New drug combination may prevent dangerous complication of bone marrow transplantation

An ongoing clinical study by Dana-Farber Cancer Institute scientists suggests that a three-drug therapy, which includes a novel medication called sirolimus, reduces graft-versus-host disease (GVHD) in stem cell transplant patients more effectively and with less toxicity than traditional treatments. The study seeks to produce better outcomes for patients receiving stem cell transplants for diseases such as leukemia and lymphoma. Such patients are at risk for GVHD, a condition in which transplanted immune cells mount an attack on patients’ own tissue and organs. The drugs tacrolimus and methotrexate, though offering moderate control of GVHD, are associated with side effects such as kidney failure, high blood pressure, mouth sores, liver damage, and lowered white blood cell counts. “These are very exciting early data,” says the study’s lead author, Joseph Antin of Dana-Farber. “We have been looking for ways of reducing transplant-related side effects for many years and these data tell us that we are on the right track. We are proceeding with further studies designed to eliminate methotrexate entirely while continuing to reduce toxicity and control GVHD.” The findings were presented at the annual meeting of the American Society of Hematology in Philadelphia on Dec. 9, 2002.