The clinical research mission of the Division of Bone Marrow Transplant and Cellular Therapies is to design and test novel, disease-specific, and biologically rational reduced-toxicity transplantation regimens for life-threatening conditions. Transplant indications span from birth through adulthood and utilize human leukocyte antigen (HLA)-matched or mismatched bone marrow, cord blood, or autologous peripheral blood stem cell grafts.
- Neurodegenerative conditions/inborn errors of metabolism
- Primary Immune Deficiencies
- Autoimmune diseases/Immune dysregulation (Crohn’s Disease, Systemic Sclerosis)
- High risk acute leukemia
- RIC-MAC for AML/MDS
- Bone marrow failures and transfusion dependent anemias (Sickle cell disease, Thalassemia, Diamond-Blackfan Anemia)
Through our BOLT-BMT study, we are coordinating a first in human clinical trial that will explore the safety and efficacy of bone marrow transplant following solid organ transplant from the same cadaveric donor to establish immunity, tolerance, and extend organ survival. Our division is also researching tandem lung transplants and bone marrow transplants for primary immunodeficiency patients with end stage lung disease.
Our cellular immune therapy research aims to test regulatory T cell therapy for refractory autoimmune diseases and control life-threatening viral infections in immunocompromised children and adults.
Our laboratory research projects aim to understand the development of immune competence alongside immune tolerance following HLA-mismatched bone marrow transplant and modulate metabolic pathways involved in graft-versus-host disease and anti-leukemia immunity.