Novel Compositions for Preventing and/or Treating Lysosomal Storage Disorders

Abstract

The present invention provides novel compositions as well as methods for preventing and/or treating lysosomal storage disorders. In particular, the present invention provides methods for preventing and/or treating Gaucher's disease.

1 - 20 . (canceled) 
     
     
         30 . A method for treating a lysosomal storage disorder in a patient diagnosed with the same, which comprises administering to the patient in need thereof an effective amount of a compound of Formula II: 
       
         
           
           
               
               
           
         
         wherein:
 R 1  is C(R 2 )(R 3 )(R 4 ); 
 R 2  is hydrogen, —OH or halogen; 
 R 3  is hydrogen, —OH, halogen or —CH 3 ; 
 R 4  is halogen, —CH 3 , phenyl, fluorophenyl, methylphenyl, cyclohexylmethyl; 
 R 3  and R 4  may join with the carbon to which they are attached to form a cycloalkyl ring, which may be optionally substituted with one or more halogen atoms; 
 R 6  is hydrogen, phenylalkyl or substituted phenylalkyl; 
 Z is optional, when present Z is —(CH 2 )—, —C(═O)—, —S(═O) 2 NH—, —S(═O) 2 —, —S(═O) 2 —CH 2 —, —C(═O)—NH—, —S(═O) 2 NR 9 —, —C(═S)—NH— or —C(═O)—CH 2 —; 
 R 9  is hydrogen or CH 3 ; 
 R 5  is hydrogen or aminophenylalkyl; 
 R 7  is —OH or halogen; and 
 R 8  is hydrogen, halogen or —CH 3 , 
 
         provided that R 2  and R 3  cannot both be hydrogen when R 4  is halogen, Z is not present, R 7  is —OH, R 5 , R 6  and R 8  are hydrogen. 
       
     
     
         31 . The method of  claim 30 , further comprising administering an effective amount of at least one other therapeutic agent. 
     
     
         32 . A method for treating a lysosomal storage disorder in a patient diagnosed with the same, which comprises administering to the patient in need thereof an effective amount of a compound of Formula III: 
       
         
           
           
               
               
           
         
         wherein:
 R 1  is C(R 2 )(R 3 )(R 4 ); 
 R 2  is hydrogen, —OH or halogen; 
 R 3  is hydrogen, —OH, halogen or —CH 3 ; 
 R 4  is halogen, —CH 3 , phenyl, fluorophenyl, methylphenyl, cyclohexylmethyl; 
 R 3  and R 4  may join with the carbon to which they are attached to form a cycloalkyl ring, which may be optionally substituted with one or more halogen atoms; 
 R 7  is —OH or halogen; and 
 R 8  is hydrogen, halogen or —CH 3 , 
 
         provided that R 2  and R 3  cannot both be hydrogen when R 4  is a halogen, R 7  is —OH and R 8  is hydrogen. 
       
     
     
         33 . The method of  claim 32 , further comprising administering an effective amount of at least one other therapeutic agent. 
     
     
         34 . The method of  claim 32 , wherein,
 R 2  is hydrogen or fluorine;   R 3  is fluorine;   R 4  is fluorine, C 1-4  alkyl, or phenyl;   R 7  is —OH; and   R 8  is hydrogen.   
     
     
         35 . The method of  claim 34 , wherein R 2  is hydrogen. 
     
     
         36 . The method of  claim 34 , wherein R 2  is fluorine. 
     
     
         37 . The method of  claim 34 , wherein R 4  is fluorine. 
     
     
         38 . The method of  claim 34 , wherein R 4  is C 1-4  alkyl. 
     
     
         39 . The method of  claim 34 , wherein R 4  is phenyl. 
     
     
         40 . A method for treating a lysosomal storage disorder in a patient diagnosed with the same, which comprises administering to the patient in need thereof an effective amount of one or more compound selected from the group consisting of: 
       
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         or pharmaceutically acceptable salts thereof. 
       
     
     
         41 . The method of  claim 40 , further comprising administering an effective amount of at least one other therapeutic agent. 
     
     
         42 . The method of  claim 40 , wherein the compound is: 
       
         
           
           
               
               
           
         
         or a pharmaceutically acceptable salt thereof. 
       
     
     
         43 . The method of  claim 40 , wherein the compound is: 
       
         
           
           
               
               
           
         
         or a pharmaceutically acceptable salt thereof. 
       
     
     
         44 . The method of  claim 40 , wherein the compound is: 
       
         
           
           
               
               
           
         
         or a pharmaceutically acceptable salt thereof. 
       
     
     
         45 . The method of  claim 40 , wherein the compound is: 
       
         
           
           
               
               
           
         
         or a pharmaceutically acceptable salt thereof. 
       
     
     
         46 . The method of  claim 40 , wherein the compound is: 
       
         
           
           
               
               
           
         
         or a pharmaceutically acceptable salt thereof.