An innovative UCF scientist has helped land two NIH grants totaling $5.5 million, the University of Central Florida announced Friday. The National Institutes of Health funding will be used to research a better treatment for hemophilia, the life-threatening blood disorder.
UCF microbiologist Henry Daniell will share the award with his colleagues at University of Florida, who will collaborate on the project. One five-year grant for $3.5 million will go toward treating hemophilia A, and a second four-year grant for $2 million will go toward treating hemophilia B. Both research projects involve developing a safer, far less expensive way of treating the disease.
Competition for NIH funding is stiff these days, said Daniell, principle investigator on the project. "Only five percent of the projects put before the 35-member NIH panel got funded. So everyone had to think this was great."
A blood disorder in which a person lacks the proteins necessary to make blood clot, hemophilia affects 20,000 Americans, mostly male, according to estimates from the Centers for Disease Control and Prevention. The inherited blood disorder affects one in 5,000 males, putting them at greater risk of spontaneous bleeding.
An injectable protein replacement is available, but more than half of all hemophiliacs develop allergic reactions to the protein, said Daniell. "If the body doesn't mesh with the protein replacement, it develops an immune reaction that is highly toxic and can be fatal."
To get around that, UCF and UF scientists are encasing the protein in genetically engineered plant material, and creating capsules that patients swallow. When delivered to the gut this way, the body tolerates the protein and doesn't fight it off.
Besides being life threatening, the current protein treatment is also expensive and must be provided in a hospital. Costs can reach up to $1 million over a patient's lifetime, according to the National Hemophilia Foundation. The plant-wrapped protein pills would be inexpensive, have a long shelf life and could be taken at home.
Daniell, who has spent the past decade developing vaccines grown inside genetically engineered lettuce, is also working on a polio vaccine that can be delivered this way for just pennies apiece. That project has been funded by the Bill & Melinda Gates Foundation, which plans to bring the vaccine to third-world countries.
On the hemophilia project, Daniell is working with UF professor Roland Herzog, whom he mentored. "The collaboration has an excellent chance of developing treatments that improve the lives of people with hemophilia and lower health care costs," said Herzog.
Their research was featured last year in the journal of the National Academy of Sciences.
Once the polio pill vaccine is available, the pill for hemophiliacs will be right behind it, Daniell said. He expects the hemophilia pill to be on the market within the next five years.