research about childhood cancer

Fact sheets

• Facts in pictures
• Publications
• Questions and answers
• Tools and toolkits
• Endometriosis
• Excessive heat
• Mental disorders
• Polycystic ovary syndrome
• All countries
• Eastern Mediterranean
• South-East Asia
• Western Pacific
• Data by country
• Country presence 
• Country strengthening 
• Country cooperation strategies 
• News releases
• Feature stories
• Press conferences
• Commentaries
• Photo library
• Afghanistan
• Cholera 
• Coronavirus disease (COVID-19)
• Greater Horn of Africa
• Israel and occupied Palestinian territory
• Disease Outbreak News
• Situation reports
• Weekly Epidemiological Record
• Surveillance
• Health emergency appeal
• International Health Regulations
• Independent Oversight and Advisory Committee
• Classifications
• Data collections
• Global Health Estimates
• Mortality Database
• Sustainable Development Goals
• Health Inequality Monitor
• Global Progress
• Data collection tools
• Global Health Observatory
• Insights and visualizations
• COVID excess deaths
• World Health Statistics
• Partnerships
• Committees and advisory groups
• Collaborating centres
• Technical teams
• Organizational structure
• Initiatives
• General Programme of Work
• WHO Academy
• Investment in WHO
• WHO Foundation
• External audit
• Financial statements
• Internal audit and investigations 
• Programme Budget
• Results reports
• Governing bodies
• World Health Assembly
• Executive Board
• Member States Portal
• Fact sheets /

Childhood cancer

• Each year, an estimated 400 000 children and adolescents of 0-19 years old develop cancer. [1, 2]
• The most common types of childhood cancers include leukemias, brain cancers, lymphomas and solid tumours, such as neuroblastoma and Wilms tumours. [1,2]
• In high-income countries, where comprehensive services are generally accessible, more than 80% of children with cancer are cured. In low- and middle-income countries (LMICs), less than 30% are cured. [2,3]
• Childhood cancer cannot generally be prevented or identified through screening.
• Most childhood cancers can be cured with generic medicines and other forms of treatment, including surgery and radiotherapy. Treatment of childhood cancer can be cost-effective in all income settings. [2]
• Avoidable deaths from childhood cancers in LMICs result from lack of diagnosis, misdiagnosis or delayed diagnosis, obstacles to accessing care, abandonment of treatment, death from toxicity, and relapse. [2,3]
• Only 29% of low-income countries report that cancer medicines are generally available to their populations compared to 96% of high-income countries. [4]
• Childhood cancer data systems are needed to drive continuous improvements in the quality of care, and to inform policy decisions.

The problem

Cancer is a leading cause of death for children and adolescents.The likelihood of surviving a diagnosis of childhood cancer depends on the country in which the child lives: in high-income countries, more than 80% of children with cancer are cured, but in many LMICs less than 30% are cured [2,3].

The reasons for lower survival rates in LMICs include: delay in diagnosis, an inability to obtain an accurate diagnosis, inaccessible therapy, abandonment of treatment, death from toxicity (side effects), and avoidable relapse. Improving access to childhood cancer care, including to essential medicines and technologies, is highly cost effective, feasible and can improve survival in all settings [4].

What causes cancer in children?

Cancer occurs in people of all ages and can affect any part of the body. It begins with genetic change in single cells, that then grow into a mass (or tumour), that invades other parts of the body and causes harm and death if left untreated. Unlike cancer in adults, the vast majority of childhood cancers do not have a known cause. Many studies have sought to identify the causes of childhood cancer, but very few cancers in children are caused by environmental or lifestyle factors. Cancer prevention efforts in children should focus on behaviours that will prevent the child from developing preventable cancer as an adult.

Some chronic infections, such as HIV, Epstein-Barr virus and malaria, are risk factors for childhood cancer. They are particularly relevant in LMICs. Other infections can increase a child’s risk of developing cancer as an adult, so it is important to be vaccinated (against hepatitis B to help prevent liver cancer and against human papillomavirus to help prevent cervical cancer) and to other pursue other methods such as early detection and treatment of chronic infections that can lead to cancer.

Current data suggest that approximately 10% of all children with cancer have a predisposition because of genetic factors [5]. Further research is needed to identify factors impacting cancer development in children.

