- Burnett School College of Medicine
At a time when research funding is hard to come by, UCF College of Medicine microbiologist Dr. Henry Daniell has landed $5.5 million in National Institutes of Health highly competitive grants for hemophilia research.
The first grant, worth $3.6 million over five years, is aimed at determining whether a green technique pioneered at the college will help make treatment of hemophilia A more effective. The second grant, worth $2 million over four years, is for similar research for the treatment of hemophilia B. The grants were awarded to the College of Medicine and the University of Florida, which applied for funding jointly thanks to a longtime collaboration between Dr. Daniell and a former mentee.
Hemophilia is an incurable bleeding condition that affects about 400,000 adults and children worldwide. Hemophilia is characterized by defects in the gene that produces proteins required for blood to clot. Hemophilia A, the most common type of hemophilia, is characterized by prolonged or spontaneous bleeding, especially in the muscles, joints or internal organs.
Treating hemophilia is challenging and dangerous because many patients suffer fatal allergic reactions to the protein that doctors use to facilitate blood clotting. Dr. Daniell’s team is working on a way to make patients resistant to any deadly allergic reactions caused by the protein.
Treatments with the protein are also expensive. They must be provided in a hospital setting under supervision, and they can cost up to $1 million over a patient’s lifetime 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.
“I am confident we will achieve success sooner than you think,” said Dr. Daniell, a microbiologist at the college’s Burnett School of Biomedical Sciences. “We are hopeful that this technique will potentially save thousands of lives.”
Dr. Daniell’s team is using genetically modified plants to encapsulate a tolerance-inducing protein within plant cells so the protein could be ingested and safely travel through the stomach before being released into the small intestines, where the immune system can act on it.
In mice with hemophilia B, when the blood clotting factor encapsulated within plant cells was delivered to the gut, it prevented fatal anaphylactic shock and complex immune reactions. The new NIH funding, which came through the National Heart, Lung and Blood Institute, will help propel the research to determine if the technique can work in other models and potentially to clinical trials thereafter.
“The collaboration has an excellent chance of developing treatments that improve the lives of people with hemophilia and, at the same time, help lower health care costs,” said Dr. Roland Herzog, a professor at UF, whom Dr. Daniell mentored.
The NIH funding has come after Dr. Daniell’s research was featured last year in Proceedings of the National Academy of Sciences, a highly acclaimed scientific journal. Bayer Healthcare of Germany, the world’s largest funder of hemophilia research, also gave Dr. Daniell a $200,000 grant in 2010 for research exploring the novel concept.
The Burnett school professor is also conducting similar research on a polio vaccine funded by the Bill and Melinda Gates Foundation and on diabetes funded by the Juvenile Diabetes Research Foundation.