Big Data Initiative Will Advance Pediatric Cancer Precision Medicine

The University of Chicago Pediatric Cancer Data Commons (PCDC) is participating in new collaborative effort to leverage big data to advance pediatric cancer precision medicine.

The global initiative, called the Leukemia and Lymphoma Society’s LLS Pediatric Acute Leukemia (PedAL) precision medicine master clinical trial, aims to improve the treatment of all children with pediatric acute leukemia.

While precision medicine is rapidly making the transition from research to clinical care for adult patients, progress for children with cancer has not kept pace.

LLS PedAL will aim to accelerate precision medicine for pediatric cancer care by testing targeted therapies for relapsed acute leukemia based on the specific genetic abnormalities driving the cancer. More than 200 sites worldwide will participate in this effort.

“Our goal is to revolutionize the standard of care for pediatric cancer patients, providing children and young adults with safer, more effective treatments that rely less on chemotherapies that harm healthy cells in the child’s body,” said Gwen Nichols, MD, the chief medical officer for LLS.

In addition to testing numerous targeted therapies, LLS PedAL will consolidate pediatric cancer data from all sites into a single data set.

“The lack of data and samples for study have been the most significant barriers to large-scale discovery and advancement in the treatment of pediatric cancers,” said pediatric oncologist Sam Volchenboum, MD, PhD, MS, director of the PCDC. “We are creating a new infrastructure to support PedAL’s wide variety of research and clinical activities.”

The team will build two data platforms for LLS PedAL. The first, called the Genomic Eligibility Algorithm at Relapse for Better Outcomes (GEARBOx), will aim to accurately and rapidly match patients who have relapsed or refractory disease to appropriate clinical trials and lifesaving treatments.

This platform will include clinical trials both within the LLS PedAL master trial, as well as other trials outside of PedAL for children with acute leukemia. GEARBOx will provide doctors with multiple trial options to help them make treatment decisions for their patients.

The second platform, the PCDC, will bring together large amounts of pediatric leukemia data into one unified space. By creating a universal international acute leukemia data dictionary, the PCDC will connect and harmonize historical and new data. The unique platform will power research by including data linkages, outcomes tracking and analysis tools, and will be accessible to scientists worldwide.

The PCDC aims to drive research and cures through the connection of dispersed data and collaborative data sharing. By working with international leaders in pediatric cancers and legal representatives from partner institutions around the world, PCDC seeks to develop a wide spectrum of clinical research.

The LLS PedAL initiative is just one part of the LLS Children’s Initiative: Cures and Care for Children, a multi-year endeavor to help children with blood cancer and their families throughout the medical journey, including research investment, education and support services, and advocacy efforts.

“Our goal is no less than a wholesale shift in the standard of care for pediatric patients, moving from toxic chemotherapies that leave survivors with lifelong health challenges to effective, safe treatments that precisely target cancer without harm to the rest of the child’s body,” said Gwen Nichols, MD, LLS Chief Medical Officer.

Through this new international initiative, researchers will continue to advance the role of precision medicine in pediatric cancer care.

“We are so excited to be building the next-generation platform for matching children with leukemia to innovative clinical trials,” Volchenboum said. “In the process, we will be creating the largest pediatric leukemia data set in the world.”