The age of precision medicine in cancer care for adults is rapidly approaching as it’s not uncommon now to select a treatment that’s actually tailored to each person’s unique tumor characteristics (biomarkers) as well as genetic changes in their specific cancer. For children, unfortunately the situation hasn’t progressed as fast due to a variety of reasons including a dearth of data. Now, in an effort to change that and work to bring precision cancer care to children, the University of Chicago Pediatric Cancer Data Commons (PCDC) has teamed up in a new global initiative known as The Leukemia & Lymphoma Society’s LLS PedAL (Pediatric Acute Leukemia) precision medicine master clinical trial, aiming to progress 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. Over 200 sites worldwide are planning on participating in this important effort.
Gwen Nichols, MD chief medical officer for LLS, commented, “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.
The Data Premise
LLS PedAL, in addition to testing a number of targeted therapies with children, will consolidate pediatric cancer data from all sites into a single data set. Sam Volchenboum, MD, PhD, MS directs University of Chicago’s PCDC and leads the LLS PedAL bioinformatics effort. The pediatric oncologist commented in the group’s recent press release, “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.” Dr. Volchenboum elaborated that in fact this group will be developing “… new infrastructure to support PedAL’s wide variety of research and clinical activities.”
The Infrastructure: Two Data Platforms
Dr. Volchenboum and team at LLS PedAL plan the development of two data platforms including 1) GEARBOx (Genomic Eligibility Algorithm at Relapse for Better Outcomes) and 2) Pediatric Cancer Data Commons. The goal is compelling and urgently needed for children with leukemia and includes two major deliverables, including 1) an advanced matching algorithm connecting leukemia patients to innovative clinical trials and, 2) the world’s largest pediatric leukemia data set.
GEARBOx will 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 offer doctors with multiple trial options to help them make treatment decisions for their patients. In this way Gearbox can be thought of as also enabling the research as a care option.
While Pediatric Cancer Data Commons (PCDC) will bring large amounts of pediatric leukemia data into one unified environment. By creating a universal international acute leukemia data dictionary, the PCDC will connect and harmonize historical and new data—again a major gap presently in children’s cancer care. The unique platform will power research by including data linkages, outcomes tracking and analysis tools, and will be accessible to scientists worldwide.
LLS PedAL involves a number of collaborators but is led by Leukemia & Lymphoma Society (LLS). The patient advocacy group has brought together leaders in childhood leukemia to develop the foundation for the collaborative master clinical trial for children with relapsed acute leukemia. The group seeks to break silos across sectors, scientific disciplines, institutions and countries in the mission to bring precision medicine and advanced cancer research as a care option to children. While the team at the University of Chicago Pediatric Cancer Commons (PCDC) develops the LLSPedAL data infrastructure described above, other collaborators are involved from pharmaceutical companies, nonprofit organizations, other sample and data management vendors, trial site organizations, genomics organizations, regulators and more.
LLS Children’s Initiative: Cures and Care for Children
The Leukemia & Lymphoma Society (LLS) strives to drive multifaceted advancements for children combating leukemia. The LLSPedAL is one piece to the LLS Children’s Initiative: Cures and Care for Children, which represents a multi-year program centering on the support of children with blood cancer, as well as their families, through every facet of the organization’s mission, from life saving research investment to education, support services and advocacy.
Dr. Gwen Nichols, MD, LLS Chief Medical Officer, reports, “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.”
About The Leukemia & Lymphoma Society (LLS)
The Leukemia & Lymphoma Society (LLS) is a 501 charitable organization founded in 1949; it happens to be the largest voluntary health organization dedicated to fighting blood cancer in the world. The organization, its leadership and staff are dedicated to cure leukemia, lymphoma, Hodgkin’s disease and myeloma, as well as materially improve the quality of life of patients and their families.
Dr. Gwen Nichols, MD, LLS Chief Medical Officer
Sam Volchenboum, MD, PhD, MS, Associate Professor of Pediatrics, Dean of Master’s Education, Associate Chief, Research Informatics Officer, Associate Director, Institute for Translational Medicine
Call to Action: Interested in getting involved with LLSPedAL as a vendor/partner? Check out their leadership. Interested in supporting the cause of precision-based care for children fighting blood cancers? See the link.