# The role of Olig2 in the tumorigenesis, progression and metastasis in MYC-amplified medulloblastoma

> **NIH NIH R01** · CHILDREN'S RESEARCH INSTITUTE · 2023 · $398,055

## Abstract

Project Summary
Medulloblastoma (MB) is the most common malignant brain tumor in children. Although treatment
combining surgery, radiation and high-dose chemotherapy has led to increased survival rates, many
MB patients still die from the disease. Moreover, surviving patients suffer severe long-term side
effects as a consequence of treatment. It is therefore crucial to develop more effective and less toxic
therapies. The most aggressive subtype of MB consists of tumors that exhibit amplification of the
MYC oncogene. Patients with MYC-amplified MB exhibit a high frequency of tumor dissemination to
the entire brain and spinal cord, often experience treatment resistance and have extremely poor
prognoses. In this application, we propose to use novel mouse MYC-driven MB and patient-derived
xenograft (PDX) models to investigate the role of oligodendrocyte lineage transcription factor 2 (Olig2)
in tumorigenesis, progression and metastasis of MYC-amplified MB. We will use these mouse models
to evaluate a novel targeted approach to treatment. To accomplish this we will: (1) evaluate whether
ablation of Olig2+ tumor cells can prevent tumor formation, progression and metastasis, (2) determine
whether deletion of the Olig2 gene prevents tumor progression and metastasis, and (3) test the
prediction that targeting Olig2 with a small molecule inhibitor can inhibit growth and prevent
metastasis in a human MYC-amplified MB PDX model. If these studies are successful, they will help
identify new targeted therapies that can be effective in treating patients with MYC-amplified MB.

## Key facts

- **NIH application ID:** 10668356
- **Project number:** 5R01CA241192-05
- **Recipient organization:** CHILDREN'S RESEARCH INSTITUTE
- **Principal Investigator:** Yanxin Pei
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2023
- **Award amount:** $398,055
- **Award type:** 5
- **Project period:** 2019-08-07 → 2025-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10668356

## Citation

> US National Institutes of Health, RePORTER application 10668356, The role of Olig2 in the tumorigenesis, progression and metastasis in MYC-amplified medulloblastoma (5R01CA241192-05). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10668356. Licensed CC0.

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