Genzyme starts dosing in Gaucher disease trial

US biotechnology major Genzyme says that it has begun to treat patients in a Phase II trial of Genz-112638, a developmental oral therapy for the treatment of Gaucher disease. The program, which is being conducted at research centers in Europe, Israel, South America and the USA, will assess the drug's efficacy as a therapy for patients with type 1 disease, the most common form of the condition.

Genz-112638 is an oral analog of the lipid ceramide and is designed to inhibit the enzyme glocosylceramide synthase, thereby reducing the level of a second lipid, glucocerebroside, which builds up in cells and tissues of people suffering from the disease. The firm explained that the drug may be of use in a variety of other conditions that are caused by malfunctions in this biochemical pathway, such as Tay-Sachs, Sandhoff and Fabry diseases.

The trial, which follows the company's extensive preclinical research program, will evaluate patient response by monitoring the change in hemoglobin and platelet levels, as well as spleen volume.