JP2017529397A5 - - Google Patents
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- Publication number
- JP2017529397A5 JP2017529397A5 JP2017535134A JP2017535134A JP2017529397A5 JP 2017529397 A5 JP2017529397 A5 JP 2017529397A5 JP 2017535134 A JP2017535134 A JP 2017535134A JP 2017535134 A JP2017535134 A JP 2017535134A JP 2017529397 A5 JP2017529397 A5 JP 2017529397A5
- Authority
- JP
- Japan
- Prior art keywords
- ald
- composition according
- mtor
- adrenoleukodystrophy
- neuropathy
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 201000011452 Adrenoleukodystrophy Diseases 0.000 claims 15
- 150000001875 compounds Chemical class 0.000 claims 6
- 239000000203 mixture Substances 0.000 claims 6
- 201000001119 neuropathy Diseases 0.000 claims 6
- 230000007823 neuropathy Effects 0.000 claims 6
- 208000033808 peripheral neuropathy Diseases 0.000 claims 6
- 102000013530 TOR Serine-Threonine Kinases Human genes 0.000 claims 5
- 108010065917 TOR Serine-Threonine Kinases Proteins 0.000 claims 5
- 239000008194 pharmaceutical composition Substances 0.000 claims 5
- 230000001919 adrenal effect Effects 0.000 claims 4
- 229940124302 mTOR inhibitor Drugs 0.000 claims 4
- 239000003628 mammalian target of rapamycin inhibitor Substances 0.000 claims 4
- 239000003550 marker Substances 0.000 claims 4
- 238000000034 method Methods 0.000 claims 4
- 230000002265 prevention Effects 0.000 claims 4
- QFJCIRLUMZQUOT-HPLJOQBZSA-N sirolimus Chemical group C1C[C@@H](O)[C@H](OC)C[C@@H]1C[C@@H](C)[C@H]1OC(=O)[C@@H]2CCCCN2C(=O)C(=O)[C@](O)(O2)[C@H](C)CC[C@H]2C[C@H](OC)/C(C)=C/C=C/C=C/[C@@H](C)C[C@@H](C)C(=O)[C@H](OC)[C@H](O)/C(C)=C/[C@@H](C)C(=O)C1 QFJCIRLUMZQUOT-HPLJOQBZSA-N 0.000 claims 4
- 210000000278 spinal cord Anatomy 0.000 claims 4
- 102100024643 ATP-binding cassette sub-family D member 1 Human genes 0.000 claims 3
- 108010049137 Member 1 Subfamily D ATP Binding Cassette Transporter Proteins 0.000 claims 3
- 210000004957 autophagosome Anatomy 0.000 claims 3
- 230000002490 cerebral effect Effects 0.000 claims 3
- 230000000694 effects Effects 0.000 claims 3
- GVJHHUAWPYXKBD-UHFFFAOYSA-N (±)-α-Tocopherol Chemical compound OC1=C(C)C(C)=C2OC(CCCC(C)CCCC(C)CCCC(C)C)(C)CCC2=C1C GVJHHUAWPYXKBD-UHFFFAOYSA-N 0.000 claims 2
- 208000018126 Adrenomyeloneuropathy Diseases 0.000 claims 2
- NKANXQFJJICGDU-QPLCGJKRSA-N Tamoxifen Chemical compound C=1C=CC=CC=1C(/CC)=C(C=1C=CC(OCCN(C)C)=CC=1)/C1=CC=CC=C1 NKANXQFJJICGDU-QPLCGJKRSA-N 0.000 claims 2
- CBPNZQVSJQDFBE-FUXHJELOSA-N Temsirolimus Chemical group C1C[C@@H](OC(=O)C(C)(CO)CO)[C@H](OC)C[C@@H]1C[C@@H](C)[C@H]1OC(=O)[C@@H]2CCCCN2C(=O)C(=O)[C@](O)(O2)[C@H](C)CC[C@H]2C[C@H](OC)/C(C)=C/C=C/C=C/[C@@H](C)C[C@@H](C)C(=O)[C@H](OC)[C@H](O)/C(C)=C/[C@@H](C)C(=O)C1 CBPNZQVSJQDFBE-FUXHJELOSA-N 0.000 claims 2
- 239000003963 antioxidant agent Substances 0.000 claims 2
- 235000006708 antioxidants Nutrition 0.000 claims 2
