# Toward Translation of an Immunotherapeutic Nanomedicine for Neuroblastoma

> **NIH NIH R01** · VANDERBILT UNIVERSITY · 2024 · $150,851

## Abstract

PROJECT SUMMARY
This proposal addresses the significant, unmet need to develop and translate new therapies for children with
advanced, high-risk neuroblastoma. Neuroblastoma (NB) is the third most common pediatric cancer and the
most common extracranial solid tumor of childhood, accounting for 15% of all pediatric cancer deaths each
year despite an intensive, multimodal, and toxic treatment regimen. Immunotherapy offers the potential for
selective targeting and killing of cancer cells and represents an appealing alternative for eradicating recurrent,
metastatic disease and achieving durable cures with minimal toxicity. However, NB has proven poorly
responsive to most immunotherapeutic modalities, notably including immune checkpoint blockade and CAR T
cell therapy. Therefore, novel immunotherapies for NB must be developed. The objective of this proposal is to
advance and mature STING-activating nanoparticles (STANs), a promising experimental immunotherapeutic
nanomedicine for enhancing immunotherapy responses in NB, towards clinical translation. To accomplish this,
we will directly address potential barriers to the clinical advancement of STANs by further optimizing their
physiochemical and biological properties via a scalable manufacturing process, elucidating key
immunopharmacological parameters in rigorous NB mouse models, and establishing rationally-designed
immunotherapy regimens that generate robust and durable responses. We will accomplish this through the
following Specific Aims. First, we will employ an integrated polymer and materials science approach to
reproducibility fabricate STANs with optimized properties via a facile and scalable flash nanoprecipitation
nanofabrication strategy. Second, we will evaluate the pharmacokinetics, biodistribution, pharmacodynamics,
safety, and therapeutic efficacy of STANs in a rigorous immunocompetent NB that mimic human disease.
Third, we will evaluate and optimize rationally-designed immunotherapy regimens combining STANs with
immune checkpoint blockade and NB-targeted CAR T cells. We expect the proposed work to address several
critical preclinical gaps that, when filled, will accelerate STANs toward clinical translation. Therefore, this
research addresses a problem of high clinical urgency by advancing a next-generation nanotechnology for
enhancing immunotherapy responses in NB.

## Key facts

- **NIH application ID:** 10924063
- **Project number:** 5R01CA274675-03
- **Recipient organization:** VANDERBILT UNIVERSITY
- **Principal Investigator:** John Tanner Wilson
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $150,851
- **Award type:** 5
- **Project period:** 2022-07-01 → 2027-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10924063

## Citation

> US National Institutes of Health, RePORTER application 10924063, Toward Translation of an Immunotherapeutic Nanomedicine for Neuroblastoma (5R01CA274675-03). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10924063. Licensed CC0.

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