2013 Fiscal Year Final Research Report
Cyclodextrins as Modifiers of Intracellular Cholesterol Trafficking in Niemann-Pick Disease Type C
| Project/Area Number |
23590642
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Research Field |
Applied pharmacology
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| Research Institution | Kumamoto University |
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Principal Investigator |
IRIE Tetsumi 熊本大学, 大学院生命科学研究部, 教授 (60150546)
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| Co-Investigator(Kenkyū-buntansha) |
ISHITSUKA Yoichi 熊本大学, 生命科学研究部, 講師 (70423655)
ARIMA Hidetoshi 熊本大学, 生命科学研究部, 教授 (50260964)
MOTOYAMA Keiichi 熊本大学, 生命科学研究部, 准教授 (50515608)
MATSUO Muneaki 佐賀大学, 医学部, 准教授 (20219398)
MOCHINAGA Sakiko 佐賀大学, 医学部, 医療技術職員 (70601045)
FUJITO Hiroshi 佐賀大学, 医学部, 教授 (50325594)
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| Project Period (FY) |
2011 – 2013
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| Keywords | Niemann-Pick Type C病 / シクロデキストリン |
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| Research Abstract |
2-Hydroxypropyl-beta-cyclodextrin (HPBCD) is an attractive drug candidate against Niemann-Pick disease Type C (NPC). However, the efficacy and safety of HPBCD treatment for NPC patients remain to be elucidated. This study was conducted to obtain the essential information on efficacy and safety of HPBCD treatment for NPC patients. 1) Weekly subcutaneous administrations of HPBCD at a dose of 1000-4000 mg/kg to Npc1-deficient mice ameliorated hepatomegaly and survival rates in the mice. 2) Compared with wild type mice, Npc1-deficient mice were substantially resistant to the lethality and the organ injury induced by HPBCD at higher doses. This study provides information on optimization of HPBCD treatment for NPC patients and also suggests Npc1 genotype should be considered in the safety evaluation of HPBCD using animals and cells.
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Research Products
(37 results)
