MYCN expression

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

Notably, MI102 effect exhibited superior tumor cell selectivity compared with the MET inhibitor tivantinib. At the transcriptional level, RNA-seq revealed that MI102 globally downregulated MYCN-associated oncogenic programs. Collectively, these findings establish pharmacological downregulation of MYCN as a promising therapeutic strategy for HCC and reveal a functional link between MET signaling and MYCN-driven oncogenic pathways.

Through extensive gene expression profiling and whole-exome sequencing of MYCN/ALK-induced clones, we identified key features of NB, including loss of NF1 and gain of 17q chromosome, which are critical for the development of malignant tumor. This model provides a valuable platform for studying the biological mechanisms driving ALK and MYCN amplification in NB derived from cNCCs.

Tumor growth decreased in animals treated with ATUX-1215, and analysis of tumor specimens confirmed decreased MYCN expression. We conclude that pharmacologic PP2A reactivation may be a relevant therapeutic component in NBL treatment through its targeting of MYCN.

In vivo, volasertib treatment attenuated fibroblast activation and collagen deposition during TGFα-induced pulmonary fibrosis. Together, these findings identify a pathogenic role for the WT1-MYCN-PLK1 axis in fibroblast activation and provide proof-of-concept evidence supporting PLK1 inhibition with volasertib as a potential therapeutic strategy for IPF.

In the myoblast model system, transduction studies with exogenous miR-17-92 or miRNA-sponge expression constructs indicated that miR-17-92 is necessary but not sufficient for oncogenic transformation. Together, these findings establish a cooperative transcriptional axis in FP-RMS involving P3F and MYCN that activates MIR17HG through a distal regulatory element, thereby contributing to oncogenic behavior and uncovering a novel mechanistic vulnerability.

Our study establishes Mycn as a key regulator of vascular development in zebrafish, acting through the PI3K signaling pathway.

Molecular docking and molecular dynamic simulation revealed that CCA-1.1 interacted with both N-Myc and c-Myc. Our findings demonstrate CCA-1.1 as a distinctive anti-cancer candidate for HCC driven by MYC activation.

We thus provide, for the first time, evidence that HMMR does have oncoprotein-like properties in neuroblastoma cells and that HMMR expression has potential as a prognostic marker. Moreover, initial biochemical analyses highlight a potential influence for HMMR in MTOR and DDR pathway regulation.

In summary, the present study demonstrated that RP11-196G11.6 may inhibit NB progression by sponging miR-376a-3p, leading to the upregulation of RYBP expression and consequently inhibiting NB progression. These findings revealed a novel lncRNA-miRNA axis involved in NB pathogenesis.

P1, N=26, Active, not recruiting, Dana-Farber Cancer Institute | Trial completion date: Oct 2025 --> Feb 2026 | Trial primary completion date: Oct 2025 --> Jan 2026