Development of a new treatment for pseudomyxoma peritoneum using proteogenomic analysis and patient-derived models

2023 Fiscal Year Final Research Report

• Project/Area Number: 21K08743
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Review Section: Basic Section 55020:Digestive surgery-related
• Research Institution: National Cancer Center Japan
• Principal Investigator: Noguchi Rei 国立研究開発法人国立がん研究センター, 研究所, 研究員 (30779682)
• Co-Investigator(Kenkyū-buntansha): 小迫 英尊 徳島大学, 先端酵素学研究所, 教授 (10291171) ; 近藤 格 国立研究開発法人国立がん研究センター, 研究所, 分野長 (30284061) ; 亀山 昭彦 国立研究開発法人産業技術総合研究所, 生命工学領域, 上級主任研究員 (80415661)
• Project Period (FY): 2021-04-01 – 2024-03-31
• Keywords: pseudomyxoma peritonei / patient-derived model / proteogenomics

Outline of Final Research Achievements

Pseudomyxoma peritoneum (PMP) is a rare cancer that manifests as symptoms caused by the dissemination of mucus-producing tumor cells into the peritoneal cavity. The mainstream treatment is a combination of excision of the primary tumor, removal of mucus, and intraperitoneal perfusion chemotherapy, which has improved the prognosis. However, postoperative recurrence and complications occur at a high rate, and the development of new treatment methods is required. The goal of the research is to find anticancer drugs and therapeutic targets for the development of treatments for PMP. In this study, we conducted drug screening using a PMP patient-derived cell line established by the applicant and identified effective anticancer drugs. We also established a protein extraction method from formalin-fixed specimens for proteogenomic analysis using large-scale specimens of PMP.

Free Research Field

Rare cancer

Academic Significance and Societal Importance of the Research Achievements

本研究成果により、腹膜偽粘液腫の患者由来細胞株を用いて、薬剤スクリーニングを行い、未だかつてない規模での薬剤の奏効性を調べることができた。また今までに取得した全ゲノムデータやRNA-Seqのデータから推測された治療標的から推測した薬剤と薬剤スクリーニングで実際奏効する薬剤の両方を検討することができた。これにより、患者由来モデルを用いての機能的アプローチと分子背景からの治療標的の同定の理論的アプローチの両方が重要であることがわかった。