Pediatric SU2C Catalyst®

The Pediatric SU2C Catalyst seeks to accelerate the development of treatments for pediatric cancers to initiate the pivotal clinical trial; and by supporting preclinical pediatric research and early phase clinical trials in children in parallel or in advance of adult clinical trials.


This cutting-edge program has a strong focus on preclinical and early, rationally-designed clinical trials in order to identify medicines that are suitable for further clinical development in pediatric oncology. The Pediatric SU2C Catalyst uses combinations of therapies and devices from other sources and companies for refractory pediatric cancers.


Pediatric SU2C Catalyst Funding

The generous funding of our charter industry leader, Bristol-Myers Squibb, supports the costs and oversight of the trials and/or translational research, including funding for the participating investigators and institutions. Our industry supporters also make available medicine(s), diagnostic test(s) and/or device(s) to Pediatric SU2C Catalyst teams.

Pediatric SU2C Catalyst Teams

Brain Tumors + Learn More

Immodulation to Treat Poor-Prognosis Pediatric Brain Tumors
Most children with brain tumors experience low survival rates even with maximal invasive treatment such as surgery, intensive chemotherapy, and radiation therapy. To avoid such invasive and potentially toxic therapies, scientists have been developing ways to help the body’s own immune system recognize cancer cells and kill them. One of the biggest developments in this area so far has been in the design of checkpoint inhibitor drugs that are able to block cancer cells’ protective mechanisms. An example of such a successful drug is the PD-1 inhibitor. Unfortunately, PD-1 inhibitors have proven ineffective for the treatment of pediatric brain tumors. There is, however, one possible way to make PD-1 inhibitors effective at killing pediatric tumor cells by combining them with drugs that mimic viral infection. These “viral mimicry” drugs can prime cancer cells to be killed by the immune system’s T-cells. In this project, the team proposes to combine these “viral mimicry” drugs with PD-1 inhibitors to see if this combination is effective in pediatric brain cancer models. The studies they propose will be carried out in mice engineered to grow aggressive, malignant pediatric brain tumors. This team’s work promises to help establish whether this novel combination therapy is safe and feasible for future clinical trials and will lead directly to future human trials in the 15-member Collaborative Network of Neuro-oncology Clinical Trials (CONNECT).

This proposal fills a critical, unmet need for children with poor-prognosis brain tumors by exploring the combination of CDK4/6 inhibitors or DNMT inhibitors and immune checkpoint blockade judiciously administered on the backbone of current therapies. Promising results in this pre-clinical research study will a) lead to a paradigm shift in the treatment of children with high-risk brain tumors for whom further intensification of conventional therapies is not an option, and b) accelerate information leading to clinical trials to determine feasibility and define efficacy.

Team Leader(s):
Maryam Fouladi, MD, MSc, Cincinnati Children’s Hospital Medical Center
James Olson, MD, PhD, Fred Hutchinson Cancer Research Center

Team Members:
Nada Jabado, MD, PhD, The Research Institute of the McGill University Health Centre
Annie Huang, MD, PhD, The Hospital for Sick Children

Patient Advocate:
Keith Desserich, Cure Starts Now

Epigenetics+ Learn More

Targeting Epigenetic Dysregulation in Pediatric Cancer
Cancer is the leading cause of death from disease in children in North America. One of the commonalities in children’s cancers is the inappropriate regulation of genes responsible for cell division and cell death. This mis-regulation is often due to mutation or amplification of a single transcription factor, which controls the degree to which a gene is expressed, or turned on. Bromodomain and Extra-Terminal motif (BET) proteins play a key role in regulating gene expression. In the laboratory, BET inhibitors have been shown to strongly decrease the expression of cancer-promoting transcription factors in a number of high-risk childhood cancers, yet a clinical trial of BET inhibitors in pediatric patients has never been done. The team proposes to test a BET inhibitor in children with cancer in the first pediatric clinical trial of BET inhibitors. In parallel, the team will perform laboratory research to identify novel drugs that, when combined with the BET inhibitor, will lead to even better anti-cancer activity. This translational work will lay the foundation for new drug combinations to treat children with cancer in second-generation clinical trials.

Team Leader(s):
Kimberly Stegmaier, MD, Dana-Farber Cancer Institute
Steven DuBois, MD, MS, Dana-Farber Cancer Institute

Team Members:
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

Hypermutant Cancers l+ Learn More

Combined Approaches by Immune Checkpoint inhibition for Hypermutant Cancers
This bold global clinical trial focuses on pediatric cancers with an extraordinary number of DNA mutations regardless of cancer type. The team has had initial success treating children with a single immunotherapy drug and is now investigating multiple immunotherapy drug combinations. The team is also looking for biological indicators that will help predict which patients will be best suited for each combination. These results could inform treatment strategies for adult tumors such as melanoma, lymphomas, and other solid tumors.

Team Leader:
Uri Y. Tabori, MD, The Hospital for Sick Children (Canada)

Team Members:
Eric Bouffet, MD, The Hospital for Sick Children (Canada)
Michael Fisher, MD, Children’s Hospital of Philadelphia
Patrick Forde, MBBCh, Johns Hopkins University
Cynthia Hawkins, MD, PhD, FRCPC, The Hospital for Sick Children (Canada)
John Maris, MD, Children’s Hospital of Philadelphia
Daniel Morgenstern, MD, The Hospital for Sick Children
Pamela Ohashi, PhD, University of Toronto (Canada)
Trevor Pugh, PhD, University of Toronto (Canada)
Adam Shlien, PhD, The Hospital for Sick Children (Canada)
Victor Velculescu, MD, PhD, Johns Hopkins University

Patient Advocate:

Denise Bebenek

Hypermutant Cancers ll+ Learn More

Clinical Translation of Novel Immune-Based Combination Therapies for Paediatric Hypermutant Cancer
Hypermutant cancers are cancers that have a high number of DNA mutations. They occur in 10-20% of cases, arise in any type of cancer from any organ, and are extremely aggressive. Problematically hypermutant cancers usually do not respond to currently available therapies such as chemotherapy and radiation. This team made an important discovery that, in children, hypermutant cancers are caused by mutations in DNA repair genes that allow many mistakes to be made when DNA is replicated as cells divide. They were also first to demonstrate that children with hypermutant cancers respond to immunotherapy, which improved long-term survivorship and quality of life. In this project, the Team proposes two groundbreaking initiatives in the advancement of childhood hypermutant cancer treatment: 1) test new promising drug candidates in combination with immunotherapy on mouse models and patient-derived cell lines, and 2) establish the first clinical trial using combination therapy through a new childhood cancer immunotherapy network (PedCITN). These initiatives will offer great hope for children with hypermutant cancers.

Team Leader(s):
Uri Tabori, MD, The Hospital for Sick Children
Daniel Morgenstern, MD, The Hospital for Sick Children

Team Members:
John Maris, MD, Children’s Hospital of Philadelphia
Crystal Mackall, MD, Stanford University

Patient Advocate:

Denise Bebenek
Jenell Holstead