Rosa Nguyen, M.D., Ph.D.

The overarching goal of my laboratory is to develop next-generation immunotherapies for children with solid tumors. We integrate cutting-edge experimental models, advanced engineering approaches, and translational research, with the ultimate objective of improving outcomes for patients with high-risk pediatric cancers, particularly neuroblastoma. I am committed to fostering a collaborative and supportive research environment where every member of the laboratory has an equal opportunity to succeed. Our research focuses on three major areas:

1. Harnessing tumor-targeting antibodies and NK cell activity in neuroblastoma

We develop and evaluate next-generation antibody-based therapeutics, including immunocytokines, bispecific T-cell engagers, and antibody-drug conjugates, to enhance anti-tumor immunity against neuroblastoma. Using preclinical models, we investigate their therapeutic efficacy, mechanisms of immune activation, and interactions with the tumor microenvironment. Our goal is to rapidly translate promising antibody platforms into investigator-initiated clinical trials for children with neuroblastoma. Building on this effort, we have initiated a Phase I clinical trial (NCT06995872) evaluating recombinant human IL-15 (rhIL-15) in combination with dinutuximab and irinotecan/temozolomide for patients with relapsed or refractory neuroblastoma.

2. Optimizing cell-based immunotherapy for pediatric solid tumors

Our laboratory develops and engineers next-generation cellular immunotherapies for pediatric solid tumors. We performed the preclinical optimization of a glypican-2 (GPC2)-targeted chimeric antigen receptor (CAR) T-cell therapy for neuroblastoma and pediatric brain tumors, which is being translated into an investigator-initiated first-in-human clinical trial within the Pediatric Oncology Branch. To further improve CAR T-cell efficacy, we engineer novel CAR constructs and cytokine support systems that enhance persistence, trafficking, and function within the immunosuppressive tumor microenvironment. In parallel, we employ genome-wide CRISPR screening and next-generation sequencing to identify mechanisms underlying tumor sensitivity, resistance, and immune evasion following adoptive cell therapy. Together, these studies aim to develop safer, more durable, and more effective cellular therapies for children with solid tumors.

3. Developing combinatorial strategies to enhance cell therapies

We are developing rational combination therapies that improve the efficacy of adoptive cell therapies against solid tumors. A major focus is the use of radiotheranostic approaches to noninvasively visualize tumor antigen expression in vivo and guide treatment strategies before cell therapy. By leveraging radiolabeled tumor-targeting antibodies and peptide-based radiopharmaceuticals, we also seek to remodel the tumor microenvironment, enhance immune cell infiltration, and improve the therapeutic activity of CAR T cells and other cellular immunotherapies. These studies integrate molecular imaging with immunotherapy to create precision combination strategies that maximize anti-tumor responses while minimizing toxicity.

I am a board-certified pediatric oncologist and completed my pediatric residency at the University of Maryland and my subspecialty training at St. Jude Children’s Research Hospital. I earned my Ph.D. in Biomedical Sciences from the University of Tennessee Health Science Center. Before starting my own research group in the Pediatric Oncology Branch at the National Cancer Institute, I trained as a postdoctoral fellow at the National Heart, Lung, and Blood Institute and the National Cancer Institute. My work has been funded by the Department of Defense, American Society of Clinical Oncology, St. Baldrick’s Foundation, Children’s Cancer Foundation, Solving Kids’ Cancer Foundation, and the B+ Foundation.