The goal of the Grohar Lab is to develop and clinically translate novel small molecule therapies for the treatment of Ewing sarcoma. Ewing sarcoma absolutely depends on the EWS-FLI1 transcription factor for cell survival. Unfortunately, there are no effective small molecule EWS-FLI1 inhibitors currently in use in the clinic. EWS-FLI1 is a challenging drug target because it is a transcription factor. Therefore, we employ a variety of complementary methods to identify and clinically translate novel EWS-FLI1 targeted therapies. These methods include:
- High Throughput Drug Screening
- siRNA screening
- Molecular Pharmacology
- Cancer Biology
- Chemical Biology
Our goal is to identify novel small molecule EWS-FLI1 inhibitors, employ analog based optimization, understand the mechanism of action of these small molecules and translate these therapies to the clinic. In addition, we are working to develop a number of novel EWS-FLI1 directed combination therapies. In the process, we hope to understand transcription factor drug targeting and apply this understanding to a variety of other tumors.