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|Title:||Method for treatment of CNS-involved lysosomal storage diseases|
|Abstract:||A method is disclosed for the in vivo treatment of patients having a lysosomal storage disease with a significant central nervous system (CNS) involvement. Said method comprises administration to said patient a small but storage-inhibitory effective amount of an N-alkyl derivative of a 1,5-iminosugar in which said alkyl group contains from about 2 to about 8 carbon atoms and said 1,5-iminosugar is 1,5-dideoxy-1,5-imino-D-glucitol, or 1,5-dideoxy-1,5-imino-D-galactitol, or an O-acylated pro-drug of said 1,5-iminosugar. In an illustrative example, CNS storage of GM2 ganglioside is inhibited in Tay-Sachs mice by administration of 1,5-(butylimino)-1,5-dideoxy-D-glucitol.|
|Inventor(s):||Platt; Frances M. (Oxford, GB), Neises; Gabrielle R. (Chesterfield, MO), Dwek; Raymond A. (Oxford, GB), Butters; Terry D. (Oxford, GB)|
|Assignee:||Monsanto Company (St. Louis, MO)|
|Filing Date:||Jan 13, 1997|
|Claims:||1. A method for the treatment of a lysosomal storage disease having a significant central nervous system (CNS) involvement in a patient in need thereof comprising administering to said patient a storage-inhibitory effective amount of an N-alkyl derivative of a 1,5-iminosugar in which said alkyl group contains from about 2 to about 8 carbon atoms and said 1,5-iminosugar is 1,5dideoxy-1,5-imino-D-glucitol or 1,5-dideoxy-1,5-imino-D-galactitol, wherein from 1 to 4 of the free hydroxyl groups of said 1,5-iminosugar are optionally O-acylated. |
2. The method of claim 1 in which the 1,5-iminosugar is 1,5dideoxy-1,5-imino-D-glucitol.
3. The method of claim 1 in which the alkyl group is butyl.
4. The method of claim 1 in which the 1,5-iminosugar is 1,5dideoxy-1,5-imino-D-glucitol and the alkyl group is butyl.
5. The method of claim 4 in which the 1,5-iminosugar is O-acylated to form the tetrabutyrate.
6. The method of any of claims 1-5 in which the lysosomal storage disease is Tay-Sachs disease.
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