Elucidation of ependymoma pathogenesis mechanism and search for therapeutic targets

2022 Fiscal Year Final Research Report

• Project/Area Number: 20K07038
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Review Section: Basic Section 47030:Pharmaceutical hygiene and biochemistry-related
• Research Institution: Musashino University
• Principal Investigator: Ohata Shinya 武蔵野大学, 薬学部, 准教授 (00442939)
• Project Period (FY): 2020-04-01 – 2023-03-31
• Keywords: 上衣腫 / 細胞内局在制御 / 創薬

Outline of Final Research Achievements

Some ependymomas, a type of brain tumor, are a rare pediatric disease with a poor prognosis, resistant to chemotherapy and radiotherapy methods, and difficult to treat surgically. In one of the poorer prognosis subclasses of ependymomas, a protein involved in carcinogenesis has been reported to be hyper-activated by fusion with other proteins, leading to ependymoma. In this research project, we sought to elucidate the molecular mechanism by which this fusion protein is over-activated and to explore which parts of the fusion protein could be targeted for therapy.

Free Research Field

薬系衛生および生物化学関連

Academic Significance and Societal Importance of the Research Achievements

上衣腫は治療が困難であることから、その発症機序の解明による治療標的の探索が求められていました。本研究によって、上衣腫の中で予後の悪いサブクラスの一つの原因となる融合タンパク質が過剰に活性化し、上衣腫を発症させる分子機構の一端が明らかになりました。今回の研究では、上衣腫原因融合タンパク質の活性化に関わる分子を同定し、両者の相互作用機序を明らかにしたことから、今後はこの部分を標的として上衣腫治療薬を開発できることが期待できます。