Inclusion Solves Insolubility : Niemann-Pick病C型治療薬開発に関する臨床⇄基礎循環型橋渡し研究
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- 資料種別
- 記事
- 著者・編者
- 入江 徹美
- 著者標目
- 並列タイトル等
- Inclusion Solves Insolubility : Translational Research Cycle from Bedside to Bench and Bench to Bedside for Drug Development Targeting Niemann-Pick Disease Type C
- タイトル（掲載誌）
- 薬学雑誌 = Journal of the Pharmaceutical Society of Japan / 日本薬学会 編
- 巻号年月日等（掲載誌）
- 142(4):2022
- 掲載巻
- 142
- 掲載号
- 4
- 掲載ページ
- 389-400
- 掲載年月日（W3CDTF）
- 2022
- ISSN（掲載誌）
- 0031-6903
- ISSN-L（掲載誌）
- 0031-6903
- 出版事項（掲載誌）
- 東京 : 日本薬学会
- 出版地（国名コード）
- JP
- 本文の言語コード
- jpn
- NDLC
- 対象利用者
- 一般
- 所蔵機関
- 国立国会図書館
- 請求記号
- Z19-411
- 連携機関・データベース
- 国立国会図書館 : 国立国会図書館雑誌記事索引
- 書誌ID（NDLBibID）
- 032103881
- 整理区分コード
- 632
- 要約等
- <p>Cyclodextrins (CDs) are used not only as pharmaceutical excipients but also as active pharmaceutical ingredients. CDs can act as artificial carriers or shuttles to ameliorate lipid transport disorders. Niemann-Pick disease type C (NPC) is an inherited, progressive neurodegenerative disorder caused by mutations in <i>NPC1</i> or <i>NPC2</i> genes, in which unesterified cholesterol accumulates in lysosomes and the transport of cholesterol from lysosomes to the endoplasmic reticulum is impaired. 2-Hydroxypropyl-β-CD (HPBCD) has activity as a cholesterol shuttle and can attenuate NPC-related manifestations in model cells and animals. HPBCD can also be an effective treatment for NPC patients, but has produced lung damage and ototoxicity at therapeutic doses in clinical trials. Like HPBCD, 2-hydroxypropyl-γ-CD (HPGCD) can normalize disrupted cholesterol homeostasis in cells derived from NPC patients and NPC model mice. HPGCD interacts with unesterified cholesterol with a mode of interaction distinct from that of HPBCD and acts as a fine-tuned cholesterol shuttle for the treatment of NPC with a wider safety margin than HPBCD in terms of ototoxicity and pulmonary toxicity. By bridging clinical and basic research, it is hoped that progress will be made in the development of therapeutic agents against neurodegenerative lipid storage disorders that share common pathogenic mechanisms with NPC.</p>
- DOI
- 10.1248/yakushi.21-00215
- オンライン閲覧公開範囲
- インターネット公開
- 連携機関・データベース
- 科学技術振興機構 : J-STAGE
- 要約等
- <p>Cyclodextrins (CDs) are used not only as pharmaceutical excipients but also as active pharmaceutical ingredients. CDs can act as artificial carriers or shuttles to ameliorate lipid transport disorders. Niemann-Pick disease type C (NPC) is an inherited, progressive neurodegenerative disorder caused by mutations in <i>NPC1</i> or <i>NPC2</i> genes, in which unesterified cholesterol accumulates in lysosomes and the transport of cholesterol from lysosomes to the endoplasmic reticulum is impaired. 2-Hydroxypropyl-β-CD (HPBCD) has activity as a cholesterol shuttle and can attenuate NPC-related manifestations in model cells and animals. HPBCD can also be an effective treatment for NPC patients, but has produced lung damage and ototoxicity at therapeutic doses in clinical trials. Like HPBCD, 2-hydroxypropyl-γ-CD (HPGCD) can normalize disrupted cholesterol homeostasis in cells derived from NPC patients and NPC model mice. HPGCD interacts with unesterified cholesterol with a mode of interaction distinct from that of HPBCD and acts as a fine-tuned cholesterol shuttle for the treatment of NPC with a wider safety margin than HPBCD in terms of ototoxicity and pulmonary toxicity. By bridging clinical and basic research, it is hoped that progress will be made in the development of therapeutic agents against neurodegenerative lipid storage disorders that share common pathogenic mechanisms with NPC.</p>
- DOI
- 10.1248/yakushi.21-00215
- オンライン閲覧公開範囲
- インターネット公開
- 関連情報（URI）
- 参照
- Cyclodextrins applied to the treatment of lysosomal storage disorders
- 参照
- Effects of intracerebroventricular administration of 2-hydroxypropyl-β-cyclodextrin in a patient with Niemann–Pick Type C diseaseβ-Cyclodextrin-threaded Biocleavable Polyrotaxanes Ameliorate Impaired Autophagic Flux in Niemann-Pick Type C DiseaseCyclodextrin Drug Carrier SystemsNiemann-Pick disease type CDirect measurement of high-density lipoprotein cholesterol in serum with polyethylene glycol-modified enzymes and sulfated alpha-cyclodextrinDevelopment of a Homogeneous Assay to Measure Remnant Lipoprotein CholesterolPharmaceutical Applications of Cyclodextrins. III. Toxicological Issues and Safety EvaluationStructure of N-Terminal Domain of NPC1 Reveals Distinct Subdomains for Binding