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.

T-cell Leukemia

DOI: 10.1084/jem.20151778

Roti et al describe the leukemia-specific delivery of mutant NOTCH1 targeted therapy with an alcohol derivative of thapsigargin conjugated to folate via a cleavable ester linkage.

Neuroblastoma

DOI: 10.1172/JCI90793

Chen et al demonstrate that MYCN upregulates EZH2 leading to inactivation of a tumor suppressor program in neuroblastoma, thus supporting testing EZH2 inhibitors in patients with MYCN-amplified neuroblastoma.

Ewing sarcoma

DOI: 10.1016/j.ccell.2017.12.009

Iniguez et al show that EWS/FLI confers tumor cell synthetic lethality to CDK12 inhibition in Ewing sarcoma.

Acute Myeloid Leukemia

DOI: 10.1038/nm.4283

Fenouille & Bassil et al report mitochondrial creatine kinase CKMT1 is necessary for survival of EVI1-positive AML and targeting CKMT1 is a promising therapeutic strategy.

News

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.

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People

Meet our lab members.

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