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New gene therapy proves promising as hemophilia treatment
Date:12/11/2013

is not without health risks. The annual cost of medications alone is about $200,000 a year.

However, about 35 percent of people with hemophilia A develop an antibody response that blocks the factor VIII therapy. They require continuous infusions of various protein factors and they face a higher mortality rate. Also, the cost of treatment can easily rise to $2 million or more a year per patient.

Nichols and David Wilcox from the Medical College of Wisconsin figured out a potential way around the antibody response in dogs with naturally occurring hemophilia A.

Using a plasmapheresis machine and a blood-enrichment technique, the research team isolated specific platelet precursor cells from three dogs that have hemophilia A. The team then engineered those platelet precursor cells to incorporate a gene therapy vector that expresses factor VIII. The researchers put those engineered platelet precursors back into the dogs. As the cells proliferated and produced new platelets, more and more were found to express factor VIII.

Then, nature took over. Platelets naturally discharge their contents at sites of vascular injury and bleeding. In this experiment, the contents included factor VIII.

In the 2 1/2 years since the dogs received the gene therapy, researchers found that factor VIII was still being expressed in platelets that were coursing throughout the vascular systems of all three dogs. All three experienced much less bleeding. In the dog that expressed the most factor VIII in platelets, the bleeding was limited to just one serious event each year over the course of three years. And such bleeding events were easily treatable with current standard therapies.

"This has been very successful," Nichols said. "And now we want to explore the possibility of moving it into human clinical trials for people with hemophilia A, similar to what Paul Monahan and Jude Samulski at UNC are currently doing for people with hemophilia B,
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Contact: Mark Derewicz
mark.derewicz@unch.unc.edu
919-923-0959
University of North Carolina Health Care
Source:Eurekalert  

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