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University of Nebraska Receives NIH Grant for New Hemophilia B Treatment

The University of Nebraska-Lincoln (UNL) has received a $9.98 million grant from the National Institutes of Health to develop a treatment for hemophilia B using factor IX (FIX) proteins from pig milk. One of the advantages of using pigs is that they have a similar biochemistry to humans. The researchers suggest that oral delivery of FIX purified from the milk of pigs, as opposed to standard intravenous infusion of factor, will bypass the inhibitor problem. Inhibitors develop when the body recognizes injected clotting factor as a foreign substance and attacks it. Although effective treatment by oral delivery requires more FIX than intravenous injection, the large amounts yielded from pig’s milk could result in a more practical, viable and inexpensive therapy.

According to Dr. William Velander, head researcher and professor of chemical engineering at UNL, “the oral therapy would be a Godsend, a true miracle. If you have an abundance of the protein, you can potentially overcome the inefficiencies of orally derived therapies.” Dr. Velander also noted that prior research in the area has been promising, “the animal testing results with oral delivery have been thus far pretty stunning.” The pigs used in this study are transgenic animals, meaning they carry a foreign gene that has been deliberately inserted into its genome. The foreign gene is constructed using recombinant DNA technology. In this case, the transgenic pigs have been produced to yield higher levels of FIX proteins in their milk. The transgenic FIX pigs are patented by the American Red Cross and the Commonwealth of Virginia.

The National Hemophilia Foundation’s Medical and Scientific Advisory Council Document #151 stated that methods to manufacture coagulation products more inexpensively, such as the use of transgenic animals, would increase supply and availability worldwide.

Source: University of Nebraska-Lincoln news release dated September 12, 2005


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