Allogeneic hematopoietic stem cell transplantation (SCT) is curative for many pediatric diseases. Approximately 4,500 SCTs are performed each year in North America in children less than 20 years of age, most of this for pediatric malignancies. There is great interest in immune modulation to harness the graft versus tumor (GVT) effects. Because severe forms of the graft-versus-host reaction leading to graft-versus-host disease (GVHD) contribute to significant morbidity and mortality following SCT, our major efforts at the NIH have focused on strategies that separate GVHD from the potentially beneficial immune reactivity against tumor. In addition to focusing on strategies to manipulate the GVT effect to increase the potency of SCT, we also have worked to develop a reduced intensity approach to SCT in attempt to minimize transplant-related complications. Our program is also studying novel immunotherapies including cancer vaccines after SCT. We are conducting studies of new agents that might be effective for post-transplant relapse. Finally, a unique area of research interest for our group is the application of SCT in the pediatric solid tumor population.
The ultimate goal of the Pediatric Transplant Section of the Pediatric Oncology Branch is to maximize immune mediated anti-tumor effects while minimizing toxicity in both pediatric solid tumors and hematologic malignancies.