Research Targets Sickle Cell Disease

A UAB researcher’s involvement in clinical trials helped approve a first-of-its-kind targeted medication to reduce pain crises for individuals with sickle cell disease.

Medical research being done in Birmingham aims to decrease painful symptoms of sickle cell disease that can have devastating effects on the patient’s health.

Sickle cell is the result of a gene mutation that causes red blood cells to produce sickle hemoglobin, which forces red cells to take on a stiff, inflexible crescent shape. The crescent cells can clog blood vessels triggering painful episodes, pneumonia, central nervous system damage, strokes and kidney failure. Sickle cell disproportionately affects African Americans.

Vaso-occlusive crises are the primary reason that people with sickle cell disease are admitted to the hospital.

The new monthly intravenous therapy for the disease, Adakveo (crizanlizumab), recently got approval by the U.S. Food and Drug Administration.

Julie Kanter, M.D., director of the University of Alabama at Birmingham Adult Sickle Cell Clinic within the Division of Hematology and Oncology and Department of Medicine, participated in the clinical trial.

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“The approval of Adakveo will decrease the rate of crisis in sickle cell disease and hopefully improve long term health outcomes in affected patients by decreasing their risk of death,” Kanter explained. “Simply put, it is a game changer. This will also allow us to see higher risk patients at least every month, which helps us recognize if other associated health problems are developing.”

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