# Pediatric Preclinical Testing Consortium: Research Programs non-CNS

> **NIH NIH U01** · UNIVERSITY OF TEXAS HLTH SCIENCE CENTER · 2021 · $315,184

## Abstract

The group at GCCRI will continue studies focused on identifying active agents and
combinations using xenograft models of pediatric sarcoma and kidney tumors. We will complete
ongoing studies, and start new studies as described below. In addition we plan to characterize
18 new sarcoma PDX models (exome seq and RNAseq), and complete characterization of 33
PDX models where we have exome sequence data from the TARGET initiative, but where we
lack RNAseq data. This will greatly expand the number of available sarcoma models that can be
used in the future PPP being developed by the FNIH. Our aims for the 6 month extension are:
Specific Aims
Proposed studies during the extension period.
1. Complete studies with Amgen 509 (ES-7, ES-3, EW-5).
2. Complete in vivo studies with ONC201.
3. Undertake screening for PPTC drugs/combinations.
4. Evaluate PLX-038A combined with vincristine in sarcoma and kidney models.
5. Evaluate Talazoparib + Radiation in CHLA258 cell in vitro and in vivo, then expand if justified
6. To characterize 33 models by RNAseq that were omitted from the ALS cohort.
7 To characterize 18 rhabdomyosarcoma and 5 Ewing sarcoma PDX models, not previously
characterized.
8. Complete submission of manuscripts reporting PPTC results.

## Key facts

- **NIH application ID:** 10293155
- **Project number:** 3U01CA199297-05S2
- **Recipient organization:** UNIVERSITY OF TEXAS HLTH SCIENCE CENTER
- **Principal Investigator:** PETER J HOUGHTON
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $315,184
- **Award type:** 3
- **Project period:** 2020-07-01 → 2021-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10293155

## Citation

> US National Institutes of Health, RePORTER application 10293155, Pediatric Preclinical Testing Consortium: Research Programs non-CNS (3U01CA199297-05S2). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10293155. Licensed CC0.

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