Therapeutic development for Inclusion Body Myositis targeting endoplasmic reticulum stress response

2015 Fiscal Year Final Research Report

• Project/Area Number: 26670270
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Applied pharmacology
• Research Institution: The University of Tokushima
• Principal Investigator: OYADOMARI Seiichi 徳島大学, 疾患プロテオゲノム研究センター, 教授 (90502534)
• Co-Investigator(Renkei-kenkyūsha): KAJI Ryuji 徳島大学, 大学院医歯薬学研究部, 教授 (00214304) ; SAKASHITA Naomi 徳島大学, 大学院医歯薬学研究部, 教授 (90284752)
• Project Period (FY): 2014-04-01 – 2016-03-31
• Keywords: 小胞体ストレス

Outline of Final Research Achievements

Inclusion Body Myositis is suggested to associate with endoplasmic reticulum stress. In this study, approximately 4000 compounds have been searched through the cell-based high-throughput assay we developed. We identified the compound which could activate the PERK signaling pathway. The identified compound was able to induce ATF4, which is biomarker of PERK signaling activation, in cultured cells but not in mice. This incompatibility could be due to poor drug delivery in vivo, as the compound had extremely poor solubility. Although it is necessary to improve the compound solubility through structural, the identified compound has a unique core structure compared with other known PERK signaling modifiers

Free Research Field

疾患ゲノム機能学