Biotechnology’s Promise: a return to normal life

In a business sense, the choice seemed shaky for a young company like Genzyme: Develop an expensive treatment for a relatively small market – the estimated five thousand Gaucher patients around the world, a fair number of them in developing countries with no means of affording the drug.

But the potential to save lives was too compelling. With Gaucher disease, lipids build up in young persons’ organs, steadily weakening their systems. Often, by the age of ten to twelve, children experience fatal convulsions.

"The first patient we treated was the son of a pediatrician," Dave Fleming remembers. "She had two sons with the disease, but we only had enough product to treat one patient. The mother chose the older boy, figuring that if the treatment worked, there would be time to develop more of it for the younger boy. That is exactly what happened. The oldest boy has graduated from college, is married, and has a child. If we identify patients early enough, they can return to normal lives."

It took ten years for Ceredase to come to market. Initially, technicians extracted the replacement enzyme from placentas (a by-product of pregnancy) collected from hospitals around Boston. As demand grew, Genzyme began collecting placentas from around the world. In 1996 the company opened a facility in Allston, near Cambridge, to produce the enzyme (renamed Cerezyme) in bioreactors, allowing for unlimited amounts and greater consistency.

"Henri Termeer said, ‘Now we have enough product to treat all the patients in the world, so go out and find them.’ "