Study show Genzyme's Cerezyme may be effective

Will GENZ stock move today on this trial data?

Genzyme Corp. is promoting results from a new clinical study that apparently validate long-term use of its enzyme replacement drug Cerezyme.

The Cambridge, Mass., company said on Monday that Cerezyme has been shown to significantly improve bone density in patients with a form of Gaucher disease.

According to the study, which was published in the January issue of the Journal of Bone and Mineral Research, 342 patients with Type 1 Gaucher disease who received treatment with Cerezyme were compared to 160 who did not receive treatment. Those who did receive treatment enjoyed a significant increase in bone mineral density.

Gaucher disease patients are at an increased risk of developing complications with their bones including fractures and osteoporosis.

What is cerezyme and Gaucher's Disease?
Cerezyme is an analog of the human enzyme ß-glucocerebrosidase, produced by recombinant DNA technology [cloned]. ß-Glucocerebrosidase is a lysosomal glycoprotein enzyme which catalyzes the hydrolysis of the glycolipid glucocerebroside to glucose and ceramide. This keeps the lysosomal proteins from aggregating on macrophages [and other cells] which then infiltrate other tissues, thus resulting in Gaucher disease.

Gaucher disease is an inherited metabolic disorder in which harmful quantities of a fatty substance called glucocerebroside accumulate in the spleen, liver, lungs, bone marrow, and sometimes in the brain. There are three types of Gaucher disease. The first category, called type 1, is by far the most common. Patients in this group usually bruise easily and experience fatigue due to anemia and low blood platelets. They also have an enlarged liver and spleen, skeletal disorders, and, in some instances, lung and kidney impairment. There are no signs of brain involvement. Symptoms can appear at any age. In type 2 Gaucher disease, liver and spleen enlargement are apparent by 3 months of age. Patients have extensive and progressive brain damage and usually die by 2 years of age. In the third category, called type 3, liver and spleen enlargement is variable, and signs of brain involvement such as seizures gradually become apparent. All Gaucher patients exhibit a deficiency of an enzyme called glucocerebrosidase that is involved in the breakdown and recycling of glucocerebroside. The buildup of this fatty material within cells prevents the cells and organs from functioning properly. Gaucher disease is one of several lipid storage diseases.

Nasdaq: GENZ on friday closed trading at down 55 cents.