The clinical mission of the Division of Blood and Marrow Transplantation and Cellular Therapies is to design and test disease-specific and biologically rational novel reduced-toxicity transplantation regimens for patients with high-risk leukemia or lymphoma, and for those afflicted with life-threatening inherited conditions that can lead to bone marrow failure, immune deficiency, autoimmune diseases, and neurodegenerative conditions including but not limited to leukodystrophies and mucopolysaccharidosis syndromes.
One of our signature protocols (ClinicalTrials: NCT01962415) has attracted patients from two dozen states, ranging from Florida to Alaska. Patients with approximately 20 unique genetic diagnoses enrolled on this reduced-intensity conditioning (RIC) trial. Diagnoses ranged from sickle cell disease, thalassemia, osteopetrosis, Krabbe disease, MLD, too many primary immune deficiency syndromes such as BLS, and XIAP deficiency. Day 100 non-relapse mortality (NRM) has remained exceptionally low; there have been no deaths during this most vulnerable transplant period. With over three dozen patients enrolled so far, one-year event-free survival exceeds 90% in the unrelated cord blood transplant setting, exceeding the results of centers of excellence worldwide.
In 2016, the division opened an institutional prospective trial for children and young adults afflicted with high-risk acute myeloid leukemia (AML), employing RIC and Myeloablative Conditioning (MAC) for HSCT in AML/MDS (ClinicalTrials: NCT02626715). To bridge the temporary post-transplant immune deficient state, we have performed therapeutic T-cell infusions with adenovirus hexon-specific interferon gamma-captured cells under IND/Institutional Review Board (IRB) approved treatment plans. These will pave the way for new protocols that we plan to open in 2018 taking advantage of a new CliniMACS® Prodigy device and conceptual advances.