The drug, called decitabine, works even in patients who don't respond to hydroxyurea, the standard treatment, and appears to produce fewer side effects.
"The results of our study are compelling," said Joseph DeSimone, professor of hematology and oncology and a member of the UIC Cancer Center. "Clearly, decitabine is an effective therapy for patients who do not benefit from the traditional treatment. It is possibly even an improvement, although more studies will have to be done."
The clinical trial was conducted at the University of Illinois Medical Center at Chicago, which houses the area's only clinic specializing in sickle cell disease. Results of the study will appear in the June 1 issue of the scientific journal Blood.
Sickle cell disease is caused by a single mutation in a gene that manufactures hemoglobin, the molecule in red blood cells that delivers oxygen to tissues throughout the body.
The defective form of hemoglobin, called hemoglobin S (for sickle), causes serious health problems. Red blood cells take on a characteristic crescent, or sickle, shape and stick to blood vessel walls.
As a result, the cells cannot squeeze through capillaries. Blood supply is blocked, depriving tissues and organs of oxygen and causing short- and long-term damage. Patients suffer episodes of intense pain, typically in the arms, legs and back, but also in the liver, kidney and abdomen.
In some severe cases, transfusions may be required because the aberrant red blood cells have a much shorter life span than normal red blood cells (10 days vs. 100) and the body can't manufacture enough new red blood cells to keep up, leading to anemia.
Patients may also suffer what is called acute chest syndrome, a particularly serious complication and th
Contact: Sharon Butler
University of Illinois at Chicago