A certain herd of 200 goats on a Massachusetts farm may look bucolic and quaint, but they actually comprise a living, breathing pharmaceutical factory, or “pharm.” The goats have been genetically engineered t0 produce a blood-thinning drug in their milk, and a report from the FDA has just declared that the drug is effective and safe for human use. An FDA advisory panel will make a recommendation this Friday on whether to approve the drug for sales; if the drug is approved, it would be the first application under new FDA regulations that allow animals to be genetically altered to produce drugs, model human disease, produce industrial or consumer products or improve their use as food [USA Today].
The goats, which are being bred by the biotech company GTC Biotherapeutics, produce a protein called antithrombin that prevents blood clotting. About 1 in 5,000 people don’t produce enough of the protein, putting them at risk of developing blood clots in their veins. Such clots can be extremely painful. If they break loose and travel through the bloodstream to the lungs or the brain, the consequences can be catastrophic. Pregnant women with the disorder are at high risk of miscarriage or stillbirth, because of blood clots in the placenta [AP]. While people with the deficiency typically manage their condition with conventional blood thinning drugs, such drugs aren’t suitable for surgeries and childbirth, when the risks of blood clots are particularly high–that’s where the new drug would come in. GTC is also developing further studies to test the drug in patients at risk for clots in non-hereditary conditions such as coronary bypass surgery [Reuters].
To make the drug, scientists at GTC put DNA for the human antithrombin protein into single cell embryos of goats. Goat embryos with the gene were then inserted into the wombs of surrogate mothers who gave birth to baby goats carrying the new trait [AP]. The transgenic female goats produce the protein in their milk, which is then refined and purified. GTC Biotherapeutics says that a single goat will produce more than six pounds of the protein in the course of a year, and also notes that the drug-producing trait will be naturally passed down to the next generation of goats.
Many biotech companies are investigating “pharming” techniques as a way to produce large quantities of protein drugs in a cheap way. However, some consumer groups are deeply wary of genetically modified animals, whether their products end up in pharmacies or grocery stores. “The regulatory process seems to have put the cart before the horse, analyzing the safety of the product before it has opined on the safety of the manufacturing process,” says Greg Jaffe of the Center for Science in the Public Interest. “FDA clearly needs to impose cradle-to-grave conditions to prevent the goats from leaving the farm or their products from entering the food supply” [USA Today].
Related Content:
80beats: Coming Soon to a Grocery Near You: Genetically Engineered Meat
80beats: Your Quarter-Pounder Just Might Have Come From a Cloned Cow (Indirectly)
Discoblog: Live From the Biggest Science Conference in the World: First GMOs, Now Pharming?
Image: flickr / avlxyz
January 10th, 2009 at 4:32 pm
This is very cool. We’ve come far along from the days of Chinese hamster ovary cells in bioreactors producting drugs for human use. I wonder how this will be received by the populace? . . . “I’m taking drugs made from what?!” Austin
http://drughealth.blogspot.com/
January 11th, 2009 at 2:46 pm
It is amazing that people still put their trust in the FDA, even after they said they are underfunded and are monetarily unable to perform thorough tests. Couple that with the fact that history shows us that new drugs that come out on the market will be the center of many deaths and lawsuits five to ten years after they are introduced and it is obvious that FDA approval is meaningless. But don’t take my word for it. Look it up. That’s what the computer/internet is for. The last paragraph of the article is right on; imagine this getting into the wild and then we realize it actually does more harm than good.
January 11th, 2009 at 6:24 pm
As one of the 1 in 5,000 people, I think this is great. For many years, I have taken the blood thinning drugs. But now I need eye surgery, so this human antithrombin protein would be well received by me. Note: The conventional blood thinning drugs are made out of some pretty weird stuff like other prestriptions. nikilee
January 13th, 2009 at 3:48 pm
Let’s see now, which would you rather take, a drug produced in Chinese Hamster Ovary cells which may or may not be totally reactive and might possibly contain fragments which cause allergic reactions, or drugs produced in E.coli which may have the same problem, OR a human drug that is totally 100% reactive with the same structure as the human protein, glycosylations, phosphorylations, sulphur groups etc produced in the milk of goats. The human protein produced in the milk of goats is produced as a protein, does not have to be “clipped” from its chinese or e.coli host, does not contain any cytoplasm The purification method is simple and the protein is pure. I prefer the all american goat thank you. The goats will not be let out in the wild, the company has too much invested in them and they already have strict security and safeguards in place as to who is allowed near the facility. Let’s see now, how do labs dispose of Chinese Hamster Ovary cells that have been modified with drugs, or E.coli..I think many of the flasks may be thrown in the trash when the cells are not 100% confluent or active. So, which is more of an issue here?