Dr. Mistry on Gaucher Disease Entering a New Era

Pramod K. Mistry, MD, PhD, Professor of Pediatrics and Internal Medicine, chief, Department of Pediatric GI/Hepatology, Yale University School of Medicine, explains that Gaucher disease does not only effect macrophage cell types. Related diseases such as cancer, Parkinson, and osteoporosis occur as a direct interaction with cells across the entire body.

Currently approved options are successful at controlling Gaucher disease but do not focus on the entire body. The next step is to have an agent that can actively regulate lipid metabolism in all cells.

The Genzyme Corporation drug eliglustat is currently under investigation and may be able to fulfill the full management of lipid metabolism. After a successful phase II trial the drug is now moving on to a phase III investigation. If approved this drug may prove useful at fulfilling the unmet needs of Gaucher disease.