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Document Type and Number:
WIPO Patent Application WO/2018/203559
Kind Code:
Provided are: pharmaceutical compositions for polyglutamine diseases and the uses thereof; methods for prevention, amelioration, progress-suppression and/or treatment of polyglutamine diseases using such pharmaceutical compositions; and a method for screening for a compound for polyglutamine diseases. One embodiment of the present invention pertains to a pharmaceutical composition that is for the prevention, amelioration, progress-suppression, and/or treatment of polyglutamine diseases, and that contains, as an active ingredient, at least one selected from the group consisting of vascular endothelial growth factor receptor (VEGFR) inhibitor compounds, compounds for inhibiting receptor tyrosine kinase (c-Met) encoded by c-Met gene, and compounds for inhibiting both VEGFR and c-Met. Another embodiment of the present invention pertains to a method for screening for a substance that has an effect on the formation of a polyglutamine aggregate, the method comprising a step for measuring, as an indicator of polyglutamine aggregates formed in a culture solution in which cells having introduced therein an expression system that expresses polyglutamine fused with a reporter protein have been cultured, a signal of the reporter protein present in a putative somatic region including a cell nucleus.

HAGIWARA Masatoshi
OKUNO Yukiko
Application Number:
Publication Date:
November 08, 2018
Filing Date:
May 01, 2018
Export Citation:
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KYOTO UNIVERSITY (36-1, Yoshida-honmachi Sakyo-ku, Kyoto-sh, Kyoto 01, 〒6068501, JP)
International Classes:
A61K45/00; A61K31/166; A61K31/404; A61K31/4162; A61K31/4412; A61K31/444; A61K31/47; A61K31/4709; A61K31/496; A61K31/5025; A61K31/5377; A61P21/02; A61P25/00; A61P25/14; A61P43/00; C12Q1/04; G01N33/15
Domestic Patent References:
Foreign References:
Other References:
STRAND, A. D. ET AL.: "Expression profiling of Huntington's disease models suggests that brain- derived neurotrophic factor depletion plays a major role in striatal degeneration", J NEUROSCI, vol. 27, no. 43, 2007, pages 11758 - 11768, XP009122871
SONG, E. K. ET AL.: "Potent anti tumor activity of cabozantinib, a c-MET and VEGFR2 inhibitor, in a colorectal cancer patient-derived tumor explant model", INT J CANCER, vol. 136, no. 8, 2015, pages 1967 - 1975, XP055562341
AKINLEYE, A. ET AL.: "MEK and the inhibitors: from bench to bedside", J HEMATOL ONCOL, vol. 6, no. 27, 2013, XP021147527
Attorney, Agent or Firm:
IKEUCHI SATO & PARTNER PATENT ATTORNEYS (26th Floor, OAP TOWER 8-30, Tenmabashi 1-chome, Kita-ku, Osaka-sh, Osaka 26, 〒5306026, JP)
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