# Novel Therapeutic Approaches for the Treatment of Rhabdomyosarcoma

> **NIH NIH R01** · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · 2021 · $410,606

## Abstract

Rhabdomyosarcoma (RMS) is the most common soft-tissue sarcoma of childhood and adolescence.
Using contemporary multimodal therapies, more than 75% of patients with localized RMS are cured
However, patients with recurrent disease experience 5-year survival rates of only 30%, and this
outcome has not significantly improved in more than 20 years. Over the past 6 years, comprehensive
genomic, epigenomic, proteomic, and drug-sensitivity profiling of RMS has been completed and
integrated into a central database (iRDb). In addition, a multidisciplinary RMS translational research
team (RMS-TRT) has been assembled to use the iRDb to identify tumor vulnerabilities that can be
exploited therapeutically. Three signaling pathways have been identified that appear to be vulnerable to
novel molecular-targeted regimens, which have shown promise in laboratory studies. In this proposal,
comprehensive preclinical testing of those regimens (e.g., pharmacokinetics, pharmacodynamics,
imaging and efficacy studies) will be performed to determine the most effective treatment. The results
from these studies will be used to design the next RMS clinical trial at St. Jude Children's Research
Hospital. Therefore, the successful completion of the Aims presented here will directly affect human
health. One unique and innovative aspect of this proposal is the Childhood Solid Tumor Network
(CSTN), a website (http://www.stjude.org/CSTN/) through which all of the iRDb data are shared freely
with the international biomedical research community with no obligation to collaborate. The majority
(>95%) of the data available through the CSTN is unpublished, but all of it is shared to accelerate
discovery and advance cures for pediatric cancer. To date, 314 requests from 122 investigators at 46
institutions across 11 countries have been fulfilled. I have a proven record of moving basic science
discoveries into clinical trials and then sharing findings with the national and international research
communities to affect the greatest change in global human health. No other center has the team,
resources, expertise, or tools available to perform the studies presented here and efficiently move the
most promising findings directly into a clinical trial.

## Key facts

- **NIH application ID:** 10134261
- **Project number:** 5R01CA219686-05
- **Recipient organization:** ST. JUDE CHILDREN'S RESEARCH HOSPITAL
- **Principal Investigator:** Michael A Dyer
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $410,606
- **Award type:** 5
- **Project period:** 2017-06-15 → 2023-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10134261

## Citation

> US National Institutes of Health, RePORTER application 10134261, Novel Therapeutic Approaches for the Treatment of Rhabdomyosarcoma (5R01CA219686-05). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10134261. Licensed CC0.

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