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.

Public Health Relevance

Studies to be undertaken will continue to identify novel new agents and drug combinations that are active in preclinical models of pediatric solid tumors. This group will focus on models of high-risk sarcoma and kidney cancers that have been molecularly characterized.

Agency
National Institute of Health (NIH)
Institute
National Cancer Institute (NCI)
Type
Research Project--Cooperative Agreements (U01)
Project #
3U01CA199297-05S2
Application #
10293155
Study Section
Program Officer
Smith, Malcolm M
Project Start
2020-07-01
Project End
2021-01-31
Budget Start
2020-07-01
Budget End
2021-01-31
Support Year
5
Fiscal Year
2021
Total Cost
Indirect Cost
Name
University of Texas Health Science Center
Department
Genetics
Type
Schools of Medicine
DUNS #
800772162
City
San Antonio
State
TX
Country
United States
Zip Code
78229