“The Ultimate Pig”

The 7th Transgenic Animal Conference held this week in Tahoe, Calif. came to a close today. But not before the “buzz” was felt about the advances in the area of genetic engineering (GE) of pigs for advancing human health. Researchers from Denmark, Germany, Ireland, and the United States astounded the over 100 scientists in the audience with their findings on GE pigs.

GE pigs are successfully being studied that will serve as animal models for human disease, including alzheimers, atherosclerosis, breast cancer, diabetes, and psoriasis. Researchers at BIO member Revivicor Inc., Blacksburg, Va., and at the German institution, the Institute of Farm Animal Genetics in Mariensee, reported that they are successfully producing pigs with up to four transgenes in their genome. And they reported that these pigs are normal functioning, happy pigs in every way.

Further, Revivicor, whose mission is regenerative medicine, noted that using pigs for replacement human cells, tissues and organs is the ONLY near-term solution to the current shortage in human organs and tissues. Their research includes pigs that provide tissues, tendons, bridge livers, and (in the long-term future) heart, kidney and livers for xenotransplantation. These products, due to the genetic engineering of the pigs, have had the protein eliminated that induces the hyper acute rejection, so rejection will not occur in the human recipient patients.

As one researcher stated, “I can create any pig you want.”

And society will be appreciative of the tenacity and resolve of these researchers who realize that GE pigs, which are physiologically so similar to you and me, will make great contributions to humankind one day when the GE pig- derived products are approved by FDA and are available for us to improve our quality of life.

The excitement of this conference will live on! But already the enthusiasm is building for the first-ever industry-led conference on GE animals to be hosted by BIO in late September, 2010. The “BIO Livestock Biotech Summit” promises to provide some cutting edge science and solutions to global issues. But more importantly, it will convene all those skeptics and aggressive adopters who will lay out the challenges to adopting GE animals in agriculture, biomedical and industrial areas. These debates will be very key because it is those folks who are a driving force for the future of the technology. As a leading Chinese researcher noted, “I think the future of GE animals is bright. In China our people want the technology because they want the choice of newly improved foods. And in China, there is 90 percent acceptance of the use of the technology for food production.” Consumer acceptance is paramount and it will be discussed at the BIO Summit. BIO looks forward to hosting this event next year in Sioux Falls, South Dakota. See you there!

New Era in Animal Biotechnology: FDA Approves Therapy Produced with Genetically Engineered Goats

As an animal scientist, today is an exciting day in my career. I know first-hand the dedication and scientific expertise needed to work towards developing technologies that use animals to improve the human condition. Animals have a long history of serving humankind, and today marks another contribution. It is a landmark day for us, and it hails the efforts of animal scientists everywhere.

The U.S. Food and Drug Administration granted the first-ever U.S. approval of a product derived from a genetically engineered animal. The product is a protein, developed to treat hereditary antithrombin deficiency, a rare clotting disorder associated with severe complications for which there are few treatment options. To give you more specifics, I’ll quote from the news release that we issued today:

“ATryn®, a recombinant form of human antithrombin developed by GTC Biotherapeutics, was approved by the FDA for the prevention of peri-operative and peri-partum thromboembolic events in hereditary antithrombin deficient patients. It is not indicated for treatment of thromboembolic events in hereditary antithrombin deficient patients. ATryn® is the first ever transgenically produced therapeutic protein and the first recombinant antithrombin approved in the United States. Along with the approval of ATryn®, the FDA’s Center for Veterinary Medicine also approved GTC’s New Animal Drug Application, the first of its kind to regulate genetically engineered animals. This is now required for a recombinant technology used to develop transgenic animals, such as the goats that produce recombinant antithrombin. GTC has granted OVATION the right to market ATryn® in the United States and pursue further clinical development.”

This has been a long road, and I am thrilled because this product will make a difference in the lives of these patients. It also means that the U.S. government regulatory process for genetically engineered animals is effective. The regulatory process was clarified in final guidance released by FDA on January 15, 2009 (link to BIO’s press release) The process ensures the products made available through various applications of genetic engineering will go through a rigorous and transparent review process before being approved for the marketplace. In short, there’s nowhere to go, but up! Biotechnology does indeed heal the world.

Learn about hereditary antithrombin deficiency:
GTC-Biotherapeutics resource page
Wikipedia’s entry
National Alliance for Thrombosis & Thrombophilia

Getting the Guidance We Need: FDA Finalizes Regulatory Guidance for GE Animals

Well, it’s finally happened. The FDA announced today that it will make the final guidance available for governing genetically engineered animals. And if I strain carefully, I can already hear them — the naysayers.

For example, back in November, the Consumers Union, the nonprofit publisher of Consumer Reports, submitted comments to the FDA containing the following statement, “A major problem with this proposal is that it is simply Guidance for Industry and so is not legally binding: “This draft guidance . . . does not create or confer any rights for on any person and does not operate to bind FDA or the public.” We urge the FDA to publish a legally binding regulation for genetically engineered animals, and not just a non-binding “guidance.””

That’s right; it’s guidance for the industry, on how to deal with the stuff that is legally binding. Submitting a product for regulatory approval is very complicated, and if the FDA never provided any guidance as to how to do that, well, we’d never have any new products.

In any event this is very exciting since scientists have been working for years to develop new products using goats, pigs, sheep, chicken, fish and cattle that will advance human health. These animals can be engineered to produce pharmaceutical proteins and replacement tissues in their milk, eggs and blood, which can be used in the treatment of human disease, to develop life-saving therapies.

That the FDA has developed this framework which is based on the Food, Drug and Cosmetic Act’s New Animal Drug (NAD) framework, means that these patients will have a greater chance of seeing these life-saving therapies. My organization put out a statement today that mentions, among other things,

BIO and its members engaged in animal biotechnology support a strong federal regulatory system to oversee development and approval of all genetically engineered (GE) animals and the products derived from them. The industry’s goal is to ultimately provide to the marketplace products that have been approved as safe and beneficial for society.”

I have to say, as a scientist and a consumer, that statement makes me proud to be a part of this industry, as I know that this is the first, in a long line of many steps, to make a product, which will ultimately save lives.