Cancer Drug May Help with Sickle-Cell Anaemia

Decitabine, a drug for leukemia and other cancers, may be a useful treatment for sickle cell anaemia when the usual therapy cannot be given, new research suggests.

Decitabine, a drug for leukemia and other cancers, may be a useful treatment for sickle cell anaemia when the usual therapy cannot be given, new research suggests. Sickle cell anaemia involves an abnormality in hemoglobin, an oxygen-carrying protein found in red blood cells. This abnormality causes the red cells to take on a rigid sickle shape when oxygen levels are low. This shape makes it difficult for the cells to pass through certain blood vessels, resulting in severe pain and other problems. This also reduces the surface area of the red cell’s and therefore further reduces oxygen capacity. Hydroxyurea, a standard treatment for sickle cell anaemia, increases levels of a fetal form of hemoglobin, which prevents sickling and the associated pain, lead author Dr. Yogen Saunthararajah and his colleagues explain. However, some patients do not respond to treatment and others cannot tolerate the drug. As an alternative, Saunthararajah, at the University of Illinois at Chicago, and his group conducted a study of decitabine in three patients resistant to hydroxyurea and five who developed side effects with the drug. Decitabine was injected under the skin one to three times per week during two 6-week periods. The researcher’s findings are reported in the medical journal Blood. Fetal hemoglobin levels increased markedly in all patients during treatment, the authors note. In fact, the peak levels achieved with decitabine were higher than those previously achieved with hydroxyurea. Moreover, features that are associated with the painful sickle cell episodes also improved dramatically, the investigators write. The only significant side effect was a drop in certain white blood cells, Saunthararajah told Reuters Health. However, a simple dose adjustment would probably solve this problem, he added. Still, the authors warn that longer follow-up is needed to rule out the possibility that decitabine could have long-term cancer-causing effects. Saunthararajah’s group has submitted applications to the NIH and the FDA for an 18-month study using a similar regimen. They hope to examine “clinical end points, such as pain crises and frequency of pneumonia,” to prove that “decitabine is clinically effective.” (Source: Blood, Karla Gale Yahoo Health News, Reuter’s December 2003)