Elucidation of preventive effect of amyloidogenesis by low molecule drugs in FAP

2015 Fiscal Year Final Research Report

• Project/Area Number: 25461275
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Neurology
• Research Institution: Shinshu University
• Principal Investigator: YAZAKI Masahide 信州大学, 学術研究院医学系, 准教授 (70372513)
• Co-Investigator(Renkei-kenkyūsha): SEKIJIMA Yoshiki 信州大学, 学術研究院医学系, 准教授 (60322715)
• Project Period (FY): 2013-04-01 – 2016-03-31
• Keywords: FAP / アミロイドーシス / アミロイド融解 / 低分子薬剤

Outline of Final Research Achievements

We studied the effectiveness of low molecule anti-transthyretin (TTR) amyloidosis drugs in TTR-related familial amyloid polyneuropathy (FAP). In liver-transplanted FAP patients, decrease in amount of amyloid deposits was seen in gastroduodenal amyloid after kiver transplantation. The composition ratios of variant and wild-type TTR fluctuated in serially biopsied samples probably due to suppress amyloidogenesis in both variant and wild-type TTR. In a domino liver-transplant recipient who had been started on oral intake of anti-TTR amyloidosis drug from preclinical stage of transmitted FAP, FAP developed 12 years after operation, suggesting that these drugs might have preventive effect against FAP in domino recipients.

Free Research Field

神経内科学