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Modified Protein Might Help Hemophiliacs

Posted on: Wednesday, 21 May 2008, 15:00 CDT

U.S. pathologists say they've created a chemically modified protein that might help people suffering hemophilia -- a hard-to-treat genetic bleeding disorder.

University of Texas Health Science Center researchers said hemophilia patients typically receive injections of blood-clotting protein Factor VIII derived from plasma or produced synthetically to control potentially life-threatening episodes of bleeding. Unfortunately as many as 1-in-3 patients produce inhibitor antibodies, which attack the externally administered FVIII and negate its blood-clotting benefits.

To combat that problem, scientists in the laboratory of Sudhir Paul at the University of Texas Medical School developed a chemically modified version of FVIII that, during laboratory tests, neutralized the inhibitor antibodies. The modification is called electrophilic FVIII analog, or E-FVIII.

It's a two-step process, said Paul. The E-FVIII permanently inactivates the antibodies that inhibit blood clotting in 20 percent to 30 percent of patients receiving Factor VIII replacement therapy. Once the antibodies are cleared, additional FVIII can be injected.

The discovery and the results of preclinical testing appeared in the May 2 issue of the Journal of Biological Chemistry.

Source: United Press International

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