The global leader in the fight against blood cancers, The Leukemia & Lymphoma Society (LLS), announced the launch of its Pediatric Acute Leukemia (PedAL) Master Clinical Trial, an unprecedented international collaboration led by LLS designed to fundamentally change how children with acute leukemias are treated.
PedAL is a key component of Dare to Dream, a bolder rebrand of the LLS Children’s Initiative, which envisions a world where childhood blood cancer patients not only survive but thrive after treatment. As a multi-year initiative, Dare to Dream also expands education and support services for patients and families, drives advocacy and policy efforts, and increases investments in childhood blood cancer research.
“Because of the project’s success over the past three years, we have increased Dare to Dream’s ambitious fundraising goal to $175 million and are committed to fundamentally changing treatment and care over the next five years through advocacy, patient support, research and PedAL,” Gwen Nichols, M.D., Chief Medical Officer at LLS and co-chair of PedAL, said.
The Need to Accelerate Safer Treatment Options for Kids
The goal of PedAL is to expedite the development of new targeted treatments for hard-to-treat childhood leukemias and replace one-size-fits-all chemotherapy with therapies tailored to each child’s unique tumor biology.
“Children are not little adults. Their bodies work in different ways, and they undergo changes as they grow, which means we need different treatment approaches, especially for children with relapsed acute leukemias,” Dr. Nichols said. “Our vision is to identify the unique tumor biology of each child’s cancer to help match them with the most promising treatment.”
Because blood cancers are more common in adults, there is a larger incentive for new treatments to be developed in that population, and progress for pediatric acute leukemia has fallen behind. For children, including those with aggressive forms of cancer, a delay in research and therapies can threaten their survival. Only 69% of kids with acute myeloid leukemia (AML) will survive more than five years. Even when treatments are effective, more than 70% of childhood cancer survivors have a chronic health condition and 42% have a severe, disabling or life-threatening condition 30 years after diagnosis.
“I lost my 9-year-old son, Zach, to AML following four years of brutal chemotherapy, full body radiation and three bone marrow transplants. Sadly, he died in the ICU on a ventilator from treatment-related toxicity. His little body couldn’t take any more,” said Julie Guillot, LLS ambassador and pediatric outreach chair. “Despite great progress, kids like Zach wait longer than adults to get access to new drugs. This is unacceptable. I’m hopeful that PedAL will enable every child – regardless of situation or location – to get access to cutting-edge personalized and targeted therapies faster. These kids want to live and deserve less toxic, more curative options.”
In the PedAL screening trial (COG Study APAL2020SC), the unique tumor biology of each child’s cancer is identified to help match them with the most promising treatment. Families can then choose to enroll eligible children in a PedAL therapeutic trial or any other clinical trial that best meets their needs.
“As we’re learning that children have a unique disease with unique targets, efforts like PedAL are critical to address the specific needs of our youngest, most vulnerable patients by using new, safer therapies,” said screening trial lead Dr. Todd Cooper, section chief of Pediatric Oncology and director of the Pediatric Leukemia/Lymphoma Program and co-director of the High-Risk Leukemia Program at Seattle Children’s. “The screening trial is significant because it will identify biological targets in a child’s specific leukemia cells and match them to clinical trials using targeted therapies. These tumor biology results will be assessed the same way from children around the globe, helping us advance research into more targeted treatments worldwide.”
Harnessing Partnerships to Accelerate Science
PedAL is the first global master clinical trial for pediatric acute leukemia to be led by a nonprofit featuring a broad, global collaboration with industry leaders and academic institutions.
Through partnerships with the National Cancer Institute (NCI), part of the National Institutes of Health; Children’s Oncology Group (COG); and the European Pediatric Acute Leukemia (EuPAL) Foundation, PedAL trials will be available to children and families worldwide – bringing the dream of safer, more effective pediatric leukemia treatments closer to home for more people.
- Multiple screening trial sites are open in Canada and the United States. The first therapeutic trial is open in the U.S. at Arkansas Children’s Hospital, with more sites to open throughout 2022 and into the next year.
- Led by the University of Chicago’s Pediatric Cancer Data Commons (PCDC), the AML Data Commons will consolidate pediatric clinical trial data from multiple international institutions into a single, unified data set to ensure consistency in data collection, analysis and reporting. This will be the largest collection of data from children with leukemia available for researchers.
- With support from LLS, the PCDC is also developing GEARBOx (Genomic Eligibility Algorithm at Relapse for Better Outcomes), a unique search tool that will help healthcare professionals all over the world quickly match patients who have relapsed or refractory disease to appropriate clinical trials and lifesaving treatments. In addition, all families and their clinicians can also access support from LLS Clinical Trial Nurse Navigators, registered nurses with oncology training who can help families understand and navigate the clinical trial enrollment process.
“PedAL brings together worldwide leaders in pediatric acute leukemia to conceive, develop and implement this groundbreaking global master clinical trial. With our partners, we will create the largest single source of data for children with acute leukemias and provide the opportunity for families to enroll their children in screening and therapeutic sites across the globe,” said E. Anders Kolb, M.D., Nemours Children’s Health, Delaware, Chief of Oncology and Hematology, chair of the COG AML Committee, and co-chair of PedAL. “We want to make PedAL accessible to as many children and their families as possible, which also aligns with the mission at Nemours Children’s Health as we seek to expand access to care and create the healthiest generations of children.”
For more information about PedAL, including trial details and who is eligible to participate, visit: lls.org/dare-to-dream.
About The Dare to Dream Project
Dare to Dream is a five-year initiative by LLS to transform treatment and care for pediatric blood cancer. Because kids are different and need to be treated differently, Dare to Dream will fund groundbreaking research and the LLS PedAL Master Clinical Trial as well as expand LLS support services and drive advocacy efforts to help all kids with blood cancers get accessible, affordable, quality healthcare.
LLS proudly and gratefully acknowledges all those contributing to the LLS mission, including corporate partners Walgreens, Burlington and Wawa, who have each pledged over $1 million to Dare to Dream. LLS also recognizes Sarah Asma and the Moore family; The Don & Lorraine Freeberg Foundation; The Harry T. Mangurian Jr. Foundation; Norcross Foundation, Inc.; Joan and Paul Rubschlager; and The Bobby Zahurak Pediatric AML Research and Patient Support Fund for their outstanding contributions to Dare to Dream.
For more information about Dare to Dream, including resources for patients and their families, trial details, and eligibility, visit: lls.org/dare-to-dream.
As part of The Dare to Dream Project, the Pediatric Acute Leukemia Master Trial (PedAL) is the first-of-its-kind global master clinical trial for pediatric acute leukemia patients that will fundamentally change how children are treated. Prior to enrolling in therapeutic trials, patients enroll in the PedAL screening trial to identify the unique tumor biology of each child’s cancer and help them to match with the most promising treatment. Multiple screening trial sites are currently open in the United States and Canada. At this time, one PedAL therapeutic trial is open in the U.S., with more planned globally. The screening trial will be available in the U.S., Canada, Australia and New Zealand. To learn more about PedAL, visit lls.org/dare-to-dream.
PedAL would not be possible without the support of major foundation donors such as Gateway for Cancer Research, which pledged $1.5 million over three years to support genomic sequencing and flow cytometry, which will be conducted through the PedAL screening trial, and the Lisa Dean Moseley Foundation, which has committed over $1.2 million over five years to support the PedAL Principal Investigators Fellowship Program, whose members will be leading the PedAL therapeutic trials.