Novel Compositions for Preventing and/or Treating Lysosomal Storage Disorders
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.