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Poster display session

4881 - Phase I study of glypican-3-derived peptide vaccine therapy for patients with refractory pediatric solid tumors


10 Sep 2017


Poster display session


Ako Hosono


Annals of Oncology (2017) 28 (suppl_5): v403-v427. 10.1093/annonc/mdx376


A. Hosono1, T. Yoshikawa2, N. Tsuchiya3, N. Fujinami3, K. Saito3, S. Mizuno3, T. Nakatsura3

Author affiliations

  • 1 Division Of Pediatric Oncology, National Cancer Center Hospital East, 277-8577 - Kashiwa/JP
  • 2 Division Of Cancer Immunotherapy, Exploratory Oncology Research And Clinical Trial Center, National Cancer Center, Chiba/JP
  • 3 Cancer Immunotherapy, Exploratory Oncology Research & Clinical Trial Center , National Cancer Center, Chiba/JP


Abstract 4881


The carcinoembryonic antigen glypican-3 (GPC3) is a good target of anticancer immunotherapy against pediatric solid tumors expressing GPC3. In this non-randomized, open-label, phase I clinical trial, we analyzed the safety and efficacy of GPC3-peptide vaccination in patients with pediatric solid tumors.


We conducted a phase I study of pediatric patients with solid tumors. GPC3 is a target of anticancer immunotherapy against some pediatric solid tumor especially hepatoblastoma. Vaccinations were carried out biweekly from the first until disease progression with the primary endpoint being the safety of GPC3-peptide vaccination and the secondary endpoints being immune response, as measured by interferon (IFN)-γ enzyme-linked immunospot assay and Dextramer staining, and the clinical outcomes of tumor response, progression free survival (PFS), and overall survival (OS).


A total of 18 patients (7 hepatoblastoma, 4 rhabdomyosarcoma, 3 brain tumor, 1 MRT, 1 pancreatoblastoma,1 Wilms tumor, 1 germ cell tumor) were enrolled from 5 hospitals, all cases showed no dose-limiting toxicity (DLT), which was the primary endpoint of this trial. No grade 3-4 hematological and non-hematological toxicity due to GPC3 vaccine therapy occurred. Clinical benefit ratio was 66.7% with six long SD (SD during more than 24 weeks; 5 hepatoblastoma,1 MRT) The GPC3-peptide vaccine induced a GPC3-specific CTL response in seven patients, with PFS and OS significantly longer in patients with high GPC3-specific CTL frequencies than in those with low frequencies. Furthermore, we established GPC3-peptide-specific CTL clones from a resected-recurrent tumor from one patient, with these cells exhibiting GPC3-peptide-specific cytokine secretion.


GPC3 peptide vaccine therapy is well tolerated in heavily pretreated pediatric patients with refractory solid tumors especially hepatoblastoma with acceptable toxicities in outpatient setting and keep good QOL.

Clinical trial identification

Legal entity responsible for the study

AKO hosono




All authors have declared no conflicts of interest.

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