Children and adolescents with cancer are living longer than in previous decades, but progress has been uneven, according to the first American Association for Cancer Research (AACR) Pediatric Cancer Progress Report, released at a congressional briefing in Washington, DC.

The 192-page report describes rapid growth in molecular diagnostics, targeted therapies, and cellular immunotherapies that are transforming outcomes for many patients. 

“Thanks to decades of federally supported research, the trajectory of pediatric cancer has changed in profound and measurable ways,” said AACR CEO Margaret Foti, MD, PhD, during the event. “The 5-year relative survival rate for all pediatric cancers now exceeds 85%.” 

Will Parsons, MD, PhD, interim director of the Texas Children’s Cancer and Hematology Center, Houston, gave a similar take on the report, saying it underscores both how far pediatric oncology has come and how dependent that progress has been on coordinated scientific and clinical efforts.

“The progress in pediatric cancer outcomes is one of the amazing accomplishments in the last decades of medical research,” he said, in an interview with Medscape Medical News. “It’s an example of a remarkable collaboration…across institutions, with the FDA, with the National Cancer Institute (NCI), with pharmaceutical companies.”

The report also describes where progress in treating pediatric cancer has been slower, stressing that survival for several childhood cancers, particularly aggressive brain tumors and certain rare solid tumors, has changed little in more than four decades, while access to care varies dramatically around the world.

Parsons cited sarcomas and brain tumors as “particularly difficult to treat” pediatric cancers. He also noted that long-term toxicities continue to affect survivors. 

“Even though we have effective treatments for many cancers, we do not have effective, highly safe and highly nontoxic treatments for most cancers,” he said.

To reduce the worst outcomes highlighted in the report, he pointed to two priorities: finding novel effective therapies for high-risk and refractory cancers and making both new and established treatments accessible and practical to deliver.

Another remaining challenge, the report states, is the dramatically varied access to care around the world.

“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,” wrote steering committee co-chair Elaine R. Mardis, PhD, of Nationwide Children’s Hospital, Columbus, Ohio, and colleagues. “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.”

The new document also underscores that childhood cancers differ biologically from adult cancers and often arise from gene fusions, epigenetic alterations, or developmental pathways rather than the higher mutational burdens typical of adult cancers. 

“It is simply not enough to study adult cancer,” co-chair Kimberly Stegmaier, MD, of Dana-Farber Cancer Institute, Boston, said during the briefing.

Genomic Profiling Moves Into Routine Care

Parsons completed his pediatric oncology training in 2007 and noted that “many of the accomplishments referenced in this report would have been thought of as virtually science fiction at that time.” 

He emphasized the importance of genomic profiling — which has been one of the leading advances in managing pediatric cancer and is increasingly reshaping routine care, according to the report — and chimeric antigen receptor (CAR) T-cell therapy.

Through the NCI’s Molecular Characterization Initiative for Childhood Cancers, launched in 2022, more than 6500 children and adolescents with cancer have undergone comprehensive molecular testing. The information obtained from this testing inform both diagnosis and risk assessment, often matching patients to emerging therapies and clinical trials, according to the report.

“Many studies have demonstrated that accurate diagnosis of pediatric cancers can be ascertained from genomic profiling tests, and this accuracy in diagnosis has been shown to improve disease outcomes,” Mardis said at the briefing.

Beyond the molecularly targeted therapies enabled by genomic profiling, CAR T-cell therapy now offers a curative option for some children with relapsed or refractory B-cell leukemia, and trials are now exploring CAR-T approaches for central nervous system (CNS) tumors, which remain far more challenging to treat than hematologic cancers, according to the report.

Five-year survival rates for diffuse midline glioma, for example, is roughly 4%, while 5-year survival for some high-grade gliomas remains near 30%. In stark contrast, 5-year survival for leukemia and Hodgkin lymphoma are approximately 87% and nearly 100%, respectively.

The most challenging cancers lack clearly actionable targets, are harder to biopsy and monitor over time, and arise in anatomic sanctuary sites such as the CNS where drug delivery is more difficult, the report notes. The tumor microenvironment in solid tumors and brain cancers can also impede immune infiltration and drug penetration, blunting the impact of therapies that work better in circulating hematologic disease.

For clinicians, the report notes, these data reinforce the importance of timely referral of children with suspected or confirmed cancer to specialized pediatric oncology centers where genomic profiling, access to CAR T-cell therapy, and enrollment in clinical trials are more likely to be available.

Survivorship and Late Effects

According to the report, more than half a million pediatric cancer survivors are living in the United States, a figure projected to increase in the coming decades. Many of these individuals face lasting effects of chemotherapy and radiation, including cardiopulmonary disease, endocrine dysfunction, neurocognitive impairment, fertility issues, and secondary malignancies.

Because treatment occurs during key developmental periods, these late effects can be especially profound and may emerge years after therapy ends. The report calls for more research on risk-adapted, less-toxic regimens and for stronger survivorship care models that bridge pediatric and adult health systems.

The AACR also recommends that primary care clinicians caring for adult survivors systematically review prior treatment exposures, screen for late effects according to established guidelines, and coordinate ongoing follow-up with survivorship clinics when available.

Disparities in Incidence and Outcomes

The report also describes structural and economic barriers that continue to limit progress and drive worse outcomes in specific groups. 

Childhood cancers are rare, and individual tumor types may be diagnosed in only a few hundred children worldwide each year. Small patient populations and limited preclinical models make drug development difficult and expensive, which can discourage investment in diseases that predominantly affect children.

Disparities add another layer of complexity. 

In the United States, Hispanic children have the highest pediatric cancer incidence rates, while non-Hispanic Black children experience the lowest survival and a higher likelihood of dying of selected pediatric cancers compared with non-Hispanic White children.

Similarly, children in rural or economically disadvantaged areas face delayed diagnosis and reduced access to specialized testing and treatment, including molecular profiling and clinical trials. 

Internationally, survival for otherwise curable childhood cancers remains below 40% in some low- and lower middle-income countries, where basic diagnostic services, reliable chemotherapy supply, and supportive care are not always available.

Closing these gaps, the report concludes, will require both new therapies and better systems to deliver existing care equitably.

For practicing clinicians, the report emphasizes attention to timely referral, enrollment of eligible patients in clinical trials, and awareness of financial and geographic barriers that may prevent families from accessing specialized care.

Data, AI, and the Next Phase of Progress

The report points to AI and large-scale data integration as potential accelerators of progress in managing pediatric cancer. 

National efforts such as the Childhood Cancer Data Initiative are assembling genomics, imaging, and longitudinal clinical data from across institutions. AI-assisted analysis of these datasets could help identify new targets, refine risk prediction, and clarify why some patients respond to therapy while others do not.

Realizing that potential, the report notes, will depend on sustained support, robust data sharing, and careful integration of AI tools into research and clinical workflows. The report also stresses that these tools must be developed and validated in diverse populations to avoid reinforcing existing disparities.

Eventually, such tools could support risk-adapted treatment decisions, automate aspects of imaging and pathology review, and help match patients to appropriate clinical trials, although the report cautions that these applications remain largely investigational.

The report was funded by AACR. Parsons and the steering committee declared no conflicts of interest.