While precision medicine — selecting a treatment tailored to each person’s unique tumor characteristics (or biomarkers) as well as the genetic changes in their cancer — is rapidly reaching adult patients, progress for children with cancer has not kept pace.
The University of Chicago Pediatric Cancer Data Commons (PCDC) is a partner in a new collaborative effort to change this paradigm, bringing the power of precision medicine to children with high-risk, hard-to-treat and relapsed leukemia.
The global initiative — The Leukemia & Lymphoma Society’s LLS PedAL (Pediatric Acute Leukemia) precision medicine master clinical trial — aims to improve the treatment of all children with pediatric acute leukemia, including myeloid leukemia and other high-risk leukemias.
LLS PedAL will test targeted therapies for relapsed acute leukemia based on the specific genetic abnormalities driving the cancer. Leaders in childhood leukemia built the foundation for this integrated, multitherapy trial. More than 200 sites worldwide will be participating.
“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.
Strength in data
In addition to testing numerous targeted therapies, LLS PedAL will consolidate pediatric cancer data from all sites into a single data set. Pediatric oncologist Sam Volchenboum, MD, PhD, MS, director of the PCDC, is leading this bioinformatics effort.
“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,” Volchenboum said. “We are creating a new infrastructure to support PedAL’s wide variety of research and clinical activities.”
Volchenboum’s team will build two data platforms for LLS PedAL:
- GEARBOx – (Genomic Eligibility Algorithm at Relapse for Better Outcomes) 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.
- Pediatric Cancer Data Commons – to 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.
“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.”
