Image Added Dr. Roscoe Brady examining a young child
About this time, two facts became known. First, the enzyme has sugar side-chains attached to amino acid backbone of its structure. Second, macrophages react with enzymes whose sugar chains end with the sugar called "mannose." The problem was that the mannose on the sugar side chains of glucocerebrosidase was inside of the chains, not on the end of the chains. The mannose therefore could not interact with macrophages. Dr. Brady's team needed to develop a method to strip off some of the sugars on the ends of the side chains to expose the mannose, which would allow the enzyme to bind to the macrophages
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Image Removed Dr
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Roscoe Brady examining a young child
In the first clinical trial with macrophage-targeted glucocerebrosidase, eight people with Gaucher disease received a fixed dose of the modified enzyme. Only the smallest one - a child - experienced beneficial effects. His hemoglobin and platelet counts increased; the size of his spleen and liver decreased; and the damage to his bones lessened. The other seven people were adults and had not received enough of the enzyme to improve their condition.
