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.


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.


DOI: 10.1038/s41588-018-0191-z

Malone et al show that selective modulation of the nuclear mRNA export protein NXT1 is a selective dependency in MYCN-amplified neuroblastomas.

Ewing sarcoma

DOI: 10.1016/j.ccell.2017.12.009

Seong et al show that TRIM8 is a dependency in Ewing sarcoma through reduced degradation of EWS/FLI.

Acute Myeloid Leukemia

DOI: 10.1038/s41375-018-0291-x

Pikman et al demonstrate in a prospective clinical trial that systematic precision medicine enables matching of patient tumor mutations with targeted therapies.


Grants and Awards


Nathaniel Mabe: NIH F32.
Jana Ellegast: Pediatric Cancer Research Foundation.
Diana Lu: NIH F30.
Neekesh Dharia: St. Baldrick's Fellowship 3rd Year.

Grants and Awards


Jana Ellegast: Lady Tata Memorial Trust.
Shan Lin: Lymphoma & Leukemia Society.
Clare Malone: NIH F32.
Alex (Bo Kyung) Seong: DoD Horizon Award.

Grants and Awards


Neekesh Dharia: Rally Foundation, Curing Kids Cancer.
Lillian Guenther: Rally Foundation.
Diana Lu: Landry Cancer Research Fellowship.

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 .

Grants and Awards

Spring 2018

Yana Pikman: NIH/NCI K08.
Neekesh Dharia: St. Baldrick's Fellowship.

Chairperson-Elect of the AACR Pediatric Cancer Working Group

February 22, 2018

Kimberly Stegmaier will assume the role of chairperson-elect of the AACR Pediatric Cancer Working Group for 2018-2019.

Damon Runyon-Jake Wetchler Award for Pediatric Innovation

November 22, 2017

Amanda Balboni Iniguez has received the 2017 Damon Runyon-Jake Wetchler Award for Pediatric Innovation. Watch Mandy talk about her research!

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.



Meet our lab members.