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FINDING CURES TOGETHER<sup>SM</sup>

Modulating Transcription Factor Abnormalities in Pediatric Cancer

Summary

Many cancer-promoting proteins are considered difficult to target or "undruggable" by standard pharmacological approaches. This is a significant problem in pediatric cancers where the tumors are often initiated by these intractable proteins. One example is Ewing sarcoma, the second most common bone tumor in children, in which the critical cancer-promoting protein, EWS-FLI, has been considered "undruggable." For children with metastatic Ewing sarcoma or those who relapse after initial treatment, the prognosis is dismal. To address this challenge, Stegmaier proposes a new drug discovery approach integrating multiple platforms and disciplines - genomic technologies, chemical biology, computational biology, proteomics, and molecular genetics. She will apply this integrative approach to target the EWS-FLI protein. Her laboratory will first determine the fingerprint of the genes that are turned on or off in the presence of EWS-FLI in Ewing tumors. Then, they will screen a library of chemicals for those that induce the gene fingerprint of the inactive EWS-FLI protein to identify potential anti-cancer drugs. They will pay particular attention to drugs that are already FDA-approved for other indications and, thus, can be rapidly brought into clinical trials for pediatric or adult patients with cancer.

Investigator

Kimberly Stegmaier, MDKimberly Stegmaier, MD, is an assistant professor in the department of pediatrics at Harvard Medical School, an independent investigator in pediatric oncology at the Dana-Farber Cancer Institute, and an attending physician in pediatric hematology-oncology at the Children's Hospital Boston and the Dana-Farber Cancer Institute. She is also an associate member of the Broad Institute of MIT and Harvard.

Stegmaier received her Bachelor of Science degree in psychology from Duke University in 1991 and her medical degree from Harvard Medical School in 1996. She completed her residency at the Children's Hospital Boston in 1999 and a postdoctoral pediatric hematology-oncology fellowship in 2006 while at the Dana-Farber Cancer Institute/Children's Hospital Boston.

Stegmaier's research program integrates chemical biology, and genomic and proteomic approaches to discover new lead compounds and protein targets for cancer therapy. One of her current research interests is the pediatric tumor Ewing sarcoma. Stegmaier will employ these integrated approaches to tackle a cancer-promoting protein in Ewing sarcoma for which there is currently no targeted therapy.

Updated: May 13, 2014