MYCN amplification

Continued progress will depend on integrating molecular profiling into clinical decision-making, refining risk-adapted treatment strategies, and expanding international collaborative research efforts. This narrative review summarizes current knowledge on neuroblastoma epidemiology, biology, staging, and treatment, highlighting recent advances and future directions aimed at improving outcomes for affected children.

Patients with relapsed HR-NBL have poor outcomes with median OS < 2 years. Time to relapse was a significant predictor of OS.

P1, N=26, Completed, Dana-Farber Cancer Institute | Active, not recruiting --> Completed

This study uncovered a novel oncogenic axis in neuroblastoma, where NeuroD1 transcriptionally upregulates USP1, promoting N-Myc stabilization and tumor progression. Furthermore, the findings highlight the therapeutic potential of repurposing Pimozide as a promising treatment strategy for this aggressive tumor subtype.

Naxitamb+GM-CSF is a good option to consolidate post-relapse CR of HR-NB. The encouraging long-term outcome cannot be attributed solely to naxitamab+GM-CSF given post-protocol therapies.

The most promising salvage options for patients responding insufficiently to treatment are the chemotherapy combinations, topotecan/vincristine/doxorubicin (TVD), topotecan/cyclophosphamide/etoposide (TCE), ifosfamide/carboplatin/etoposide (ICE) or topotecan/cyclophosphamide (TopoCy), or &lsqb;131I]-mIBG therapy. Early-phase clinical trials are also a possible option in this setting.

In conclusion, this study addresses a fundamental systems biology and translational research gap by establishing a data-driven framework for selecting NB cell lines that accurately reflect patient-derived tumor biology with direct implications for prioritizing therapeutically relevant drug candidates. Future studies should prioritize the top CMRs as in vitro models to enhance translational relevance and accelerate precision drug discovery in high-risk pediatric NB.

BMP7 was a prognostic maker of neuroblastoma.

To the best of our knowledge, this is the first reported case of neonatal neuroblastoma with concurrent MYCN amplification and 1p deletion achieving favorable outcome through comprehensive multimodal therapy. This case underscores the importance of early diagnosis, genetic profiling, and aggressive treatment in managing high-risk neuroblastoma in neonates.

In parallel, the effects of the compounds on non-malignant cell lines were assessed to obtain an indication of their selectivity toward tumor cells. Compound 17, a structural analog lacking the second aromatic ring in the ex-aldehyde portion, displayed a distinct profile with a limited anticancer activity, underscoring the importance of this structural fragment for antiproliferative efficacy.

P1, N=99, Completed, Children's Oncology Group | Active, not recruiting --> Completed | Trial completion date: Sep 2025 --> Dec 2025