- 230000004900 autophagic degradation Effects 0.000 claims 2
- AQHHHDLHHXJYJD-UHFFFAOYSA-N propranolol Chemical compound C1=CC=C2C(OCC(O)CNC(C)C)=CC=CC2=C1 AQHHHDLHHXJYJD-UHFFFAOYSA-N 0.000 claims 2
- ATHGHQPFGPMSJY-UHFFFAOYSA-N spermidine Chemical compound NCCCCNCCCN ATHGHQPFGPMSJY-UHFFFAOYSA-N 0.000 claims 2
- 229960000235 temsirolimus Drugs 0.000 claims 2
- QFJCIRLUMZQUOT-UHFFFAOYSA-N temsirolimus Natural products C1CC(O)C(OC)CC1CC(C)C1OC(=O)C2CCCCN2C(=O)C(=O)C(O)(O2)C(C)CCC2CC(OC)C(C)=CC=CC=CC(C)CC(C)C(=O)C(OC)C(O)C(C)=CC(C)C(=O)C1 QFJCIRLUMZQUOT-UHFFFAOYSA-N 0.000 claims 2
- HDTRYLNUVZCQOY-UHFFFAOYSA-N α-D-glucopyranosyl-α-D-glucopyranoside Natural products OC1C(O)C(O)C(CO)OC1OC1C(O)C(O)C(O)C(CO)O1 HDTRYLNUVZCQOY-UHFFFAOYSA-N 0.000 claims 1
- FFHPXOJTVQDVMO-DSYKOEDSSA-N (2r,3s,5r)-5-(6-aminopurin-9-yl)-2-methyloxolan-3-ol Chemical compound C1[C@H](O)[C@@H](C)O[C@H]1N1C2=NC=NC(N)=C2N=C1 FFHPXOJTVQDVMO-DSYKOEDSSA-N 0.000 claims 1
- AGBQKNBQESQNJD-SSDOTTSWSA-N (R)-lipoic acid Chemical compound OC(=O)CCCC[C@@H]1CCSS1 AGBQKNBQESQNJD-SSDOTTSWSA-N 0.000 claims 1
- ZOBPZXTWZATXDG-UHFFFAOYSA-N 1,3-thiazolidine-2,4-dione Chemical compound O=C1CSC(=O)N1 ZOBPZXTWZATXDG-UHFFFAOYSA-N 0.000 claims 1
- SGTNSNPWRIOYBX-UHFFFAOYSA-N 2-(3,4-dimethoxyphenyl)-5-{[2-(3,4-dimethoxyphenyl)ethyl](methyl)amino}-2-(propan-2-yl)pentanenitrile Chemical compound C1=C(OC)C(OC)=CC=C1CCN(C)CCCC(C#N)(C(C)C)C1=CC=C(OC)C(OC)=C1 SGTNSNPWRIOYBX-UHFFFAOYSA-N 0.000 claims 1
- SHGAZHPCJJPHSC-ZVCIMWCZSA-N 9-cis-retinoic acid Chemical compound OC(=O)/C=C(\C)/C=C/C=C(/C)\C=C\C1=C(C)CCCC1(C)C SHGAZHPCJJPHSC-ZVCIMWCZSA-N 0.000 claims 1
- PWKSKIMOESPYIA-BYPYZUCNSA-N L-N-acetyl-Cysteine Chemical compound CC(=O)N[C@@H](CS)C(O)=O PWKSKIMOESPYIA-BYPYZUCNSA-N 0.000 claims 1
- WHXSMMKQMYFTQS-UHFFFAOYSA-N Lithium Chemical compound [Li] WHXSMMKQMYFTQS-UHFFFAOYSA-N 0.000 claims 1
- ZFMITUMMTDLWHR-UHFFFAOYSA-N Minoxidil Chemical compound NC1=[N+]([O-])C(N)=CC(N2CCCCC2)=N1 ZFMITUMMTDLWHR-UHFFFAOYSA-N 0.000 claims 1
- 102100031455 NAD-dependent protein deacetylase sirtuin-1 Human genes 0.000 claims 1
- 229940126033 PPAR agonist Drugs 0.000 claims 1
- JDUWHZOLEDOQSR-JKPSMKLGSA-N Penitrem A Chemical compound O([C@H]1[C@H]2[C@@](C(N3)=C11)(C)[C@@]4(C)CC[C@@H]5O[C@@H]([C@@H]([C@H]6O[C@@]56[C@]4(O)CC2)O)C(=C)C)C(C)(C)[C@H]2C[C@H]4[C@]2(O)C2=C1C3=CC(Cl)=C2CC4=C JDUWHZOLEDOQSR-JKPSMKLGSA-N 0.000 claims 1
- QNVSXXGDAPORNA-UHFFFAOYSA-N Resveratrol Natural products OC1=CC=CC(C=CC=2C=C(O)C(O)=CC=2)=C1 QNVSXXGDAPORNA-UHFFFAOYSA-N 0.000 claims 1
- 229940121908 Retinoid X receptor agonist Drugs 0.000 claims 1
- 108010041191 Sirtuin 1 Proteins 0.000 claims 1
- NAVMQTYZDKMPEU-UHFFFAOYSA-N Targretin Chemical compound CC1=CC(C(CCC2(C)C)(C)C)=C2C=C1C(=C)C1=CC=C(C(O)=O)C=C1 NAVMQTYZDKMPEU-UHFFFAOYSA-N 0.000 claims 1
- 229940123464 Thiazolidinedione Drugs 0.000 claims 1
