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 innovative experimental systems to develop novel immunotherapies for patients with 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 pediatric solid tumors

We developed next-generation antibody cytokine fusion proteins and will assess their therapeutic efficacy in preclinical neuroblastoma models. The aim of these studies is to translate these novel antibody cytokine fusion proteins to an investigator-initiated clinical trial for GD2-expressing malignancies.

2. Optimizing cell-based immunotherapy for pediatric neuroblastoma:

We have demonstrated preclinical activity of GPC2-targeting chimeric antigen receptor (CAR) T cell therapy in preclinical neuroblastoma models. 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 focuses on CAR engineering to manipulate the CAR structure, conditionally alter CAR expression, and exploit the use of transgenic cytokines. We use genome-wide CRISPR screens to understand the mechanisms of tumor response to and evasion from adoptive CAR T therapy. Altogether, these efforts are geared towards finding new adoptive therapies to treat patients with neuroblastoma.


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. degree in Biomedical Sciences from the University of Tennessee Health Science Center and trained as a postdoctoral fellow at the National Heart, Lung, and Blood Institute and the National Cancer Institute. I am the recipient of the 2018 Conquer Cancer ASCO Young Investigator Award and I am a 2022 St. Baldrick’s Foundation Scholar. My work has also been funded by the Department of Defense Peer-Reviewed Cancer Research Program, the 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 Monday, November 13, 2023