ALPL-1 是骨肉瘤嵌合抗原受体治疗的靶点。

ALPL-1 is a target for chimeric antigen receptor therapy in osteosarcoma.

机构信息

Translational Research Unit, Department of Cellular Therapy, Oslo University Hospital, Oslo, Norway.

Josep Carreras Leukemia Research Institute, Barcelona, Spain.

出版信息

Nat Commun. 2023 Jun 8;14(1):3375. doi: 10.1038/s41467-023-39097-x.

Abstract

Osteosarcoma (OS) remains a dismal malignancy in children and young adults, with poor outcome for metastatic and recurrent disease. Immunotherapies in OS are not as promising as in some other cancer types due to intra-tumor heterogeneity and considerable off-target expression of the potentially targetable proteins. Here we show that chimeric antigen receptor (CAR) T cells could successfully target an isoform of alkaline phosphatase, ALPL-1, which is highly and specifically expressed in primary and metastatic OS. The target recognition element of the second-generation CAR construct is based on two antibodies, previously shown to react against OS. T cells transduced with these CAR constructs mediate efficient and effective cytotoxicity against ALPL-positive cells in in vitro settings and in state-of-the-art in vivo orthotopic models of primary and metastatic OS, without unexpected toxicities against hematopoietic stem cells or healthy tissues. In summary, CAR-T cells targeting ALPL-1 show efficiency and specificity in treating OS in preclinical models, paving the path for clinical translation.

摘要

骨肉瘤(OS)仍然是儿童和青年的一种令人沮丧的恶性肿瘤,转移性和复发性疾病的预后不良。由于肿瘤内异质性和潜在可靶向蛋白的大量非靶向表达,免疫疗法在 OS 中的效果不如某些其他癌症类型那么有希望。在这里,我们表明嵌合抗原受体(CAR)T 细胞可以成功靶向碱性磷酸酶(ALPL)的一种同工酶,该同工酶在原发性和转移性 OS 中高度特异性表达。第二代 CAR 构建体的靶识别元件基于两种先前显示针对 OS 反应的抗体。转导这些 CAR 构建体的 T 细胞在体外环境中和最先进的原发性和转移性 OS 原位同种异体模型中针对 ALPL 阳性细胞介导有效的细胞毒性,而对造血干细胞或健康组织没有意外的毒性。总之,针对 ALPL-1 的 CAR-T 细胞在治疗 OS 的临床前模型中显示出效率和特异性,为临床转化铺平了道路。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/125f/10250459/6280fd911fb4/41467_2023_39097_Fig1_HTML.jpg

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