Cancer Therapeutics Program, Peter MacCallum Cancer Centre, East Melbourne, VIC, Australia;
Blood. 2014 Feb 27;123(9):1341-52. doi: 10.1182/blood-2013-03-488114. Epub 2014 Jan 10.
Epigenetic modifying enzymes such as histone deacetylases (HDACs), p300, and PRMT1 are recruited by AML1/ETO, the pathogenic protein for t(8;21) acute myeloid leukemia (AML), providing a strong molecular rationale for targeting these enzymes to treat this disease. Although early phase clinical assessment indicated that treatment with HDAC inhibitors (HDACis) may be effective in t(8;21) AML patients, rigorous preclinical studies to identify the molecular and biological events that may determine therapeutic responses have not been performed. Using an AML mouse model driven by expression of AML1/ETO9a (A/E9a), we demonstrated that treatment of mice bearing t(8;21) AML with the HDACi panobinostat caused a robust antileukemic response that did not require functional p53 nor activation of conventional apoptotic pathways. Panobinostat triggered terminal myeloid differentiation via proteasomal degradation of A/E9a. Importantly, conditional A/E9a deletion phenocopied the effects of panobinostat and other HDACis, indicating that destabilization of A/E9a is critical for the antileukemic activity of these agents.
表观遗传修饰酶,如组蛋白去乙酰化酶(HDACs)、p300 和 PRMT1,被 AML1/ETO 募集,AML1/ETO 是 t(8;21)急性髓系白血病(AML)的致病蛋白,为靶向这些酶治疗这种疾病提供了强有力的分子依据。尽管早期临床评估表明,HDAC 抑制剂(HDACi)治疗 t(8;21)AML 患者可能有效,但尚未进行严格的临床前研究来确定可能决定治疗反应的分子和生物学事件。使用由 AML1/ETO9a(A/E9a)表达驱动的 AML 小鼠模型,我们证明了用 HDACi 帕比司他治疗携带 t(8;21)AML 的小鼠会引起强烈的抗白血病反应,而不需要功能性 p53 或激活传统的凋亡途径。帕比司他通过蛋白酶体降解 A/E9a 触发终末髓样分化。重要的是,条件性 A/E9a 缺失模拟了帕比司他和其他 HDACi 的作用,表明 A/E9a 的不稳定性对于这些药物的抗白血病活性至关重要。