Patrick Grohar
Faculty Member
Last active: 3/27/2014


Our lab is focused on developing new therapies for pediatric sarcomas. Many of these tumors are driven by oncogenic transcription factors generated by reciprocal chromosomal translocations. These mutations are responsible for the continued proliferation of these tumors and occur in a genetic background characterized by a limited number of cooperating somatic mutations. Unfortunately, these fusion proteins are transcription factors and therefore represent challenging small molecule drug targets. Our work is focused on developing methods and compounds to target these specific transcription factors. To date, we have primarily worked to identify compounds that inhibit the dominant oncogene of Ewing sarcoma, the EWS-FLI1 transcription factor. We have completed a high throughput screen to identify mithramycin as an inhibitor of EWS-FLI1. We have translated this compound to the clinic in a phase I/II trial currently accruing patients at the National Cancer Institute. In addition, we have utilized clinical observations to identify trabectedin as an inhibitor of EWS-FLI1. We are now working to improve the activity of both compounds by studying novel analogs and developing novel mechanism-based combination therapies. We are studying the mechanism of action of these and other compounds known to interfere with specific transcription factors. Finally, we are working to understand the most effective way to utilize small molecules to poison specific transcription factors.


The following timeline graph is generated from all co-authored publications.

Featured publications are shown below:

  1. A Decade in Banking Ewing Sarcoma: A Report from the Children's Oncology Group. Borinstein SC, Beeler N, Block JJ, Gorlick R, Grohar P, Jedlicka P, Krailo M, Morris C, Phillips S, Siegal GP, Lawlor ER, Lessnick SL, COG Ewing Sarcoma Biology Committee (2013) Front Oncol : 57
    › Primary publication · 23519678 (PubMed) · PMC3602933 (PubMed Central)
  2. Rapamycin induces feedback activation of Akt signaling through an IGF-1R-dependent mechanism. Wan X, Harkavy B, Shen N, Grohar P, Helman LJ (2007) Oncogene 26(13): 1932-40
    › Primary publication · 17001314 (PubMed)