Hemophilia Research Earns UCF Prestigious Award

Hemophilia Research Earns UCF Prestigious Award

UCF College of Medicine Professor Henry Daniell is one of only five scientists and clinicians worldwide to earn a Special Project Award from one of the world’s largest funders of hemophilia research.

Bayer HealthCare, of Germany, awarded Daniell the grant to continue his cutting-edge research into hemophilia, an incurable bleeding condition that affects about 400,000 worldwide. Hemophilia is characterized by defects in the gene that produces a protein required for blood to clot. Hemophilia A, the most common type of hemophilia, is characterized by prolonged or spontaneous bleeding, especially into the muscles, joints or internal organs.

Treating the diseases is challenging and dangerous because many patients suffer fatal allergic reactions to the expensive protein that doctors use to make their blood clot.  Treatments must be provided in a hospital setting under supervision, and they can cost up to $1 million because of the required hospital stays and blood transfusions. Average annual treatment costs are $60,000 to $150,000, according to the National Hemophilia Foundation.

To prevent the potentially deadly reactions, Daniell and his team at the University of Central Florida want to help patients develop a tolerance to the therapeutic protein before they seek treatment.

The researchers are using genetically modified plants to encapsulate a tolerance-inducing protein within plant cell walls so that it could be ingested and safely travel through the stomach before it’s released into the small intestines, where the immune system can act on it.

In hemophilic mice, when blood clotting factor IX bioencapsulated in plant cells was delivered to the gut, it prevented fatal anaphylactic shock and complex immune reactions. If future research bears out, this approach would be much safer and potentially much less expensive to deliver.

Daniell’s research was featured in Proceedings of the National Academy of Sciences, a highly acclaimed U.S. scientific journal, in April.

“It’s quite an honor to be ranked first in a global competition,” Daniell said. “This grant will certainly help us to move this research forward and potentially save thousands of lives.”

Daniell completed his research in collaboration with a former student, Roland Herzog, an associate professor at the University of Florida’s College of Medicine.

Since its founding in 2002, Bayer HealthCare has awarded 175 grants, totaling more than $20 million, to researchers, clinicians and caregivers from 28 countries.

The Bayer Hemophilia Awards Program supports basic and clinical research and education in hemophilia. Through grants provided to early career researchers, fellows in training and other hemophilia care professionals, the program seeks to support the next generation of care and treatment options for people with hemophilia worldwide.

For the past two decades, Daniell has developed transgenic plants for producing and delivering oral vaccines and immune-tolerant therapies. He has used similar techniques to create potential vaccines against malaria and cholera and genetically engineered insulin into plants to help prevent diabetes.

Daniell joined UCF’s Burnett School of Biomedical Sciences in 1998. His research led to the formation of the university’s first biotechnology company. Daniell also became only the 14th American in the last 222 years to be elected the Italian National Academy of Sciences. In 2007, he was named a Fellow of the American Association for the Advancement of Sciences.

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