Adjunctive therapy for Hurler syndrome

Enzyme replacement therapy (ERT) remains an essential way to treat MPS disease. This therapy works because lysosomal enzymes made in the lab can be taken into cells and delivered to the lysosome with the help of a carbohydrate-dependent receptor. Once in the lysosome, the replaced enzyme helps clear storage. How well this therapy works depends on the ability of the replaced enzyme to efficiently reach and enter certain affected organs like the brain and heart. We propose to use a drug that is already approved by the Federal Drug Administration (FDA) as a means of enhancing delivery of the therapeutic enzyme. We will study how the drug works in MPS cells and then test its ability to improve ERT in a mouse model for MPS-I. Our hope is that this drug, when used in combination with ERT, will not only increase the efficiency of ERT but also lower its cost.

–¬†Vito Ferro, PhD

University of Queensland
Brisbane, Queensland Australia