# Osteosarcoma: Patient Derived Xenograft Preclinical Testing

> **NIH NIH U01** · UNIVERSITY OF TX MD ANDERSON CAN CTR · 2020 · $177,926

## Abstract

Osteosarcoma is a disease in which the recent lack of progress in improving patient
survival strongly suggests the need for novel treatment approaches. A panel of well
characterized osteosarcoma patient derived xenografts with genetic changes recapitulating the
disease, have been tested by our laboratory as part of the Pediatric Preclinical Testing Program.
These efforts thus far have been highly successful in identifying several drugs which are
currently being or about to be tested in osteosarcoma clinical trials conducted by the Children’s
Oncology Group – specifically eribulin and glembatumumab vedotin. Numerous additional
osteosarcoma patient derived xenograft models have been developed potentially permitting
expansion of the panel of tumors assessed. In this proposal exploration of using a more efficient
approach to screen for drug activity in the established models may permit utilization of a broader
panel of osteosarcoma patient derived xenografts. In this proposal eltrombopag will be explored
for activity in the treatment of osteosarcoma based on promising in vitro data. Building on prior
findings, eribulin will be tested for additive activity with agents routinely used in the treatment of
osteosarcoma including cisplatin, cyclophosphamide and etoposide. Finally, a novel ALK
inhibitor that also has IGF-1R inhibitory activity, certinib, will be tested as a single agent and in
combination with rapamycin for activity in the osteosarcoma patient derived xenografts. These
results can be informative for future osteosarcoma clinical trials development as well as
potentially identify novel agents that can be considered for clinical trials.

## Key facts

- **NIH application ID:** 10075727
- **Project number:** 3U01CA199221-06S1
- **Recipient organization:** UNIVERSITY OF TX MD ANDERSON CAN CTR
- **Principal Investigator:** Richard G. Gorlick
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $177,926
- **Award type:** 3
- **Project period:** 2015-07-10 → 2021-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10075727

## Citation

> US National Institutes of Health, RePORTER application 10075727, Osteosarcoma: Patient Derived Xenograft Preclinical Testing (3U01CA199221-06S1). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10075727. Licensed CC0.

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