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

Lasker Clinical Research Scholar

Pediatric Oncology Branch


Building 10-CRC, Room 1-5816
Bethesda, MD 20892


Research Topics

The goal of my laboratory is to apply cutting-edge experimental systems and methods to develop new immunotherapies for patients with solid tumors, such as neuroblastoma. I am committed to creating an environment in my laboratory where everyone has an equal opportunity to succeed. I believe that diversity is integral to fostering a productive work environment for scientific discovery and collaboration, and I look forward to serving as a mentor to students and post-doctoral fellows. My laboratory focuses on two main areas:

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

We design next-generation antibodies, such as immunocytokines, bispecific T-cell engagers, and antibody-drug conjugates, and assess their therapeutic activity and immunologic effect in preclinical models of neuroblastoma. The aim of our studies is to translate these new antibody formats into investigator-initiated clinical trials for children with neuroblastoma.

2. Optimizing cell-based immunotherapy for pediatric solid tumors

We performed preclinical optimization of a GPC2-targeting chimeric antigen receptor (CAR) T-cell therapy to treat neuroblastoma and brain tumors. This work will be translated into an investigator-initiated first-in-human clinical trial for patients with neuroblastoma in the Pediatric Oncology Branch. To further optimize CAR T-cell therapy, our laboratory leverages CAR engineering approaches and transgenic cytokines to augment CAR function in the tumor microenvironment. We use genome-wide CRISPR screens and next-generation sequencing to understand the mechanisms of tumor response to and evasion from adoptive T-cell therapy. Altogether, these efforts are geared toward finding new cellular therapies to treat children with solid tumors.


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, and Solving Kids’ Cancer Foundation.

Selected Publications

  1. Sun M, Cao Y, Okada R, Reyes-González JM, Stack HG, Qin H, Li N, Seibert C, Kelly MC, Ruppin E, Ho M, Thiele CJ, Nguyen R. Preclinical optimization of a GPC2-targeting CAR T-cell therapy for neuroblastoma. J Immunother Cancer. 2023;11(1).
  2. Nguyen R, Zhang X, Sun M, Abbas S, Seibert C, Kelly MC, Shern JF, Thiele CJ. Anti-GD2 Antibodies Conjugated to IL15 and IL21 Mediate Potent Antitumor Cytotoxicity against Neuroblastoma. Clin Cancer Res. 2022;28(17):3785-3796.

Related Scientific Focus Areas

This page was last updated on Tuesday, February 13, 2024