- LUKBXSAWLPMMSZ-OWOJBTEDSA-N Trans-resveratrol Chemical compound C1=CC(O)=CC=C1\C=C\C1=CC(O)=CC(O)=C1 LUKBXSAWLPMMSZ-OWOJBTEDSA-N 0.000 claims 1
- HDTRYLNUVZCQOY-WSWWMNSNSA-N Trehalose Natural products O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)O[C@@H]1O[C@@H]1[C@H](O)[C@@H](O)[C@@H](O)[C@@H](CO)O1 HDTRYLNUVZCQOY-WSWWMNSNSA-N 0.000 claims 1
- 229930003427 Vitamin E Natural products 0.000 claims 1
- 229960004308 acetylcysteine Drugs 0.000 claims 1
- 239000000556 agonist Substances 0.000 claims 1
- 229960001445 alitretinoin Drugs 0.000 claims 1
- AGBQKNBQESQNJD-UHFFFAOYSA-N alpha-Lipoic acid Natural products OC(=O)CCCCC1CCSS1 AGBQKNBQESQNJD-UHFFFAOYSA-N 0.000 claims 1
- IYIKLHRQXLHMJQ-UHFFFAOYSA-N amiodarone Chemical compound CCCCC=1OC2=CC=CC=C2C=1C(=O)C1=CC(I)=C(OCCN(CC)CC)C(I)=C1 IYIKLHRQXLHMJQ-UHFFFAOYSA-N 0.000 claims 1
- 229960005260 amiodarone Drugs 0.000 claims 1
- 230000001315 anti-hyperlipaemic effect Effects 0.000 claims 1
- 229940121363 anti-inflammatory agent Drugs 0.000 claims 1
- 239000002260 anti-inflammatory agent Substances 0.000 claims 1
- 229960002938 bexarotene Drugs 0.000 claims 1
- 210000004027 cell Anatomy 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 claims 1
- LDCRTTXIJACKKU-ONEGZZNKSA-N dimethyl fumarate Chemical compound COC(=O)\C=C\C(=O)OC LDCRTTXIJACKKU-ONEGZZNKSA-N 0.000 claims 1
- 229960004419 dimethyl fumarate Drugs 0.000 claims 1
- 229940079593 drug Drugs 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- -1 fluspirylene Chemical compound 0.000 claims 1
- WIGCFUFOHFEKBI-UHFFFAOYSA-N gamma-tocopherol Natural products CC(C)CCCC(C)CCCC(C)CCCC1CCC2C(C)C(O)C(C)C(C)C2O1 WIGCFUFOHFEKBI-UHFFFAOYSA-N 0.000 claims 1
- 229940121372 histone deacetylase inhibitor Drugs 0.000 claims 1
- 239000003276 histone deacetylase inhibitor Substances 0.000 claims 1
- 239000000411 inducer Substances 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 239000003112 inhibitor Substances 0.000 claims 1
- 235000019136 lipoic acid Nutrition 0.000 claims 1
- 229910052744 lithium Inorganic materials 0.000 claims 1
- 229960001078 lithium Drugs 0.000 claims 1
- RDOIQAHITMMDAJ-UHFFFAOYSA-N loperamide Chemical compound C=1C=CC=CC=1C(C=1C=CC=CC=1)(C(=O)N(C)C)CCN(CC1)CCC1(O)C1=CC=C(Cl)C=C1 RDOIQAHITMMDAJ-UHFFFAOYSA-N 0.000 claims 1
- 229960001571 loperamide Drugs 0.000 claims 1
- 229960003632 minoxidil Drugs 0.000 claims 1
- JDUWHZOLEDOQSR-UHFFFAOYSA-N penitrem A Natural products C1CC2(O)C34OC4C(O)C(C(=C)C)OC3CCC2(C)C(C(N2)=C34)(C)C1C3OC(C)(C)C1CC3C1(O)C1=C4C2=CC(Cl)=C1CC3=C JDUWHZOLEDOQSR-UHFFFAOYSA-N 0.000 claims 1
- 239000002307 peroxisome proliferator activated receptor agonist Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- YVUQSNJEYSNKRX-UHFFFAOYSA-N pimozide Chemical compound C1=CC(F)=CC=C1C(C=1C=CC(F)=CC=1)CCCN1CCC(N2C(NC3=CC=CC=C32)=O)CC1 YVUQSNJEYSNKRX-UHFFFAOYSA-N 0.000 claims 1
- 229960003634 pimozide Drugs 0.000 claims 1
