An “All‐In‐One” Immunomodulator‐Engineered Clinical Translatable Immunotherapy of Advanced Hepatocellular Carcinoma

Author:

Xie Ting1,Huang Cong1,Wang Yuqing1,Zhang Haitao1,Guo Pei1,Phann Thuy Thu1,Cheng Yao1,Lei Longtianyang1,Tao Zhenghao1,Gao Qing1,Wei Hua1ORCID,Yu Cui‐Yun12

Affiliation:

1. Hunan Province Cooperative Innovation Center for Molecular Target New Drug Study School of Pharmaceutical Science Hengyang Medical School University of South China Hengyang 421001 China

2. Affiliated Hospital of Hunan Academy of Chinese Medicine Hunan Academy of Chinese Medicine Changsha 410013 China

Abstract

AbstractClinical treatment of advanced hepatocellular carcinoma (HCC) remains a significant challenge. Utilizing 1‐bromoacetyl‐3,3‐dinitroazetidine (RRx‐001) to downregulate the expression of innate immune checkpoint molecule, cluster of differentiation 47 (CD47), provides a powerful means for treating advanced HCC containing abundant immunosuppressive macrophages. Herein engineering of a previously optimized Doxorubicin (DOX)‐delivery nanoplatform based on sodium alginate is reported to further co‐deliver RRx‐001 (biotinylated aldehyde alginate–doxorubicin micelle prodrug nanoplatform, BEA‐D@R) for efficient immunotherapy of advanced HCC. This groundbreaking  technique reveals the “all‐in‐one” immunotherapeutic functionalities of RRx‐001. Besides the previously demonstrated functions of downregulating CD47 expression and increasing reactive nitrogen species (RNS) generation, another key function of RRx‐001 for downregulating the expression of the adaptive immune checkpoint molecule programmed cell death 1 ligand 1 (PDL1) is first uncovered here. Combined with the reactive oxygen species (ROS) generation and an upregulated “eat me” signal level of DOX, BEA‐D@R collectively increases RNS generation, enhances T‐cell infiltration, and maximizes macrophage phagocytosis, leading to an average of 40% tumor elimination in a mice model bearing an initial tumor volume of ≈300 mm3 that mimics advanced HCC. Overall, the “all‐in‐one” immunotherapeutic functionalities of a clinical translatable nanoplatform are uncovered for enhanced immunotherapy of advanced HCC.

Publisher

Wiley

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