How Cerezyme works
Gaucher disease is a rare genetic disorder caused by mutations in the GBA gene, which encodes for an enzyme called beta-glucocerebrosidase. This enzyme is responsible for breaking down glucocerebroside (a component of cell membranes) into glucose (a simple sugar) and ceramide (a simple fat molecule). Mutations in GBA reduce the activity of beta-glucocerebrosidase present in the cells. Without enough of this enzyme, waste products build up in cells to a toxic level, causing symptoms of the disease. Type 1 is the most common form of Gaucher disease.
Cerezyme consists of an artificially produced beta-glucocerebrosidase enzyme which is made using recombinant DNA technology: plant or animal cells are used to produce large quantities of the enzyme, which is injected into a patient’s bloodstream.
A previous version of this therapy, called Ceredase, was purified from human placental tissue, but this method required expensive transport of human tissue for manufacturing and carried the risk of transmitting disease from the donor tissue, and increased the possibility of an immune reaction.
Cerezyme in clinical trials
The results of an early clinical trial comparing the effects of Ceredase and Cerezyme to treat Gaucher patients were published in the scientific journal Annals of Internal Medicine. A total of 15 patients with type 1 Gaucher disease (four children and 11 adults) were randomly assigned to receive Ceredase, and 15 patients (three children and 12 adults) were assigned to receive Cerezyme. Patients were treated every two weeks for nine months.
Both treatments were equally effective in treating Gaucher syndrome as determined by hemoglobin levels, platelet counts, and blood enzyme tests. Researchers also measured the incidence of antibody formation against either treatment, which was higher in patients treated with Ceredase (40%) than in patients treated with Cerezyme (20%). No major allergic side effects occurred in either group. Researchers concluded that Cerezyme is as effective at treating Gaucher disease as Ceredase, but is easier to produce and may be less likely to cause an immune response.
A Phase 3 clinical trial (NCT03485677) is currently recruiting type 1 or type 3 Gaucher disease patients, from 2 to 17 years old. A total of 60 patients will be randomly assigned to receive Cerezyme and Cerdelga (eliglustat), or Cerdelga alone. The trial is recruiting participants in Canada, France, Italy, Russian Federation, Spain, Sweden, and Turkey, and is expected to be completed in March 2023.
Cerezyme can cause side effects such as fatigue, fever, and chills. Some patients may also experience redness or rash near the injection site.
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