“Decades of research, collaboration, advocacy, and legislative and policy actions have transformed the outlook for cancers affecting children and adolescents. According to the AACR Pediatric Cancer Progress Report 2025:
- In the United States, the five-year survival rate for all pediatric cancers combined has increased from 63% in the mid-1970s to 87% in 2015-2021.
- Pediatric cancer mortality in the United States declined by 57% between 1970 and 2000, and by a further 19% from 2001 to 2023, reflecting advances in risk-stratified therapy, precision medicine, and supportive care.
- Over the past few decades, research has uncovered the unique biological underpinnings of pediatric cancers, which are rare compared with adult cancers and are biologically distinct in their cellular origins, genomic drivers, tumor types, and therapeutic vulnerabilities.
- Between 2015 and 2025, the U.S. Food and Drug Administration (FDA) approved more than 20 molecularly targeted therapies and more than 10 immunotherapies for pediatric cancers. Indeed, the proportion of drugs approved specifically for pediatric use rose from 5.9% between 2012-2016 to 13.8% between 2017-2021. While these numbers are significantly lower than what has been approved for adult cancers, the new treatments have been transformative for certain patients.
- Advances in precision medicine not only have led to new anticancer therapeutics but also have allowed clinicians to tailor treatment intensity based on the molecular profile of a child’s or adolescent’s cancer, reducing therapy intensity for pediatric patients with a favorable disease profile and increasing it for pediatric patients with a higher risk of recurrence.
- Technologies such as gene editing, liquid biopsy, and artificial intelligence are deepening our understanding of pediatric cancers and paving the way for additional breakthroughs.”
