Experimental drug shows promise in treating severe, often-lethal complication of stem cell transplants

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Drug derived from pig’s intestines

An experimental drug called defibrotide reversed severe veno-occlusive disease (VOD) of the liver in more than one third of the stem cell transplant recipients enrolled in a study. VOD is a type of potentially fatal liver damage that can result from the high doses of chemotherapy given prior to a transplant. The findings, to be published in the Dec. 15, 2002, issue of Blood, have been posted as a “First Edition Paper” on the journal’s web site ( “Stem cell transplant patients suffering from severe veno-occlusive disease are at a very high risk of death, with a mortality rate in excess of 90 percent. The results from this study are compelling, especially given the remarkable safety of the drug in this extremely sick population,” says Paul G. Richardson, a hematologic oncologist at Dana-Farber who led the multi-center study. The study was funded in part by the Orphan Products Development Program of the Food and Drug Administration. Defibrotide is manufactured by Gentium SpA of Como, Italy.