First “Pharming” Drug Approved

Dolly the sheep was cloned because the administrator Ian Wilmut, and the team that did the deed, hoped to create a herd of genetically altered sheep through cloning and inserting human genes that would result in the sheep producing milk containing properties that could be extracted and turned into medicine—a process dubbed “pharming.” That enterprise failed financially and Wilmut went onto human cloning research before quitting that—good for him—to pursue induced pluripotent stem cell (IPSC) investigations.

Where Wilmut and team failed, a different group succeeded. The first medicine derived through pharming has received FDA approval. From the story:

U.S. health officials on Friday approved the first drug made using genetically engineered animals despite lingering concerns over health and environmental implications. The drug, GTC Biotherapeutics Inc’s anti-clotting therapy Atryn, is an intravenous therapy made using a human protein gathered from female goats specially bred to produce it in their milk…

GTC’s goats are bred using cells injected with human DNA in a process that it says is a cost effective way to produce human antithrombin, a natural protein to prevent blood from clotting. The company has a herd of about 200 at its Massachusetts facility that it says is otherwise normal and healthy.

The FDA looked at the impact of goats as they aged and reproduced. “We have looked carefully at seven generations of these (generically engineered) goats; all of them are healthy and we haven’t seen any adverse effects,” said Bernadette Dunham, head of the FDA’s Center for Veterinary Medicine.

Some worry that the goats could get into the food supply and object on ethical grounds to genetically engineering animals. I get the concern. However, it seems to me that if efficacious medicine can be obtained in this way more efficiently and productively than is otherwise available—assuming that proper safety precautions have been taken—it is a positive achievement. It could reduce the cost of medicine and make therapies available to relatively small patient groups because drug companies would find it easier to make a profit. I know many will disagree, but that sound you hear is me applauding.

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