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An Advanced Intrahepatic Cholangiocarcinoma Patient Benefits from Personalized Immunotherapy

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Abstract

Advanced intrahepatic cholangiocarcinoma (ICC) is a highly aggressive malignancy characterized by limited response to standard therapeutic modalities, such as radiotherapy, chemotherapy, and targeted therapy. The prognosis for patients with advanced ICC is exceedingly bleak, with an overall survival of less than 1 year. In recent years, personalized neoantigen vaccines have emerged as a promising approach to augment the immune response against tumors. Clinical investigations are currently underway to evaluate the efficacy of neoantigen-based peptide, DNA, and dendritic cell vaccines. Herein, we present a noteworthy case of advanced ICC patients who experienced disease progression following relapse and subsequently received immunotherapy with a personalized neoantigen nanovaccine. This innovative treatment strategy involved the administration of a custom-designed neoantigen-based peptide nanovaccine tailored to the patient’s specific gene mutation profile subsequent to failure of first-line therapy. The clinical efficacy and anti-tumor immune responses were evaluated using various methods, including imaging, interferon-γ ELISPOT assay, and intracellular cytokine staining. Notably, the neoantigen nanovaccine elicited a robust and specific tumor-killing effect mediated by T cells, resulting in a durable response lasting up to 25 months. These findings highlight the potential of neoantigen-based immunotherapy as a novel therapeutic avenue for the management of advanced ICC.

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Data Availability

The raw data supporting the conclusions of this article will be made available by the authors, without undue reservation.

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Acknowledgements

We thank the patient and her families, as well as the members who participated in the study, for making this research possible.

Funding

The study was supported by the National Natural Science Foundation of Nanjing University of Chinese Medicine (No. XZR2023075), the Hospital Management Research of Nanjing Drum Tower Hospital (No. NDYGN2023002), and Medical Research of Jiangsu Health Committee (No. H2023068).

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Author notes

  1. Sihui Zhu and Chenxi Liu equally contributed to this work.

Authors and Affiliations

  1. Comprehensive Cancer Centre, Department of Oncology, Nanjing Drum Tower Hospital, The Affiliated Hospital of Nanjing University Medical School, 321 Zhongshan Road, Jiangsu Province, 210008, Nanjing, China

    Sihui Zhu, Chenxi Liu, Yunchen Jin, Mingzhen Zhou, Chen Xu, Jie Shao, Qin Liu, Jia Wei, Jie Shen & Baorui Liu

  2. Comprehensive Cancer Centre of Nanjing International Hospital, Medical School of Nanjing University, Nanjing, China

    Sihui Zhu & Chen Xu

  3. Clinical Cancer Institute of Nanjing University, 321 Zhongshan Road, Jiangsu Province, 210008, Nanjing, China

    Sihui Zhu, Chenxi Liu, Yunchen Jin, Mingzhen Zhou, Chen Xu, Jie Shao, Qin Liu, Jia Wei, Jie Shen & Baorui Liu

  4. Department of Radiology of Drum Tower Hospital, Medical School of Nanjing University, Nanjing, China

    Hailong Zhang

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  1. Sihui Zhu
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Contributions

JShen and BRL designed the clinical trial. SHZ, CXL, JShen, and BRL drafted the manuscript and provided figures. SHZ, CXL, YCJ, HLZ, MZZ, CX, QL, JW, JShao, and JShen acquired, analyzed, and interpreted the data. JShen and BL revised the manuscript critically for important intellectual content and agreed to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved. All authors contributed to the article and approved the submitted version.

Corresponding authors

Correspondence to Jie Shen or Baorui Liu.

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The patients/participants provided written informed consent to participate in this study. Written informed consent was obtained from the individual(s) for the publication of any potentially identifiable images or data included in this article.

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The authors declare that they have no competing interests.

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Cite this article

Zhu, S., Liu, C., Jin, Y. et al. An Advanced Intrahepatic Cholangiocarcinoma Patient Benefits from Personalized Immunotherapy. Inflammation (2024). https://doi.org/10.1007/s10753-024-02003-8

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  • DOI: https://doi.org/10.1007/s10753-024-02003-8

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