A Plant Cell–Expressed Enzyme for Gaucher Disease

Gaucher disease is characterized by glycolipid accumulation secondary to inherited defects in an enzyme, glucocerebrosidase; the chief clinical manifestations are pancytopenia, hepatosplenomegaly, and bone disease. Controlling the disorder is accomplished by enzyme replacement, but these products are costly and occasionally in short supply.

To examine the safety and efficacy of taliglucerase alfa — a novel, plant cell–expressed glucocerebrosidase — international investigators conducted a randomized, double-blind, phase III trial involving 32 patients with confirmed Gaucher disease with splenomegaly (≥8 × normal volume) and thrombocytopenia. Patients received 30 U/kg or 60 U/kg intravenous injections of taliglucerase alfa every 2 weeks.

Result…