9 June 2021

Discovering More Efficient Immunotherapies for the Treatment of Neuroblastoma

Researcher to Watch: Rosa Nguyen, MD, PhD

Neuroblastoma is a type of cancer that forms in certain types of nerve tissue, most commonly affecting children age two or younger. Neuroblastoma accounts for 12% of all cancer-related deaths in children with 650 new cases each year in North America. Despite intensive treatment, 33% of patients experience disease progression or reoccurrence within the first two years of therapy.

Dr. Rosa Nguyen is a pediatric oncologist, translational researcher, and clinical scientist who first became interested neuroblastoma research during her clinical training while caring for children afflicted with this debilitating cancer. The most significant breakthrough in their patient care was the incorporation of immunotherapy into the current standard of care which significantly extended the survival of affected children. However, little was known about the underlying mechanism. Dr. Nguyen was inspired to understand the immune destruction of neuroblasts through immunotherapy with the goal of informing new therapies for patients with neuroblastoma.

In a Geneva managed study titled “New antibody-cytokine fusion proteins for the therapy of neuroblastoma,” Dr. Nguyen aims to determine the role of cytokines in maximizing inherent cell-mediated immunity of patients as a cancer-fighting modality. Her research is being performed in at the National Institutes of Health (NIH) National Cancer Institute (NCI) and is being funded by the Department of Defense through the FY19 Peer Reviewed Cancer Research Program Horizon Award.

Cytokines are small immunoregulatory proteins that can induce important functional changes in tissues and immune cells. Antibody-cytokine fusion proteins are a novel class of biopharmaceuticals showing enormous potential. IL-21, a pleiotropic, or having multiple effects, cytokine that also activates natural killer (NK) cells, has not been broadly studied in neuroblastoma.

Dr. Nguyen is engineering and testing two novel antibody-cytokine fusion proteins to investigate their effect on stimulating the immune system as compared to an interleukin-fusion protein (hu14.18-IL-2) previously tested in a phase II clinical trial.

If she is successful, Dr. Nguyen’s research could have extended impacts on future clinical trials and contribute to the basic understanding of anti-tumor immunity in neuroblastoma.


Disclaimer: The views expressed do not reflect the official policy of the Army, the Department of Defense, or the U.S. Government.

Oncology scientist