CNS Tumor

P1, N=12, Not yet recruiting, Neonc Technologies, Inc. | Trial completion date: Oct 2025 --> Oct 2026 | Trial primary completion date: Oct 2025 --> Oct 2026

Although intergroup differences were limited, our intragroup analyses revealed reduced satiety persistence and earlier appetite rebound, particularly in patients with HyOb and severe HI, providing new insights into the complex, multifactorial pathophysiology of HyOb associated with aCP.

Six PMS2 variants were associated with either early or later-onset CMMRD. Future validation through larger prospective cohort studies is necessary to confirm our findings and better understand the natural history of PMS2-CMMRD to inform clinical decision-making in PMS2-Lynch syndrome (PMS2-LS).

P2, N=33, Active, not recruiting, Children's Oncology Group | Trial completion date: Mar 2026 --> Jul 2027

IFI44 may drive glioma progression through dual mechanisms of immune microenvironment remodeling and promotion of tumor cell aggressiveness, supporting its potential as a prognostic biomarker and therapeutic target. Although preliminary knockdown and overexpression assays were performed, the underlying mechanisms of IFI44-mediated immune regulation and tumor progression require further investigation.

These findings contribute to the literature suggesting that, rarely, GPV in aoCPG may contribute to cancer diagnoses in children, raising the question of how tumors in these cases may present differently in children than adults. Increased knowledge about potential childhood cancer risks related to what have historically been considered aoCPG could modify predictive genetic testing recommendations for children and enhance existing cancer screening protocols.

In this large cohort with extended follow-up, STR patients exhibited a second recurrence peak at 7 to 10 years postoperatively. Adjuvant GKRS showed long-term benefit, supporting its selective use and long-term surveillance beyond 10 years.

RECQL4 knockdown in glioma and MPNST cell lines resulted in increased apoptosis and susceptibility to ATR-inhibitors. Our findings show that RECQL4 expression has divergent patterns across tumor types and that targeting RECQL4 may dampen tumor survival and enhance susceptibility to ATR inhibitor therapy in CNS tumors.

The therapeutic and diagnostic applications of nanoMIPs in neurodegeneration, brain tumors, and CNS infections are also highlighted. Finally, current limitations and future perspectives are discussed, including biocompatibility, large-scale production, and regulatory considerations, positioning nanoMIPs as a next-generation platform for overcoming BBB-associated barriers, and advancing precision brain therapeutics.

Immunofluorescence, NGS analysis, and DNA methylation profiling revealed that the PDTs faithfully mirrored the cellular nature, the genetic and epigenetic landscape of their matched primary tumors. Our study shows the feasibility of generating PDTs, even from rarer entities, that recapitulate the genetic and epigenetic features of primary tumors, highlighting their potential as models for tumor biology studies and precision medicine.

Our findings revealed critical insights into the immune landscape of NSCLC BrM under ICI therapy, highlighting CD4+PDCD1+CXCR6+ T cells in CSF as a promising biomarker and illuminating fundamental mechanisms underlying ICI efficacy.

Our results provide the first pooled evidence prevalence estimate of OPG in children and adolescents with NF1 and highlight the importance of close monitoring, reinforcing guidelines that favor a symptom-based MRI approach and suggesting no clear advantage of routine imaging.