Regeneron Pharmaceuticals, Inc., Tarrytown, NY, 10591, United States.
Sci Rep. 2019 Dec 27;9(1):20068. doi: 10.1038/s41598-019-56670-x.
Bispecific antibodies (bsAb) that bridge tumor cells and CD3-positive effector T cells are being developed against many tumor cell targets. While tumor cell factors other than target expression level appear to play a role in determining the efficacy of CD3 bsAb, the identity of such factors remains largely unknown. Using a co-culture system of primary human T cells and B lymphoma cell lines, we demonstrate a range of sensitivities to CD20xCD3 bsAb that is independent of CD20 surface expression. To identify genes that modulate tumor cell sensitivity to CD3 bsAb, we employed a genome-scale CRISPR activation screen in a CD20xCD3-sensitive human B lymphoma cell line. Among the most highly enriched sgRNAs were those targeting genes with predicted effects on cell-cell adhesion, including sialophorin (SPN). Increased expression of SPN impeded tumor cell clustering with T cells, thereby limiting CD3 bsAb-mediated tumor cell lysis. This inhibitory effect of SPN appeared to be dependent on sialylated core 2 O-glycosylation of the protein. While SPN is not endogenously expressed in the majority of B cell lymphomas, it is highly expressed in acute myeloid leukemia. CRISPR-mediated SPN knockout in AML cell lines facilitated T cell-tumor cell clustering and enhanced CD3 bsAb-mediated AML cell lysis. In sum, our data establish that the cell cross-linking mechanism of CD3 bsAb is susceptible to subversion by anti-adhesive molecules expressed on the tumor cell surface. Further evaluation of anti-adhesive pathways may provide novel biomarkers of clinical response and enable the development of effective combination regimens for this promising therapeutic class.
双特异性抗体(bsAb)可桥接肿瘤细胞和 CD3 阳性效应 T 细胞,针对许多肿瘤细胞靶标进行开发。虽然除靶标表达水平之外的肿瘤细胞因子似乎在决定 CD3 bsAb 的疗效方面发挥作用,但这些因子的身份在很大程度上仍然未知。使用原代人 T 细胞和 B 淋巴瘤细胞系的共培养系统,我们证明了对 CD20xCD3 bsAb 的一系列敏感性,这与 CD20 表面表达无关。为了确定调节肿瘤细胞对 CD3 bsAb 敏感性的基因,我们在 CD20xCD3 敏感的人类 B 淋巴瘤细胞系中使用了全基因组 CRISPR 激活筛选。在高度富集的 sgRNA 中,有一些靶向基因具有预测的细胞间粘附效应,包括唾液酸蛋白(SPN)。SPN 的表达增加会阻碍肿瘤细胞与 T 细胞的聚集,从而限制 CD3 bsAb 介导的肿瘤细胞裂解。SPN 的这种抑制作用似乎依赖于该蛋白的唾液酸化核心 2 O-糖基化。虽然 SPN 不在大多数 B 细胞淋巴瘤中内源性表达,但它在急性髓系白血病中高度表达。CRISPR 介导的 AML 细胞系中的 SPN 敲除促进了 T 细胞-肿瘤细胞聚集,并增强了 CD3 bsAb 介导的 AML 细胞裂解。总之,我们的数据表明,CD3 bsAb 的细胞交联机制容易受到肿瘤细胞表面表达的抗粘附分子的颠覆。进一步评估抗粘附途径可能为临床反应提供新的生物标志物,并为这一有前途的治疗类别开发有效的联合治疗方案。