Enzyme-Replacement Therapy for Gaucher's Disease

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Abstract
The development of successful enzyme-replacement therapy for Gaucher's disease is a major achievement. For the first time, physicians can offer patients with this relatively common disorder a safe and effective treatment. Unfortunately, the preparation of modified glucocerebrosidase, Ceredase, is very costly to produce and therefore very expensive. The price of 1 IU is $3.50. If the drug is given in a dose of 60 IU per kilogram of body weight every two weeks, as recommended by Barton et al. (May 23 issue)1 for routine use, the cost of treating a 70-kg adult would be $382,200 per year for the drug alone; for a 35-kg child the cost would be a more moderate $191,100. The cost of the weekly doses recommended by Barton et al. for severely ill patients would be twice this.