Pain Relief for Sickle Cell Patients

Pain Relief for Sickle Cell Patients

The Food and Drug Administration (FDA) awarded Deepa Manwani, M.B.B.S., and colleagues $1.2 million over four years to study the effectiveness and safety of Gamunex, a drug that may offer pain relief to patients with Sickle Cell disease (SCD— an inherited blood disorder in which red blood cells assume a sickled shape due to a mutation affecting the protein hemoglobin. A major symptom of SCD is periodic episodes of pain, called crises, caused by white and sickled red blood cells clogging blood vessels. Previous studies found that giving Gamunex intravenously blocks the interaction of white and red cells and improves blood flow. The FDA funding will support a phase 2 clinical trial, in which a specific dosage of Ganumex will be tested in a double-blind, placebo-controlled study to determine its safety and effectiveness in relieving pain in patients. Dr. Manwani is associate professor of clinical pediatrics.