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3d
Efficacy and safety of sequential therapy with Venetoclax, Azacitidine, and Homoharringtonine in elderly patients with de novo AML: A prospective, single-arm, multicenter, exploratory clinical trial (ChiCTR2500112019)
P=N/A, N=40, Not yet recruiting, the First Affiliated Hospital, Zhejiang University School of Medicine; The People's Hospital Affiliated to Ningbo University
New trial
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Venclexta (venetoclax) • azacitidine • Synribo (omacetaxine mepesuccinate)
3d
SRSF2 mutations drive daunorubicin resistance in acute myeloid leukemia via THBS1 stabilization. (PubMed, J Exp Clin Cancer Res)
SRSF2 mutations promoted DNR resistance through multiple mechanisms, and targeted combination therapy with PDGFB pathway inhibitors may represent a novel strategy to improve therapeutic outcomes in patients with mutations.
Journal
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SRSF2 (Serine and arginine rich splicing factor 2) • THBS1 (Thrombospondin 1) • PDGFB (Platelet Derived Growth Factor Subunit B)
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SRSF2 mutation
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Venclexta (venetoclax) • daunorubicin • Synribo (omacetaxine mepesuccinate)
5d
Targeting c-Myc-p300-CARM1 complex induces ferroptosis and reduces CD8+ T cell exhaustion in esophageal squamous cell carcinoma. (PubMed, Int J Biol Sci)
These findings revealed that SGC2085 and MYCi975 could disrupt the transcriptional complex CPC, affect metabolic pathways, and reprogram the immune microenvironment. This study provides a potential therapeutic strategy for ESCC patients.
Journal
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MYC (V-myc avian myelocytomatosis viral oncogene homolog) • CD8 (cluster of differentiation 8)
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MYCi975
20d
OMO-103-02: Study to Evaluate the Safety, PK, and Efficacy of the Myc Inhibitor OMO-103 Administered iv in Patients With PDAC (clinicaltrials.gov)
P1, N=26, Active, not recruiting, Peptomyc S.L. | Recruiting --> Active, not recruiting | Trial primary completion date: Dec 2025 --> May 2026
Enrollment closed • Trial primary completion date
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gemcitabine • albumin-bound paclitaxel • OMO-103
20d
Krüppel-Like Factor 4, a Hub Gate for Cell Crosstalk in Tumor Microenvironment. (PubMed, Cancer Med)
KLF4 serves as a hub gate orchestrating cell crosstalk within the TME. Understanding its context-dependent functions may facilitate the development of KLF4-targeted therapies for precision oncology.
Review • Journal
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KLF4 (Kruppel-like factor 4)
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APTO-253
26d
Discovery of novel and potent harringtonine derivative P2 via systematic structure-activity Optimization: Semi-Synthesis, anti-leukemia activity, and mechanism study. (PubMed, Eur J Med Chem)
P2 exhibited a ∼10-fold increase in antiproliferative potency against human leukemic cell lines compared to homoharringtonine (HHT)...Our findings provide valuable insights to guide the future structural optimization of harringtonine derivatives. Furthermore, P2 has been identified as a promising anti-leukemic candidate and warrants further development.
Journal
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MYC (V-myc avian myelocytomatosis viral oncogene homolog) • MCL1 (Myeloid cell leukemia 1)
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Synribo (omacetaxine mepesuccinate)
1m
pH-triggered Mn-ZIF8 nanoreactor enhances tumor immunotherapy by amplifying the cGAS-STING pathway through a self-reinforcing gas-metal synergy strategy. (PubMed, J Nanobiotechnology)
In this study, we propose a Gas-Metal Synergy Strategy, which integrates immune activation and biosafety, by engineering a pH-responsive manganese-based zeolitic imidazolate framework (named MRPH) nanoplatform co-loaded with the nitric oxide (NO) donor RRX-001...Both in vitro and in vivo studies demonstrate that MRPH significantly enhances gas-amplified metalloimmunotherapy. This work pioneers a low-toxicity paradigm that integrates gas therapy and metal-based immunotherapy, offering a transformative approach to solid tumor immunotherapy.
Journal
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CD8 (cluster of differentiation 8)
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nibrozetone (RRx-001)
1m
Homoharringtonine suppresses acute myeloid leukemia progression by orchestrating EWSR1 phase separation in an m6A-YTHDF2-dependent mechanism. (PubMed, Imeta)
In vivo, the anti-leukemic efficacy of HHT was significantly diminished upon EWSR1 knockdown, demonstrating that EWSR1 was required for therapeutic response. Collectively, these findings uncover a phase separation-centric mechanism by which HHT exerts anti-AML activity, establish the EWSR1-YTHDF2-m6A axis as a critical regulator of leukemia progression, and position EWSR1 as both a functional target and a predictive biomarker for optimizing HHT-based therapies.
Journal
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EWSR1 (EWS RNA Binding Protein 1) • HMOX1 (Heme Oxygenase 1) • YTHDF2 (YTH N6-Methyladenosine RNA Binding Protein 2) • TNFRSF1B (TNF Receptor Superfamily Member 1B)
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Synribo (omacetaxine mepesuccinate)
1m
Transcriptome-Guided Drug Repurposing Identifies Homoharringtonine (HHT) as a Candidate for Radiation-Induced Pulmonary Fibrosis. (PubMed, Pharmaceutics)
This study highlights the value of computational drug repurposing platforms for accelerating therapeutic discovery. Further preclinical investigations are warranted to evaluate HHT's in vivo efficacy and clinical applicability in RPF.
Journal
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CCND1 (Cyclin D1) • RHOA (Ras homolog family member A)
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Synribo (omacetaxine mepesuccinate)
1m
Organoid-based drug screening identifies homoharringtonine as a therapeutic agent for anaplastic thyroid cancer via TFEB-mediated lysosomal dysfunction. (PubMed, Cell Oncol (Dordr))
Our study demonstrates the utility of cancer organoids in drug discovery and identifies HHT as a promising therapeuticagent for ATC.
Journal
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TFEB (Transcription Factor EB 2)
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Synribo (omacetaxine mepesuccinate)
2ms
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS (clinicaltrials.gov)
P1/2, N=28, Active, not recruiting, University of Colorado, Denver | Trial primary completion date: Aug 2026 --> Oct 2025
Trial primary completion date
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azacitidine • Synribo (omacetaxine mepesuccinate)
2ms
Extensive myeloid sarcoma presenting with esophageal compression and dysphagia as the initial manifestation. (PubMed, Medicine (Baltimore))
Infiltrative mediastinal/retroperitoneal MS may present with esophageal obstruction and mimic lymphoma or carcinoma. High-index suspicion, targeted biopsy with high-power morphology, and a focused immunohistochemical panel are critical for timely diagnosis and treatment initiation.
Journal
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KIT (KIT proto-oncogene, receptor tyrosine kinase) • CD20 (Membrane Spanning 4-Domains A1) • CD34 (CD34 molecule) • PTPRC (Protein Tyrosine Phosphatase Receptor Type C) • CD5 (CD5 Molecule) • CD79A (CD79a Molecule) • CD7 (CD7 Molecule) • KRT19 (Keratin 19) • CD2 (CD2 Molecule) • CD99 (CD99 Molecule)
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cytarabine • Synribo (omacetaxine mepesuccinate) • aclarubicin