A drug normally used to treat leukemia may prove to be the first effective drug to alleviate the pain and suffering of sickle-cell anemia, according to a Johns Hopkins Medical Institutions researcher.
The drug hydroxyurea appears to turn on a gene that directs the production of fetal hemoglobin, an immature version of the oxygen-carrying red pigment in the blood.
In patients with sickle-cell anemia, a genetic defect in the production of adult hemoglobin causes red blood cells, which normally have a donut shape, to deform into a sickled shape, permanently destroying the cells and causing them to become stuck in clumps, thus blocking blood vessels.
Generally, the disease, which affects primarily blacks, is treated with transfusions to replace damaged red blood cells as well as with antibiotics and pain relievers. Hydroxyurea attempts to turn on the production of fetal hemoglobin, normally turned off in adults, to provide a sufficient supply of normal hemoglobin.
Dr. Samuel Charache at Hopkins and researchers at several other universities are beginning phase-one trials to determine the safety of using hydroxyurea. Later studies will determine whether the drug can effectively treat sickle-cell anemia. (Anyone interested in participating in the trial should contact Johns Hopkins.)