Yurkevicz Annali M, Liu Yanfeng, Katz Samuel G, Glazer Peter M
Department of Therapeutic Radiology, Yale University School of Medicine, New Haven, Connecticut.
Department of Genetics, Yale University School of Medicine, New Haven, Connecticut.
Mol Cancer Ther. 2025 Jan 2;24(1):105-117. doi: 10.1158/1535-7163.MCT-23-0809.
Identifying an optimal antigen for targeted cancer therapy is challenging as the antigen landscape on cancerous tissues mimics that of healthy tissues, with few unique tumor-specific antigens identified in individual patients. pH low insertion peptide (pHLIP) acts as a unique delivery platform that can specifically target the acidic microenvironment of tumors, sparing healthy tissue in the process. We developed a pHLIP-peptide conjugate to deliver the SIINFEKL peptide, an immunogenic fragment of ovalbumin (OVA), to tumor cells in vivo. When processed intracellularly, SIINFEKL is presented for immune recognition through the major histocompatibility complex (MHC) class I pathway. We observed selective delivery of pHLIP-SIINFEKL both in vitro and in vivo using fluorescently labeled constructs. In vitro, treatment of melanoma tumor cells with pHLIP-SIINFEKL resulted in recognition by SIINFEKL-specific T cells (OT1), leading to T-cell activation and effector function. Mechanistically, we show that this recognition by OT1 T cells was abrogated by siRNA/shRNA knockdown of multiple components within the MHC class I pathway in the target tumor cells, indicating that an intact antigen processing pathway in the cancer cells is necessary to mediate the effect of pHLIP-directed SIINFEKL delivery. In vivo, pHLIP-SIINFEKL treatment of tumor-bearing mice resulted in the recruitment of OT1 T cells and suppression of tumor growth in two syngeneic tumor models in immunocompetent mice, with no effect when mutating either the pHLIP or SIINFEKL components of the conjugate. These results suggest that pHLIP-mediated peptide delivery can be used to deliver novel artificial antigens that can be targeted by cell-based therapies.
识别用于靶向癌症治疗的最佳抗原具有挑战性,因为癌组织上的抗原格局与健康组织相似,在个体患者中很少能鉴定出独特的肿瘤特异性抗原。pH低插入肽(pHLIP)作为一种独特的递送平台,可特异性靶向肿瘤的酸性微环境,在此过程中使健康组织免受影响。我们开发了一种pHLIP-肽偶联物,用于在体内将卵清蛋白(OVA)的免疫原性片段SIINFEKL肽递送至肿瘤细胞。当在细胞内进行加工时,SIINFEKL通过主要组织相容性复合体(MHC)I类途径呈递以供免疫识别。我们使用荧光标记构建体在体外和体内均观察到pHLIP-SIINFEKL的选择性递送。在体外,用pHLIP-SIINFEKL处理黑色素瘤肿瘤细胞导致被SIINFEKL特异性T细胞(OT1)识别,从而导致T细胞活化和效应功能。从机制上讲,我们表明,通过对靶肿瘤细胞中MHC I类途径内的多个成分进行siRNA/shRNA敲低,OT1 T细胞的这种识别被消除,这表明癌细胞中完整的抗原加工途径对于介导pHLIP定向的SIINFEKL递送的作用是必要的。在体内,用pHLIP-SIINFEKL处理荷瘤小鼠导致OT1 T细胞募集,并在免疫活性小鼠的两种同基因肿瘤模型中抑制肿瘤生长,当突变偶联物的pHLIP或SIINFEKL成分时则无此效果。这些结果表明,pHLIP介导的肽递送可用于递送可被基于细胞的疗法靶向的新型人工抗原。