Translational research on the effects of intracerebroventricular administration of 2-hydroxypropyl-beta-cyclodextrin against Niemann-Pick disease type C
Project/Area Number |
26460221
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Research Category |
Grant-in-Aid for Scientific Research (C)
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Allocation Type | Multi-year Fund |
Section | 一般 |
Research Field |
Medical pharmacy
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Research Institution | Kumamoto University |
Principal Investigator |
Irie Tetsumi 熊本大学, 大学院生命科学研究部(薬), 教授 (60150546)
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Co-Investigator(Kenkyū-buntansha) |
松尾 宗明 佐賀大学, 医学部, 教授 (20219398)
中潟 直己 熊本大学, 生命資源研究・支援センター, 教授 (30159058)
有馬 英俊 熊本大学, 大学院生命科学研究部(薬), 教授 (50260964)
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Co-Investigator(Renkei-kenkyūsha) |
ERA Takumi 熊本大学, 発生医学研究所, 教授 (00273706)
ISHITSUKA Yoichi 熊本大学, 生命科学研究部, 准教授 (70423655)
KONDO Yuki 熊本大学, 生命科学研究部, 助教 (90721879)
OKADA Yasuyo 武庫川女子大学, 薬学部, 講師 (70211117)
|
Project Period (FY) |
2014-04-01 – 2017-03-31
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Project Status |
Completed (Fiscal Year 2016)
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Budget Amount *help |
¥5,070,000 (Direct Cost: ¥3,900,000、Indirect Cost: ¥1,170,000)
Fiscal Year 2016: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2015: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
Fiscal Year 2014: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
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Keywords | niemann-pick病C型 / cyclodextrin / 脳室内投与療法 / Niemann-Pick病Type C / NPC |
Outline of Final Research Achievements |
This translational research was conducted to evaluate the effects of intracerebroventricular administration of 2-hydroxypropyl-beta-cyclodextrin (HPBCD) against Niemann-Pick disease type C models and patients. Although a pre-symptomatic injection of HPBCD showed more potent effects on the lifespan and Purkinje cell loss in NPC model mice, a post-symptomatic HPBCD also significantly attenuated the disease states compared with control group. We proved that HPBCD had an optimal concentration range to exert attenuating effects against NPC manifestation in model cells. In additon, physical and chemical properties, such as degrees of substitution of the hydroxypropyl group, also were assessed. Furthermore, we also perfomed pharmacokinetic analysis in NPC patients treated with HPBCD. The results of this study will provide a rationale for the optimization of intracerebroventricular HPBCD therapy for NPC.
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Report
(4 results)
Research Products
(56 results)
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[Journal Article] In vitro evaluation of 2-hydroxyalkylated β-cyclodextrins as potential therapeutic agents for Niemann-Pick Type C disease2016
Author(s)
Kondo Y, Tokumaru H, Ishitsuka Y, Matsumoto T, Taguchi M, Motoyama K, Higashi T, Arima H, Matsuo M, Higaki K, Ohno K, Irie T
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Journal Title
Molecular Genetics and Metabolism
Volume: 118
Issue: 3
Pages: 214-219
DOI
Related Report
Peer Reviewed / Acknowledgement Compliant
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[Journal Article] Cholesterol-Lowering Effect of Octaarginine-Appended β-Cyclodextrin in <i>Npc1</i>-Trap-CHO Cells2016
Author(s)
K. Motoyama, R. Nishiyama, Y. Maeda. T. Higashi, Y. Kawaguchi, S. Futaki, Y. Ishitsuka, Y. Kondo, T. Irie, T. Era, H. Arima
