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from the issue of September 15, 2005
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Grant aids hemophilia treatment
BY KIM HACHIYA, UNIVERSITY COMMUNICATIONS
Seventeen years of research have brought scientists to the brink of developing an effective, low-cost treatment for Hemophilia B, a debilitating genetic bleeding disorder.
And, a $9.98 million grant to a team led by a UNL biomedical engineer offers a glimmer of hope for clinical trials within five years.
UNL's William Velander heads the team that includes scientists from three universities and two private entities. The grant from the National Heart, Lung, and Blood Institute within the National Institutes of Health, allows the team to "transition the science to the level of technology" needed for remaining clinical studies in animals and humans, Velander said.
Velander is a professor of chemical engineering at UNL. Funds from the Nebraska Tobacco Settlement Biomedical Research Fund were used to support the recruitment of Velander and to support his research program, making this grant possible.
The product is a blood protein known as Factor IX. When genetics conspire to disable this factor, the person suffers from hemophilia, a disorder characterized by uncontrolled or abnormal bleeding often in the joints and sometimes the brain. Therapy for the hemophilia patient involves transfusions of Factor IX. But, the substance is difficult to isolate from blood plasma.
The UNL project will shepherd the transition from basic science that has already confirmed the abundance and basic quality of the genetically engineered Factor IX, to trialing it in hemophilic dogs. The key to reaching this point, Velander said, was the team's previous work in developing an abundant and safe source of Factor IX protein, from the milk of genetically engineered pigs. Velander recently presented this work to the World Federation of Hemophilia at its October 2004 meeting in Bangkok.
Earlier work, Velander said, focused on producing Factor IX in a laborious but traditional method in stainless steel bioreactors called upon to mimic the natural synthesis that occurs in the liver. That process worked, but created such tiny amounts of the product that the supply was limited and costly. While Factor IX is produced in the body by the liver, scientists sought an alternative tissue that also makes abundant complex proteins and would be easier to harvest, so they turned to the mammary glands of livestock.
While some teams focused on ruminants - sheep, cows and goats - Velander's team looked at pigs, whose biochemistry is close to humans.
Turns out, the pig team got it right.
A gene that turned on the production of Factor IX was introduced into pig chromosomes, creating what Velander called "a self-replicating bioreactor that follows the genetic laws of nature." These genetically engineered pigs produced large quantities of Factor IX in their milk and both the new gene and the Factor IX was harmless to the pigs.
The process could provide treatment costing $2,000 to $10,000 per year, he said. Current annual treatments can be over $100,000.
Hemophilia B affects approximately 15,000 people in the United States. Some 500,000 and 1 million people worldwide have some type of hemophilia. Factor IX is a treatment for Hemophilia Type B, which afflicts about 20 percent of hemophiliacs. Hemophilia Type A is treated by Factor VIII which has the same lack of abundance as Factor IX.
"The developmental lessons learned with Factor IX will lead directly to Factor VIII product," Velander said. "Factor VIII is only three years behind the pace set by Factor IX."
UNL scientists working with Velander on the project include Kevin Van Cott, a professor of Chemical Engineering as well Professors Michael Meagher and Todd Swanson of the Biological Process Development Facility.
GO TO: ISSUE OF SEPTEMBER 15
NEWS HEADLINES FOR SEPTEMBER 15
Perlman outlines plan for campus childcare facility
Fall enrollment numbers improve
Grant aids hemophilia treatment
UNL opens doors to hurricane-affected students
FROM THE ARCHIVES
Libraries receive 5,000-plus Sandhills images
NU offers tuition scholarships to Katrina victims
Research gauges impact of New Orleans port disruption
UNL food allergen test commercialized
732204S34925X
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