Improving outcomes of childhood cancer

Because it is generally not possible to prevent cancer in children, the most effective strategy to reduce the burden of cancer in children and improve outcomes is to focus on a prompt, correct diagnosis followed by effective, evidence-based therapy with tailored supportive care.

Early diagnosis

When identified early, cancer is more likely to respond to effective treatment and result in a greater probability of survival, less suffering, and often less expensive and less intensive treatment. Significant improvements can be made in the lives of children with cancer by detecting cancer early and avoiding delays in care. A correct diagnosis is essential to treat children with cancer because each cancer requires a specific treatment regimen that may include surgery, radiotherapy, and chemotherapy.

Early diagnosis consists of 3 components [6]:

• awareness of symptoms by families and primary care providers;
• accurate and timely clinical evaluation, diagnosis, and staging (determining the extent to which a cancer has spread); and
• access to prompt treatment.

Early diagnosis is relevant in all settings and improves survival for many cancers. Programmes to promote early and correct diagnosis have been successfully implemented in countries of all income levels, often through the collaborative efforts of governments, civil society and nongovernmental organizations, with vital roles played by parent groups. Childhood cancer is associated with a range of warning symptoms, such as fever, severe and persistent headaches, bone pain and weight loss, that can be detected by families and by trained primary health-care providers [6].

Screening is generally not helpful for childhood cancers. In some select cases, it can be considered in high-risk populations. For example, some eye cancers in children can be caused by a mutation that is inherited, so if that mutation or disease is identified in the family of a child with retinoblastoma, genetic counselling can be offered and siblings monitored with regular eye examinations early in life. Genetic causes of childhood cancers are relevant in only a small proportion children with cancer. There is no high-quality evidence to support population-based screening programmes in children.

A correct diagnosis is essential to prescribe appropriate therapy for the type and extent of the disease. Standard therapies include chemotherapy, surgery and/or radiotherapy. Children also need special attention to their continued physical and cognitive growth and nutritional status, which requires a dedicated, multi-disciplinary team. Access to effective diagnosis, essential medicines, pathology, blood products, radiation therapy, technology and psychosocial and supportive care are variable and inequitable around the world.

However, cure is possible for more than 80% of children with cancer when childhood cancer services are accessible. Pharmacological treatment, for example, includes inexpensive generic medications included on the WHO List of Essential Medicines for Children. Children who complete treatment require ongoing care to monitor for cancer recurrence and to manage any possible long-term impact of treatment.

Palliative care

Palliative care relieves symptoms caused by cancer and improves the quality of life of patients and their families. Not all children with cancer can be cured, but relief of suffering is possible for everyone. Paediatric palliative care is considered a core component of comprehensive care, starting when the illness is diagnosed and continuing throughout treatment and care, regardless of whether or not a child receives treatment with curative intent.

Palliative care programmes can be delivered through community and home-based care, providing pain relief and psychosocial support to patients and their families. Adequate access to oral morphine and other pain medicines should be provided for the treatment of moderate to severe cancer pain, which affects more than 80% of cancer patients in the terminal phase.

WHO response

In 2018, WHO launched, with the support of St. Jude Children’s Research Hospital, the Global Initiative for Childhood Cancer, to provide leadership and technical assistance to governments to support them in building and sustaining high-quality childhood cancer programmes [4]. The goal is to achieve at least 60% survival for all children with cancer by 2030. This represents an approximate doubling of the current cure rate and will save an additional one million lives over the next decade. 

The CureAll framework and its accompanying technical package have been developed to support implementation of the Initiative. The package helps governments and other stakeholders assess current capacity, set priorities, generate investment cases, develop evidence-based standards of care and monitor progress. An information-sharing portal has been created to facilitate sharing of expertise between countries and partners.  

In December 2021, WHO and St Jude Children’s Research Hospital launched the Global Platform for Access to Childhood Cancer Medicines, the first of its kind, to provide an uninterrupted supply of quality-assured childhood cancer medicines with end-to-end support from selecting to dispensing medicines according to best possible care standards.

WHO and the International Agency for Research on Cancer (IARC) collaborate with the International Atomic Energy Agency (IAEA) and other UN organizations and partners, to:

• increase political commitment for childhood cancer control;
• support governments to develop high-quality cancer centres and regional satellites to ensure early and accurate diagnosis and effective treatment;
• develop standards and tools to guide the planning and implementation of interventions for early diagnosis, treatment and palliative and survivorship care,
• improve access to essential medicines and technologies; and
• support governments to safeguard families of children with cancer from financial harm and social isolation as a result of cancer care.