and Transfer of CholesterolImproved systemic AAV gene therapy with a neurotrophic capsid in Niemann–Pick disease type C1 miceEvaluation of the vancomycin dosage regimen based on serum creatinine used in the neonatal intensive care unitRole of 6-O-α-maltosyl-β-cyclodextrin in lysosomal cholesterol deprivation in Npc1-deficient Chinese hamster ovary cellsReply: Lung toxicity of hydroxyl-β-cyclodextrin infusionInfluence of Npc1 genotype on the toxicity of hydroxypropyl-β-cyclodextrin, a potentially therapeutic agent, in Niemann–Pick Type C disease modelsPopulation pharmacokinetics of doxapram in low-birth-weight Japanese infants with apneaImpact of the Niemann–Pick c1 Gene Mutation on the Total Cellular Glycomics of CHO CellsComparative effects of respiratory stimulants on hypoxic neuronal cell injury in <scp>SH‐SY5Y</scp> cells and in hippocampal slice cultures from rat pupsDifferential Effects of 2-Hydroxypropyl-Cyclodextrins on Lipid Accumulation in Npc1-Null CellsIntracerebroventricular Treatment with 2-Hydroxypropyl-β-Cyclodextrin Decreased Cerebellar and Hepatic Glycoprotein Nonmetastatic Melanoma Protein B (GPNMB) Expression in Niemann–Pick Disease Type C Model MiceLong-Term Parenteral Administration of 2-Hydroxypropyl-β-Cyclodextrin Causes Bone LossCyclodextrins in peptide and protein deliveryHomogeneous assay for measuring low-density lipoprotein cholesterol in serum with triblock copolymer and α-cyclodextrin sulfateSevere hypervitaminosis A in siblings: Evidence of variable tolerance to retinol intakeHPGCD Outperforms HPBCD as a Potential Treatment for Niemann-Pick Disease Type C During Disease Modeling with iPS CellsIn vitro evaluation of 2-hydroxyalkylated β-cyclodextrins as potential therapeutic agents for Niemann-Pick Type C diseaseFertility of cold-stored mouse sperm is recovered by promoting acrosome reaction and hyperactivation after cholesterol efflux by methyl-beta-cyclodextrin2-Hydroxypropyl-β-Cyclodextrin Acts as a Novel Anticancer AgentIn Vitro and In Vivo Evaluation of 6-O-α-Maltosyl-β-Cyclodextrin as a Potential Therapeutic Agent Against Niemann-Pick Disease Type CIn vivo Efficacy and Safety Evaluation of Lactosyl-β-cyclodextrin as a Therapeutic Agent for Hepatomegaly in Niemann-Pick Type C DiseaseDifferential mode of cholesterol inclusion with 2‐hydroxypropyl‐cyclodextrins increases safety margin in treatment of Niemann‐Pick disease type CDifferential effects of α‐, β‐ and γ‐cyclodextrins on human erythrocytesHydroxypropylcyclodextrins in Parenteral Use. II: Effects on Transport and Disposition of Lipids in Rabbit and HumansPredicting the Binding Mode of 2-Hydroxypropyl-β-cyclodextrin to Cholesterol by Means of the MD Simulation and the 3D-RISM-KH TheoryCyclodextrins and Iatrogenic Hearing Loss: New Drugs with Significant RiskIntrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trialMethyl-Beta-Cyclodextrin Improves Fertilizing Ability of C57BL/6 Mouse Sperm after Freezing and Thawing by Facilitating Cholesterol Efflux from the Cells1Population pharmacokinetics of theophylline in very premature Japanese infants with apnoeaCyclodextrins as catalysts for the removal of cholesterol from macrophage foam cells.Drug solubilizers to aid pharmacologists: Amorphous cyclodextrin derivativesNiemann–Pick type C disease involves disrupted neurosteroidogenesis and responds to allopregnanoloneLung toxicity of hydroxypropyl-β-cyclodextrin infusionReversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the <i>npc1</i> <sup>−/−</sup> mouseAminophylline, administered at usual doses for rodents in pharmacological studies, induces hippocampal neuronal cell injury under low tidal volume hypoxic conditions in guinea-pigsHydroxypropylcyclodextrins in Parenteral use. I: Lipid Dissolution and Effects on Lipid Transfers in VitroEffects of cyclodextrins on chlorpromazine-induced haemolysis and central nervous system responsesEffects of cyclodextrin in two patients with Niemann–Pick Type C diseaseStructure of a cholesterol-binding protein deficient in Niemann–Pick type C2 diseasePARTICIPATION OF CYCLODEXTRIN INCLUSION CATALYSIS IN PHOTOLYSIS OF CHLORPROMAZINE TO GIVE PROMAZINE IN AQUEOUS SOLUTIONEfficacy of 2-Hydroxypropyl-b-cyclodextrin in Niemann-Pick disease type C model mice and its pharmacokinetic analysis in a patient with the diseaseCyclodextrin-induced hemolysis and shape changes of human erythrocytes in vitro.
- 連携機関・データベース
- 国立情報学研究所 : CiNii Research
- 提供元機関・データベース
- Japan Link Center雑誌記事索引データベースCrossrefPubMedCrossref
- 書誌ID（NDLBibID）
- 032103881