Use
Imiglucerase, a mannose-terminated form of human placental
glucocerebrosidase produced by recombinant technology, was introduced for the
treatment of type-1 Gaucher's disease. lmiglucerase catalyzes the hydrolysis of
glucocerebroside and thus prevents accumulation of this lipid in organs and tissues.
Compared with the natural enzyme, the modification by removal of the terminal
sugars resulted in strong binding of the exposed mannose to lectins expressed on
the surface of macrophages, which greatly increases the amount of enzyme taken
up by the cells. In 6-month clinical trials, its unequivocal efficacy was shown by
improvement in all patients with type-1 Gaucher's disease, leading to an increased
hemoglobin levels and platelet counts, decreased incidence of epistaxis and
bruising, reduced spleen and liver sizes, and improved skeletal parameters. This
enzyme replacement therapy is well tolerated with few mild adverse reactions.