An Advanced Intrahepatic Cholangiocarcinoma Patient Benefits from Personalised Immunotherapy and FGFR2 Inhibiter

Advanced intrahepatic cholangiocarcinoma (ICC) is a highly aggressive malignancy which shows poor response to first-line treatments including radiotherapy, chemotherapy and targeted therapy. Its prognosis is extremely poor, with an overall survival of less than one year. Individualised neoantigen vaccines have shown to be effective in boosting the immune response against tumours, and neoantigen-based peptide as well as DNA and dendritic cell vaccines are now under investigations in clinical trials. Here we reported a case of a patient with advanced ICC who progressed after receiving targeted combination immunotherapy after relapsing and received immunotherapy based on personalised neoantigen nanovaccine. This patient administered a neoantigen-based peptide nanovaccine customised according to her gene mutation spectrum after progression on first-line therapy. Clinical efficacy and anti-tumour immune responses were assessed by imaging, interferon-γ ELISPOT assay and intracellular cytokine staining. A specific tumour killing effect was effectively produced by the T cells mediated by neoantigen nanovaccine, and the response lasted up to 25 months. This provides a new possibility to treat advanced ICC with neoantigen-based immunotherapy.