I am a physician scientist whose most immediate goal is to develop safe and effective methods for conditioning patients prior to bone marrow transplantation, thereby expanding the use of this curative treatment. In addition to improving treatment and, potentially, the prevention of hematologic malignancies, I aspire to expand the utility of transplantation for other blood and immune diseases, including genetic diseases and autoimmune diseases, and for the induction of tolerance. Toward this goal I completed clinical training in pediatrics and pediatric hematology/oncology. In my clinical practice I see patients in the pediatric hematopoietic stem cell transplant clinic and in an inpatient capacity, with a focus on caring for patients with bone marrow failure and pre-malignant conditions. My scientific research program complements these clinical interests and is focused on understanding how hematopoietic stem cells (HSCs) interact with their microenvironment in health and disease and then modulating these interactions with antibodies and small molecules for therapeutic and research purposes. As a graduate student in Irv Weissman’s laboratory, I was the first to demonstrate that inhibition of stem cell factor signaling through antagonistic antibodies to CD117 could lead to host HSC depletion, and that this could be used as an effective strategy for enhanced engraftment of donor HSCs, which we showed is otherwise limited without conditioning. As a post-doctoral fellow in Derrick Rossi’s laboratory, I further showed that this could be improved using CD117 antibody-drug-conjugates, which enabled the replacement of donor HSCs across disease settings with minimal perturbation to the mature blood and immune system. Multiple therapies are in development stemming from my pre-clinical work, with the most advanced in a clinical trial at Stanford testing antagonistic anti-CD117 antibody conditioning in immunodeficient patients.

Our lab is now focused on targeting additional antigens and better understanding how such conditioning regimens truly work, where they work, and how the resulting microenvironment compensates for their perturbations. We are also through multiple collaborations testing the use of our conditioning methods across malignant and non-malignant diseases. Further, we are investigating combination strategies with complementary immune suppression—which is required to prevent rejection and induce tolerance in the allogeneic setting—and gene therapy/gene editing to apply such conditioning in the autologous setting. I believe my clinical and scientific training has prepared me well to be a true concurrent physician and scientist, with an aim to improve our understanding of normal and diseased hematopoiesis and ultimately have an important impact on many patients.