GTC Biotherapeutics and Tufts pair up on new 'pharming' initiative
Monday, 17 March 2008
Big pharmaceutical companies looking to cut back on the exorbitant costs of discovering, developing, and manufacturing effective drugs might do well to seek a solution in an old-phasioned place - the phamily pharm.
Both GTC Biotherapeutics in Framingham and the Tufts University Cummings School of Veterinary Medicine in Grafton are helping to pioneer the practice of "pharming," a combination of old-school animal husbandry and agriculture and cutting edge biotech and pharmaceutical production.
Super Secret Locale
Pharming uses genetically modified, or "transgenic," livestock animals such as goats, pigs, and cows to produce effective human proteins in their blood or milk, explained Dr. George Saperstein, chairman of the department of environmental and population health and director of contract research at the Cummings school.
GTC uses genetically modified goats to produce a highly specialized anti-clotting human protein in their milk. The goats are raised, and their precious milk harvested, at a specialized phacility (OK, I'll stop now) in the vicinity of Charlton. The exact location is kept secret so the company can avoid curious passersby, according to Tom Newberry, vice president of corporate communications for GTC.
"The farm we have operating is designed from the ground up for drugs," Newberry said. "The quality of the land, the water from the wells, everything is qualified for drug production. While we call it a farm, really at all levels of concept, it's a drug production facility. This isn't just Old MacDonald's farm retrofitted."
GTC currently employs 167 at both its Charlton farm and Framingham headquarters.
In many ways, Newberry said, GTC's facility is similar to the massive Bristol-Myers Squibb bio-manufacturing plant in Devens currently under construction.
Both produce a human protein using another animal's cells. Bristol-Myers uses Chinese hamster cells, while GTC uses goats mammary gland cells. Both can produce massive quantities of the desired protein in a relatively short period of time.
But there's a crucial difference between Bristol-Myers' traditional bio-reactor production method and GTC's goat-assisted delivery of protein.
The Price Is Right
The BMS facility is projected to cost upward of $800 million when completed. GTC's fully functioning farm cost about $30 million to bring online, Newberry said.
It's a cost savings that can't be ignored for too much longer, according to Newberry.
The types of anti-clotting proteins that GTC produces are currently produced for commercial use in very small quantities in the traditional way. There currently exists a billion-dollar market for the agents, and worldwide production is roughly about one kilogram per year, Newberry said.
"That's how this technology becomes vitally important," Newberry said. "If we can liberate the supply to develop these treatments as true therapeutics, and we're able to get very large quantities in a cost-effective way, we'd be dramatically lowering a huge cost of the health care system."
It seems to hold a tremendous amount of promise, but "pharming," like most biotech work, is not without its ethical, scientific and health concerns, warned Saperstein.
For example, animals carry a wide range of viruses that are harmless for them, but harmful to humans. One need look only as far as the recent bird flu outbreaks and the global rise of the mutated ape AIDS virus as proof that friendly farm animals can carry some nasty critters in them.
Additionally, Saperstein warned, herds of transgenic animals must be kept under rigorous scrutiny to avoid outside contamination from normal livestock and other wildlife. In other words, Saperstein said, a "pharm" must be "bio-secure" on an almost Fort Knox level.
Keeping the pharm bio-secure and ensuring for the humane treatment of the animals is the job of the veterinarian, Saperstein said. The vet must also be familiar with all aspects of a given animal's physiology and history, to know if ancient viruses carried within it may be potentially harmful to humans.
Saperstein said Tufts is not quite yet churning out scientist/vet/farmers but it won't be long, he predicted.
"Once the first product is approved by the FDA in this country, that would be the watershed event needed to get companies interested in this process, "Saperstein said. "It's certainly something we need to keep the costs of production down."
GTC's ATryn anti-clotting therapeutic is well underway with clinical trials in Europe, Newberry said, and is aggressively trying to prove its safety to the FDA.
Stay tuned.
