The physicians at UPMC Children’s Hospital of Pittsburgh's Blood and Marrow Transplantation and Cellular Therapies (BMTCT) program continuously strive to improve cellular therapies for treatment of childhood cancer and blood diseases.
The division’s research is pursuing new treatment methods, such as reduced-intensity conditioning, that will make marrow and cord blood transplants kinder, gentler, and safer for pediatric patients. The team’s ultimate goals are to reduce the complication rate and improve the quality of life for children undergoing these specialized procedures.
In pursuit of these goals, investigators are active on multiple fronts, using both basic laboratory research and clinical trials. In the laboratory, scientists are studying immune reconstitution, tolerance and alloreacivity, and cellular immunotherapy. Clinical investigations are underway to trial novel therapies for treating Crohn’s disease and other autoimmune diseases, Krabbe disease and other leukodystrophies, MPS syndromes, and high-risk leukemias.
Other BMTCT studies are focused on understanding the relationship between individual genetic variability and post-transplant outcomes, including graft-versus-host disease (GVHD). Our investigators have learned that individual variation in immune-response genes influences a child’s outcome after transplantation. This has led to further clinical research focused on therapies that move away from the "one size fits all" approach to tailored treatments that are based on a each patient’s specific metabolic needs.
