The 2026 Childhood Cancer Report
From diagnosis to research: measuring momentum in the search for cures
Childhood Cancer Research Milestones
Just two decades ago, pediatric cancer reached a major milestone: the first ever FDA-approval of a drug specifically to treat cancer in children.49 The chemotherapy drug, clofarabine, was given accelerated approval for the treatment of some types of relapsed pediatric leukemia. This “first in kids” approval was a rarity; every single other pediatric cancer drug was either first or simultaneously approved for the treatment of adult cancers.
Since 1950, just over 60 drugs have been approved by the FDA for children with cancer. That’s the same number the FDA typically approves for adults in a single year.50
Despite limited approvals, scientific discoveries in childhood cancer have continuously led the way for all types of cancer research. In the 1940s, Sidney Farber, the father of modern chemotherapy, showed how folic acid antagonist drugs could be used to achieve remission in kids with leukemia. These drugs, which later came to include the foundational chemotherapy methotrexate, are a type of cellular poison that work to block the fuel that cancer cells need to divide and replicate. Later, in the 1950s, this research would lead to the successful treatment of Wilms tumor, a rare pediatric variant of kidney cancer.51
Progress in leukemia treatment continues to be the standard to which all cancer treatments aspire to. In 2016, 5-year survival rates for pediatric leukemia as a whole reached 89%52—a substantial improvement from the survival rates of less than 10% when Sidney Farber made his early discoveries.* The past several decades have shown what is possible for kids, but the progress has not been uniform across childhood cancer types nor has it been whole or complete within types.
This section details research and progress milestones by childhood cancer type, offering insights on the characteristics of these unique diseases. Included are the current standard treatments—the frontline treatments that children typically receive, but that will vary by patient age, cancer stage, and doctor recommendations. It also provides some historical context to the search for cures (see Section 4 for more information). Also included are comprehensive reviews of the progress in the treatment of a handful of childhood cancer types (leukemia, lymphoma, brain tumors, spinal cord tumors, retinoblastoma, neuroblastoma, and sarcomas). This list is not exhaustive—there are several more types including hepatoblastoma (liver cancer); Wilms tumor (mentioned above); pediatric cases of ovarian, testicular, pancreatic, and skin cancers; and rare tumors that have been diagnosed and documented in literature only a handful of times.
In addition, this section contains a focus on childhood cancer predisposition factors and syndromes. Scientists estimate that between 10-18% of all cancers are driven by predisposition.53 While predisposition itself is not a specific cancer type, it is a critical area of research interest. RB1, the gene that drives some types of the childhood cancer retinoblastoma, was discovered in 1985, opening the door to the study of predisposition in both adult and childhood cancers.
Taken together, these milestones show both the remarkable progress and the persistent gaps in childhood cancer research. Understanding the history of these advances provides a critical foundation for charting the path forward.
* This includes all types of leukemia diagnosed in children ages 0-19. Some leukemia types still have poor survival rates for children.