Researchers work to treat blood clot condition

UCF is making a name for itself in the field of medicine with a little help from a $5.5 million grant.

The National Institutes of Health granted UCF and the University of Florida money for their partnered science research study on hemophilia A and B. These were joint applications by Dr. Henry Daniell's, UCF, and Dr. Roland Herzog's, UF, labs for funding. One of the two applications was in response to a request by the NIH for applications in bioengineering research partnerships.

"I am sure every medical school in the United States that has a research component applies for these types of grants," said Herzog, professor of pediatrics, molecular genetics and microbiology at UF.

According to the U.S. National Library of Medicine, hemophilia is a bleeding condition where your body does not produce the proteins you require for your blood to clot. This is a genetic condition that is typically found in males.

"People could just scratch themselves and die from bleeding because the blood will never clot," said Daniell, the first Pegasus Professor in the Burnett College of Biomedical Sciences, trustee chair and scientist on the project.

Zenaida Kotala of UCF News & Information said that the first grant, worth $3.6 million over five years, was to help make a treatment for hemophilia A, and the second grant, worth $2 million over four years, is for research on a treatment for hemophilia B.

There is currently a treatment for those with hemophilia that injects the proteins needed for the blood to clot, but the treatment is dangerous. Those with hemophilia were born without the proteins required for the blood to clot, so their immune systems are rejecting the treatment and sending them into anaphylactic shock; some result in death.

Daniell and Herzog are working on a way to make the immune system not reject the proteins needed.

The treatment is also expensive. According to the Northern Ohio Hemophilia Foundation, the average cost of treatment can run a patient $60,000 to $260,000 per year.

"We are working on inducing the immune tolerance to the therapeutic clotting factor so that the immune system does not reject the therapy," Herzog said.

Herzog is Daniell's former student and runs the hematology and immunology lab at UF that is housing mice with hemophilia B that are being tested during the research.

"We are researching and creating the vaccine, and UF is testing it on the mice," research associate Dr. Dheeraj Verma said.

After receiving their grant, they have found a way to make the protein needed inside plant cells, put them in a capsule and orally deliver them to the mice. The plant cells take them into the mice's stomach and into their gut and then the protein is released, Daniell said.

"We developed a method that actually helped stop this immune reaction," Daniell said.

When they release it little by little, the body eventually accepts that this is something that is important to the body and then it lets it go. After all of this, the immune reaction that was happening to patients has stopped. This is because the gut has the largest immune system in the body, he said. They are anticipating that the treatment will only need to be given once every 10 years.

"Oral delivery would be much less invasive than the frequent intravenous injections of clotting factor that are currently required," Herzog said.

Eventually, this new research will be tested on dogs at Duke University. If everything goes well, it will be used on humans.

"If we can achieve oral tolerance in patients and thus prevent the immune complication, I would expect that patients can save several hundred thousands of dollars," Herzog said.

"This is the absolute, low cost technology," Daniell said. "All that the companies need to do is grow more plants, harvest the leaves, dry them, powder them and put them in a capsule...That is it."

Daniell and Herzog hope to propose their first clinical trial five years from now.