Research Overview

Kimberly Stegmaier’s laboratory champions the importance of cancer target and therapy discovery in academic centers for malignancies affecting children not likely to be pursued by the pharmaceutical industry. Her research program focuses on the integration of genomic, genetic, and proteomic approaches to identify new protein targets and small-molecule modulators of malignancy with an eye toward clinical translation. In particular, she developed a chemical genomic approach to drug screening, Gene Expression-based High-throughput Screening (GE-HTS), in which gene expression signatures serve as surrogates for different biological states. This is a generic method and a departure from traditional target-based or phenotype-based screening. Her laboratory has applied the GE-HTS approach to the alteration of the malignant state (i.e., induction of acute myeloid leukemia (AML) and neuroblastoma differentiation) and to the modulation of intractable protein targets such as transcription factor abnormalities in Ewing sarcoma and T-cell acute lymphoblastic leukemia. Compounds emerging from screening efforts have served as clinical leads for trial development as well as tool compounds for probing mechanism of oncogenesis and for the identification of new cancer targets. Ongoing work in the laboratory focuses on applying these approaches to:

  1. Identification of new cancer-promoting protein targets
  2. Modulation of pharmacologicaly challenging oncoproteins (e.g., transcription factors)
  3. Identification of new small-molecule inducers of cancer cell differentiation

The laboratory’s ultimate goal is the translation of compelling findings to clinical trial. Clinical trials for AML and Ewing sarcoma have resulted from this research.

About Kimberly Stegmaier

Kimberly Stegmaier is a physician-scientist who received an MD from Harvard Medical School. She completed her Pediatrics internship/residency at Children's Hospital Boston and a Pediatric Hematology/Oncology fellowship at the Children's Hospital Boston/Dana-Farber Cancer Institute where she also served as the Chief Fellow. Dr. Stegmaier 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 (DFCI), and an attending physician at the Children’s Hospital Boston (CHB) and DFCI. She is also an Associate Member of the Broad Institute of Harvard and MIT.

Dr. Stegmaier has been the recipient of an American Society of Hematology Fellow Scholar Award, Hood Foundation Child Health Research Grant, Sidney Kimmel Cancer Foundation Scholar Award, Smith Family New Investigator Award and HHMI Physician-Scientist Early Career Award. She was recently named a Stand-Up-to-Cancer Innovative Research Grant Recipient and received a number of other awards, including, the ASH Joanne Levy, MD, Memorial Award for Outstanding Achievement (2006), the 2007 Genome Technology “Tomorrow’s Principal Investigator” Award and the Society for Pediatric Research Young Investigator Award (2012). Outside of her scientific and clinical endeavors, Dr. Stegmaier has a passion for ballet and has performed professionally with Ballet Theater of Boston and the Cambridge Chamber Ballet.

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Researcher Areas

  • Pediatric and Other Malignancies

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