Hematology, Oncology, Stem Cell Transplant, and Regenerative Medicine, Department of Pediatrics, Stanford University, Stanford, CA, USA.
Section for Bioinformatics, Department of Health Technology, Technical University of Denmark, Kongens Lyngby, Denmark.
Nat Commun. 2023 May 22;14(1):2935. doi: 10.1038/s41467-023-38456-y.
Resistance to glucocorticoids (GC) is associated with an increased risk of relapse in B-cell progenitor acute lymphoblastic leukemia (BCP-ALL). Performing transcriptomic and single-cell proteomic studies in healthy B-cell progenitors, we herein identify coordination between the glucocorticoid receptor pathway with B-cell developmental pathways. Healthy pro-B cells most highly express the glucocorticoid receptor, and this developmental expression is conserved in primary BCP-ALL cells from patients at diagnosis and relapse. In-vitro and in vivo glucocorticoid treatment of primary BCP-ALL cells demonstrate that the interplay between B-cell development and the glucocorticoid pathways is crucial for GC resistance in leukemic cells. Gene set enrichment analysis in BCP-ALL cell lines surviving GC treatment show enrichment of B cell receptor signaling pathways. In addition, primary BCP-ALL cells surviving GC treatment in vitro and in vivo demonstrate a late pre-B cell phenotype with activation of PI3K/mTOR and CREB signaling. Dasatinib, a multi-kinase inhibitor, most effectively targets this active signaling in GC-resistant cells, and when combined with glucocorticoids, results in increased cell death in vitro and decreased leukemic burden and prolonged survival in an in vivo xenograft model. Targeting the active signaling through the addition of dasatinib may represent a therapeutic approach to overcome GC resistance in BCP-ALL.
糖皮质激素(GC)耐药与 B 细胞祖细胞急性淋巴细胞白血病(BCP-ALL)的复发风险增加相关。在健康 B 细胞祖细胞中进行转录组学和单细胞蛋白质组学研究,我们在此确定糖皮质激素受体途径与 B 细胞发育途径之间的协调。健康的前 B 细胞最能表达糖皮质激素受体,并且这种发育表达在患者诊断时和复发时的原发性 BCP-ALL 细胞中是保守的。体外和体内糖皮质激素处理原发性 BCP-ALL 细胞表明,B 细胞发育与糖皮质激素途径之间的相互作用对于白血病细胞中的 GC 耐药至关重要。在 GC 治疗后存活的 BCP-ALL 细胞系中的基因集富集分析显示 B 细胞受体信号通路的富集。此外,体外和体内 GC 治疗后存活的原发性 BCP-ALL 细胞表现出晚期前 B 细胞表型,PI3K/mTOR 和 CREB 信号激活。达沙替尼是一种多激酶抑制剂,最有效地靶向 GC 耐药细胞中的这种活跃信号,当与糖皮质激素联合使用时,可导致体外细胞死亡增加,并在体内异种移植模型中减少白血病负担和延长生存时间。通过添加达沙替尼靶向这种活跃信号可能代表克服 BCP-ALL 中 GC 耐药的一种治疗方法。