Blood Cancer United has announced the launch of Dare to Dream REALworld: AML, a patient-centered initiative co-founded with the Rally Foundation for Childhood Cancer Research to construct a comprehensive real-world evidence base for pediatric acute myeloid leukemia. Announced in Washington, the project is designed to engage current and former pediatric patients alongside their families, capturing electronic health records and clinical experiences to inform ongoing research, enhance future clinical trial designs, and accelerate the development of targeted therapies for children affected by blood cancer.
Developing therapies for rare pediatric conditions presents severe logistical challenges because small patient cohorts restrict the scale and pace of traditional clinical investigations. Gwen Nichols, M.D., chief medical officer of Blood Cancer United, emphasized that time is critical for young patients facing acute myeloid leukemia, noting that research frequently moves more slowly than affected families can afford. Nichols explained that REALworld: AML provides children and families an opportunity to directly contribute to scientific investigation while supplying researchers with the evidence required to design more efficient studies and advance therapeutic options.
The initiative derives its name from Real-world Evidence Accelerating New Treatments, reflecting an operational model that bridges patient communities and clinical investigators. Supported by foundational backing from the Rally Foundation for Childhood Cancer Research, the project incorporates technological infrastructure from Tempus, including its specialized personal health concierge application, Olivia. Dean Crowe, founder and chief executive officer of Rally Foundation, stated that families confronting rare childhood cancers urgently require research frameworks designed around their clinical realities, emphasizing that the initiative establishes a patient-centered model capable of benefiting other rare oncology indications.
Families enrolling in the initiative gain access to the Olivia platform, which aggregates disparate medical records into a centralized digital hub to assist participants in accessing, organizing, and interpreting health data. In addition to record consolidation, participants receive educational resources curated by Blood Cancer United that correspond directly to their child’s specific diagnosis, treatment course, and ongoing support requirements. Chris Scotto DiVetta, senior vice president of Olivia at Tempus, noted that the platform aims to ensure patients experience the full clinical benefit of modern medicine while establishing pathways for families to actively advance pediatric oncology discovery.
A primary objective of REALworld: AML is the creation of a rigorous, standardized dataset that researchers can leverage to analyze pediatric acute myeloid leukemia disease biology and refine future clinical protocols. Over time, investigators intend to utilize the aggregated real-world data to construct external control arms for comparative clinical assessments. When scientifically and regulatorily justified, these external controls permit researchers to compare patients receiving investigational treatments against validated data from historically treated cohorts, thereby allowing more enrolled trial participants to access novel investigational therapies rather than standard regimens.
The deployment of the data registry aligns with ongoing regulatory initiatives by the U.S. Food and Drug Administration, which continues to refine guidance regarding the application of real-world evidence in clinical research and regulatory decision-making. Regulatory acceptance of real-world data carries significant implications for rare childhood malignancies, where conventional randomized controlled trials often face recruitment limitations due to narrow patient populations.
The personal significance of real-world data collection is reflected in the participation of the Breyfogle family, whose twin daughters, Kenedi and Kendal, were both diagnosed with acute myeloid leukemia shortly after birth. Following six months of inpatient chemotherapy, both children entered clinical remission; however, Kendal subsequently suffered a relapse and died in September 2017, while Kenedi remains in remission today. Abby Breyfogle has shared Kenedi’s electronic health records and plans to incorporate Kendal’s medical history into the platform, ensuring that data from both sisters inform pediatric leukemia research.
Currently, the REALworld: AML initiative accepts electronic health record contributions from families of children who are currently living with or in clinical remission from acute myeloid leukemia. Project organizers have also highlighted the clinical necessity of analyzing longitudinal data from children who succumbed to the disease, with plans underway to expand the data platform to include historical records from deceased patients in the near future.
REALworld: AML functions as a core component of Blood Cancer United’s broader Dare to Dream program, which aims to transform pediatric leukemia treatment through laboratory discovery, advocacy, and clinical innovation. The centerpiece of this broader effort is the PedAL master clinical trial, which provides comprehensive genomic screening to characterize the unique biological profiles of pediatric leukemia patients and direct them toward specialized subtrials. To date, PedAL has screened more than 660 children across more than 100 medical centers globally, maintaining treatment arms that evaluate therapies including venetoclax and the menin inhibitor ziftomenib, alongside compassionate use initiatives that preserve drug access for aggressive leukemia subtypes.
The launch reflects an expanding collaboration among established advocacy and philanthropic bodies, supported by contributing partners including the Big Nova Foundation and The Harry T. Mangurian Jr. Foundation. Blood Cancer United, originally founded in 1949 as The Leukemia & Lymphoma Society, represents the largest global nonprofit dedicated to blood cancer patient support and research across more than 100 malignant conditions. Partnering organization Rally Foundation for Childhood Cancer Research contributes two decades of pediatric oncology advocacy, having awarded $47.5 million in research grants and secured $356 million in federal biomedical research allocations through the Congressionally Directed Medical Research Program.
Abby Breyfogle reflected on the urgency driving participation, explaining that living through pediatric acute myeloid leukemia demonstrates how desperately families require better therapeutic options. She noted that knowing researchers can learn from both Kenedi’s ongoing remission and Kendal’s fatal progression offers profound meaning, expressing hope that their family’s experience can assist the next household confronting a childhood cancer diagnosis.
Subject of Research: Pediatric acute myeloid leukemia real-world data registry and research initiative
Article Title: Blood cancer united launches REALworld: AML to accelerate new treatments for children with blood cancer
Article References: Blood cancer united launches REALworld: AML to accelerate new treatments for children with blood cancer. Original publication (Bibliographic details unavailable.)
Image Credits: AI Generated
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Keywords: pediatric acute myeloid leukemia, real-world evidence, Blood Cancer United, Rally Foundation for Childhood Cancer Research, Dare to Dream Project, electronic health records, Olivia health concierge app, Tempus, PedAL master clinical trial, external control arms, genomic screening, venetoclax, ziftomenib, menin inhibitor, Food and Drug Administration guidance, compassionate use program
