UF scientists awarded $4.7M for hemophilia research

All three researchers are members of the university's Genetics Institute.

Published: Thursday, January 13, 2011 at 1:21 p.m.
Last Modified: Friday, January 14, 2011 at 12:04 a.m.

University of Florida researchers have received four grants totaling almost $4.7 million to develop therapies for improving the health and quality of life of people with hemophilia.

In those with hemophilia, a gene that produces a protein required for blood to clot is defective.

Hemophiliacs can suffer from spontaneous internal bleeding, and even a minor injury can cause severe bleeding.

Throughout history, it has been tied with such notables as Genghis Khan; the Tsarevitch Alexei, crown prince of Russia; British actor Richard Burton; and American teenager Ryan White, who acquired HIV after a transfusion of tainted blood to treat his hemophilia.

New treatments for the condition are urgently needed. Therapeutic approaches now in use often are rejected by the patient's immune system. They also are expensive, in some cases costing $1 million for a round of treatment.

Two grants from the National Institutes of Health and two others from Bayer HealthCare will help investigators test new approaches.

Roland Herzog, an associate professor in the UF College of Medicine's department of pediatrics, is principal investigator on the NIH grants and a co-principal investigator on the Bayer awards.

"This portfolio of grants allows us to build and sustain a robust hemophilia research program covering both forms of the disease — hemophilia A and B — and fulfill an unmet need in Florida," Herzog said Wednesday.

The other principal investigators are Arun Srivastava and Sergei Zolotukhin.

All three researchers are members of the UF Genetics Institute and on the faculty of the pediatrics division of cellular and molecular therapy.

A four-year, $2.6 million grant from the NIH's National Heart, Lung and Blood Institute will be used to develop and test better gene therapy methods for hemophilia B.

A five-year, $1.7 million grant to Herzog from the same agency will support work to prevent the immune system from rejecting gene or protein therapies.

A pair of $200,000 Bayer awards will fund work on hemophilia A in an attempt to develop tolerance to protein therapy.

An estimated one in 5,000 boys is born with hemophilia A, and standard treatment is intravenous infusion of the missing clotting factor protein.

Boys get the disease, which is linked to the X chromosome, while girl "carriers" rarely show symptoms.

Patients with hemophilia B risk death from severe allergic reactions if exposed to the protein used in therapy.

Both the risk and the cost are extremely high, researchers say.

Herzog and his colleagues will focus on how to safely administer the clotting factor protein.

"There have to be better ways to do this," he said.

Contact Diane Chun at 374-5041 or chund@gmail.com.

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