Thalidomide-like drug treats blood disorder

From San Diego, at a meeting of the American Society of Hematology

A novel drug appears to help people with myelodysplasia, a persistent condition that leaves them short of crucial blood components. The drug could become the first treatment specifically for the condition, says Alan List of the University of South Florida in Tampa.

In people with myelodysplasia, underproductive bone marrow fails to produce enough platelets and blood cells. The resulting syndrome features excess bleeding, anemia, and infections and may lead to a severe form of leukemia.

The drug thalidomide can sometimes increase red-cell numbers in myelodysplasia, but the treatment reduces white cells and causes neurological side effects. Because thalidomide and other treatments are often ineffective, many people with the syndrome need frequent transfusions of blood products and die early.

In search of an alternative treatment, List and his colleagues treated 45 myelodysplasia patients with CC5033, a new compound that’s chemically related to thalidomide. The volunteers received either 10 milligrams or 25 mg of CC5033 per day.

Two-thirds of the volunteers showed higher concentrations of red cells after 16 weeks of treatment. More than half of these patients haven’t needed a blood transfusion for more than 40 weeks.

The 25 mg/day dose of CC5033 invariably reduced volunteers’ white-cell counts, possibly increasing infection risk. However, only about half of the patients getting 10 mg/day for 3 out of every 4 weeks experienced that side effect.


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