- 239000011148 porous material Substances 0.000 claims 1
- 229960003712 propranolol Drugs 0.000 claims 1
- 230000016314 protein import into mitochondrial matrix Effects 0.000 claims 1
- VMXUWOKSQNHOCA-LCYFTJDESA-N ranitidine Chemical compound [O-][N+](=O)/C=C(/NC)NCCSCC1=CC=C(CN(C)C)O1 VMXUWOKSQNHOCA-LCYFTJDESA-N 0.000 claims 1
- 229960000620 ranitidine Drugs 0.000 claims 1
- 235000021283 resveratrol Nutrition 0.000 claims 1
- 229940016667 resveratrol Drugs 0.000 claims 1
- 229940063673 spermidine Drugs 0.000 claims 1
- 229960001603 tamoxifen Drugs 0.000 claims 1
- 229960002663 thioctic acid Drugs 0.000 claims 1
- 229940074410 trehalose Drugs 0.000 claims 1
- 125000000647 trehalose group Chemical group 0.000 claims 1
- ZEWQUBUPAILYHI-UHFFFAOYSA-N trifluoperazine Chemical compound C1CN(C)CCN1CCCN1C2=CC(C(F)(F)F)=CC=C2SC2=CC=CC=C21 ZEWQUBUPAILYHI-UHFFFAOYSA-N 0.000 claims 1
- 229960002324 trifluoperazine Drugs 0.000 claims 1
- 229960001722 verapamil Drugs 0.000 claims 1
- 235000019165 vitamin E Nutrition 0.000 claims 1
- 229940046009 vitamin E Drugs 0.000 claims 1
- 239000011709 vitamin E Substances 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP14382353.2A EP2997977A1 (en) | 2014-09-19 | 2014-09-19 | Specific mtor inhibitors in the treatment of x-linked adrenoleukodystrophy |
| EP14382353.2 | 2014-09-19 | ||
| PCT/EP2015/071563 WO2016042166A1 (en) | 2014-09-19 | 2015-09-21 | Specific mtor inhibitors in the treatment of x-linked adrenoleukodystrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2017529397A JP2017529397A (ja) | 2017-10-05 |
| JP2017529397A5 true JP2017529397A5 (enExample) | 2018-11-01 |
Family
ID=51752074
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017535134A Pending JP2017529397A (ja) | 2014-09-19 | 2015-09-21 | Ｘ連鎖性副腎白質ジストロフィーの治療における特異的ｍＴＯＲ阻害剤 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US10071063B2 (enExample) |
| EP (2) | EP2997977A1 (enExample) |
| JP (1) | JP2017529397A (enExample) |
| CN (1) | CN107206087A (enExample) |
| CA (1) | CA2961729A1 (enExample) |
| MA (1) | MA39496A (enExample) |
| WO (1) | WO2016042166A1 (enExample) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3335730A1 (en) * | 2016-12-15 | 2018-06-20 | Fundació Institut de Recerca Biomédica de Bellvitge (IDIBELL) | Compounds for treating x-linked adrenoleukodystrophy |
| BR112020009765A2 (pt) * | 2017-12-20 | 2020-11-03 | Société des Produits Nestlé S.A. | composições e métodos que usam uma combinação de indutor de autofagia e alto teor de proteína para indução de autofagia |
| AU2018390799B2 (en) * | 2017-12-20 | 2024-10-03 | Societe Des Produits Nestle S.A. | Compositions and methods using high protein for induction of autophagy |
| CN108827747A (zh) * | 2018-06-04 | 2018-11-16 | 华中科技大学苏州脑空间信息研究院 | 一种多色荧光标记样本的塑性包埋方法 |
