Synribo (omacetaxine mepesuccinate)

We report a case of a 42-year-old patient with EVI1-positive AML harboring the MLL-AF6 fusion gene, who failed to achieve remission after undergoing standard "IA" induction therapy and was then treated with VAH (venetoclax, azacitidine, and homoharringtonine) consolidation chemotherapy. This case suggests that the combination of ATRA with the VAH regimen may demonstrate promising efficacy and an acceptable safety profile in patients with EVI1-positive AML who are refractory to conventional chemotherapy. However, further clinical studies are required to confirm its wider applicability.

P1/2, N=682, Recruiting, Ascentage Pharma Group Inc. | N=458 --> 682

In a clinical trial (ChiCTR2500097714), erlotinib (an EGFR inhibitor) combined with azacitidine plus the HAG regimen, which consists of homoharringtonine, a low dose of cytarabine, and granulocyte colony-stimulating factor priming, achieves a remission rate of 75% in relapsed/refractory AML, likely via MMRN1/EGFR axis blockade. Our findings establish MMRN1 as a dual-functional target for LSC maintenance and immune evasion and propose that disrupting MMRN1 or EGFR remodels the immunosuppressive tumor microenvironment, offering a promising strategy for AML immunotherapy.

P=N/A, N=20, The FIrst Affiliated Hospital, College of Medicine, Zhejiang University; The FIrst Affiliated Hospital, College of Medicine, Zhejiang University

P=N/A, N=60, The First Hospital of China Medical University; The First Hospital of China Medical University

Furthermore, we identify a synergistic antitumor effect between KPT-330 and translation inhibitors, including everolimus and homoharringtonine. Notably, the disruptive impact of KPT-330 on CRC homeostasis extends to other cancer cell lineages, underscoring its broad mechanistic relevance. Collectively, our findings elucidate a novel mechanism through which KPT-330 destabilizes CRC via translational dysregulation and highlight its potential therapeutic utility in combination regimens for DDLPS.

P1, N=94, Not yet recruiting, Zhongda Hospital

P2, N=40, Enrolling by invitation, Guangdong Second Provincial General Hospital

PLD1-inhibitor remodeled phospholipid metabolism, induced ferroptosis and restored QUIZOM response in LSC. Our findings provided the therapeutic and resistant mechanisms of QUIZOM and paved the way for targeted interventions in this AML subtype.

P2, N=61, Not yet recruiting, First People's Hospital of Hangzhou