We hope that information obtained during the trial will benefit future patients with Gaucher disease. We do not know if patients participating in the trial will benefit directly from it. Bone marrow transplantation . Bone marrow transplantation has been used successfully in the past to treat a few people with Gaucher disease. However the treatment requires a marrow donor and has a 10 percent to 25 percent risk of fatal complications, which make it an unacceptable approach for most patients with Gaucher disease.
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.