US2019076376A1PendingUtilityA1

Methods for treating mucopolysaccharidosis

Assignee: UNIV MIAMIPriority: Jul 10, 2015Filed: Jul 8, 2016Published: Mar 14, 2019

Est. expiryJul 10, 2035(~9 yrs left)· nominal 20-yr term from priority

Inventors:Shaun Brothers Claes Wahlestedt Claude Henry Volmar

A61K 9/0053A61K 9/0019A61K 31/05A61P 3/00A61K 31/03A61K 31/235A61K 31/166A61K 31/36A61K 31/09A61K 31/69A61K 31/7004A61K 31/194A61K 31/185A61K 31/423A61K 45/06

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Claims

Abstract

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

Claims

exact text as granted — not AI-modified

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.

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