Calling an early halt to a national trial because of promising results, doctors Monday announced the first treatment for sickle cell anemia that attacks the underlying cause of the disease rather than simply combats its painful symptoms.
The National Institutes of Health said it was issuing a clinical alert to thousands of doctors nationwide informing them that the drug hydroxyurea should be considered for many adult patients with the crippling blood disease.
The institutes had sponsored the trial of the drug at nearly two dozen centers to see if it could reduce the painful episodes characteristic of the disease, which in this country primarily afflicts AfricanAmericans.
“This is the first effective treatment for sickle cell disease and we are elated,” said Dr. Samuel Charache of Johns Hopkins University Medical School, the principal investigator and coordinator of the planned five-year study, “but we want to emphasize that hydroxyurea is a treatment for the disease and not a cure.”
Moreover, doctors cautioned that there could be adverse effects in some patients and that the drug should not be given to women who plan to have children nor, for now, to children.
Experts estimate, between 70,000 and 80,000 people in the United States have the disease.