Development of a treatment for PAX7-FOXO1 alveolar rhabdomyosarcoma; a study using a patient-derived cancer model

2023 Fiscal Year Final Research Report

• Project/Area Number: 21K16669
• Research Category: Grant-in-Aid for Early-Career Scientists
• Allocation Type: Multi-year Fund
• Review Section: Basic Section 56020:Orthopedics-related
• Research Institution: Osaka University (2022-2023); National Cancer Center Japan (2021)
• Principal Investigator: Sin Yooksil 大阪大学, 感染症総合教育研究拠点, 特任研究員(常勤) (70761352)
• Project Period (FY): 2021-04-01 – 2024-03-31
• Keywords: 肉腫 / 横紋筋肉腫 / 患者由来細胞株

Outline of Final Research Achievements

Rhabdomyosarcoma is a soft tissue sarcoma. In the high-risk group of patients with alveolar rhabdomyosarcoma (aRMS), standard treatment has not yet been established. Patient-derived cell lines are useful for therapeutic development, but are difficult to obtain for rare cancers, including aRMS. It has also been pointed out that data obtained from patient-derived cell lines do not necessarily reflect the nature of the clinical tumor. Using surgical specimens, the principal investigator has successfully established an aRMS cell line carrying the PAX7-FOXO1 fusion gene. This study used this patient-derived cell line to identify effective anticancer drugs. And the investigator also identified culture conditions that can mimic the expression profile of cancer-related proteins in the patient-derived cell line in vitro to that of clinical tumors.

Free Research Field

分子生物学

Academic Significance and Societal Importance of the Research Achievements

薬剤スクリーニングの結果、PAX7-FOXO1融合遺伝子を有する胞巣型横紋筋肉腫の患者由来細胞株の増殖を抑制する効果のある抗がん剤をいくつか特定できた。しかし従来の培養法で得られたデータは臨床腫瘍を反映しているとは言い難い。そこで本研究では、臨床腫瘍の性質をより反映可能な患者由来細胞株の培養条件を特定を目指した試行を行い、実現に至った。本研究で有用性を見出した手法はあらゆる疾患由来の細胞株に応用可能であることから、本研究成果は希少がんに留まらず、疾患研究に必須の培養細胞株全般から得られるデータの信頼性を向上させ得るものであるといえる。