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St. Baldrick’s Foundation-SU2C Pediatric Cancer Dream Team: Immunogenomics to Create New Therapies for High-Risk Childhood Cancers

John M. Maris, MD
Leader

John M. Maris, MD

Director, The Center for Childhood Cancer Research
The Children’s Hospital of Philadelphia

Crystal L. Mackall, MD
Co-leader

Crystal L. Mackall, MD

Associate Director, Stanford Cancer Institute
Co-Medical Director, Stanford Laboratory for Cell and Gene Medicine

Overview

The St. Baldrick’s Foundation – SU2C Pediatric Cancer Dream Team is a collaboration between pediatric cancer researchers in the largely disparate disciplines of cancer genomics and immunotherapeutics. The team will focus on developing new, targeted immunotherapeutics for the most difficult-to-cure childhood cancers.

New classes of therapeutics are needed to improve the survival of children with cancer and decrease the physical, emotional, and financial life-altering costs of curative therapies. The Team has used new technologies in the field of cancer genomics, epigenetics and proteomics to discover and validate new targets for immunotherapy. They have built new antibodies, antibody-drug conjugates and CAR T cells to attack these targets. The team has innovated in developing new immunotherapies, discovering basic mechanisms of effectiveness (or lack thereof) in both antibody and cellular engineering, and developed novel methods to monitor clinical effectiveness and toxicity. The team has opened 25 clinical trials and has treated 688 pediatric patients with cancers that have resisted treatment. They demonstrated the potency of immunotherapy against acute lymphocytic leukemia (ALL), as well as defined mechanisms for how these cancers cells develop resistance. They have also made progress against childhood solid cancers, with many emerging therapeutics just entering the clinics, or scheduled to enter testing over the next one to three years.

Originally launched as the SU2C-St. Baldrick’s Foundation Pediatric Cancer Dream Team, the team will continue its work with funding from St. Baldrick’s while remaining under the SU2C umbrella.

Progress to Date

The St. Baldrick’s Foundation-SU2C Pediatric Cancer Dream Team has major contributions to the treatment of cancer in children by fusing the fields of genomics and immunotherapeutics. Its work contributed to the historic approval of a T-cell therapy for acute lymphocytic leukemia.

In its work to date, the Dream Team has:

  • Launched 25 clinical trials and treated 688 pediatric patients with cancers that have resisted existing treatments.
  • Pioneered work in immunotherapy, including chimeric antigen receptor T cell (CAR-T) therapy that has resulted in the previously unheard-of rate of complete response in more than 70 percent of children treated who have a type of acute lymphocytic leukemia (ALL).
  • Made major contributions to the work that led to FDA approval of tisagenlecleucel, the first CAR T-cell therapy approved for ALL.
  • Helped identify biomarkers predicting which patients receiving T-cell therapy will encounter severe Cytokine Release Syndrome (CRS), “cytokine storm,” a life-threatening complication that sometimes occurs with the use of antibody therapies.
  • Developed a standardized diagnosis and management plan for the management of cytokine storm, which is now widely used.
  • Made progress against childhood solid cancers, with many emerging therapeutics against diseases such as neuroblastoma, glioblastoma, medulloblastoma, osteosarcoma, Ewing sarcoma and rhabdomyosarcoma just now entering the clinics, or scheduled to enter into Phase 1 testing over the next one to three years.
  • Generated 105 peer-reviewed publications and 16 patent applications.

Amount Of Funding

$14.5 million

Principals

  • ​Poul Sorensen, MD, PhD, University of British Columbia, Vancouver, Canada
  • Donald W. Parsons, MD, PhD, Baylor College of Medicine, Houston, Texas
  • Michael D. Taylor, MD, PhD, The Hospital for Sick Children, Toronto, Canada
  • Michael C. Jensen, MD, Seattle Children’s Hospital, Seattle, Washington
  • Paul Sondel, MD, PhD, University of Wisconsin, Madison, Wisconsin

Advocates

  • ​Kelly Lynn Cotter
  • Jay Scott
  • Patrick James Sullivan
  • Lisa W. Tichenor