|Posted date||Jan 24, 2018|
|International application number||2017JP023162|
|International publication number||WO 2017222040|
|Date of international filing||Jun 23, 2017|
|Date of international publication||Dec 28, 2017|
|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||
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||
|IPC(International Patent Classification)||
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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