Advancing Childhood and Adolescent & Young Adult (AYA) Cancer Research

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Topic Description

Post Date: April 15, 2026

Expiration Date: April 15, 2027

Purpose

The National Institutes of Health (NIH) aims to accelerate progress in preventing, diagnosing, and treating pediatric and AYA patients diagnosed with cancer by supporting innovation and discovery across the full continuum of cancer research and care. This continuum spans molecular characterization, mechanisms of oncogenesis and cancer progression, genetic epidemiology, functional genomics, therapeutic development, preclinical testing, clinical trials, population-based surveillance, and survivorship studies.

research is sought on the use and development of artificial intelligence (AI) and other machine learning (ML) approaches to analyze and integrate existing and new pediatric cancer data, address small sample sizes, and enable privacy-preserving data sharing. It also encourages the creation of tools that make these data AI-ready and accessible, with the goal of accelerating discovery and improving outcomes for children and AYAs with cancer.

Background

Pediatric, adolescent, and young adult (AYA) cancers present unique challenges due to small patient numbers, diverse and often rare disease subtypes, and biologic features that differ from adult cancers. Current standard-of-care therapy for these cancers can result in acute and long-term toxicities (e.g., secondary malignancies, chronic health conditions) that affect survival and quality of life. Lack of robust preclinical models, limited availability of well-annotated biospecimens, and data silos all constrain the pace of translational research. Disparities in access to care, molecular testing, and clinical trials further compound these challenges, particularly for geographically remote populations. More research is needed to better understand the molecular mechanisms driving these cancers and related adverse outcomes and to ultimately inform prevention, early diagnosis, as well as novel targeted approaches for risk assessments to reduce mortality and morbidity. 

As the volume and complexity of pediatric and AYA cancer data continue to grow, there is potential to use advanced computational methods to facilitate pediatric cancer research and optimize care. Examples of these data types include multi-omics data, epidemiologic/risk factor data, imaging, pathology, electronic health records, clinical trial data, real-world evidence, and long-term follow-up and survivorship cohort studies. Computational tools, including artificial intelligence (AI) and machine learning (ML), can rapidly integrate diverse data streams, reveal patterns, and generate testable hypotheses about disease biology, treatment response, toxicity, and late effects.

Participating ICOs

National Cancer Institute (NCI)

Research areas include but are not limited to: 

  • AI/ML approaches where applicable (data harmonization, integration, and incorporation of real-world data sources).
  • Mechanisms of tumor initiation, progression, treatment response, relapse, and resistance in fusion-driven cancers and other molecular subtypes.
  • Therapeutic targets using functional genomics and other synthetic lethality approaches.
  • Immune responses, tumor microenvironment modulation, and development of rational therapeutic combinations.
  • Late effects and identification of individuals at risk based on treatment exposures and genetic predispositions.
  • Preventive interventions to reduce impact of cancer treatment on survival and quality of life.
  • Clinical trial access and increased accrual through novel trial designs (e.g., decentralized trials) and data-driven insights.
ICO Scientific Contact:
Subhashini Jagu
[email protected]

National Institute of Dental and Craniofacial Research (NIDCR)

NIDCR encourages childhood and AYA research related to dental, oral, and craniofacial (DOC) cancers and DOC complications from cancer treatments, all sites, including:

  • Strategies to prevent, detect, treat, and monitor cancers affecting DOC structures, including jaw bones (e.g., osteosarcoma), muscles (e.g., rhabdomyosarcoma), teeth (e.g., ameloblastoma), oral cavity (e.g., Fanconi anemia-associated squamous cell carcinoma), and salivary glands
  • Secondary analyses of existing cancer-related DOC data and samples
  • Approaches to prevent, detect, treat, and monitor treatment-related DOC complications, including mucositis, xerostomia, craniofacial growth disruption, dental abnormalities, and osteonecrosis
  • Long-term DOC function preservation studies in cancer survivors
  • Dental-medical integration to optimize DOC outcomes and oral health related quality of life
  • Patient-centered approaches to improve dental care access and continuity during cancer treatment and follow-up
IC may give special consideration to support meritorious applications in this topic area.
ICO Scientific Contact:
Lorena Baccaglini, DDS, MS, PhD
[email protected]

Zhong Chen, MD, PhD
[email protected]

Office of Disease Prevention (ODP)

For this topic, ODP is particularly interested in projects that test interventions and approaches to improve health and well-being and prevent co-morbidities and adverse health outcomes among children, adolescents, and young adults with cancer.

This office does not award grants. Applications must be relevant to the objectives of at least one of the participating Institutes or Centers listed in this topic.
ICO Scientific Contact:
Elizabeth L. Neilson, PhD, MPH, MSN
[email protected]


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