Novel Drug Prevents Common Viral Disease in Stem-Cell Transplant Patients

A new drug can often prevent a common, sometimes severe viral disease in patients receiving a transplant of donated blood-making stem cells, a clinical trial led by researchers at Dana-Farber Cancer Institute and Brigham and Women's Hospital indicates.

In a paper in the Sept. 26 issue of the New England Journal of Medicine, the researchers report that patients who took the drug CMX001 shortly after transplant were far less likely to develop cytomegalovirus (CMV) infection than were patients who took a placebo. CMV disease is a common source of illness in transplant patients and can cause pneumonia, diarrhea and digestive tract ulcers, or other problems. Although some antiviral drugs, when administered at the earliest signs of CMV infection, often forestall CMV disease, they often cause kidney dysfunction or impair patients' ability to make new blood cells.

"With current agents, between 3 and 5 percent of allogeneic [donor] transplant patients develop CMV disease within six months of transplantation, and a small number of them may die of it," says study lead author Francisco Marty, MD, of Dana-Farber and Brigham and Women's. "There clearly is a need for better treatments with fewer adverse effects. This clinical trial examined whether the disease can be prevented, rather than waiting for blood tests to show that treatment is needed."

CMV is a herpes-like virus that infects the majority of Americans by adulthood. Most often, it is held in check by the immune system and produces no symptoms. Most people are unaware they are infected, Marty explains.


In stem-cell transplant patients, however, the immune system -- based in the blood-making tissue of the bone marrow -- is replaced with donor blood-making cells after patients receive high doses of chemotherapy. During this transition period, long-dormant viruses like CMV may have an opportunity to become active. The result can be CMV disease.

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