This trial is open to enrollment.

Targeting Epigenetic Dysregulation

Kimberly Stegmaier, MD

Co-Director of the Pediatric Hematologic Malignancy ProgramDana-Farber Cancer InstituteView Bio

Stegmaier is an Associate Professor of Pediatrics at Harvard Medical School, a Principal Investigator in Pediatric Oncology at the Dana-Farber Cancer Institute (DFCI), and an Attending Physician at the Boston Children’s Hospital Boston (BCH) and DFCI. She is the Co-director of the Pediatric Hematologic Malignancies Program at DFCI and BCH and is also an Associate Member of the Broad Institute of Harvard and MIT. She received her B.S. from Duke University and her M.D. from Harvard Medical School. She completed her residency at BCH and a post-doctoral pediatric hematology-oncology fellowship at DFCI/BCH. In 2006, she launched her own laboratory effort at DFCI.

Stegmaier’s laboratory integrates chemical biology, genomic, and proteomic approaches to discover new lead compounds and protein targets for cancer therapy. She has focused her efforts on the acute leukemias and two pediatric solid tumors of childhood: Ewing sarcoma and neuroblastoma.

Steven DuBois, MD, MS

Director, Experimental TherapeuticsDana-Farber Cancer InstituteView Bio

DuBois is director of experimental therapeutics at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. He is a pediatric oncologist with an active clinical and translational research program focused on patients with advanced neuroblastoma and Ewing sarcoma. He conducts phase I and II clinical trials of novel targeted agents. His clinical practice focuses on providing outstanding team-based care for children and young adults with solid cancers. He is also a member of the American Pediatric Society.

View Full Team

Meet the Team

  • Kimberly Stegmaier, MD, Dana-Farber Cancer Institute
  • Steven DuBois, MD, MS, Dana-Farber Cancer Institute
  • David Kirsch, MD, PhD, Duke University
  • Elizabeth Lawlor, MD, PhD, University of Michigan
  • Peter Dirks, MD, PhD, The Hospital for Sick Children


  • Patient Advocate: Kathleen Malcolmson
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About This SU2C Catalyst Clinical Trial

Cancer is the leading cause of death from disease in children in North America. Many children’s cancers stem from faulty regulation of genes responsible for cell division and cell death. This misregulation is often due to mutation or amplification of a single type of protein called a transcription factor, which controls the degree to which a gene is expressed, or turned on.

What is epigenetics?

The study of how external or environmental factors turn genes on and off.

What is a BET inhibitor?

An anti-cancer drug that blocks proteins important in reading DNA, which is a process important for cancer cells.

This SU2C Catalyst Team hypothesizes that one way to stop abnormal gene expression and to eradicate cancer cells may be to target BET proteins, a type of protein that plays a key role in regulating gene expression. In the laboratory, drugs that inhibit BET activity have been shown to strongly decrease the expression of cancer-promoting transcription factors in a number of high-risk childhood cancers, yet until now a clinical trial of BET inhibitors in pediatric patients has never been done.

In this clinical trial, the team is evaluating a BET inhibitor from Bristol-Myers Squibb and, in parallel, performing laboratory research to identify other drugs that, when combined with the BET inhibitor, will lead to even better anticancer activity. This team’s work will lay the foundation for new drug combinations to treat children with cancer in phase II clinical trials.

We Need You

Why Your Participation Matters

This project is the first test of BET inhibitor therapy in children with the highest-risk pediatric cancers. It will allow us to (1) establish a safe and active dose to be further studied in phase II clinical trials, (2) investigate why specific cancers respond to (or resist) treatment with BET inhibitor therapy, and (3) potentially provide benefit to children participating in this trial. The team will also identify drugs that may be used in combination with BET inhibitors to make treatment more effective. These combinations will then be studied in additional clinical trials across a group of high-risk pediatric cancers.


There are additional criteria that must be met to participate, or that may prevent a patient from participating.

Key criteria are summarized below and can be found in detail on Interested patients will need to review their medical histories with a clinical trial patient coordinator before they can be accepted to participate in this trial.

Male or Female
Age Range:
1 - 21 years of age at enrollment
Relapsed or refractory (resistant to treatment) pediatric cancers including solid tumor, lymphoma, and specific primary brain tumors.


Participants must have active disease seen on scans, including solid tumors or lymphoma.

Patients must have fully recovered from the acute toxic effects of all prior anticancer therapy.

Patients must not have received prior therapy with other BET inhibitors.

Before beginning the trial, your child must be able to function well enough to care for him/herself most of the time without assistance, as appropriate to the child’s age.

Participants must meet specific organ function criteria.

Patients must be able to swallow intact pills.

Female patients who are pregnant or nursing will not be eligible.

Certain foods/juices and medications are prohibited prior to enrolling and while being treated in this clinical trial.

Patients with primary or metastatic CNS tumors will not be eligible unless they meet predefined genomic criteria.

Once enrolled, patients will take the BET inhibitor by mouth once a day for 5 days followed by 2 days off.

Patients are seen at the study center once a week during the first 28-day cycle. Visits become less frequent after the first cycle.

Patients may receive up to 26 cycles as long as scans do not show worsening of the disease and side effects are not severe.

The trial is open to patients with a range of tumor types and tumor biological features, but slots are always available for patients with tumors that have specific genetic changes predicted to make the tumors more sensitive to BET inhibitors. Some of these genetic changes include extra copies of the genes MYC or MYCN. In addition, patients with a rare cancer called NUT midline carcinoma can enroll at any time.


You play a vital role.

Here is the location where you can currently participate in this clinical trial. If you or a loved one is interested in enrolling and learning more about this study, please contact the patient coordinator below. The patient coordinator is there to help you understand every aspect of the clinical trial process and answer any questions you may have.

Once you contact the patient coordinator, he or she will start by reviewing your medical history with you to see whether you meet all the criteria to participate. The coordinator will then guide you through a review of the study and detailed “informed consent” documents that you are required to sign when you enroll in a clinical trial.

Boston, Massachusetts

Dana-Farber Cancer Institute
Steven G DuBois, MD, MS
P: 617-632-5460

Durham, North Carolina

Duke University Medical Center
David Van Mater, MD, PhD
P: 919-681-3401

Ann Arbor, Michigan

University of Michigan, C.S. Mott Children’s Hospital
Rajen Mody, MD
P: 734-764-7126

Toronta, Ontario

The Hospital for Sick Children
Daniel Morgenstern, MD
P: 416-813-7654 x227565

Cincinnati, OH

Cincinnati Children's Hospital
Trent Hummel, MD
P: 513-803-1140


Support for this study comes from the Bristol-Meyers Squibb Company.
Learn more about BMS +
Here are some links to organizations that provide more information about Pediatric Hypermutant Cancers, and may provide information on patient support, treatment options, and current clinical trials.
Dana-Farber/Boston Children’s Cancer and Blood Disorders Center +
For additional resources, please visit +

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Even if you do not meet the requirements for this trial, there may be other trials for you. Get started with the SU2C Clinical Trial Finder, a free and confidential cancer clinical trial matching and referral service.
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