The Walter and Eliza Hall Institute of Medical Research, Melbourne, VIC, Australia.
Department of Medical Biology, University of Melbourne, Melbourne, VIC, Australia.
Blood. 2021 May 20;137(20):2721-2735. doi: 10.1182/blood.2020010167.
Selective targeting of BCL-2 with the BH3-mimetic venetoclax has been a transformative treatment for patients with various leukemias. TP-53 controls apoptosis upstream of where BCL-2 and its prosurvival relatives, such as MCL-1, act. Therefore, targeting these prosurvival proteins could trigger apoptosis across diverse blood cancers, irrespective of TP53 mutation status. Indeed, targeting BCL-2 has produced clinically relevant responses in blood cancers with aberrant TP-53. However, in our study, TP53-mutated or -deficient myeloid and lymphoid leukemias outcompeted isogenic controls with intact TP-53, unless sufficient concentrations of BH3-mimetics targeting BCL-2 or MCL-1 were applied. Strikingly, tumor cells with TP-53 dysfunction escaped and thrived over time if inhibition of BCL-2 or MCL-1 was sublethal, in part because of an increased threshold for BAX/BAK activation in these cells. Our study revealed the key role of TP-53 in shaping long-term responses to BH3-mimetic drugs and reconciled the disparate pattern of initial clinical response to venetoclax, followed by subsequent treatment failure among patients with TP53-mutant chronic lymphocytic leukemia or acute myeloid leukemia. In contrast to BH3-mimetics targeting just BCL-2 or MCL-1 at doses that are individually sublethal, a combined BH3-mimetic approach targeting both prosurvival proteins enhanced lethality and durably suppressed the leukemia burden, regardless of TP53 mutation status. Our findings highlight the importance of using sufficiently lethal treatment strategies to maximize outcomes of patients with TP53-mutant disease. In addition, our findings caution against use of sublethal BH3-mimetic drug regimens that may enhance the risk of disease progression driven by emergent TP53-mutant clones.
选择性靶向 BCL-2 的 BH3 模拟物 venetoclax 已成为治疗各种白血病患者的变革性治疗方法。TP-53 在上游控制着 BCL-2 及其生存相关蛋白(如 MCL-1)的凋亡。因此,靶向这些生存相关蛋白可以触发各种血液癌症的凋亡,而与 TP53 突变状态无关。事实上,针对 BCL-2 的治疗已在具有异常 TP-53 的血液癌症中产生了临床相关的反应。然而,在我们的研究中,TP53 突变或缺失的髓系和淋巴白血病细胞会与具有完整 TP-53 的同基因对照竞争,除非应用足够浓度的靶向 BCL-2 或 MCL-1 的 BH3 模拟物。令人惊讶的是,如果抑制 BCL-2 或 MCL-1 的作用不足以致死,肿瘤细胞会逃脱并随着时间的推移而茁壮成长,部分原因是这些细胞中 BAX/BAK 的激活阈值增加。我们的研究揭示了 TP-53 在塑造对 BH3 模拟药物的长期反应中的关键作用,并调和了 venetoclax 初始临床反应的不同模式,随后是 TP53 突变慢性淋巴细胞白血病或急性髓系白血病患者的治疗失败。与针对 BCL-2 或 MCL-1 的 BH3 模拟物剂量单独亚致死相比,针对两种生存相关蛋白的联合 BH3 模拟物方法增强了杀伤力,并持久地抑制了白血病负担,而与 TP53 突变状态无关。我们的研究结果强调了使用足够致死的治疗策略来最大化 TP53 突变疾病患者结局的重要性。此外,我们的研究结果警告不要使用可能增加由新兴 TP53 突变克隆驱动的疾病进展风险的亚致死 BH3 模拟药物方案。