Methods for treating mucopolysaccharidosis

Abstract

The present invention provides methods and compositions for the treatment of mucopolysaccharidoses (MPS).

1 . A method of treating mucopolysaccharidosis (MPS) in a subject in need thereof, comprising administering to the subject a therapeutically effective amount of a pharmaceutical composition comprising a stilbene or stilbenoid compound or a pharmaceutically acceptable salt, ester, or prodrug thereof. 
     
     
         2 . The method of  claim 1 , wherein the MPS is MPS I-H, MPS I-H/S, MPS I-H/S, MPS II, MPS III A, MPS III B, MPS III C, MPS III D, MPS IV A, MPS IV B, MPS VI, MPS VII or MPS IX. 
     
     
         3 - 14 . (canceled) 
     
     
         15 . The method of  claim 1 , wherein the compound is a modified stilbene having from one to ten substituents. 
     
     
         16 . The method of  claim 15 , wherein the substituents are independently selected from hydroxyl, alkyl, alkenyl, carboxyl, alkyloxy, amino, amido, aryl, or halogen. 
     
     
         17 . The method of  claim 15 , wherein the stilbene or stilbenoid compound is of the formula: 
       
         
           
           
               
               
           
         
         wherein each of R 1  to R 10  is H or a substituent selected from hydroxyl, alkyloxy, alkyl, alkenyl, C(O)OH, alkyl ester, amino, alkylaryl, or halo, and 
         wherein any two adjacent R substituents may form a 5- or 6-membered carbocyclic or heterocyclic ring. 
       
     
     
         18 . The method of  claim 17 , wherein from 2 to 5 of R 1 -R 10  are not hydrogen. 
     
     
         19 . The method of  claim 1  any one of  claims 1 , wherein the stilbene or stilbenoid compound is trans-resveratrol, piceatannol, CTI-110, CTI-111, CTI-112 or CTI-113. 
     
     
         20 - 24 . (canceled) 
     
     
         25 . The method of  claim 1 , wherein the pharmaceutical composition is formulated for oral administration. 
     
     
         26 . The method of  claim 25 , wherein the pharmaceutical composition is formulated as a capsule or a tablet. 
     
     
         27 . The method of  claim 25 , wherein the pharmaceutical composition is formulated as a liquid. 
     
     
         28 . The method of  claim 1 , wherein the pharmaceutical composition is formulated for parenteral administration. 
     
     
         29 . The method of  claim 28 , wherein the pharmaceutical composition is formulated for intravenous injection. 
     
     
         30 . The method of  claim 1 , wherein the stilbene or stilbenoid compound can cross the blood-brain barrier. 
     
     
         31 . The method of  claim 1 , wherein the pharmaceutical composition is administered at least once daily. 
     
     
         32 . The method of  claim 31 , wherein the therapeutically effective amount is from about 10 mg to about 5000 mg daily.