A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 31/70 (2006.01) A61K 31/445 (2006.01)
Patent
CA 2278507
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.
L'invention concerne un procédé de traitement in vivo de patients atteints d'une maladie de surcharge lysosomiale ayant des répercussions importantes sur le système nerveux central. Ce procédé consiste à administrer à ces patients une dose petite mais efficace sur le plan de l'inhibition de la surcharge, d'un dérivé N-alkyle d'un 1,5-iminosucre, dans lequel le groupe alkyle contient environ 2 à environ 8 atomes de carbone, ledit 1,5-iminosucre étant un 1,5-didéoxy-1,5-imino-D-glucitol, ou un 1,5-didéoxy-1,5-imino-D-galactitol, ou un promédicament acylé en O dudit 1,5-iminosucre. A titre d'exemple, on empêche la surcharge de ganglioside GM2 dans le au système nerveux central de souris atteintes de la maladie de Tay-Sachs, en administrant à celles-ci du 1,5-(butylimino)-1,5-didéoxy-D-glucitol.
Butters Terry D.
Dwek Raymond A.
Neises Gabrielle R.
Platt Frances M.
Monsanto Company
Osler Hoskin & Harcourt Llp
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