Treatment of MeCP-2 Associated Disorders

Patent metadata

Field	Value
Publication number	US-2015265554-A1
Application number	US-201514733621-A
Country	US
Kind code	A1
Filing date	Jun 8, 2015
Priority date	Oct 25, 2010
Publication date	Sep 24, 2015
Grant date	—

Abstract

The invention relates to the use of cystamine, cysteamine, or a salt thereof, or of calcineurin inhibitors for treating a MeCP2-associated disorder such as Rett syndrome.

First claim

1 . A method of treating a neurological and/or neurodevelopmental disorder that is caused by MeCP2 expression defects in a patient comprising administering to said patient cystamine or cysteamine, or a salt thereof in an amount effective to treat an MECP2-associated disorder.

2 . The method according to claim 1 , wherein the neurological and/or neurodevelopmental disorder that is caused by MeCP2 expression defects is selected from the group consisting of Rett syndrome, autism, Pervasive development disorder, non-syndromic mental retardation, idiopathic neonatal encephalopathy and idiopathic cerebral palsy.

3 . The method according to claim 2 , wherein the neurological and/or neurodevelopmental disorder is Rett syndrome.

4 . The method according to any of claims 1 to 2 , further comprising administering cystamine or cysteamine, or a salt thereof in combination with another pharmaceutically active compound.

5 . A method of treating a neurological and/or neurodevelopmental disorder that is caused by MeCP2 expression defects in a patient comprising administering to said patient a calcineurin inhibitor in an amount effective to treat an MECP2-associated disorder.

6 . The method according to claim 5 , wherein the calcineurin inhibitor is a macrolide.

7 . The method according to claim 5 , wherein the calcineurin inhibitor is selected from the group consisting of calcipressins, tacrolimus and tacrolimus analogs, cyclosporine A and cyclosporine A analogs, LxPV proteins, 2,6-diaryl-substitued pyrimidine derivatives and FK506-binding proteins.

8 . The method according to claim 7 , wherein the calcineurin inhibitor is selected from the group consisting of calcipressin 1, calcipressin 2, calcipressin 3, tacrolimus, ascomycin, sirolimus, pimecrolimus, cyclosporine A, voclosporine, LxPVc1, LxPVc2, LxPVc3, 6-(3,4-dichloro-phenyl)-4-(N,N-dimethylaminoethylthio)-2-phenyl-pyrimidine and FK506-binding protein 8.

9 . The method according to claim 8 , wherein the calcineurin inhibitor is tacrolimus.

10 . The method according to claim 8 , wherein the calcineurin inhibitor is cyclosporin A.

11 . The method according to claim 5 , wherein the neurological and/or neurodevelopmental disorder that is caused by MeCP2 expression defects is selected from the group consisting of Rett syndrome, autism, Pervasive development disorder, non-syndromic mental retardation, idiopathic neonatal encephalopathy and idiopathic cerebral palsy.

12 . The method according to claim 11 , wherein the disorder is Rett syndrome.

13 . The method according to claim 5 further comprising administering a calcineurin inhibitor, or a salt thereof in combination with another pharmaceutically active compound.

14 . The method of claim 1 or 2 , wherein the cysteamine or cystamine or salt thereof is administered orally.

Assignees

• Univ Aix Marseille
• Inserm Inst Nat De La Santé Et De La Rech Médicale
• Centre Nat Rech Scient
• Inst Curie

Inventors

• Roux Jean-Christophe
• Villard Laurent
• Saudou Frédérie

Classifications

• A61P43/00

  Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 · CPC title

• A61P25/24

  Antidepressants · CPC title

• A61P25/28

  for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia · CPC title

• A61P25/22

  Anxiolytics · CPC title

• C07D491/06

  Peri-condensed systems · CPC title