# MicroRNA Targeted Therapeutic Approach for Pediatric High-Grade Glioma

> **NIH NIH R21** · UNIVERSITY OF ILLINOIS AT CHICAGO · 2020 · $411,143

## Abstract

ABSTRACT
Various strategies have been taken for cancer chemotherapy, however, treatment-related toxicity
and immune response are still major issues due to the lack of targeted delivery to cancer cells. In
particular, pediatric high grade gliomas (pHGGs) are currently often difficult to treat, largely due to
locations of the disease.
 High levels of oncogenic miRNA (oncomiR) inhibit expression of tumor suppressor proteins,
thus critically implicated in initiation and progression of pHGGs. Anti-miRNA can neutralize
oncomiRNA that are aberrantly expressed in pHGG, and restore the expression of tumor
suppressor proteins that inhibit tumor growth. However, cellular delivery of anti-miRNA is currently
one of the largest challenges for advancement into the clinic. Peptide-mediated anti-miRNA
delivery that efficiently and specifically targets cancer cells may offer a solution.
 Our innovative approach potentially overcomes these problems for the following reasons: 1) a
novel non-toxic CPP, p28, selectively enters cancer cells; 2) p28 crosses the blood-brain barrier
and can serve as a cancer-targeting delivery system when functional molecules are conjugated; 3)
p28-conjugated with anti-miRNAs, which is highly specific to their targets, will be expected to
specifically inhibit growth of pHGGs.
 Our supporting data suggest that p28 conjugated with anti-miR20 (anti-miR20-p28), the
overexpressed oncomiR in pHGG compared to adult high-grade glioma (aHGG) and healthy brain
tissue, preferentially and successfully delivers functional anti-miR20 into pHGGs. The overall goal
of this proposal is to take a strategic approach to evaluate the effects of anti-miRNAs conjugated
with non-toxic tumor-targeting peptide (p28) on pHGGs. If successful, our approaches may
potentially overcome the major limitation of current anti-miRNA-based therapy and improve the
treatment of childhood brain tumors without damaging healthy tissues.

## Key facts

- **NIH application ID:** 10043989
- **Project number:** 1R21CA252370-01
- **Recipient organization:** UNIVERSITY OF ILLINOIS AT CHICAGO
- **Principal Investigator:** Tohru Yamada
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $411,143
- **Award type:** 1
- **Project period:** 2020-09-01 → 2023-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10043989

## Citation

> US National Institutes of Health, RePORTER application 10043989, MicroRNA Targeted Therapeutic Approach for Pediatric High-Grade Glioma (1R21CA252370-01). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10043989. Licensed CC0.

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