Targeting a Sickle-Cell Problem

Targeting a Sickle-Cell Problem

Sickle cell disease (SCD), a genetic disorder in which red blood cells are misshapen, causes serious complications. One SCD complication is painful leg ulcers, for which there is no approved treatment. Following up on her successful phase 1 study, the U.S. Food and Drug Administration has awarded Caterina Minniti, M.D., a four-year, $1.9 million grant to conduct a phase 2 trial of sodium nitrite cream for treating leg ulcers in SCD patients. When the cream is applied to the wound, sodium nitrite is converted into nitric oxide (NO), which causes blood vessels to dilate and also kills bacteria, stimulates the proliferation of keratinocytes (the main cell type in the skin’s outer layer) and decreases inflammation. These NO- induced changes aid healing. In the phase 1 study, the sodium nitrite cream decreased pain at the wound site and reduced patient’s narcotic use. Dr. Minniti is professor of medicine and of pediatrics at Einstein and director of the Sickle-Cell Center for Adults at Montefiore. (1R01FD005729-01)