MYC (V-myc avian myelocytomatosis viral oncogene homolog)

P3, N=156, Not yet recruiting, Ruijin Hospital

In an orthotopic 4T1 tumor model, TO-ISe (0.5 mg kg-1) with irradiation achieved up to 72.8% tumor growth inhibition. These results highlight TO-ISe as a dual-function rG4-targeting photodynamic immunotherapeutic agent.

The RUVBL proteins form a complex with ATPase activity that has broad cellular functions and we demonstrate that pharmacological inhibition of this protein complex results in a strong reduction of MYC(N) signaling, cell-cycle arrest, DNA damage, and apoptosis. We confirmed the association with MYCN and identified the RUVBL genes as independent prognostic biomarkers in human primary neuroblastoma data.

ATAD2 drives immunotherapy resistance in LUAD by activating an ATAD2-LDHA-LA axis that impairs CD8+ T cell function. Targeting ATAD2 may broadly restore antitumor immunity and enhance the efficacy of T cell-based immunotherapies.

These results suggest that RSV may offer a promising therapeutic approach for osteosarcoma, modulating key pathways involved in tumour progression, metastasis and chemoresistance. Further studies are required to assess its clinical applicability.

This study deciphered a novel druggable metabolic-epigenetic pathway (lactate-H3K18la-KIF20A-Myc-PD-L1) responsible for immune evasion in HCC. Targeting this axis might offer a promising strategy to reprogram the tumor microenvironment and restore immunotherapy sensitivity.

FSD1L is a novel oncogenic driver in HCC that is selectively enriched in metastatic and lenvatinib-resistant contexts. It activates the WNT/β-catenin pathway to regulate HCC proliferation, EMT and lenvatinib resistance. FSD1L holds promise as a dual biomarker for diagnosis/prognosis and a potential therapeutic target for lenvatinib resistance in HCC.

Based on our experimental data, we propose a translational pathway, from preclinical intraperitoneal validation to clinically feasible local delivery (e.g., endoscopic submucosal injection or retention enema), with dose translation and follow-up endpoint suggestions. This study demonstrates the potential of advanced biomaterials and nucleic acid therapeutics for genetic/rare diseases, providing a basis for personalized FAP prevention.

This study provides valuable real-world insight into the clinical landscape of DLBCL in Saudi Arabia. Overall outcomes are consistent with international data, although older age and comorbidities remain associated with poorer prognosis. As advanced therapies such as chimeric antigen receptor T-cell therapy and bispecific antibodies become more available, further improvements in survival are expected. These findings underscore the need for a national lymphoma registry and continued investment in research infrastructure to guide evidence-based, personalized care.

The authors further evaluate the potential of SOX1 as a biomarker and therapeutic target in cancer diagnosis, treatment, prognosis, and associated complications. Although the functional heterogeneity of SOX1 presents challenges for clinical application, therapeutic modulation of its upstream and downstream pathways, as well as methylation-based assays for early detection, remain clinically promising.

Thus, our work defines C1orf35 as a dual-function oncoprotein that promotes CRC progression by coordinately enhancing tumor-intrinsic growth via the c-Myc/PYCR2 axis and fostering an immune-suppressive niche. These findings nominate C1orf35 as a promising multi-faceted therapeutic target and prognostic biomarker in CRC.

Xenograft studies further validated the therapeutic efficacy of CPM4, showing a significant reduction in tumor growth. These results underscore the therapeutic potential of CPM4 as an effective drug candidate for inhibiting c-MYC-driven tumor growth.