• E-mail
• Print
• Comment
• Font Size
• Digg
• del.icio.us
• Discuss article

Grant to Help UNL Hemophilia study

Posted on: Tuesday, 13 September 2005, 21:00 CDT

A nearly $10 million federal grant will help a University of Nebraska-Lincoln researcher and his team take the next steps toward developing an effective, safe and lower-cost treatment for one type of hemophilia, a debilitating genetic bleeding disorder.

The target of the research is a human blood clotting protein called Factor IX, which is deficient in people with hemophilia B.

Currently, supplies of the protein are limited and costly -- too costly for most patients in the United States to receive treatment for anything but bleeding episodes and too costly for most in the developing world to receive any treatment, said William Velander, UNL professor of chemical engineering.

So Velander and his team worked to produce the clotting factor in the milk of genetically engineered pigs.

The $9.98 million in funding from the National Heart, Lung, and Blood Institute of the National Institutes of Health will allow the team to standardize the purification of the protein and begin trials in hemophiliac dogs.

If successful, the team -- which includes researchers from two other universities and two private groups -- hopes to begin clinical trials in humans within five years, Velander said.

The researchers already have seen positive results in hemophiliac mice, with the clotting lasting twice as long as with currently available treatments.

Health ministries from several countries are interested in getting involved in the research, he said.

"The NIH grant gives us tremendous credibility in the eyes of the world," Velander said. "The scientific credibility of the past results that we've gotten and the future path that we've chosen has been validated. That's very important."

Currently, therapy for the disorder involves transfusions of protein derived from human blood plasma or produced in culture. Just treating bleeding episodes can cost $50,000 to $200,000 a year, he said. Few can afford more frequent preventive treatments. And intravenous treatments for young children can be problematic. Not treating the disorder can result in crippling joint disease and other problems.

Ultimately, the researchers would like to develop an oral treatment, Velander said.

The pigs -- roughly a dozen of them -- are housed in an isolated facility in Virginia.

Hemophilia B is one of the two most common types of hemophilia, accounting for about 20 percent of hemophiliacs. Some 500,000 to 1 million people worldwide suffer from hemophilia. The researchers also are working to produce another clotting factor used to treat hemophilia A. That work is about three years behind the work on hemophilia B.

UNL researchers working with Velander on the project include Kevin Van Cott, Michael Meagher and Todd Swanson. Also involved are researchers from the University of North Carolina at Chapel Hill, LifeSci Partners of California, ProGenetics LLC of Virginia and Colorado State University.

Source: Omaha World - Herald

More News in this Category