Hemophilia Research Earns Dr. Daniell 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 Dr. 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, Dr. Daniell and his team at the College of Medicine’s Burnett School of Biomedical Sciences 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 intestine, 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.
Dr. 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,” Dr. Daniell said. “This grant will certainly help us to move this research forward and potentially save thousands of lives.”
Dr. Daniell completed his research in collaboration with his former student, Roland Herzog, now 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.