The Global Initiative for Childhood Cancer is part of the response to the World Health Assembly resolution Cancer Prevention and Control through an Integrated Approach (WHA70.12), focused on the reduction of premature mortality from NCDs and the achievement of universal health coverage.

• Steliarova-Foucher E, Colombet M, Ries LAG, et al. International incidence of childhood cancer, 2001-10: a population-based registry study. Lancet Oncol. 2017;18(6):719-731.
• World Health Organization. (‎2021)‎. CureAll framework: WHO global initiative for childhood cancer: increasing access, advancing quality, saving lives. World Health Organization. https://apps.who.int/iris/handle/10665/347370  
• Lam CG, Howard SC, Bouffet E, Pritchard-Jones K. Science and health for all children with cancer. Science. 2019 Mar 15;363(6432):1182-1186. doi: 10.1126/science.aaw4892. PMID: 30872518.
• World Health Organization. (‎2020)‎. Assessing national capacity for the prevention and control of noncommunicable diseases: report of the 2019 global survey. World Health Organization. https://apps.who.int/iris/handle/10665/331452  
• Zhang J, Walsh MF, Wu G, Edmonson MN, Gruber TA, et al. Germline Mutations in Predisposition Genes in Pediatric Cancer. N Engl J Med. 2015 Dec 10;373(24):2336-2346.
• Pan-American Health Organization. (2014). Early diagnosis of childhood cancer. Pan-American Health Organization. https://iris.paho.org/handle/10665.2/34850  

More on childhood cancer

Knowledge Action Portal for noncommunicable diseases

Children's Cancer Research Fund

• Get Involved Overview
• Ways to Donate
• Honor Someone
• Be A Fundraiser
• Planned Giving
• Partner with Us
• About Us Overview
• What We Fund
• Staff & Board
• Education & Research Overview
• Research Grants
• Research Updates
• Brain & Neural Tumors
• Survivorship
• Other Cancers
• For Families Overview
• Big Dreams Tour
• Camp Norden
• Share Your Story

Childhood Cancer Facts

We’ve gathered answers to your most frequently asked questions about childhood cancer, including statistics, resources and more.

How common is childhood cancer?

About 1 in 285 children will develop cancer before the age of 20.

How many children are diagnosed with cancer each day?

47 children are diagnosed with cancer every day in the U.S.

How many types of childhood cancer are there?

There are more than 12 major types of childhood cancers and over 100 subtypes.

What is the survival rate for childhood cancer?

1 in 7 children diagnosed with cancer in the U.S. will not survive. Survival rates differ for different types of childhood cancers – but overall, childhood cancer remains the most common cause of death by disease among children in the U.S.

What are the most common types of childhood cancer?

The two most common types of childhood cancers are leukemias and brain/central nervous system cancers. 

Is childhood cancer rare?

Technically, yes. A rare disease is defined as one that affects a population smaller than 200,000 – meaning that all childhood cancers are technically considered rare. But we believe no childhood cancer is too rare to deserve research funding. Read our article: Why childhood cancers are considered “too rare” to get research funding.

How much funding goes to childhood cancer research each year?

It’s hard to pinpoint exactly how much funding goes to childhood cancer research, but it pales in comparison to funds available for research into adult cancers. In fact, as a percentage of cancer research funding from the federal government, childhood cancer research funding is in the single digits. Over half of all funds dedicated to childhood cancer research are from donors like you. In short, without your support, vital childhood cancer research would not move forward.

How does childhood cancer affect families?

Childhood cancer impacts the whole family – in fact, one in four families lose more than 40% of their annual household income as a result of childhood cancer treatment-related work disruption. One in three families face other work disruptions such as having to quit work or change jobs.

Siblings of children with cancer are at risk for emotional and behavioral difficulties, such as anxiety, depression and post-traumatic-stress disorder.

Symptoms of post-traumatic stress disorder are well documented for parents whose children have completed cancer treatment.

Is childhood cancer different from adult cancers?

