I am a pediatric oncologist and physician-scientist focused on the development of immunotherapies for pediatric cancer. I first became interested in pursuing a career in laboratory research during my fellowship in at the NIH, where I took care of some of the first children with leukemia to receive CD19 CAR T cells. Witnessing the early success of CAR T cells awoke in me a realization that physician-scientists can translate discoveries from their laboratories to generate effective cures for patients in need. In pediatrics, where market forces do not effectively drive capital investment for clinical development, it is imperative that physician-scientists focus on developing drugs within academia, shepherding them from the laboratory through early phase clinical trials.

The core mission of my laboratory is to leverage discoveries in basic immunology and cancer biology to develop and test new immunotherapeutics in children. As an example, during my postdoctoral studies, we discovered that GD2 is expressed at high levels on Diffuse Intrinsic Pontine Glioma (DIPG), a universally fatal pediatric brain tumor, and that anti-GD2 chimeric antigen receptor (CAR) T cells are effective in preclinical models of this disease (Mount/Majzner et al., Nature Medicine, 2017). Within four years of publication, we translated this to a clinical trial that I co-led. Excitingly, we observed radiographic and/or clinical responses in eight of the first ten patients treated, including a 17-year-old with a sustained complete response (Majzner/Ramakrishna et al., Nature, 2022). Concurrently, my laboratory focuses on the development of novel immunotherapies, including engineering CAR T cells with innovative designs that are capable of transforming how we target cancer (Tousley et al, Nature, 2023). By fostering a research program spanning basic science with translational immunotherapy, my goal as a physician-scientist is to make discoveries that enable translation of effective therapies for children with otherwise incurable cancers.