A drug nearing approval by the Food and Drug Administration could soon bring Duke University Medical Center up to $8 million a year in royalties.
Myozyme would be the first treatment available for Pompe disease, which causes the body to be unable to break down carbohydrates, resulting in muscle deterioration. It is a rare, inherited glycogen-storage disorder that is lethal for infants and debilitating in adults.
"Rarely do you get an opportunity to do something this dramatic," said Vice Chancellor for Corporate and Venture Development Dr. Bob Taber. "It's the first drug we've had where the drug is actually in people, and we're getting royalties."
Twelve years ago, Dr. Yuan-Tsong Chen, chief of the division of medical genetics in the department of pediatrics, pioneered the development of enzyme replacement therapy as a treatment for the disease.
Chen and his team spent more than five years developing his idea into a viable enzyme-production method. The University, however, lacked the facilities to develop the treatment fully and test it on humans. Consequently, Duke partnered with Synpac (North Carolina) Inc., a drug development company located in Research Triangle Park. In March 2000, Synpac licensed the technology to Genzyme, a biotechnology company based in Cambridge, Mass., and the developer of Myozyme.
"Genzyme made the case to us that they would be in the best case to manufacture and commercialize it," said Jim Verdonik, a spokesperson for Synpac.
Genzyme also has some unique ties to Duke-Dr. Victor Dzau, chancellor for health affairs and president and CEO of Duke University Health System, is a member of its board of directors.
"Genzyme has been working to develop a treatment for Pompe disease for about seven years, and we've invested approximately $500 million to date in developing a treatment," said Bo Piela, a spokesperson for Genzyme. "This is the largest research and development program in our history."
The company had been working to develop a treatment by deriving the enzyme from the milk of genetically modified rabbits, but researchers felt that the method would not produce sufficient amounts of the enzyme to supply patient needs.
"It was also risky in the sense that if the rabbits became sick, it would compromise our ability to make the drug," Piela said.
After licensing the cell line developed by Duke and Synpac, Genzyme ultimately decided to use its own line taken from the cells of Chinese hamsters. The University could garner between $1 million and $8 million a year through royalties, Taber said. The new funds may be small change when compared to Duke's operating budget of several billion dollars a year, but it will significantly increase the annual $4 million that the University currently receives through licensing deals.
The advent of Myozyme could provide new hope to families affected by Pompe disease. The drug would be used to treat the infantile form of the disease and may also be used to treat the juvenile and adult forms, Taber said. It would be administered to patients intravenously once every two weeks, and those afflicted with the disease would receive treatments for the rest of their lives.
Administrators are optimistic about Myozyme and other drugs that may be developed by Duke researchers in the future. "We would like to do more things in-house, and we're thinking about ways to do that. But inevitably you have to put it out in the commercial world because we're a university," Taber said.
Piela said Genzyme had yet to specify a price for the drug, which is expected to be on the market by early 2006. But the cost may seem a small price to pay for a second chance at life. "You have to see these children. When they come in they're almost flaccid-they can't move, they can't roll over," Taber said. "Then you see a child who's been treated who seems normal. The transformation is just remarkable. These are kids who would have died."
Get The Chronicle straight to your inbox
Signup for our weekly newsletter. Cancel at any time.