TEAMWORK between an anticancer agent and a hematopoietic growth factor may provide the first effective treatment for sickle cell anemia.
Promising data from a small study in which the drug hydroxyurea was used to treat patients with sickle cell anemia have prompted the expansion of trials to six centers. Pediatrician George J. Dover, MD, and internist Samuel Charache, MD, who have been using hydroxyurea to treat 2 patients since 1983, have expanded treatment to 38 patients in the enlarged study.
In a pilot study involving six patients, three of whom were treated at Johns Hopkins, three patients demonstrated marked clinical improvement. However, it was pathological rather than clinical findings that stimulated enthusiasm for hydroxyurea. The drug appeared to turn on the gene responsible for the production of the gamma protein chain, which in turn can combine with the alpha protein chain to form the fetal hemoglobin molecule.
The gene for