GLYPICAN 3 targeted therapy in pediatric solid malignant tumors.

【目的】本研究的目的是分析Glypican3作为一种新的诊断儿童实体恶性肿瘤的肿瘤标志物，并建立新的诊断体系。最终目标是开发针对glypican的免疫基因疗法。【方法】对病理材料及患者血清进行Glypican3、AFP的免疫组化分析和血清学分析。【结果】多数肝母细胞瘤和恶性生殖细胞瘤的免疫组化和血清学检测均为Glypican3阳性，三分之一的肾母细胞瘤、肺泡型横纹肌肉瘤和未分化肉瘤检测均为Glypican3阳性。AFP在肝母细胞瘤和恶性生殖细胞瘤中多呈阳性。非肿瘤患者出生后血清糖化甘油3含量较高，随年龄增长逐渐降低。按1年标准化的水平。【讨论】Glypican3被确认为儿童实体恶性肿瘤的新型诊断标志物。基于本研究，对于1年以上的儿童实体恶性肿瘤难治性病例，这种针对glypican 3的新的治疗免疫基因疗法将有望出现。

【Purpose】The purpose of this study is to analyze the usefulness of Glypican3 as a novel diagnostic tumor marker for pediatric solid malignant tumor, and to establish the new diagnostic system. Final goal is to develop the immuno-genetic therapy targeted for the Glypican3.【Method】The immunohistochemical analysis and serological analysis of Glypican3 and AFP was performed for the pathological materials and patient’s serum. 【Results】Immunohistochemlically and serologically, most cases of hepatoblastoma and malignant germ cell tumor showed positive for Glypican3, and one of thirds Wilms tumor, alveolar type rhabdomyosarcoma and undifferentiated sarcoma were also positive. Concerning AFP, most cases of hepatoblastoma and malignant germ cell tumor showed positive. Concerning non-neoplastic patients’, serum Glypican 3 was high just after birth and gradually decreased according to their age. The level normalized by the 1 year.【Discussion】Glypican3 was confirmed as the novel diagnostic marker for pediatric solid malignant tumors. Based on this research, for the refractory cases of pediatric solid malignant tumor over 1 year, such new treatment immuno-genetic therapy targeted forGlypican 3 will be expected.