Macrophages come from the bone marrow. The fact that bone marrow transplantation can successfully treat Gaucher disease is significant. Enzyme replacement therapy . Enzyme replacement therapy with modified GC has been available as a treatment for Gaucher disease since the early 1990s. The GC used for therapy is extracted from human placentas or generated in the laboratory and the chemically modified to target it to macrophages.
Patients receive the modified enzyme by intravenous infusion.
Treatments are usually given every two weeks, and it takes about two hours for a person to receive the treatment. Approximately 800 people worldwide are receiving enzyme replacement therapy. The disease has stopped getting worse in the majority of these patients, and most have also experienced a reversal of disease symptoms. The main drawback to enzyme replacement therapy is that it involves lifelong infusions. It is also very expensive.