Synribo (omacetaxine mepesuccinate)

P1/2, N=28, Active, not recruiting, University of Colorado, Denver | Trial primary completion date: Aug 2026 --> Oct 2025

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.

P1/2, N=24, Terminated, M.D. Anderson Cancer Center | Completed --> Terminated; The study terminated early because the company was longer providing the investigational product.

We then evaluated the response of these mutant cells to a panel of compounds targeting protein synthesis at various levels-including an MNK1 inhibitor, metformin, silvestrol, homoharringtonine, anisomycin, resveratrol, and hygromycin B-as well as cytarabine, a chemotherapeutic agent commonly used in T-ALL treatment. Our results showed that the RPL5-I60V mutation confers increased sensitivity to most of these compounds, with the exception of hygromycin B. This study advances our understanding of how oncoribosomes contribute to cancer pathogenesis and highlights the therapeutic potential of directly or indirectly targeting altered ribosomes, offering insights for the development of personalized treatment strategies.

P1/2, N=62, Not yet recruiting, The First Affiliated Hospital, Zhejiang University College of Medicine; The First Affiliated Hospital of Zhejiang University Medical College

P1/2, N=28, Active, not recruiting, University of Colorado, Denver | Trial primary completion date: Aug 2025 --> Aug 2026

P1/2, N=458, Recruiting, Ascentage Pharma Group Inc. | N=284 --> 458 | Trial completion date: Aug 2026 --> Sep 2029 | Trial primary completion date: Aug 2025 --> Sep 2028

Clinical responses were seen exclusively in patients with MDS, which suggests that dose optimization or combination with cytoreductive agents may be necessary for eliciting clinical activity in AML. This trial was registered at www.ClinicalTrials.gov as #NCT04874194.

P1/2, N=62, Not yet recruiting, First Affiliated Hospital of Zhejiang University

Methyl-angolensate, byssochlamic-acid, homoharringtonine, piperacillin and cephaeline were potentially targeted therapeutic compounds for hub genes based on molecular docking. Our study firstly provides new insight into the genetic crosstalk between metastatic melanoma and vitiligo that may facilitate the development of personalized treatments.

P2/3, N=610, Recruiting, Institute of Hematology & Blood Diseases Hospital, China | Not yet recruiting --> Recruiting

We established the stromal-AML co-culture system (HS-5 cells with U937, HL-60, and primary AML cells) and the humanized mouse AML model to systematically evaluate eIF4E's role in modulating homoharringtonine (HHT) and arsenic trioxide (ATO) cytotoxicity within the leukemic microenvironment. Genetic perturbation studies demonstrated that eIF4E knockdown sensitized AML cells to HHT/ATO, whereas its overexpression conferred therapeutic resistance. eIF4E may play an essential role in mediating the cytotoxic effects of HHT and ATO, representing a novel therapeutic target in AML.