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Glypican 3 expression in pediatric malignant solid tumors
Yoshiaki Kinoshita1, Sakura Tanaka1, Ryota Souzaki1
1Department of Pediatric Surgery, Kyushu University, Fukuoka, Japan.
Purpose:
Glypican 3 (GPC3) is one of the cell surface heparan sulfate proteoglycans that binds to the cell membrane, and it is known as an oncofetal protein in adult malignant tumors. Clinical trials using a GPC3 peptide vaccine have already been started in Japan as a new immunotherapy for hepatocellular carcinoma in adult patients. To investigate the possibility of GPC3 immunotherapy for pediatric malignant tumors, we assessed the expression of GPC3 in pediatric malignant tumors.
Methods:
Immunohistochemically, the GPC3 expression was examined in 159 pediatric solid tumors, including 35 cases of neuroblastoma, 30 cases of Wilms tumor, 10 cases of hepatoblastoma, 25 cases of germ cell tumors, 56 cases of rhabdomyosarcoma, and 3 cases of other tumors. In addition, to clarify the physiological expression during the fetal to neoinfantile period, autopsy specimens of subjects without any neoplastic diseases were assessed in 9 fetal cases and 21 neoinfantile cases. The serum levels of GPC3 were also analyzed using specimens obtained from 53 subjects by the sandwich enzyme-linked immunosorbent assay method.
Results:
Histologically, a high rate of GPC3 expression was noted in 10 (90.9%) of the 11 subjects with yolk sac tumors and 6 (60.0%) of the 10 subjects with hepatoblastoma. In addition, 9 (30.0%) of the 30 subjects with Wilms tumors and 14 (25.0%) of the 56 subjects with rhabdomyosarcoma were positive for the expression of GPC3. Concerning autopsy specimens, most of the 23 subjects younger than 7 months showed positive findings in the liver (94.7%) and kidney (81.8%). Two subjects (100%) with yolk sac tumors and six (75.0%) of the eight subjects with hepatoblastoma serologically demonstrated a high rate of positive expression. Concerning the distribution of the serum GPC3 level according to age, 8 (80.0%) of the 10 subjects younger than 1 year showed a positive finding, while only 16 (37.3%) of the 43 subjects older than 1 year showed a positive finding.
Conclusion:
Most cases of hepatoblastoma and yolk sac tumor, and some cases of other tumors were found to express GPC3 either histologically or serologically. On the other hand, GPC3 was physiologically expressed during the fetal and neoinfantile period under 1 year of age. Although, more preliminary data and experience are required, patients older than 1 year that show a positive finding for GPC3 are considered to be appropriate candidates to receive the new immunotherapy using GPC3 peptide vaccination.
Insights
Glypican 3 (GPC3) is expressed in pediatric tumors like hepatoblastoma and yolk sac tumors, suggesting its potential for immunotherapy. GPC3 is also physiologically present in infants under one year old.
Area of Science:
- Oncology
- Immunology
- Biochemistry
Background:
- Glypican 3 (GPC3) is an oncofetal protein found on cell surfaces, implicated in adult cancers.
- GPC3-targeted immunotherapy is under investigation for adult hepatocellular carcinoma.
Purpose of the Study:
- To evaluate Glypican 3 (GPC3) expression in pediatric malignant tumors.
- To determine the feasibility of GPC3-based immunotherapy for pediatric cancers.
Main Methods:
- Immunohistochemical analysis of GPC3 expression in 159 pediatric solid tumors.
- Assessment of GPC3 serum levels using sandwich ELISA in 53 subjects.
- Examination of GPC3 expression in fetal and neoinfantile autopsy specimens.
Main Results:
- High GPC3 expression observed in yolk sac tumors (90.9%) and hepatoblastoma (60.0%).
- GPC3 positivity also noted in Wilms tumors (30.0%) and rhabdomyosarcoma (25.0%).
- Physiological GPC3 expression was prevalent in infants under 7 months; serum GPC3 levels were higher in subjects under 1 year.
Conclusions:
- Hepatoblastoma and yolk sac tumors frequently express GPC3, indicating potential therapeutic targets.
- GPC3 expression is developmentally regulated, occurring physiologically in early infancy.
- Pediatric patients over 1 year old with GPC3-positive tumors may be candidates for GPC3 peptide vaccination immunotherapy.
