Uniting the world-class strengths of different institutions to tackle the same problems can result in greater progress than any single institution could achieve. The Pediatric Cancer Dependencies Accelerator is doing this by leveraging the expertise and capabilities of the Broad Institute of MIT and Harvard, Dana-Farber Cancer Institute, and St. Jude Children’s Research Hospital.

The project is accelerating the identification of vulnerabilities in pediatric cancers and translating them into better treatments by marrying innovation in mapping cancer dependencies to pediatric cancer expertise. This vision of transformative progress is supported by joint funding of more than $60 million from all three institutions over five years. The investment supports infrastructure development and scientific work by a team of more than 100 investigators, data scientists, trainees, and research staff.

The project is co-led by Charles W.M. Roberts, MD, PhD, St. Jude Executive Vice President and Comprehensive Cancer Center director, Kimberly Stegmaier, MD, vice chair for Pediatric Oncology Research, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and institute member at the Broad Institute, and Francisca Vazquez, PhD, Broad Institute Cancer Dependency Map director.

“Despite many advances, cancer remains the number one cause of death by disease for children in the U.S.,” Roberts said. “It can take decades in a research lab to understand mechanisms and develop new treatments. Through this project, we believe we can now leapfrog barriers to rapidly identify therapeutic vulnerabilities in childhood cancer and translate those into targeted therapies in the clinic much faster.”

Through the collaboration, cross-functional groups of investigators focus on three core disease areas (brain tumors, hematological malignancies, and solid tumors) and two cross-cutting interest areas (pan-cancer and data science). These working groups accelerate progress by combining unique strengths, resources, and technologies.

Charles W.M. Roberts, MD, PhD

Through this project, we believe we can now leapfrog barriers to rapidly identify therapeutic vulnerabilities in childhood cancer and translate those into targeted therapies in the clinic much faster.

Charles W.M. Roberts, MD, PhD

Executive Vice President; Director, Comprehensive Cancer Center

The project is advancing diverse scientific aims, which include:

  • developing and deploying genome-editing approaches to identify hidden dependencies in a range of high-risk childhood cancers
  • leveraging emerging technologies to characterize pediatric cancers’ genetic and epigenetic landscape
  • developing model systems where none currently exist for high-risk childhood cancers that have poor outcomes
  • identifying effective combination therapies and mechanisms of drug resistance and shortening the timeline for developing new therapies
  • developing computational approaches to mine and integrate data and building innovative software tools for data sharing

This collaboration builds on groundbreaking research initiatives. The St. Jude–Washington University Pediatric Cancer Genome Project yielded rich insights into the genomic landscape of pediatric cancers, including the discovery that most disease-driving genetic mutations are not druggable. The Cancer Dependency Map (DepMap) Initiative from the Broad Institute, in collaboration with Dana-Farber investigators, developed extensive, world-class datasets and computational infrastructure that impacted research and target discovery programs worldwide. Launched in 2015, the Pediatric Cancer Dependency Map Project served as a proof of concept for applying the DepMap approach to childhood cancers. That effort created infrastructure and expertise that is being leveraged and expanded through the Pediatric Cancer Dependencies Accelerator.

The Pediatric Cancer Dependencies Accelerator is a testament to collaboratively uniting diverse expertise to pursue opportunities with transformative potential.