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Keri Smith, Ph.D.
assistant professor of pathology and laboratory medicine, MS
Research Work: Novel Covalent Antigens to Induce B Cell Tolerance
Treating a cut with soap, water, peroxide and a bandage to stop the bleeding seems like an effective remedy. However, people with hemophilia need much more than a first aid remedy and a bandage to heal their wounds.
Hemophilia is a genetic bleeding disorder where blood does not clot normally due to a missing protein. Doctors treat patients with hemophilia by using replacement therapy, which essentially replaces the missing protein. However, this mode of therapy does not work for every patient.
“Patients who have hemophilia are missing a vital protein in the clotting cascade known as Factor VIII (FVIII),” said Keri Smith, Ph.D. “To stop bleeding, doctors inject hemophiliac patients with replacement FVIII. Unfortunately, giving replacement FVIII is not effective in up to 30 percent of patients.”
Smith said the ineffectiveness is because certain white blood cells, known as B cells, recognize replacement FVIII as a foreign protein and attack it by making antibodies against it.
“These antibodies are specific proteins that bind to the FVIII and render it ineffective,” she explained.
Smith’s work focuses on preventing the B cells from making antibodies against FVIII, while searching for better and more effective treatments for hemophilia.
“Our laboratory has developed a novel compound that we believe will bind specifically to the problem-causing B cells and make them stop producing antibodies against FVIII,” Smith said.
By using mice genetically altered to have hemophilia, Smith studies the reaction that occurs after injecting them with replacement FVIII to find that the mice also develop B cells that make antibodies against FVIII.
Smith’s findings may lead to ways to deactivate the B cells and make replacement FVIII therapy more effective for more patients or open doors to new lines of treatment.
“I am hopeful that I can be a part of the development of new treatments for hemophilia, while gaining more insight into the workings of the immune system,” she said.
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