CNS Tumor

Noninvasive imaging parameters extracted from QSM images can reflect glioma WHO grade, molecular markers, prognosis, and recurrence risk. These preliminary findings showed that the QSM might be a noninvasive imaging biomarker for comprehensive evaluation of adult-type diffuse gliomas.

The six identified hub genes are consistently downregulated in glioma and exhibit strong diagnostic potential. Their close association with the immune microenvironment and tumor-cell lineage highlights their value as biomarkers and potential therapeutic targets.

EF24 disrupts this complex through suppressing NRF2 mRNA stability and impairing its protein translation, ultimately depleting GPX4 and triggering ferroptosis cascades. For the first time, our study proposes an epitranscriptomic axis of METTL3/YTHDF1/NRF2/GPX4 as a regulator of ferroptosis in glioma, which may be targeted to design an effective and safe therapeutic strategy.

The protective effect of Faecalibacterium sp002160895 on GBM was mediated by 7.59% of the expression level of CD4 in Treg cells. Our study, through MR analysis, revealed the causal relationship between gut microbiota and the susceptibility to glioma, and for the first time proposed the important role of circulating immune cells in this process, providing new potential biomarkers for the early diagnosis and treatment of glioma.

Definitive diagnosis requires pathology, though sampling bias poses challenges. Advanced imaging (contrast-enhanced 3D T2-FLAIR MRI) and biomarkers (BRAF V600E, beta-catenin, p53, Ki-67) show promise in improving diagnosis, predicting recurrence, and guiding treatment.

The clinical applicability of these tests must be corroborated with clinical presentation and neurosurgical resection to highlight progression and recurrence rates. Standardized protocols and molecular-based evidence are essential for optimizing patient management and outcomes.

Differential considerations, including epithelioid sarcoma, poorly differentiated chordoma, CRINET, choroid plexus carcinoma, and rare composite tumors, further complicate the diagnostic landscape. A comprehensive, multimodal diagnostic approach combining histologic, immunophenotypic, and molecular data is essential to accurately identify AT/RT and guide clinical management, particularly in diagnostically ambiguous or atypical cases.

Finally, we outline a translational roadmap that pairs patient-derived iPSC/organoid and organ-on-a-chip platforms to stratify EndMT states and prioritize targets. We also explore combination regimens that integrate multi-pathway modulation with epigenetic and immune approaches, aiming to deliver clinically meaningful anti-fibrotic benefits while better preserving physiological signaling.

Overall, the R273C mutation, although mechanistically unclear, is more prevalent than other TP53 variants and defines a distinct biological subset of LGIMAs, marked by increased Ki-67 and female predominance. Incorporating TP53 and broader genetic profiling via NGS could improve our understanding of LGIMAs and support a refined classification system, enhancing diagnostic and prognostic accuracy.

Finally, we propose future directions focused on decoding tissue-specific immunodynamics and developing spatiotemporally controlled, multiorgan immunoregulatory frameworks. Together, this review underscores cGAS-STING as a promising therapeutic axis in the evolving landscape of neuroimmunology.

Dabrafenib plus trametinib has shown superiority over chemotherapy in pediatric low-grade gliomas and activity against high-grade diseases. Larotrectinib and entrectinib provide tumor-agnostic options for NTRK-fusion-positive tumors with central nervous system penetration. Selumetinib offers clinical benefits in NF1-associated plexiform neurofibromas and shows promise for treating NF1-related low-grade gliomas. Tovorafenib, a type II RAF inhibitor active in BRAF-altered tumors (including BRAFKIAA1549 fusion), achieved robust responses, thereby leading to FDA approval. ONC201 (dordaviprone) has received accelerated approval for the treatment of H3 K27M-mutant diffuse midline gliomas, with Japanese trials and patient-initiated programs expanding access. Abemaciclib, a CDK4/6 inhibitor, is under phase II evaluation for pediatric high-grade glioma and diffuse midline glioma, including sites in Japan. Neurosurgeons play a pivotal role in securing high-quality biopsies, thus enabling comprehensive molecular diagnostics and facilitating enrollment in international trials. This review summarizes current targeted therapies and ongoing studies and outlines practical considerations for integrating precision oncology into pediatric neuro-oncology in Japan.

In here we report a case of a female patient who developed LMD from a Pleomorphic Xanthoastrocytoma (PXA), BRAFV600-mutated, who has shown successful response to treatment with BRAF/MEKi (Encorafinib/Binimetinib) for over 3 years since initial LMD diagnosis. The effectiveness of therapy in this patient was initially observed as stable disease, with radiographic progression when BRAF/MEKi were withheld, and immediate tumor control achieved when reinstated. Despite being just one case, this hopefully could serve as proof-of-concept for use of targeted therapy for BRAF V600E-mutated tumors with LMD progression, sparing patients from alternative tumor control options such as radiation therapy.