MYCN amplification

Maximal safe resection remains the cornerstone of management, with radiotherapy or chemotherapy reserved for selected cases. Despite potential morbidity, long-term survival and functional outcomes are favorable for many patients.

P1/2, N=374, Active, not recruiting, Memorial Sloan Kettering Cancer Center | Trial completion date: May 2026 --> May 2027 | Trial primary completion date: May 2026 --> May 2027

We discuss emerging strategies to disrupt MYCN interactions and to enable its degradation through disruption of protein stabilisation mechanisms or the use of degraders such as PROTACs.  By integrating knowledge of MYCN biology, molecular structures and chemical biology, these approaches provide promising routes towards targeted therapies for MYCN-driven neuroblastoma.

P2, N=42, Completed, National Cancer Institute (NCI) | Active, not recruiting --> Completed | Trial completion date: Sep 2026 --> Mar 2026

Combined CHK1/CHK2 inhibitor therapy shows a synergistic anti-tumor effect against neuroblastoma cells. Combination therapy impairs DNA damage response pathways and drives accelerated cell cycle progression in neuroblastoma cells. Combination therapy increases DNA damage, replication stress, and apoptosis marker expression. Combined CHK1/CHK2 inhibition holds therapeutic potential for the treatment of neuroblastoma.

Therefore, we propose that immune IL4i1 is permissive for NB growth and survival. IL4i1 produces context-dependent oncometabolites and, as a secreted enzyme, represents a target for cell death manipulation in cancers sensitive to oxidative stress-driven cell death.

Our review did not uncover features to improve grading of oligodendroglioma, IDH-mutant and 1p/19q-codeleted, although CNS WHO grade 3 tumors with elevated mitotic rates, yet lacking necrosis, microvascular proliferation, CDKN2A/B homozygous deletion and MRI contrast enhancement, may be associated with extended survival. Implementing evidence-based criteria for risk stratification and grading will improve guidance for clinical decision-making.

CDCA7 may serve as a prognostic marker for neuroblastoma. Its silencing leads to G1 cell cycle arrest and a reduction in CDK6 expression, ultimately inhibiting NB cell proliferation.

This case supports the role of subtotal resection followed by proton beam therapy as a viable treatment strategy for MYCN-amplified intradural extramedullary anaplastic ependymomas, particularly when gross-total resection is contraindicated. https://thejns.org/doi/10.3171/CASE25804.

Pulmonary toxicity was observed in patients receiving lorlatinib combined with anti-GD2 immunotherapy or chemotherapy. These preliminary findings suggest potential efficacy of frontline lorlatinib in ALK-driven neuroblastoma, highlight molecular determinants of sensitivity, and reveal challenges of resistance and treatment-related toxicity.

The NB‑MSI2 + MYCN+ subtype defines a clinically aggressive, therapy‑refractory state characterized by high proliferation, metabolic reprogramming, and immunosuppression. For patients with MYCN-amplified NB, MSI2 is both a prognostic biomarker and a candidate therapeutic target.

Racotumomab elicited immune responses without serious related adverse events in HR-NB patients.