Phase II study of the GPC3-derived peptide vaccine as an adjuvant therapy for hepatocellular carcinoma patients

Yu Sawada, Toshiaki Yoshikawa, Kazuya Ofuji, Mayuko Yoshimura, Nobuhiro Tsuchiya, Mari Takahashi, Daisuke Nobuoka, Naoto Gotohda, Shinichiro Takahashi, Yuichiro Kato, Masaru Konishi, Taira Kinoshita, Masafumi Ikeda, Kohei Nakachi, Naoya Yamazaki, Shoichi Mizuno, Tadatoshi Takayama, Kenji Yamao, Katsuhiko Uesaka, Junji FuruseItaru Endo, Tetsuya Nakatsura

Research output: Contribution to journalArticlepeer-review

127 Citations (Scopus)


The recurrence rates of Hepatocellular carcinoma (HCC) are high, necessitating novel and effective adjuvant therapies. Therefore, we conducted a phase II study of glypican-3 (GPC3) peptide vaccine as an adjuvant therapy for HCC patients. Forty-one patients with initial HCC who had undergone surgery or radiofrequency ablation (RFA) were analyzed in this phase II, open-label, single-arm trial. Ten vaccinations were performed for 1 y after curative treatment. We also investigated case-control subjects, where selected patients treated surgically during the same period were analyzed. The expression of GPC3 in the available primary tumors was determined by immunohistochemical analysis. Six patients received RFA therapy while 35 received surgery. The recurrence rate tended to be lower in the 35 patients treated with surgery plus vaccination compared to 33 patients who underwent surgery alone (28.6% vs. 54.3% and 39.4% vs. 54.5% at 1 and 2 y, respectively; p = 0.346, 0.983). Twenty-five patients treated with surgery and vaccination had GPC3-positive tumors; the recurrence rate in this group was significantly lower compared to that in 21 GPC3-positive patients who received surgery only (24% vs. 48% and 52.4% vs. 61.9% at 1 and 2 y, respectively; p = 0.047, 0.387). The GPC3 peptide vaccine improved the 1-y recurrence rate in patients with GPC3-positive tumors. This study demonstrated that GPC3 expression by the primary tumor may be used as a biomarker in a putative larger randomized clinical trial to determine the efficacy of the GPC3-derived peptide vaccine.

Original languageEnglish
Article numbere1129483
Issue number5
Publication statusPublished - 2016 May 3
Externally publishedYes


  • Adjuvant therapy
  • CTL
  • Glypican-3
  • HCC
  • Peptide vaccine

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology
  • Oncology


Dive into the research topics of 'Phase II study of the GPC3-derived peptide vaccine as an adjuvant therapy for hepatocellular carcinoma patients'. Together they form a unique fingerprint.

Cite this