A Message from the AACR

We are witnessing a transformative era in cancer science and medicine. In the United States, overall cancer mortality has been declining consistently since the early 1990s, thanks to decades of sustained federal investment in scientific innovation and collaborations. Significant progress has also been made against pediatric cancers, with 5-year relative survival rate for all pediatric cancers combined now exceeding 85 percent.

The American Association for Cancer Research (AACR) has a longstanding and unwavering commitment to advancing pediatric cancer research. The inaugural AACR Pediatric Cancer Progress Report 2025 represents a historic milestone as the first-of-its-kind report that is dedicated to educating the public, Congress, and the scientific community about the research-driven breakthroughs against pediatric cancers and the barriers to further progress that remain. We trust that this report will catalyze increased federal and private investments in pediatric cancer research, ensuring that children and adolescents benefit from the same advances transforming adult oncology.

Cancer is a devastating diagnosis for anyone, but it is especially tragic when cancer affects a child or an adolescent, as it endangers the prospect of a full life and deeply impacts patients and their families. Pediatric cancers are rare compared to adult cancers and biologically distinct in their cellular origins, genomic drivers, tumor types, and therapeutic vulnerabilities. Although highly effective therapies have been developed for certain pediatric cancers, treatments for many others have not fundamentally changed in more than four decades. Continued reliance on cytotoxic chemotherapy, surgery, and radiotherapy to treat pediatric cancers means that childhood and adolescent cancer survivors often face lifelong challenges, including risks of second primary cancers, chronic health conditions, and profound psychosocial and financial hardships that can shorten life expectancy and diminish quality of life. Many children and adolescents in high-income countries have access to cutting-edge medicine, but most pediatric patients in low-income and lower middle-income countries lack even the basic diagnostic and therapeutic resources. This report emphasizes the need for strengthening cross-sector collaborations, both nationally and internationally, that are proving to be the most effective approach in accelerating the pace of progress against pediatric cancers and addressing global inequities in pediatric cancer care.

Encouragingly, groundbreaking scientific and clinical advances are beginning to reshape the landscape of pediatric oncology. Comprehensive molecular profiling is enabling precise diagnoses, guiding risk-adapted therapy, and revealing inherited susceptibilities that can inform lifelong surveillance and care. In some high-income countries, such as Germany and Australia, the success of national molecular profiling programs has led governments of these countries to cover the cost of these tests for all newly diagnosed patients. Large-scale studies have revealed that 10–18 percent of pediatric patients develop cancer due to inherited genetic predisposition, underscoring the importance of early detection, surveillance, and genetic counseling. In parallel, cutting-edge technologies—such as liquid biopsies, functional genomics, and patient-derived model systems—are expanding our understanding of disease mechanisms and accelerating the development of safer and more effective therapies.

As highlighted in this report, several new therapies for pediatric cancers have been approved recently by the US Food and Drug Administration, leading to improved outcomes for certain cancer types. Many of these new treatments belong to the latest pillars of cancer medicine—molecularly targeted therapy and immunotherapy. Notable examples include the CAR T-cell therapy tisagenlecleucel and the bispecific T-cell engager blinatumomab for acute lymphoblastic leukemia, the monoclonal antibody dinutuximab for high-risk neuroblastoma, and the first menin-targeted therapy revumenib for leukemia carrying certain biomarkers. These therapeutics are offering hope to pediatric patients with cancer—some of whom are featured in this report—and helping save and extend lives, thus exemplifying the enormous return on investment from federal support for medical research. Therefore, it is concerning that pediatric cancer research currently represents only less than 5 percent of the National Cancer Institute’s annual budget. A significant increase in federal investment is urgently needed to fuel new discoveries, develop effective drugs, improve survivorship care, and reduce the lifelong health and economic burden experienced by pediatric cancer survivors.

Despite major advances, many pediatric cancer patients, particularly those diagnosed with osteosarcoma, metastatic Ewing sarcoma, alveolar rhabdomyosarcoma, high-grade glioma, or acute myeloid leukemias, have experienced minimal to no improvements in treatment or outcomes. Progress against these rarer forms of pediatric cancer is constrained by the scarcity of experimental models and the lack of incentives to develop drugs for small patient populations. Eliminating these barriers and addressing the unmet needs in treating rarer forms of pediatric cancer require partnerships among government agencies, academic institutes, biopharmaceutical companies, and professional organizations. These partnerships are also vital to increasing investments in basic and translational research, developing innovative model systems, implementing novel clinical trial designs, and accelerating drug development for rare and ultrarare pediatric cancers.

For many years, AACR has championed efforts to advance pediatric cancer research. Our organization has funded pediatric cancer research since 1999, driving innovations in diagnostics and therapeutics and fostering scientific careers. In 2011, AACR established the AACR Pediatric Cancer Working Group, which has become a focal point for the scientific community to identify research and policy priorities. The AACR Special Conferences on Pediatric Cancer Research, now in their third iteration, have emerged as a premier platform to disseminate the latest advances in the field. The AACR Childhood Cancer Predisposition Workshops, the most recent of which was held in 2023, have spearheaded the development of new evidence-based standards of clinical care for children and adolescents with cancer predisposition syndromes. Recognizing the importance of collaborative data-sharing to accelerating progress, the global pediatric cancer community is increasingly utilizing federated databases that allow analyses across institutions—an approach that can expand drug discovery and development. In this regard, the AACR Project GENIE®, whose consortium members include major children’s hospitals and cancer centers, houses clinico-genomic data from nearly 10,000 pediatric patients and growing; these datasets are publicly available to researchers globally to accelerate the pace of discovery and precision oncology.

AACR is deeply committed to working with academic institutes, biopharmaceutical partners, policymakers, patient advocates, and all other stakeholders in the medical research community to catalyze the next generation of breakthroughs in pediatric cancer prevention, diagnosis, treatment, and survivorship. By increasing public and private partnerships and investments, expanding global collaborations, and ensuring equitable access to clinical trials and innovative therapies, we can transform the future for children and adolescents with cancer. The inaugural AACR Pediatric Cancer Progress Report 2025 stands as both a celebration of scientific progress and an urgent call to action. With bold vision, unwavering dedication, and sustained support, together we can turn today’s challenges into tomorrow’s cures and bring new hope to children and adolescents affected by cancer.

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