# Tumor Antigen Targeted Nanoparticle Therapy for Glioblastoma (GBM)

> **NIH NIH R42** · NANOVALENT PHARMACEUTICALS, INC. · 2024 · $1,000,000

## Abstract

Project Summary
The goal of this Fast Track STTR project is to determine proof-of-principle and efficacy
of a novel blood brain barrier (BBB) penetrating therapeutic nanoparticle for the potential
treatment of otherwise intractable brain tumors like glioblastoma multiforme (GBM). This
project seeks to demonstrate that novel, targetable nanoparticles can delivery
therapeutic substances to human brain tumor cells and reduce tumor burden in brain
cancer and prolong patient survival. The therapeutic cargos are encapsulated cytotoxic
drugs for otherwise intractable brain tumors. After exiting the brain vasculature, upon
recognition by the tumor cell, the nanoparticle binds, gets taken into the cell
(endocytosed) and the nanoparticle cargo is released, ultimately allowing availability of
the drug to kill the cancer cell. This project fits well within the mission of the NCI, to
develop new nanotechnology-based therapeutics, especially for high-risk tumors.
Historically successful cancer chemotherapy, while vastly increasing survival in non-
CNS tumors, has failed to do so for brain tumors in children and adults alike. GBM
remains the most malignant primary central nervous system tumor, where the median
overall survival is 15–23 months and 5-year survival is less than 6%. The incidence of
brain metastases is increasing with an estimated 69,950 adults age 40+ in 2021 in the
US alone. Brain tumors represent the highest per-patient initial cost of care for any
cancer group. Estimations from Surveillance, Epidemiology, and End Results (SEER) on
annualized mean net cost of care approach $150,000 per patient. These patients have
the highest annualized mean net costs for last-year-of-life care, relative to other cancers,
at $135,000 to $210,000 (depending on age and gender). There us thus dramatic unmet
need to prevent morbidity and mortality while improving an otherwise dismal survival rate.
Treatment-resistant metastases are the ultimate cause of death in most cancer patients.
For brain cancer treatment, systemic therapy for metastases is generally ineffective due
to the inability to get therapeutic doses across the blood brain barrier. A reliable, low-
toxic, highly effective therapy is urgently needed to treat patients with primary tumors
and treatment-resistant metastases. The specific aims of this proposal are therefore
efficient encapsulation cancer drugs inside the targeted HPLNs that cross the blood
brain barrier, demonstrate safety and efficacy in killing cancer cells in a spectrum of
humanized xenograft mouse models of human GBM. NanoValent's goal, at the
conclusion of the Fast Track proposal is to have a promising optimized formulation that
can be that can ultimately be GMP manufactured and submitted for IND approval with
the FDA.

## Key facts

- **NIH application ID:** 11081824
- **Project number:** 4R42CA281707-02
- **Recipient organization:** NANOVALENT PHARMACEUTICALS, INC.
- **Principal Investigator:** JON Owen NAGY
- **Activity code:** R42 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $1,000,000
- **Award type:** 4N
- **Project period:** 2023-08-08 → 2026-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/11081824

## Citation

> US National Institutes of Health, RePORTER application 11081824, Tumor Antigen Targeted Nanoparticle Therapy for Glioblastoma (GBM) (4R42CA281707-02). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/11081824. Licensed CC0.

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