Until this week, medical science held out little hope for victims of sickle cell anemia, an inherited blood disorder that causes organ damage, episodes of extreme pain and premature death. The announcement Monday that researchers at the Johns Hopkins University Medical School have developed the first effective treatment for severe forms of the illness means that sickle cell may one day become a manageable, chronic illness rather than the relentless killer it is today.
Between 70,000 and 80,000 people in the United States suffer from the disease. In the United States it primarily affects African Americans, but it is also common among people whose ancestors came from the Middle East, the Mediterranean and India. The disease is produced by a genetically programmed deformation in the oxygen-carrying red blood cells, which prevents them from circulating freely through the body's network of tiny blood vessels. It takes its name from the characteristic sickle shape of the affected red cells, which in healthy people are roughly spherical in form.
The Hopkins researchers, led by Dr. Samuel Charache of the medical school, found that a drug called hydroxyurea, which is used to treat some cancer patients, reduced by 50 percent the number of pain episodes, hospitalizations, blood transfusions and life-threatening complications among patients with severe forms of the illness. The National Institutes of Health found the initial results of hydroxyurea therapy so encouraging that it ended clinical trials several months early and advised doctors to consider using the drug immediately for adult sickle cell patients. Further trials will be needed to determine if the drug is safe for use by children and pregnant women.
Dr. Charache was careful to note that the new treatment is not a cure. Nor do researchers yet understand exactly how hydroxyurea suppresses the most severe symptoms of the disorder, though they suspect it involves stimulating the body's own production of fetal hemoglobin, a type of red blood cell that the body normally stops making during infancy and that does not deform into the sickle shape. In adults who took hydroxyurea, the level of fetal hemoglobin rose by as much as 20 percent.
The Food and Drug Administration still must approve hydroxyurea's use for sickle cell patients before most health insurers will pay for the drug, which costs about $100 a month. The cost is a hardship for many patients. That is why the FDA should expedite approval so that victims of this cruel disease can begin enjoying the life-saving benefits of the treatment as soon as possible.