| CN115678960B (zh) * | 2022-09-13 | 2024-03-19 | 澳门大学 | 一种缓冲液、试剂盒和蛋白酶体检测方法 |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5262165A (en) | 1992-02-04 | 1993-11-16 | Schering Corporation | Transdermal nitroglycerin patch with penetration enhancers |
| US6010715A (en) | 1992-04-01 | 2000-01-04 | Bertek, Inc. | Transdermal patch incorporating a polymer film incorporated with an active agent |
| US5362718A (en) | 1994-04-18 | 1994-11-08 | American Home Products Corporation | Rapamycin hydroxyesters |
| UA48973C2 (uk) | 1995-06-07 | 2002-09-16 | Орто-Макнейл Фармасьютікалз Інк. | Трансдермальний пластир на основі 17-деацетилноргестимату для попередження овуляції |
| US6465627B2 (en) * | 1995-07-28 | 2002-10-15 | The Regents Of The University Of California | DAX-1 protein, methods for production and use thereof |
| US5948433A (en) | 1997-08-21 | 1999-09-07 | Bertek, Inc. | Transdermal patch |
| US8029561B1 (en) | 2000-05-12 | 2011-10-04 | Cordis Corporation | Drug combination useful for prevention of restenosis |
| DE60130673T2 (de) * | 2000-09-29 | 2008-07-17 | Ethicon Inc. | Beschichtungen für medizinische vorrichtungen |
| ES2303441B1 (es) | 2006-05-05 | 2009-06-22 | Fundacio Privada Institut D`Investigacio Biomedica De Bellvitge (Idibell) | Uso del acido valproico o de una sal farmaceuticamente aceptable del mismo para la prevencion y/o el tratamiento de la adenoleucodistrofia ligada a x. |
| TW201035104A (en) | 2009-03-27 | 2010-10-01 | Chunghwa Chemical Synthesis & Biotech Co Ltd | Improved synthesis method for Temsirolimus and its similar compounds |
| WO2011066430A2 (en) * | 2009-11-25 | 2011-06-03 | Duke University | Therapeutic agents for neurodegenerative diseases |
| ES2377381B1 (es) * | 2010-05-17 | 2013-03-14 | Fundació Institut De Investigació Biomèdica De Bellvitge (Idibell) | Uso de una combinación de n-acetil-cisteína y ácido lipoico para la preparación de un medicamento útil para el tratamiento de una enfermedad con daño axonal y lesiones oxidativas concomitantes. |
| SG189110A1 (en) * | 2010-09-29 | 2013-05-31 | Oxyrane Uk Ltd | De-mannosylation of phosphorylated n-glycans |
| US9670549B2 (en) | 2011-11-10 | 2017-06-06 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Gene expression signatures of neoplasm responsiveness to therapy |
-
2014
- 2014-09-19 EP EP14382353.2A patent/EP2997977A1/en not_active Withdrawn
-
2015
- 2015-09-21 CN CN201580062585.2A patent/CN107206087A/zh active Pending
- 2015-09-21 EP EP15766167.9A patent/EP3193937A1/en not_active Withdrawn
- 2015-09-21 MA MA039496A patent/MA39496A/fr unknown
- 2015-09-21 WO PCT/EP2015/071563 patent/WO2016042166A1/en not_active Ceased
- 2015-09-21 CA CA2961729A patent/CA2961729A1/en not_active Abandoned
- 2015-09-21 JP JP2017535134A patent/JP2017529397A/ja active Pending
- 2015-09-21 US US15/512,392 patent/US10071063B2/en not_active Expired - Fee Related
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