Validation of a Novel Treatment for Glioblastoma

? DESCRIPTION (provided by applicant): The goal of this application is to study in vivo efficacy of CT102, a newly discovered inhibitor of the OLIG2 transcription factor in animal models of glioblastoma (GBM), a highly underserved area of pharmacotherapy. In this proposal, the experienced drug discovery group at Curtana Pharmaceuticals Inc. will conduct a pre-clinical evaluation of selectivity and in vivo distribution of CT102 to determine protocols for efficacy studies in orthotopic models of GBM. CT102 will be studied at UCSD to confirm potency of newly synthesized materials, assess activity in an orthotopic model of GBM and conduct in vitro and in vivo profiling to evaluate CT102 as a sensitizer to chemo- or radiotherapy. A successful outcome may result in a potential small molecule therapeutic for GBM. PUBLIC HEALTH RELEVANCE PUBLIC HEALTH RELEVANCE: The objective is to evaluate the efficacy of an inhibitor of the transcription factor, OLIG2 in pre-clinical animal models of glioblastoma, a