Neuroblastoma

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.

Furthermore, transcriptomic analysis of zebrafish brain tissue confirmed significant dysregulation of genes involved in mitochondrial function. Overall, our study establishes that mitochondrial dysfunction and exaggerated mitophagy contribute to L-BMAA-induced injury in both zebrafish brains and SH-SY5Y cells, offering a potential therapeutic target for treating therapy-refractory neurodegenerative diseases caused by environmental factors.

P1, N=48, Recruiting, St. Jude Children's Research Hospital | N=32 --> 48 | Trial completion date: Mar 2027 --> Mar 2028 | Trial primary completion date: Mar 2026 --> Mar 2027

P1/2, N=76, Not yet recruiting, National Cancer Institute (NCI) | Initiation date: May 2026 --> Jul 2026

P2, N=28, Recruiting, Instituto do Cancer do Estado de São Paulo | Active, not recruiting --> Recruiting

P1, N=30, Recruiting, New York Medical College | Trial completion date: Dec 2024 --> Dec 2027 | Trial primary completion date: Dec 2023 --> Dec 2026

Emerging platforms, including oncolytic virotherapy, NK cell engagers, and neoantigen vaccines, offer rational strategies to convert immunologically cold tumors into treatment-responsive phenotypes. Together, these advances point toward a future of combination immunotherapy tailored to the distinct immune biology of childhood cancers.

It promotes the pathogenesis of this condition by directly targeting FBXW7 and inhibiting mitochondrial pathway-mediated apoptosis. These findings highlight a novel role for platelet miRNAs in the neuropathogenesis of HFMD and suggest the miR-223-3p/FBXW7 axis as a potential therapeutic target.

This pilot study suggests that genetic variations in the miRNA-processing pathway may affect NB susceptibility. Further research involving larger, ethnically diverse cohorts, along with expanded genotype-expression analyses and functional validation, is essential before clinical or prognostic applications can be established.