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PHARMACEUTICAL COMPOSITION

Foreign code F180009326
File No. (AF13P005)
Posted date Jan 24, 2018
Country WIPO
International application number 2017JP023162
International publication number WO 2017222040
Date of international filing Jun 23, 2017
Date of international publication Dec 28, 2017
Priority data
  • P2016-124992 (Jun 23, 2016) JP
Title PHARMACEUTICAL COMPOSITION
Abstract The present invention provides a pharmaceutical composition which can be administered to a human body safely and is effective for the treatment of PolyQ diseases. The present invention provides: a pharmaceutical composition which contains arginine, a physiologically acceptable salt of arginine or a solvate of arginine or the physiologically acceptable salt as an active ingredient and can be used for the treatment or prevention of a PolyQ disease; and the pharmaceutical composition described above, wherein the PolyQ disease is Huntington's disease, hereditary spinocerebellar ataxia, dentatorubropallidoluysial atrophy or spinal and bulbar muscular atrophy.
Outline of related art and contending technology BACKGROUND ART
In accordance with the aging population, age-related neurodegenerative diseases andcorticosteroids continues to increase and, because there is no effective treatment has been a serious social problem. Therefore, these effective for the neurodegenerative disease is demanded development of a drug.
Age-related neurodegenerative diseases andcorticosteroids PolyQ to one of the diseases. PolyQ diseases include, Huntington's Disease, spinocerebellar ataxia and the like of the various, cause abnormal extension of the chain in the protein (40 amino acids>) PolyQ by hereditary neurodegenerative disorders 9 to develop one generic. Is about 7000-8000 of a person suffering from Japan and the user is estimated, is not yet effective methods for treating intractable. PolyQ individuals, an abnormality cause by extension of PolyQ misfolded protein chain, resulting in the aggregation, as a result are considered to cause neurodegeneration.
Chemical chaperones, to stabilize the native conformation of the protein, protein folding rate of the reaction or affect the fidelity by, an anti-agglomeration properties are exhibited. Arginine, and expressed in E. coli protein aggregation of various recombinant and in order to increase the solubility, the most commonly used one of the additive (non-patent document 1). From the effects of anti-coagulant this nonspecific, arginine, such as Parkinson's disease and Alzheimer's disease by misfolding of a protein to be effective in the treatment. In fact, arginine, amyloid β peptide (1-42) is to prevent aggregation of the reported (Non-Patent Document 2).
Arginine, including a human organism species of amino acids constituting the 1, can be safely administered to an animal such as a human can. Arginine is, other than disease PolyQ disease in a clinical trial, safety is established (non-patent document 3, 4). Also, arginine, the blood-brain barrier (BBB) to pass through known (Non-Patent Document 5).
Scope of claims (In Japanese)[請求項1]
アルギニン若しくはその生理学的に許容される塩、又はそれらの溶媒和物を有効成分とし、PolyQ病の治療又は予防に用いられる、医薬用組成物。
[請求項2]
前記PolyQ病が、ハンチントン病、遺伝性脊髄小脳失調症、歯状核赤核淡蒼球ルイ体萎縮症、又は球脊髄性筋萎縮症である、請求項1に記載の医薬用組成物。
[請求項3]
前記PolyQ病が、遺伝性脊髄小脳失調症1型、遺伝性脊髄小脳失調症3型、又は球脊髄性筋萎縮症である、請求項1に記載の医薬用組成物。
  • Applicant
  • ※All designated countries except for US in the data before July 2012
  • JAPAN SCIENCE AND TECHNOLOGY AGENCY
  • Inventor
  • NAGAI Yoshitaka
  • POPIEL Helena Akiko
  • MINAKAWA Eiko
IPC(International Patent Classification)
Specified countries National States: AE AG AL AM AO AT AU AZ BA BB BG BH BN BR BW BY BZ CA CH CL CN CO CR CU CZ DE DJ DK DM DO DZ EC EE EG ES FI GB GD GE GH GM GT HN HR HU ID IL IN IR IS JO JP KE KG KH KN KP KR KW KZ LA LC LK LR LS LU LY MA MD ME MG MK MN MW MX MY MZ NA NG NI NO NZ OM PA PE PG PH PL PT QA RO RS RU RW SA SC SD SE SG SK SL SM ST SV SY TH TJ TM TN TR TT TZ UA UG US UZ VC VN ZA ZM ZW
ARIPO: BW GH GM KE LR LS MW MZ NA RW SD SL SZ TZ UG ZM ZW
EAPO: AM AZ BY KG KZ RU TJ TM
EPO: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
OAPI: BF BJ CF CG CI CM GA GN GQ GW KM ML MR NE SN ST TD TG
Reference ( R and D project ) CREST Creation of a Novel Technology towards Diagnosis and Treatment based on Understanding of Molecular Pathogenesis of Psychiatric and Neurological Disorders AREA
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