Patrick J. Grohar, M.D., Ph.D.

faculty photo
Associate Professor of Pediatrics (Oncology)
Director, Translational Research, Center for Childhood Cancer Research, Children’s Hospital of Philadelphia, University of Pennsylvania
Senior Research Scientist, Division of Oncology, Children's Hospital of Philadelphia Research Institute, University of Pennsylvania
Department: Pediatrics
Graduate Group Affiliations

Contact information
Children's Hospital of Philadelphia
Division of Oncology
Department of Pediatrics
3401 Civic Center Boulevard
Philadelphia, PA 19104
BS (Chemistry)
Villanova University, 1995.
PhD (Chemistry and Biochemistry)
Wayne State University, 2001.
Wayne State University, 2003.
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Description of Research Expertise

The Grohar Lab focuses on developing and clinically translating novel small molecule therapies for the treatment of pediatric solid tumors. A number of pediatric solid tumors have clear oncogenic driver mutations that are considered by many to be “undruggable.” The lab has primarily focused on Ewing sarcoma and the development of therapies that target the EWS-FLI1 transcription factor. More recently, we have extended our focus to other solid tumors with the goal of better understanding therapeutic vulnerabilities at the interface of transcription and epigenetics. We have a number of compounds in various stages of clinical development including a clinical trial based on our laboratory work that is actively accruing patients. We employ high-throughput genetic and chemical screens to identify novel targets and compounds, molecular pharmacology to understand the mechanism of action, and a variety of next-generation sequencing assays to delineate the effects genome-wide.

Selected Publications

Grohar, P. J., Ballman, K., Heise, R. Glod, J., Wedekind, M. F., Mascarenhas, L., Gedminas, J., DuBois, S., Maki, R., Hayashi, M., Peer, C., Lindenberg, L., Menagonzalez, E., Bagatell, R., Laetsch, T. W., Widemann, B.C., Reinke, D. K., Chugh, R.: Results of a phase I study of trabectedin administered as a 1-hour infusion in combination with low dose irinotecan in relapsed/refractory Ewing sarcoma patients (pts). Annual Meeting of the American Society of Clinical Oncology June 2023 Notes: SARC037: Accepted for poster discussion.

Kinn-Gurzo, S., Kitchen-Goosen, S. M., Chasse M. H., Adams, M., Kaufman, R., Grohar, P. J.: Heterogeneity of the EWS-FLI1 transcriptome drives differences in the cellular phenotype. Peer Reviewed Poster Presentation, Annual Meeting of the American Association for Cancer Research, Orlando, FL April 2023.

Veluvolu, S. M., Grohar, P. J.: Importance of pharmacologic considerations in the development of targeted anticancer agents for children. Curr Opin Pediatr 35(1): 91 - 96, February 2023.

Grohar, P.J. (Lead author), Dirksen, U., DuBois, S., Binitie, O., Hogendoorn, P. C. W., Laack, N.: Ewing sarcoma. Pediatric Oncology Pizzo and Poplack (eds.). 2023.

Shulman, D. S., Whittle, S. B., Surdez, D., Bailey, K. M., de Alava, E., Yustein, J. T., Shlien, A., Hayashi, M., Bishop, A. J., Crompton, B. D., DuBois, S. G., Shukla, N., Leavey, P. J., Lessnick, S. L., Kovar, H., Delattre, O., Grunewald, T. G. P., Antonescu, C., Roberts, R. D., Toretsky, J. T., Tirode, F., Gorlick, R., Janeway, K. A., Reed, D. R., Lawlor, E. R., Grohar, P. J.: An international working group consensus report for the prioritization of molecular biomarkers for Ewing sarcoma. NPJ Precision Oncology 17(6(1)): 65, September 2022.

Maki, R.G., Grohar, P. J., Antonescu, C. R.: Ewing sarcoma and related FET family translocation-associated round cell tumors: A century of clinical and scientific progress. Genes Chromosomes Cancer 61(8): 509-517, August 2022.

Gedminas, J. M., Kaufman, R., Boguslawski, E., Gross, A., Adams, M., Beddows I, Kitchen-Goosen, S.M., Roberts, R., Grohar, P. J.: Lurbinectedin inhibits the EWS-WT1 transcription factor in Desmoplastic small round cell tumor. Molecular Cancer Therapeutics (2022) Molecular Cancer Therapeutics 21(8): 1296 - 1305, August 2022.

Cash, T., Reed, D. R., Gorlick, R., DuBois, S., Grier, H., Grohar, P. J., Binitie, O., Pawel, B., Healey, J., Krailo, M., Buxton, A., Womer, R. B., Janeway, K. A. : Long term outcomes in patients with localized Ewing sarcoma treated with interval compressed chemotherapy: A long-term follow-up report from Children’s Oncology Group study AEWS0031. Peer Reviewed Oral Presentation (T. Cash), Annual meeting of the American Society of Clinical Oncology June 2022.

Chen, S., Shenoy, A., Al-Ibraheemi, A., Bush, J., Davis, J. L., Grohar, P., Binitie, O., Krailo, M.D., Reed, D.A., Janeway, K. A.: A report on the review of archived osteosarcoma and Ewing sarcoma specimens at the Biopathology Center. Peer Reviewed Poster Presentation, Annual meeting of the American Society of Clinical Oncology June 2022 Notes: Bone Sarcoma Committee, Children’s Oncology Group.

Deng, Qu, Natesan, R., Cidre-Aranaz, F., Arif, S, Liu, Y., Rasool, R., Wang, P., Mitchell-Velasquez, E., Das, C. K., Vinca, E., Cramer, Z., Grohar, P., Chou, M., Kumar-Sinha, C., Weber, K., Eisinger-Mathason, K., Grillet, N., Grunewald, T., Asangani, I. A.: Oncofusion-driven de novo enhancer assembly promotes malignancy in Ewing sarcoma via aberrant expression of the stereociliary protein, LOXHD1. Cell Reports 39(11): 110971, June 2022.

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Last updated: 04/25/2023
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