Delivery of therapeutic compounds to the brain and other tissues
First Claim
1. A method for treating a mammal suffering from a lysosomal storage disease of the brain, spinal cord or meninges associated with diminished activity of an enzyme comprising intrathecally administering directly into the cerebrospinal fluid (CSF) of the mammal a pharmaceutical composition comprising said enzyme in an amount effective to ameliorate symptoms of said lysosomal storage disease, wherein said symptoms are selected from the group consisting of an increased amount of storage granules, an increased amount of glycosaminoglycans, meningeal swelling, spinal cord compression and nystagmus.
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Abstract
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 α-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.
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Citations
49 Claims
- 1. A method for treating a mammal suffering from a lysosomal storage disease of the brain, spinal cord or meninges associated with diminished activity of an enzyme comprising intrathecally administering directly into the cerebrospinal fluid (CSF) of the mammal a pharmaceutical composition comprising said enzyme in an amount effective to ameliorate symptoms of said lysosomal storage disease, wherein said symptoms are selected from the group consisting of an increased amount of storage granules, an increased amount of glycosaminoglycans, meningeal swelling, spinal cord compression and nystagmus.
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