First AACR Pediatric Cancer Progress Report Highlights Prominent Progress, Impending Issues in the Field

The American Association for Cancer Research (AACR) has published its innaugural Pediatric Cancer Progress Report, which showcased newsworthy advancements and persisting challenges while urging for action by federal institutions in the field of cancer research for children and adolescents (ages 0-19).1,2

Since the 1970s, U.S. pediatric cancer data have steadily improved, with 5-year survival rates improving from 63% in the mid-1970s to 87% in 2015-2021, in addition to mortality rates decreasing by a cumulative 76% since 1970, according to the report.1,2 These improved data come from a more comprehensive understanding of pediatric cancer that accounts for distinct characteristics like clinically actionable genetic alterations and complex genomic rearrangements in pediatric tumors, along with inherited variants in cancer predisposition genes that occur at rates of nearly 70%, 50%, and 18% respectively.

However, despite improvements in pediatric cancer care and treatments, certain cancer types, such as glioma and sarcoma, have persistently low rates of 5-year survival (25%). Moreover, there will be an estimated 15,000 U.S. diagnoses of pediatric cancer in 2025, and the rarity of pediatric cancer care centers puts certain patient groups such as minorities and rural residents at a disadvantage. In the U.S., patients diagnosed with pediatric cancer who are non-Hispanic black have a 30% higher chance of death from pediatric cancer compared with patients who are non-Hispanic white.

“With this inaugural report, we are highlighting the tremendous progress achieved against pediatric and adolescent cancers and are discussing the remaining challenges,” Elaine R. Mardis, PhD, FAACR, said in a news release.1 “The report elucidates how innovative research, often funded by the National Institutes of Health [NIH], has improved outcomes for many of our youngest cancer patients. We also document that in several lethal cancer types, there remains a significant need for new insights, novel therapies, and international clinical trials to achieve similar progress.”

Mardis is a cochair for the steering committee of the report for the AACR in Philadelphia, Pennsylvania.

What were the additional notable advances found in the report?

The report underscored growing clinical attention to pediatric cancer in the last decade, noting more than 20 molecularly targeted therapies and over 10 immunotherapies that were approved by the FDA for pediatric cancers in addition to the proportion of pediatric cancer drugs approved by the FDA rising from 5.9% to 13.8% from 2012 to 2021.2 The report noted that proliferated FDA approvals in tandem with rising survival statistics are supported by a refined perception of pediatric cancer which is then applied to cutting-edge treatments and technologies.

Integration of tumor and germline sequencing in patients with pediatric cancer indicated that in pediatric tumors, 70% or more have alterations that are clinically actionable, and 18% harbor mutations that are both inherited and predispose patients to cancer. Enhancing knowledge of pediatric cancer and its origin is possible with tools like CRISPR gene editing, according to the report. CRISPR is a gene editing tool that can alter DNA on a granular level with the ability to recreate genetic mutations, giving researchers improved opportunities to see the impacts they have on patients.The report outlined another tool in liquid biopsies, which puts a better understanding of pediatric cancer gained from tools like CRISPR into practice.

Liquid biopsies are minimally invasive cancer screening techniques that utilize cerebrospinal fluid and blood to detect tumor DNA in cells. Liquid biopsies have already improved detection timetables by months in patients with Li-Fraumeni syndrome compared with standard imaging and have shown promise in being applied to other tumor types, according to the report. Moreover, the report touched on other modern cancer screening tools, specifically those that utilize artificial intelligence (AI). In separate studies, AI-powered pediatric cancer screening tools in patients with either retinoblastoma or cancer predisposition syndrome achieved accuracy rates of 97% and 99.5% respectively.

The advances in pediatric cancer research highlighted in the report have helped the number of pediatric cancer survivors rise from 270,000 patients in 1997 to over 521,000 in 2022.

What were the other pressing challenges that efforts should be focused on?

Despite encouraging developments, pediatric cancer still has major areas of improvement that need to be addressed, the report noted. Globally, socioeconomic factors play a crucial role in pediatric cancer, with at least 80% of 400,000 pediatric cancer cases each year happening in low- or middle-income countries. The report noted that this subset of patients also has lower survival rates for cancers like retinoblastoma (57%) due to lower access to resources compared with survival rates for patients in high-income countries with robust, easily accessible resources like the U.S. (98%). Five-year survival rates across all pediatric cancers in low-income countries are no greater than 30% vs the rate of 85% in the U.S. According to the report, building stronger and wider networks of care is an essential step in filling in pediatric cancer gaps between low- and high-income countries.

The report pointed out that with higher amounts of pediatric cancer survivors, other problems arise. Patients who are pediatric cancer survivors experience several challenges throughout their lives, which can be mental, physical, financial, or social. The report added that up to 90% of pediatric cancer survivors undergo almost twice the number of chronic health conditions as the average person by the age of 50. Moreover, a review of pediatric cancer survivors (n = 389,000) in the report showed that pediatric cancer survivors had lower rates of college graduation, marriage, and having children, at rates of 30%, 30%, and 40% respectively.

What are the next steps for pediatric cancer research?

Suggested next steps for pediatric cancer research were laid out by the report, mainly focusing on federal funding for government institutions. The report called for yearly budgets of $51.303 and $7.934 billion for the NIH and National Cancer Institute, respectively. Along with proposed 2026 fiscal year budgets, the report suggested pediatric cancer research focusing on continuing to modernize care for patients through regulatory and policy reform for clinical trials to include patients from low-income countries and harness the potential of available data with AI tools. Investing in an equitable pediatric cancer care future could save 6.2 million lives and yield almost $2 billion, according to the report.

“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,” said Mardis.1 “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.”

References

1. AACR Pediatric Cancer Progress Report 2025 release. December 4, 2025. Accessed December 15, 2025. https://www.aacr.org/about-the-aacr/newsroom/news-releases/aacr-releases-inaugural-pediatric-cancer-progress-report/
2. AACR Pediatric Cancer Progress Report 2025. December 4, 2025. Accessed December 15, 2025. https://cancerprogressreport.aacr.org/wp-content/uploads/sites/2/2025/12/AACR_PCPR_2025.pdf