Pediatric Oncology

Our research program focuses on the integration of "omic" approaches for the identification of new protein targets and small-molecule modulators of malignancy with an eye toward clinical translation.

Cancer discovery efforts in our laboratory have focused on the alteration of the malignant state (e.g., AML and neuroblastoma differentiation) and the modulation of pharmacologically challenging oncoproteins (e.g., EWS/FLI in Ewing sarcoma, MYCN in neuroblastoma, and NOTCH1 in T-ALL.) Most recently, we are applying an integrated approach to discover new therapeutic opportunities in these malignancies with deep genomic characterization of primary tumors, kinase activity profiling for immediately druggable targets, functional genomic screening for new tumor dependencies and chemical screening for modulators of relevant oncogenic drivers. Clinical trials for patients with AML and Ewing sarcoma have resulted from our research and a trial testing BET bromodomain inhibitors in patients with MYCN-amplified neuroblastoma is in development.

Research

Highlights of our recent work.

Pediatric Dependency Map

DOI: 10.1016/j.ccell.2018.11.005

Dharia et al demonstrate a first generation pediatric cancer dependency map that suggests that pediatric cancers have vulnerabilities that are distinct from those in adult cancers.

Neuroblastoma

DOI: 10.1038/s43018-022-00405-x

Mabe and Huang et al show that GD2 is downregulated in mesenchymal neuroblastoma and is reversible with EZH2 inhibitors.

Ewing Sarcoma

DOI: 10.1038/s41556-022-01059-8

Lu DY, Ellegast JM et al show that ETV6 is a dependency in Ewing sarcoma through modulating EWS-FLI1 binding to chromatin.

Leukemia

DOI: 10.1158/2159-8290.CD-21-0956

Ellegast et al identified IRF2BP1 as an enriched dependency in AML through deregulation of inflammatory signaling.

News

Cancer Grand Challenges

2024

Dr. Stegmaier will be part of the PROTECT Cancer Grand Challenges team to develop new therapeutics to target oncogenic drivers in children.

U01 grant funded

2024

The NCI announced the funding of a U01 grant co-led by Kimberly Stegmaier and Robbie Majzner to interrogate ganglioside targeting in sarcomas.

Grants and Awards

2022-2023

Constanze Schneider: Helen Gurley Brown award.
Nathaniel Mabe: NIH K99/R00 Pathway to Independence.
Yara Zabala Rodriguez : HHMI Hanna H. Gray Fellow.
Susu Zhang: Alex's Lemonade Stand Young Investigator.
Stefan Bjelosevic: Leukemia & Lymphoma Society Fellow.

Cancer Moonshot Grant

October 2018

A Dana-Farber and Boston Children’s Hospital team, led jointly by Kimberly Stegmaier, MD and Scott Armstrong, MD, PhD, wins Cancer Moonshot grant to therapeutically target EWS-oncoproteins.

Professor of Pediatrics

July 2018

Kimberly Stegmaier was promoted to Professor of Pediatrics at Harvard Medical School!

Save Lives Now

April 25, 2018

Kimberly Stegmaier featured along with other cancer researchers and the Avengers on American Airlines planes for Stand Up To Cancer!

Casty Family Achievement in Mentoring

April 2018

Kimberly Stegmaier was recognized as an outstanding faculty mentor who has positively shaped the careers of individuals dedicated to advancing cancer medicine .

Robert J. Arceci Innovation Award

April 27, 2017

Kimberly Stegmaier was named winner of the 2017 Robert J. Arceci Innovation Award by the St. Baldrick's Foundation.

Endowed Ted Williams Chair

September 30, 2016

Kimberly Stegmaier was named the Ted Williams Chair. This Chair is designated for an outstanding investigator in the Department of Pediatric Oncology at Dana-Farber Cancer Institute.

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People

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