小児・若年成人の高悪性度神経膠腫に対する治療標的候補を特定(Study Identifies Candidate Treatment Targets for High-Grade Glioma in Children and Young Adults)

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2026-09-17 マウントサイナイ医療システム(MSHS)

高悪性度神経膠腫は小児・若年成人に発生する悪性度の高い脳腫瘍で、研究チームは腫瘍細胞の遺伝子・分子特性を解析し、腫瘍の増殖や生存に関与する経路を特定することで、将来の治療標的となり得る分子を探索した。研究では、腫瘍組織などの分子データを用いて、患者間で共通する生物学的特徴と腫瘍のサブタイプによる違いを分析している。その結果、特定の分子経路を標的とする候補が複数見いだされ、個々の腫瘍の分子特性に基づいて治療法を選択する精密医療につながる可能性が示された。ただし、候補標的の発見は治療効果の確立とは異なり、実際の患者治療への有効性・安全性については今後の前臨床・臨床研究による検証が必要である。

小児・若年成人の高悪性度神経膠腫に対する治療標的候補を特定(Study Identifies Candidate Treatment Targets for High-Grade Glioma in Children and Young Adults)
Proteogenomic profiling of high-grade glioma from infancy through age 40 shows age-related tumor changes distinct from normal brain development, candidate kinase targets, and differences between male and female patients.

<関連情報>

小児およびAYA世代の高悪性度神経膠腫のプロテオゲノム解析により、年齢依存的な生物学的特徴、男女差、およびキナーゼ標的が明らかになった Proteogenomic analysis of pediatric and AYA high-grade glioma reveals age-dependent biology, female-male differences, and kinase targets

Nicole L. Tignor ∙ Mateusz Koptyra ∙ Shrabanti Chowdhury ∙ … ∙ Philadelphia Coalition for a Cure ∙ Children’s Brain Tumor Network ∙ Clinical Proteomic Tumor Analysis Consortium
Cell Reports Medicine  Published:September 17, 2026
DOI:https://doi.org/10.1016/j.xcrm.2026.103024

Highlights

  • Deep multi-omics characterization of 112 pediatric, adolescent, and young adult HGGs
  • Distinct genetic, transcriptomic, and proteomic features across age and sex groups
  • Druggable targets revealed by causal network analysis of kinase activities
  • Proteins and immune signatures linked to poor outcomes

Summary

High-grade gliomas (HGGs) in children and adolescents and young adults (AYA) exhibit distinct biology across the neurodevelopmental spectrum. To dissect tumor-intrinsic molecular characteristics independent of developmental variation, we perform comprehensive proteogenomic analyses of tumors from 112 HGG patients aged 0–40 years. Our multi-omics analysis identifies two AYA subgroups—adolescents (aged 15–26 years) and young adults (aged 26–40 years)—with distinct molecular profiles and survival outcomes. Tumor-normal comparisons and survival modeling highlight roles of oxidative phosphorylation and neuronal system biology in glioma progression. Causal network analysis and cell line studies provide a rationale for personalized therapies targeting candidate kinases, such as CDK8. Survival modeling, clustering, and immune-landscape analyses identify proteins, post-translational modifications, and immune signatures linked to outcomes and reveal clinically relevant differences between male and female patients.

医療・健康
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