MYCN amplification

While CSF remains the optimal matrix for diagnosis and characterization, advances in ultrasensitive detection methods increasingly support the feasibility of plasma and urine for longitudinal follow-up. Integrating liquid biopsy with advanced imaging and tissue-based data will likely transform diagnostic accuracy, therapeutic decision-making, and real-time monitoring of CNS tumors.

MYCN promotes ferroptosis in RB via xCT and transsulfuration pathways. Targeting the MYCN-xCT-transsulfuration axis may offer a novel therapeutic approach for MYCN-amplified RB.

This integration constructs more precise disease classification models and facilitates the development of personalized treatment plans. This review emphasizes the critical roles of transcriptomics, radiomics, digital pathology analysis, and multi-omics fusion strategies in enhancing diagnostic precision for neuroblastoma and optimizing treatment decisions.

Targeting ZDHHC22 suppresses neuroblastoma cell growth in vitro and in vivo, particularly in refractory patient-derived models. Collectively, our findings uncover a biological function of ZDHHC22 in regulating N-Myc transcriptional activation and indicate that ZDHHC22 is a promising therapeutic target for N-Myc-driven high-risk neuroblastoma, especially in MYCN-amplified patients.

Our findings reveal a new role for the transcriptional regulator NONO in maintenance of the cellular redox balance and justify the strategy of therapeutic targeting of MYCN-amplified tumors vulnerable to oxidative stress.

These findings indicate that Msi1 could serve as a novel therapeutic target for high-risk, treatment-refractory neuroblastoma.

Our findings enhance understanding of the genomic landscape of Chinese pediatric cancer populations. Further research is required to validate these findings and fully explore the potential of genomic data to improve outcomes for pediatric cancer patients.

Notably, LY6E emerged as the most prognostically significant gene within the signature. More importantly, we revealed that LY6E modulates M2-type macrophage polarization in neuroblastoma for the first time, suggesting a novel mechanism through which it may contribute to shaping an immunosuppressive tumor microenvironment.

P2, N=10, Not yet recruiting, Shaare Zedek Medical Center

P2, N=153, Completed, St. Jude Children's Research Hospital | Active, not recruiting --> Completed

Clinically, TWIST1+TAC+ CTCs were significantly enriched in the blood samples of metastatic NB patients compared to the non-metastatic group. As a proof-of-principle study, we further demonstrated that the TACR1 antagonist aprepitant could effectively suppress endothelial senescence, tumorigenesis, and CTC-mediated metastatic progression in vivo, presenting a potential therapeutic strategy for NB patients with TAC1-TACR1 activation.

We show that CDK2-Cyclin A2 bind ASCL1 in less responsive cells, with CDK-mediated phosphorylation of ASCL1 limiting the ability of ASCL1 to drive differentiation. Implications: Our study reveals that context-dependent interactions of ASCL1 with protein partners on the chromatin control its ability to re-engage a differentiation program in neuroblastoma.