New Approaches To Make Bone Marrow Transplantation Safer And More Effective

Armen Hareyan's picture

Bone Marrow Transplantation

Radical new treatment approaches at Children's Hospital of Pittsburgh of UPMC are allowing more and more young cancer patients to receive life-saving bone marrow transplants.

Children's oncologists led by Rakesh Goyal, MD, director of the Blood and Marrow Transplantation Program, are at the forefront of developing reduced-intensity bone marrow transplants (BMT) and are exploring cutting edge cancer therapies such as arsenic, best known as a deadly poison.

Reduced intensity transplants involve using lower doses of chemotherapy before transplant to greatly reduce the risk of complications after transplant.

"By making BMT safer, we hope to make it a more practical option for pediatric patients suffering from life-threatening diseases that once were considered incurable such as high-risk leukemias and other forms of cancer, sickle cell disease and severe aplastic anemia," said Dr. Goyal, also an associate professor of Pediatrics at the University of Pittsburgh School of Medicine. "Through our approaches, we are reaching patient survival rates that often exceed national averages."


Also, Children's has become one of only a handful of pediatric cancer centers in the country to use a revolutionary new therapy, arsenic trioxide to treat a form of leukemia known as acute promyelocytic leukemia (APL). This arsenic compound was first used as a treatment in native Chinese medicine and in recent years has proven successful in treating adults with cancer in clinical trials sponsored by the National Cancer Institute.

A 3-year-old patient from Washington County with APL recently became Children's first, and one of the nation's youngest, to be treated with arsenic. Treatment with arsenic trioxide causes programmed cancer cell death, working similarly to chemotherapy agents yet without the toxic side effects, according to Lakshmanan Krishnamurti, MD, an oncologist at Children's.

"Because we were able to bring our patient's APL into remission using arsenic and without chemotherapy, which would have wiped out his immune system, we were able to perform this crucial bone marrow transplant," said Dr. Krishnamurti, also an associate professor of pediatrics at the University of Pittsburgh School of Medicine and the director of Children's Sickle Cell Program.

April is Donate Life Month and Children's is celebrating Organ Donor Awareness Week this week to raise awareness about the need for organ, tissue and marrow donors. Children's BMT Program was established in 1991 and earlier this year earned prestigious accreditation from the Foundation for the Accreditation of Cellular Therapy.

Dr. Krishnamurti also has pioneered reduced-intensity BMT as a cure for sickle cell disease, performing the first-such procedure in the tristate region in 2003 in a young boy who no longer has the disease.

Children's BMT Program utilizes bone marrow, peripheral