Claims
- 1. A method for treating a glycolipid storage-related disorder, comprising administering a therapeutically effective amount of an inhibitor of glycolipid synthesis in combination with an agent capable of increasing the rate of glycolipid degradation.
- 2. The method of claim 1, wherein the inhibitor of glucosylceramide synthesis is an imido sugar.
- 3. The method of claim 2, wherein the imido sugar is selected from the group consisting of N-butyldeoxynojirimycin (NB-DNJ), N-butyldeoxygalactonojirimycin (NB-DGN), and N-nonyldeoxynojirimycin (NN-DNJ).
- 4. The method of claim 3, wherein the imido sugar is N-butyldeoxygalactonojirimycin (NB-DGN)
- 5. The method of claim 1, wherein the inhibitor is selected from the group consisting of 1-phenyl-2-decanoylamino-3-morpholino-1-propanol (PDMP), D-threo-1-phenyl-2-decanoylamino-3-morpholino-1-propanol or a structurally related analogue thereof.
- 6. The method of claim 1, wherein the inhibitor is a nucleic acid encoding a peptide or protein capable of inhibiting glycolipid synthesis.
- 7. The method of claim 6, wherein the nucleic acid is an antisense sequence.
- 8. The method of claim 6, wherein the nucleic acid is a catalytic RNA capable of interfering with the expression of enzymes responsible for glycolipid synthesis.
- 9. The method of claim 1, wherein the inhibitor of glycolipid synthesis is an inhibitor of neuronal glycolipid synthesis.
- 10. The method of claim 1, wherein the agent capable of increasing the rate of glycolipid degradation is an enzyme involved in glycolipid degradation.
- 11. The method of claim 10, wherein the enzyme is selected from the group consisting of glucocerebrosidase, lysosomal hexoseaminidase, galactosidase, sialidase, and glucosylceramide glucosidase.
- 12. The method of claim 1, wherein the agent capable of increasing the rate of neuronal glycolipid degradation is a molecule which increases the activity of a glycolipid degrading enzyme.
- 13. The method of claim 1, wherein the agent capable of increasing the rate of neuronal glycolipid degradation is a nucleic acid sequence which encodes a neuronal glycolipid degrading enzyme.
- 14. The method of claim 1, wherein the glycolipid storage-related disorder is selected from the group consisting of Gaucher disease, Sandhoff's disease, Fabry's disease, Tay-Sach's disease, Niemann-Pick disease, GM1 gangliosidosis, Alzheimer's disease, stroke, and epilepsy.
- 15. The method of claim 1, wherein the inhibitor of glycolipid synthesis and the agent capable of increasing the rate of glycolipid degradation are given simultaneously, sequentially, or separately.
- 16. A method for treating a glycolipid storage-related disorder, comprising administering a therapeutically effective amount of an inhibitor of glycolipid synthesis in combination with bone marrow transplantation.
- 17. The method of claim 16, wherein the inhibitor of glucosylceramide synthesis is an imido sugar.
- 18. The method of claim 17, wherein the imido sugar is selected from the group consisting of N-butyldeoxynojirimycin (NB-DNJ), N-butyldeoxygalactonojirimycin (NB-DGN), and N-nonyldeoxynojirimycin (NN-DNJ).
- 19. The method of claim 18, wherein the imido sugar is N-butyldeoxygalactonojirimycin (NB-DGN)
- 20. The method of claim 16, wherein the inhibitor is selected from the group consisting of 1-phenyl-2-decanoylamino-3-morpholino-1-propanol (PDMP), D-threo-1-phenyl-2-decanoylamino-3-morpholino-1-propanol or a structurally related analogue thereof.
- 21. The method of claim 16, wherein the inhibitor is a nucleic acid encoding a peptide or protein capable of inhibiting glycolipid synthesis.
- 22. The method of claim 21, wherein the nucleic acid is an antisense sequence.
- 23. The method of claim 21, wherein the nucleic acid is a catalytic RNA capable of interfering with the expression of enzymes responsible for glycolipid synthesis.
- 24. The method of claim 16, wherein the inhibitor of glycolipid synthesis is an inhibitor of neuronal glycolipid synthesis.
- 25. A pharmaceutical composition useful for the treatment of glycolipid storage-related disorders, comprising a therapeutically effective amount of an inhibitor of glycolipid synthesis, an agent capable of increasing the rate of glycolipid degradation, and a pharmaceutically acceptable carrier.
- 26. The pharmaceutical composition of claim 25, wherein the inhibitor of glucosylceramide synthesis is an imido sugar.
- 27. The pharmaceutical composition of claim 26, wherein the imido sugar is selected from the group consisting of N-butyldeoxynojirimycin (NB-DNJ), N-butyldeoxygalactonojirimycin (NB-DGN), and N-nonyldeoxynojirimycin (NN-DNJ).
- 28. The pharmaceutical composition of claim 27, wherein the imido sugar is N-butyldeoxygalactonojirimycin (NB-DGN)
- 29. The pharmaceutical composition of claim 25, wherein the inhibitor is selected from the group consisting of 1-phenyl-2-decanoylamino-3-morpholino-1-propanol (PDMP), D-threo-1-phenyl-2-decanoylamino-3-morpholino-1-propanol or a structurally related analogue thereof.
- 30. The pharmaceutical composition of claim 25, wherein the inhibitor is a nucleic acid encoding a peptide or protein capable of inhibiting glycolipid synthesis.
- 31. The pharmaceutical composition of claim 30, wherein the nucleic acid is an antisense sequence.
- 32. The pharmaceutical composition of claim 30, wherein the nucleic acid is a catalytic RNA capable of interfering with the expression of enzymes responsible for glycolipid synthesis.
- 33. The pharmaceutical composition of claim 25, wherein the inhibitor of glycolipid synthesis is an inhibitor of neuronal glycolipid synthesis.
- 34. The pharmaceutical composition of claim 25, wherein the agent capable of increasing the rate of glycolipid degradation is an enzyme involved in glycolipid degradation.
- 35. The pharmaceutical composition of claim 34, wherein the enzyme is selected from the group consisting of glucocerebrosidase, lysosomal hexoseaminidase, galactosidase, sialidase, and glucosylceramide glucosidase.
- 36. The pharmaceutical composition of claim 25, wherein the agent capable of increasing the rate of neuronal glycolipid degradation is a molecule which increases the activity of a glycolipid degrading enzyme.
- 37. The pharmaceutical composition of claim 25, wherein the agent capable of increasing the rate of neuronal glycolipid degradation is a nucleic acid sequence which encodes a neuronal glycolipid degrading enzyme.
- 38. The pharmaceutical composition of claim 25, wherein the glycolipid storage-related disorder is selected from the group consisting of Gaucher disease, Sandhoff's disease, Fabry's disease, Tay-Sach's disease, Niemann-Pick disease, GM1 gangliosidosis, Alzheimer's disease, stroke, and epilepsy.
Priority Claims (1)
Number |
Date |
Country |
Kind |
9909066.4 |
Apr 1999 |
GB |
|
RELATED PATENT APPLICATIONS
[0001] This application is a continuation of PCT/GB00/01560 filed Apr. 20, 2000, which application is herein specifically incorporated by reference.
Continuations (1)
|
Number |
Date |
Country |
Parent |
PCT/GB00/01560 |
Apr 2000 |
US |
Child |
10042527 |
Oct 2001 |
US |