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Drug Boosts Platelet Production, Reversing Immune Thrombocytopenic Purpura

Attacking a platelet-depleting autoimmune disease in a whole new way, an experimental drug is helping patients with immune thrombocytopenic purpura (ITP) once again// produce healthy amounts of platelets -- with no major side effects.

That's the conclusion of a new, multicenter study led by Dr. James B. Bussel, professor of pediatrics at Weill Cornell Medical College, attending pediatrician at NewYork-Presbyterian Hospital/Weill Cornell Medical Center, and director of the Hospital's Program for Platelet Disorders.

The new drug, a novel protein called AMG 531, successfully boosted platelet production in patents with chronic ITP, a serious autoimmune disorder that affects more than 16,000 adult Americans, and perhaps as many children, each year.

In ITP, immune system antibodies mysteriously begin to attack and destroy blood platelet cells. In some cases, the disease goes into spontaneous remission, but for many patients it remains a chronic condition. Impaired clotting leaves many patients, especially the elderly, vulnerable to serious or fatal hemorrhage.

Up till now, ITP patients have typically turned to powerful drugs such as corticosteroids or intravenous immune globulin, which work by inhibiting platelet destruction. These drug therapies can have limited success, but they also have serious side effects. For some ITP patients splenectomy (surgical removal of the spleen) is another treatment option.

AMG 531 fights ITP in a totally different way.

"Experts have long realized that ITP not only destroys platelets, it also inhibits platelet production in the marrow," explains Dr. Bussel.

In fact, prior work in the 1990s had focused on a type of recombinant thrombopoietin, called PEG-MGDF, that researchers hoped would stimulate platelet production.

The drug did have success. "However, Dr. David Kuter at Massachusetts General Hospital showed that some patients -- and even hea
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