Carlos Jorge Antonio Elias Godoy, Tavares Mauricio Temotheo, Lima Keli, de Almeida Larissa Costa, de Barros Waitman Karoline, Costa-Lotufo Leticia Veras, Parise-Filho Roberto, Machado-Neto João Agostinho
Department of Pharmacology, Institute of Biomedical Sciences, University of São Paulo, São Paulo, Brazil.
Department of Pharmacy, Faculty of Pharmaceutical Science, University of São Paulo, São Paulo, Brazil.
Cell Death Discov. 2025 Apr 6;11(1):147. doi: 10.1038/s41420-025-02446-4.
Epigenetic modifications significantly influence gene expression and play crucial roles in various biological processes, including carcinogenesis. This study investigates the effects of novel purine-benzohydroxamate compounds, particularly 4 f, as hybrid kinase/histone deacetylase (HDAC) inhibitors in hematological malignancies, focusing on acute myeloid leukemia (AML). Our results demonstrate that these compounds selectively reduce cell viability in blood cancer cells, with inhibitory concentration values indicating higher potency against neoplastic cells compared to normal leukocytes. Mechanistically, 4 f induces apoptosis and cell cycle arrest, promoting differentiation in leukemia cells, while effectively inhibiting HDAC activity. Furthermore, 4 f enhances the therapeutic efficacy of venetoclax, a BCL2 inhibitor, in AML models sensitive and resistant to this drug. The combination treatment significantly increases apoptosis and reduces cell viability, suggesting a synergistic effect that may overcome drug resistance. This study provides valuable insights into the potential of HDAC inhibitors, particularly 4 f, as a promising therapeutic strategy for treating resistant hematological malignancies. Our findings underscore the importance of further exploring hybrid kinase/HDAC inhibitors in combination therapies to improve outcomes in patients with acute leukemias and other hematological malignancies.
表观遗传修饰显著影响基因表达,并在包括致癌作用在内的各种生物学过程中发挥关键作用。本研究调查了新型嘌呤 - 苯甲羟肟酸化合物,特别是4f,作为血液系统恶性肿瘤中混合激酶/组蛋白去乙酰化酶(HDAC)抑制剂的作用,重点关注急性髓性白血病(AML)。我们的结果表明,这些化合物选择性地降低血液癌细胞的活力,抑制浓度值表明其对肿瘤细胞的效力高于正常白细胞。从机制上讲,4f诱导细胞凋亡和细胞周期停滞,促进白血病细胞分化,同时有效抑制HDAC活性。此外,在对BCL2抑制剂维奈托克敏感和耐药的AML模型中,4f增强了其治疗效果。联合治疗显著增加细胞凋亡并降低细胞活力,表明可能克服耐药性的协同效应。本研究为HDAC抑制剂,特别是4f,作为治疗耐药血液系统恶性肿瘤的有前景的治疗策略的潜力提供了有价值的见解。我们的研究结果强调了进一步探索混合激酶/HDAC抑制剂联合疗法以改善急性白血病和其他血液系统恶性肿瘤患者预后的重要性。