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Journal Title
Biological and Pharmaceutical Bulletin
Volume: 39
Issue: 11
Pages: 1823-1829
DOI
NAID
ISSN
0918-6158, 1347-5215
Related Report
Peer Reviewed / Open Access
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[Journal Article] Efficacy of 2-Hydroxypropyl-β-cyclodextrin in Niemann–Pick Disease Type C Model Mice and Its Pharmacokinetic Analysis in a Patient with the Disease2015
Author(s)
Tanaka Y, Yamada Y, Ishitsuka Y, Matsuo M, Shiraishi K, Wada K, Uchio Y, Kondo Y, Takeo T, Nakagata N, Higashi T, Motoyama K, Arima H, Mochinaga S, Higaki K, Ohno K, Irie T
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Journal Title
Biological and Pharmaceutical Bulletin
Volume: 38
Issue: 6
Pages: 844-851
DOI
NAID
ISSN
0918-6158, 1347-5215
Related Report
Peer Reviewed / Open Access / Acknowledgement Compliant
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[Journal Article] HPGCD outperforms HPBCD as a potential treatment for Niemann-Pick disease type C during disease modeling with iPS cells2015
Author(s)
Soga M, Ishitsuka Y, Hamasaki M, Yoneda K, Furuya H, Matsuo M, Ihn H, Fusaki N, Nakamura K, Nakagata N, Endo F, Irie T, Era T
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Journal Title
Stem Cells
Volume: 33
Issue: 4
Pages: 1075-1088
DOI
Related Report
Peer Reviewed / Open Access
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[Journal Article] Population pharmacokinetics of doxapram in low-birth-weight Japanese infants with apnea2015
Author(s)
Ogawa Y, Irikura M, Kobaru Y, Tomiyasu M, Kochiyama Y, Uriu M, Ishitsuka Y, Kondo Y, Yukawa E, Kamada N, Ohno H, Yamazaki T, Irie T
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Journal Title
European Journal of Pediatrics
Volume: 174
Issue: 4
Pages: 509-518
DOI
Related Report
Peer Reviewed
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[Journal Article] Differences in reaction specificity toward lipoprotein X and abnormal LDL among six homogenous assays for LDL-cholesterol2015
Author(s)
Matsushima K, Sugiuchia H, Nishimura H, Manabe M, Ikeda K, Ando Y, Kondo Y, Ishitsuka Y, Irikura M, Irie T
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Journal Title
Clinica Chimica Acta,
Volume: 439
Pages: 29-37
DOI
Related Report
Peer Reviewed
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[Journal Article] Effects of intracerebroventricular administration of 2-hydroxypropyl-β-cyclodextrin in a patient with Niemann-Pick Type C disease2014
Author(s)
Matsuo M, Shraishi K, Wada K, Ishitsuka Y, Doi H, Iimori Y, Mizoguchi T, Eto J, Mochinaga S, Arima H, Irie T
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Journal Title
Molecular Genetics and Metabolism Reports
Volume: 1
Pages: 391-400
DOI
Related Report
Peer Reviewed / Open Access / Acknowledgement Compliant
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[Presentation] 1.Niemann-Pick病C型 治療薬としての2-Hydroxypropyl-cyclodextrinsの有効性・安全性評価2015
Author(s)
山田侑世, 石塚洋一, 近藤悠希, 竹尾 透, 中潟直己, 江良択実, 東 大志, 本山敬一, 有馬英俊, 松尾宗明, 檜垣克美, 大野耕策, 入江徹美
Organizer
第32回日本薬学会九州支部大会
Place of Presentation
宮崎県延岡市 九州保健福祉大学
Year and Date
2015-11-28
Related Report
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[Presentation] 3.Niemann-Pick 病 C 型治療薬 2-Hydroxypropyl-β-cyclodextrin の安全性評価2015
Author(s)
山田侑世, 石塚洋一, 田中雄太, 近藤悠希, 徳丸博子, 白石広葵, 内尾有史朗, 田口真紀子, 山縣美月, 廣瀬優美子, 武氏志保里, 堀越裕佳, 竹尾 透, 中潟直己, 江良択実, 東 大志, 本山敬一, 有馬英俊, 松尾宗明, 檜垣克美, 大野耕策, 入江徹美
Organizer
第 8 回トランスポーター研究会九州部会
Place of Presentation
鹿児島市 鹿児島大学医学部
Year and Date
2015-07-18
Related Report
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