VUMC’s bone marrow transplant efforts get infusion of expertise
Bone marrow transplantation at Vanderbilt is getting a boost with the recruitment of two specialists — both of them trained at the institution that pioneered the procedure 30 years ago — to strengthen Vanderbilt's programs for adults and children.
Dr. Friedrich Schuening has joined Vanderbilt as director of Hematology and the Bone Marrow Transplant Program. A member of the Vanderbilt-Ingram Cancer Center, Schuening also has been named Ingram Professor of Cancer Research, an endowed professorship funded through the ongoing Imagine a World Without Cancer Campaign.
Schuening most recently headed the Bone Marrow Transplant Program at the University of Wisconsin, where the annual number of procedures doubled under his four-year leadership. Prior to that appointment, Schuening spent more than a decade at the Fred Hutchinson Cancer Center in Seattle, where the first bone marrow transplants in the United States were performed in the late 1960s.
Meanwhile, Dr. Haydar Frangoul has joined Vanderbilt Children's Hospital and the VICC as associate professor of Pediatric Hematology-Oncology to build Vanderbilt's pediatric bone marrow transplantation program. Frangoul comes to Vanderbilt from Fred Hutchinson, where he served as a senior fellow in pediatric hematology-oncology. Previously, Frangoul spent three years as a general pediatrician in Charleston, W.Va., after completing a pediatric residency and one year of pediatric hematology-oncology fellowship at Duke University.
The two physicians, who did not know each other previously, say it is a fortunate coincidence that they have arrived at the same time and can build the two programs in a parallel fashion.
"Having a strong adult program will be of great benefit," Frangoul said. "I believe that we see things eye-to-eye and can develop a strong collaboration because we've been trained with the same philosophy."
Schuening agreed. "The idea is that this will be a strong program that will bring together activity in the adult program at Vanderbilt, the pediatric program at Vanderbilt as well as the program at the Veterans Affairs Medical Center," he said.
Bone marrow transplants (BMT) are used to treat a number of diseases, including many forms of leukemia, lymphoma, anemias and some solid tumors. The bone marrow, which is found inside the hollow bones, produces the body's platelets (for clotting), red blood cells (for carrying oxygen) and white blood cells (primary agents of the immune system). In a BMT, high-dose chemotherapy and/or radiation is used to destroy the bone marrow, and healthy marrow cells (stem cells) are infused into the patient to replace it. In cases of leukemias and other blood diseases, the transplant is used to replace diseased marrow; in treatment of solid tumors, the transplant is used to permit higher doses of chemotherapy.
There are three types of BMT: autologous, in which some of a patient's own stem cells are collected before chemotherapy and infused after treatment; related allogeneic, in which a family member's marrow is used; and unrelated allogeneic, in which a matched donor who is not a family member is identified.
Unrelated BMTs are more challenging to do because they carry a greater risk of complications, including rejection of the donor marrow and a condition called graft-versus-host disease (GVHD). In GVHD, the donor marrow cells recognize the patient's body as "foreign" and react against it.
At Vanderbilt Children's Hospital, Frangoul and his colleagues in Pediatric Hematology-Oncology are busy making preparations to begin doing unrelated allogeneic BMTs later this fall. "We hope that many of our patients will no longer need to be referred to other centers and that we can expand our service area into Knoxville and Chattanooga," Frangoul said.
Also on the horizon: offering "cord blood transplants" at Vanderbilt sometime next year. In this procedure, the immature stem cells — which "grow up" to become red cells, white cells or platelets — are harvested from umbilical cords donated at birth and infused into patients requiring BMT.
Frangoul and Schuening say they expect a number of advances in the near future to improve effectiveness of BMT techniques and to reduce complications, including better treatments and preventions of GVHD.
For instance, Schuening hopes to establish use at Vanderbilt of new approaches to eliminate "minimum residual disease," leftover tumor cells that remain in the body even after high-dose chemotherapy. "One such approach is an immunotherapeutic one, in which one would specially educate or prime the patient's immune system cells against the tumor cells and transfuse those into the patient after BMT," Schuening said. "The goal would be to get rid of the few remaining cancer cells in the body and prevent recurrence."
Another investigational but promising approach that Vanderbilt plans to begin offering in the future is "mini-transplantation," which combines lower doses of chemotherapy with use of donor-derived immune system cells that can recognize and fight tumor cells in a "graft versus tumor" effect. "We would hope to achieve cure with a less toxic approach," Schuening said. "We might be able to use this technique with patients who are not eligible for current transplant approaches because of advanced age, pulmonary or cardiac problems, or other health concerns. We are very excited about this possibility."
Schuening's own basic research interest focuses on transfering therapeutic genes into the stem cells to treat genetic diseases and some forms of cancer. "The stem cells are the only cells in the body that continue to perpetuate for the lifetime of the patient, so the effect of the gene therapy, if successful, could be longterm," he said.
A native of Germany, Schuening was working with a colleague to establish that country's first BMT program in the early 1980s when he decided to come to the United States for more in-depth training at Fred Hutchinson. He had intended to return to Germany in three years but decided to stay on when given an opportunity to continue his laboratory research.
Frangoul received his medical education at the American University of Beirut in Lebanon before coming to the United States for residency training. Although he always had an interest in pediatric oncology, he says that his experience as a general pediatrician in private practice was a beneficial one. "I think having that experience has made me a better doctor than I would have been otherwise," Frangoul said.