Historic Drug Got Its Start In North Grafton
There has been a lot of excitement around genetic-based cures for different diseases, but a relatively local case is a great example of how long it can take between an idea, the resulting research and its approval for use in humans by the federal Food and Drug Administration.
On Feb. 6, Framingham’s GTC Biotherapeutics announced that the FDA had approved its ATryn drug, which is the first drug produced by livestock. It started out with one goat, although there is now a large herd of them at the company’s farm in Charlton.
That announcement was 20 years in the making. And it’s also pretty cool that the expertise to start the process was located here in Central Massachusetts. But let’s start at the beginning.
Animal Farm
In the late 1980s, Karl Ebert, an associate professor in Tufts University’s Department of Anatomy and Cellular Biology in the Schools of Veterinary Medicine, Dental Medicine, and Medicine, was approached by Genzyme Corp., which wanted him to develop livestock that could eventually produce a drug that could be safely used by humans.
“They originally wanted cattle, but I said I wouldn’t do that,” Ebert said in a recent interview at the Cummings School of Veterinary Medicine in North Grafton. The gestation period for cattle is much longer than of goats, which Ebert told them were better bets. Ebert has retired, but two of the people who helped him with the original program, Gail Zamarchi, contract research officer, and Dr. Sandra Ayres, who is a veterinarian and an assistant professor of biomedical sciences, are still at Cummings.
At that time, the company dealing with Ebert was Integrated Genetics, which was soon bought by Genzyme Corp. in 1989. Genzyme Transgenics was a separate business within Genzyme, but then became GTC Biotherapeutics in 1993.
The collaborative work between Cummings and GTC also shows how economic development can sprout, and served as a model for those who followed them.
From the late 1980s through the mid-1990s, work progressed at Cummings resulting in the first transgenic goat. The creation of “Number One,” as she was christened, showed it was possible to inject a goat’s embryo with human DNA, implant the embryo in another goat, and raise genetically engineered goats to produce a drug that was eventually approved for use in humans by U.S. regulators. The goats produce milk that has additional antithrombin, the blood-thinning protein, in their milk. The protein in the milk is purified, used to produce ATryn and then given to patients through an IV.
This is just the first of what will likely be a number of other drugs developed in the same way. GTC has a patent in the U.S. until 2021 covering the production of any therapeutic protein in the milk of any transgenic animal, according to the company.
It is also working on human coagulation factors that could be used to treat hemophilia.
And it is not only recently that the process has received a lot of press. From the 1990s on, the work has been documented in the mainstream press in national magazines as well as in agricultural and other scientific magazines.
Ebert documented the coverage as it went along, and recently shared three large binders full of stories about Cummings, the goats and what Genzyme, and then GTC, were trying to accomplish.
It’s also fun to hear about the animals behind the work.
“She was the most photogenic animal there was. When she saw a camera, she posed,” Ebert said. “We would joke that she would smile,” said Zamarchi added.
Federal regulators also made frequent visits to Cummings to see for themselves how the goats were faring. “They (USDA officials) were always impressed with how socialized and friendly the goats were: it means you’re doing your job well,” said Ayres, who was a veterinarian that was part of the project. The veterinarians were on call around the clock and no veterinarian wanted to miss a birth, it was “all hands on deck,” Ayres said. And everyone loved caring for the baby goats, which had to be fed by hand.
Got news for our Biotech Buzz column? E-mail Eileen Kennedy at ekennedy@wbjournal.com.
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