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Enzyme replacement therapy (ERT) is a well-established means of treating lysosomal storage disease (LSD). This method of treatment is dependent on the delivery of intravenously delivered enzyme to cell-surface receptors of the affected tissues. In the past, many LSDs have been successfully treated by this method. In many cases, however, infusion of enzyme produces less than ideal results. In some cases, rapid clearance of infused enzyme from the circulation by insulin like-growth factor II/cation-independent mannose 6-phosphate receptor (IFG-II/MPR) in the liver or spleen lead to the lack of delivery of enzyme across the blood-brain barrier (BBB). Some of LSDs like MPS II have neurodegenerative phenotype. Thus, IV infusion of enzyme has limitation for central nervous system (CNS) defect in MPS II. And most of recombinant enzymes for LSDs are often high volume formulations thus developed primarily as intravenous formulations. Such parenteral drugs need, however, outpatient clinic, or specialty pharmacy solely for drug therapy. Not only do these issues raise health care costs, but also decrease patient satisfaction and compliance. In response to this challenge from parental IV formulation for LSDs, we have investigated modified ERT method and evaluated the efficacy in animal model.

Development of New Strategies for Enzyme Replacement Therapy for Lysosomal Storage Disorders