Department of Morphology, Surgery and Experimental Medicine, University of Ferrara, Ferrara, Italy.
Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy.
J Cell Physiol. 2018 Oct;233(10):6440-6454. doi: 10.1002/jcp.26539. Epub 2018 Apr 18.
Despite considerable progress in treatment protocols, B-lineage acute lymphoblastic leukemia (B-ALL) displays a poor prognosis in about 15-20% of pediatric cases and about 60% of adult patients. In addition, life-long irreversible late effects from chemo- and radiation therapy, including secondary malignancies, are a growing problem for leukemia survivors. Targeted therapy holds promising perspectives for cancer treatment as it may be more effective and have fewer side effects than conventional therapies. The phosphatidylinositol 3-phosphate kinase (PI3K)/Akt/mechanistic target of rapamycin (mTOR) signaling pathway is a key regulatory cascade which controls proliferation, survival and drug-resistance of cancer cells, and it is frequently upregulated in the different subtypes of B-ALL, where it plays important roles in the pathophysiology, maintenance and progression of the disease. Moreover, activation of this signaling cascade portends a poorer prognosis in both pediatric and adult B-ALL patients. Promising preclinical data on PI3K/Akt/mTOR inhibitors have documented their anticancer activity in B-ALL and some of these novel drugs have entered clinical trials as they could lead to a longer event-free survival and reduce therapy-associated toxicity for patients with B-ALL. This review highlights the current status of PI3K/Akt/mTOR inhibitors in B-ALL, with an emphasis on emerging evidence of the superior efficacy of synergistic combinations involving the use of traditional chemotherapeutics or other novel, targeted agents.
尽管在治疗方案方面取得了相当大的进展,但 B 系急性淋巴细胞白血病(B-ALL)在约 15-20%的儿科病例和约 60%的成年患者中预后较差。此外,化疗和放疗带来的终身不可逆转的晚期副作用,包括继发性恶性肿瘤,是白血病幸存者日益面临的问题。靶向治疗为癌症治疗带来了有希望的前景,因为它可能比传统疗法更有效且副作用更少。磷脂酰肌醇 3-激酶(PI3K)/Akt/雷帕霉素(mTOR)靶蛋白(mTOR)信号通路是一个关键的调节级联,它控制着癌细胞的增殖、存活和耐药性,在不同亚型的 B-ALL 中经常上调,在疾病的病理生理学、维持和进展中发挥重要作用。此外,该信号级联的激活预示着儿科和成年 B-ALL 患者的预后较差。PI3K/Akt/mTOR 抑制剂的有前景的临床前数据记录了它们在 B-ALL 中的抗癌活性,其中一些新型药物已进入临床试验,因为它们可能导致无事件生存时间延长,并减少 B-ALL 患者的治疗相关毒性。这篇综述强调了 PI3K/Akt/mTOR 抑制剂在 B-ALL 中的现状,重点介绍了涉及使用传统化疗药物或其他新型靶向药物的协同组合的优越疗效的新出现证据。