Matar Sittana, Skah Seham, Diomande Liza E, Buss Tim, Hagland Hanne R, Yadav Ajay, Forstrøm Rune J, Dalhus Bjørn, Hestdal Kjetil, Pettersen Rolf D, Richartz Nina
Caedo Oncology AS, Oslo, Norway.
Department of Chemistry, Bioscience and Environmental Engineering, University of Stavanger, Stavanger, Norway.
Mol Cancer Ther. 2025 Jun 4;24(6):816-827. doi: 10.1158/1535-7163.MCT-24-0917.
Therapeutic anti-CD47 monoclonal antibodies (mAbs) are designed to block the CD47-SIRPα checkpoint and promote immune-mediated recognition and elimination of cancer cells. However, current anti-CD47 mAbs have limitations, including off-tumor toxicity and reduced effectiveness in advanced cancers. Additionally, CD47 serves as a death receptor that mediates programmed cancer cell death (PCCD), a mechanism that has not been fully explored in current therapies. In this study, we introduce CO-001, a chimeric bifunctional IgG4 mAb, and its optimized variant CO-005, a bivalent humanized single-chain fragment variable-fragment crystallizable fusion protein. Both CO-001 and CO-005 promoted phagocytosis and PCCD. CO-005, specifically engineered to overcome the safety limitations associated with anti-CD47 antibodies, demonstrates a superior hematologic safety profile in vitro and ex vivo compared with benchmark anti-CD47 antibodies. Notably, CO-005 exhibited no binding to red blood cells, limited binding to white blood cells, and showed no hemagglutination activity. In preclinical models, CO-005 demonstrated potent antitumor activity in B-cell precursor acute lymphoblastic leukemia and Raji lymphoma xenograft models through the dual action of PCCD induction and enhancement of phagocytosis. The ability of CO-005 to trigger strong PCCD while preserving conventional immune responses provides a novel and promising approach for CD47-targeted cancer therapy. Its favorable safety profile, observed in both in vitro and ex vivo studies, positions CO-005 as a promising candidate with potential therapeutic advantages over existing anti-CD47 treatments.
治疗性抗CD47单克隆抗体(mAb)旨在阻断CD47-SIRPα检查点,并促进免疫介导的癌细胞识别和清除。然而,目前的抗CD47单克隆抗体存在局限性,包括肿瘤外毒性和在晚期癌症中疗效降低。此外,CD47作为一种死亡受体,介导程序性癌细胞死亡(PCCD),这一机制在目前的治疗中尚未得到充分探索。在本研究中,我们引入了嵌合双功能IgG4单克隆抗体CO-001及其优化变体CO-005,一种二价人源化单链可变片段-可结晶片段融合蛋白。CO-001和CO-005均促进吞噬作用和PCCD。专门设计用于克服与抗CD47抗体相关的安全局限性的CO-005,与基准抗CD47抗体相比,在体外和体内均表现出卓越的血液学安全性。值得注意的是,CO-005与红细胞无结合,与白细胞的结合有限,且无血凝活性。在临床前模型中,CO-005通过诱导PCCD和增强吞噬作用的双重作用,在B细胞前体急性淋巴细胞白血病和Raji淋巴瘤异种移植模型中显示出强大的抗肿瘤活性。CO-005在触发强烈PCCD的同时保留传统免疫反应的能力,为CD47靶向癌症治疗提供了一种新颖且有前景的方法。在体外和体内研究中观察到的良好安全性,使CO-005成为一种有前景的候选药物,与现有的抗CD47治疗相比具有潜在的治疗优势。