Delivery of therapeutic compounds to the brain and other...

A - Human Necessities – 61 – K

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Details

A61K 38/43 (2006.01) C12N 9/00 (2006.01) C12N 9/04 (2006.01) C12N 9/24 (2006.01) C12N 9/36 (2006.01) C12N 9/44 (2006.01)

Patent

CA 2537238

The present invention relates to the intrathecal (IT) administration of recombinant enzyme to treat lysosomal storage disorders. In an exemplary embodiment, intrathecal administration of human .alpha.-L-iduronidase (rhIDU) injections in MPS I affected animals resulted in significant enzyme uptake, significant rh-iduronidase activity in brain and meninges and a decrease of glycosaminoglycan (GAG) storage in cells of MPS I subjects to that of normal subjects. Intrathecal administration proved more effective than intravenous treatment at alleviating MPS I symptoms, indicating it is a useful method of treating lysosomal storage disorders.

Cette invention concerne l'administration intrathécale (IT) d'une enzyme recombinante pour traiter des maladies lysosomales. Dans un mode de réalisation donné à titre d'exemple, l'administration intrathécale d'injections d'.alpha.-L-iduronidase (rhIDU) humaine à des animaux atteints de la mucopolysaccharidose de type I (MPS I) a provoqué une importante absorption d'enzymes, une importante activité de la rh-iduronidase dans le cerveau et les méninges, ainsi qu'une réduction de l'accumulation de glycosaminoglycane (GAG) dans les cellules de sujets atteints de MPS I par rapport aux sujets sains. L'administration intrathécale s'est révélée plus efficace qu'un traitement intraveineux pour atténuer les symptômes de la MPS I, ce qui indique qu'il s'agit d'une méthode utile pour le traitement de maladies lysosomales.

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