Nash Amanda, DeBonis Jonathon, Murungi Danna, Castillo Bertha, Kim Boram, Hu Fangheng, Chambers Courtney, Nguyen Annie, Hernandez Andrea, Wang Zeshi, Rios Peter D, Ghani Sofia, Joshi Ira, Isa Douglas, Zheng Ningbo, Peng Weiyi, Igoshin Oleg A, Oberholzer Jose, Hodges H Courtney, Reticker-Flynn Nathan, Veiseh Omid
Department of Bioengineering, Rice University, Houston, Texas, USA.
Department of Molecular and Cellular Biology, Baylor College of Medicine, Houston, Texas, USA.
J Immunother Cancer. 2025 Apr 1;13(4):e010685. doi: 10.1136/jitc-2024-010685.
Curative responses to immunotherapy require the generation of robust systemic immunity with limited toxicity. Recruitment of T cell populations such as precursor exhausted T cells (Tpex) from lymphoid tissues to tumors is a hallmark of effective treatment. However, the ability to efficiently induce this recruitment is lacking in current immunotherapy approaches. Furthermore, systemic administration of immunotherapies frequently results in dose-limiting toxicities, yielding an inadequate therapeutic window for eliciting durable responses.
In this investigation, we evaluated the safety and antitumor efficacy of locally administered interleukin 12 (IL-12) using a clinically translatable cytokine delivery platform (NCT05538624) to identify Tpex recruitment capabilities at tolerable cytokine doses.
We show IL-12 cytokine factories can effectively treat a broad spectrum of cancer types. Single-cell RNA sequencing data suggests that the antitumor efficacy seen in our studies was due to retinal pigmented epithelial cells-mIL12 treatment inducing differentiation of Tpex cells within the tumor microenvironment. When administered in combination with checkpoint therapy, IL-12 cytokine factory treatment generated systemic abscopal immunity, preventing subcutaneous tumor outgrowth in 8/9 mice with colorectal cancer and lung metastasis in mice with melanoma. Furthermore, this platform was well tolerated in a non-human primate without signs of toxicity.
Our new immunotherapy approach provides a robust strategy for inducing Tpex recruitment and systemic immunity against a range of solid peritoneal malignancies, many incurable with current immunotherapy strategies. Notably, these features were achieved using IL-12, and by leveraging our technology, we avoided the toxicities that have prevented the translation of IL-12 to the clinic. Our findings provide a strong rationale for the clinical development of IL-12 cytokine factories.
免疫疗法的治愈反应需要在毒性有限的情况下产生强大的全身免疫。将T细胞群体(如前体耗竭T细胞,Tpex)从淋巴组织募集到肿瘤是有效治疗的一个标志。然而,目前的免疫疗法缺乏有效诱导这种募集的能力。此外,免疫疗法的全身给药经常导致剂量限制性毒性,从而产生一个不充分的治疗窗口来引发持久反应。
在本研究中,我们使用一个具有临床可转化性的细胞因子递送平台(NCT05538624)评估了局部施用白细胞介素12(IL-12)的安全性和抗肿瘤疗效,以确定在可耐受的细胞因子剂量下Tpex的募集能力。
我们表明IL-12细胞因子工厂可以有效治疗多种癌症类型。单细胞RNA测序数据表明,我们研究中观察到的抗肿瘤疗效是由于视网膜色素上皮细胞-mIL12治疗诱导了肿瘤微环境中Tpex细胞的分化。当与检查点疗法联合使用时,IL-12细胞因子工厂治疗产生了全身远隔效应免疫,在8/9只患有结直肠癌的小鼠中预防了皮下肿瘤生长,在患有黑色素瘤的小鼠中预防了肺转移。此外,该平台在非人类灵长类动物中耐受性良好,没有毒性迹象。
我们的新免疫疗法为诱导Tpex募集和针对一系列实体腹膜恶性肿瘤的全身免疫提供了一个强大的策略,这些肿瘤中有许多是目前免疫疗法无法治愈的。值得注意的是,这些特性是通过使用IL-12实现的,并且通过利用我们的技术,我们避免了那些阻碍IL-12转化为临床应用的毒性。我们的发现为IL-12细胞因子工厂的临床开发提供了有力的理论依据。