Pediatric Cancer Research

Improving the Odds

Advances and opportunities in blood and marrow transplantation

When other treatments have failed, a blood or marrow transplant (BMT) gives some cancer patients another chance for a cure.

In a BMT, a patient's damaged or diseased blood-making cells are replaced with healthy ones. And while it is a potentially lifesaving procedure, it is also a very risky one. For patients who do not have a tissue type family match, an unrelated donor must be found through the national registry, or a family member who is not a full match may be used. It is not uncommon for patients who receive cells from an unrelated donor to contract graft-versus-host disease (GVHD), a serious complication that occurs when the donor's cells attack the patient's tissues.

Children's Hospital makes transplantation safer for patients who are at risk for GVHD. As part of an ongoing clinical trial, CHOP staff use a special device to remove the blood cells thai cause GVHD from the donor blood-making cells prior to the transplant. The technique makes transplantation a more viable option for the many patients who are not able to find a well-matched donor.

"There are very few centers that do this," says Nancy J. Bunin, M.D., director of Blood and Marrow Transplant at Children's Hospital.

Henry Korneffel, now a healthy 9-year-old, underwent this procedure six years ago. Since then, his family has given generously to Bunin's program for years and provided the funds to purchase the newer devices used in the procedure.

Another trial, which grew out of laboratory research led by Stephan A. Grupp, M.D., Ph.D., explores the use ofsirolimus, an immunosuppressant, to help prevent relapse and GVHD in acute lymphoblastic leukemia patients who receive a blood or marrow transplant. The trial was piloted at CHOP and is now part of a national study, giving new hope to children with a particularly grim prognosis.

Bunin's team also participated in a pilot study led by the University of Pennsylvania's David Porter, M.D., in which patients who had relapsed after transplant were given donor immune cells that were activated by a special procedure available at Penn's Abramson Cancer Center, making them more effective at killing cancer cells. The treatment was shown to be beneficial for some leukemia and lymphoma patients, but CHOP researchers need additional funds to continue and expand this exciting new technique.

Bunin is also seeking funding to conduct mulricenter studies that would explore the long-term neuropsychological and physical effects of transplantation on children. Recommendations that result from such studies could have a profound impact on patients' futures because as much as researchers have learned about how to improve outcomes for transplant recipients, many questions still remain.

Source: Cause for Hope: CHOP Cancer Center Annual Report 2009