticleNeuro-oncology advances
Immunogenomic profiling reveals targets for gene therapy in pediatric brain tumors.
Article in Neuro-oncology advances. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Background: Pediatric brain tumors, particularly pontine diffuse midline glioma (pDMG), remains lethal with limited therapeutic options. Improved stereotactic biopsy techniques and advances in bioinformatics are progressively enabling deeper exploration of immunological vulnerabilities empowering novel strategies, including adoptive cell and gene therapies (ACGTs). We aimed to integrate genomic, transcriptomic, and immunological analyses to identify actionable pathways, surface antigens, and neoantigens that could inform next-generation immunotherapies, including Chimetic Antigen Receptor (CAR)-T cells and T cell Receptor (TCR)-T cell strategies. Methods: Primary pDMG samples ( Results: Transcriptional drug response profiling revealed heterogeneous but actionable pathway dependencies. B4GALNT1 expression varied across tumors, identifying a subset with GD2 levels compatible with CAR-T targeting. Tumor microenvironment profiling showed enrichment of dendritic cells and M2 macrophages, with scarce CD8 Conclusion: This integrated profiling approach identifies targetable pathways, surface antigens, and neoantigens in pDMG, supporting the development of CAR-T and TCR-T therapies. These insights also suggest potential applicability to other cancers harboring shared mutations.
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