A new age dawns as FDA approves drug from goats with human gene

Earlier this month, the U.S. Food and Drug Administration approved the first drug produced from animals that have been injected with a human gene, paving the way for a new class of biotechnology drugs which could potentially be mass-produced at much lower cost than current therapies.

The drug utilizes an anti-clotting protein called antithrombin, which can prevent fatal blood clots caused by a rare disorder in people who are deficient in the protein.

GTC Biotherapeutics, which markets its product as ATryn, said that the milk of just one of its 200 genetically altered goats can produce the equivalent of 90,000 plasma donations of the blood-thinning protein, reported The New York Times.

Americans paid $286.5 billion in pharmaceutical costs in 2007. In today’s shrinking economy, paying for prescription medicines is an increasingly crippling expense for many, so this first step to what could be a new era in cheaper, more plentiful drugs from “pharm animals,” as they are called, is welcome news.

There are critics of the procedure. Animal rights activists oppose the use of genetically altered animals. The Humane Society of the United States, in a position statement, expressed its “grave concern about subjecting sentient animals to commercial utilization through genetic engineering,” and warned of potential “unpredictable occurrences.”

The FDA, for its part, has said that ATryn is “safe and effective,” based on the evaluation of a panel of outside experts. The agency’s Center for Veterinary Medicine examined seven generations of the goats and found no adverse effects on their health.

They also found that GTC has “adequate procedures in place to ensure that food from these goats does not enter the food supply,” and that they cause no significant impact on the environment. The goats are housed on a Massachusetts farm in a strictly controlled environment.

Producing drugs from animals is by no means new. Insulin to treat diabetes originally came from cows, and later from pigs, before human insulin was produced in the 1980s. The animal-produced insulin worked well, but because it was non-human protein, patients could develop allergies or reactions.

Kirstin Matthews, Ph.D., is a fellow in science and technology policy at the Baker Institute at Rice University whose research concentrates on the intersection between traditional biomedical research and public policy. She sees this latest development as a positive one: “It’s exciting,” she told the Chronicle, “that the FDA has, for the first time, approved this new way of producing proteins for pharmaceutical use, especially if it improves people’s access to cheaper, more plentiful drugs.”

It looks like a positive, constructive advance that will not harm the health of living animals — the goats breed conventionally after the human gene is implanted in a single-cell embryo, which is then implanted in the womb of a surrogate mother goat. It is definitely a boon for treating human illnesses, and opens the door for others to develop biological products using similar methods.

Especially in this era of escalating drug costs and diminishing resources to pay for them, it sounds like a good prescription.