Yes – the two most common causes of cancer in adults are smoking and obesity. In children, the most common cause of cancer is randomly acquired DNA mutation. In short, childhood cancer is a disease of unfortunate random chance.

For many adult cancers, we know what puts adults at higher risk, and we can prevent many cancers by changing behaviors or treat them effectively by screening and catching them early. For kids, prevention may not be possible – some childhood cancers begin developing before the child is even born.

How can I support a family facing childhood cancer?

If you feel compelled to help a family experiencing cancer, know that your thoughtfulness could be a huge stress relief that’s remembered for a lifetime. Here are 5 ways to support a family facing cancer.

Looking for even more? Check out our blog post: “Is there anything I can do?” There is, and here’s how.

What should I write in a card to a child who has cancer?

You don’t want to write the wrong thing, but you want them to know you care… so where to start? Here are 5 things to write in a card to a child or family dealing with childhood cancer.

Looking to go above and beyond a card? Read our blog: Tips for bringing a meal to a cancer family.

What should I not say to a family facing cancer?

A family that has just received a new cancer diagnosis may be feeling very fragile – meaning even the most well-intentioned questions or comments can sting. Read our blog: What to Say (and What Not to Say) to a Family Facing Cancer where cancer parents share what they’ve found helpful, and what is better left unsaid.

What are the side-effects of childhood cancer treatment?

Side-effects of cancer treatment can range widely, and they are dependent on a child’s specific treatments. But some of the most common side-effects of childhood cancer treatment include:

Acute side-effects:

• Mouth, gum and throat sores
• Weight changes
• Increased risk of infection

What are some late-effects of childhood cancer treatment?

Because kids are still growing and developing during cancer treatment, and these treatments are so harsh, many survivors face late-effects years or even decades after treatment is over. Learn more about childhood cancer survivorship here.

Some common late-effects include:

• Mental health issues and memory loss
• Hearing loss
• More cavities
• Heart and other organ damage
• Increased risk of secondary cancers
• Infertility
• Nerve damage, pain and weakness
• Stunted bone growth

Looking for more? Check out our blog: Seven childhood cancer facts to share this childhood cancer awareness month.

What is Childhood Cancer Awareness Month?

Childhood Cancer Awareness Month is held every September to raise awareness about childhood cancer, its impact on children and families, and the need for improved research, treatment, and support. It aims to educate the public, advocate for increased funding and resources, and foster community engagement to ultimately improve the lives of children diagnosed with cancer.

Research on Childhood Cancers

In 2013, Phineas Sandi received CAR T-cell therapy for acute lymphoblastic leukemia while participating in an NCI clinical trial. Phineas, holding the syringe that was used to infuse his CAR T cells, is cancer-free.

Why Research is Critical to Progress against Childhood Cancer

Cancer is the leading cause of death from disease among children in the United States. Although substantial progress has been made in the treatment of several types of childhood cancer over the past five decades, progress against other types has been limited. Even when long-term survival is achieved, many survivors of childhood cancer may experience long-term  adverse effects from the disease or its treatment.

Clearly, more research is needed to develop new, more-effective, and safer treatments for childhood cancer. And infrastructure and practices that allow researchers to learn from every child with cancer need to be put in place.

NCI has a number of programs that address childhood cancers specifically, and many of the institute’s other research programs are applicable to children with cancer even if they aren’t focused specifically on pediatric cancers. The institute supports a broad range of biomedical research that is relevant to this population, including:

• Basic research to enhance our understanding of the fundamental mechanisms of cancer
• Clinical research to test new treatments for safety and effectiveness
• Survivorship research to reduce the long-term adverse effects of cancer and its treatment

Dr. Brigitte Widemann Appointed as the Special Advisor to the NCI Director for Childhood Cancer

Brigitte Widemann, MD, Chief of the NCI Center for Cancer Research Pediatric Oncology Branch and practicing pediatric oncologist, has been appointed as Special Advisor to the NCI Director for Childhood Cancer.

Challenges in Childhood Cancer Research

One challenge in conducting research on childhood cancer is that cancers in children and adolescents are relatively uncommon. Childhood cancers represent less than 1% of all new cases of cancer diagnosed in the United States each year. Because the number of children with cancer is small and patients are treated at many different institutions, answering complex biological questions about childhood cancer requires collaboration.

As clinical trials are increasingly restricted to smaller numbers of patients who are defined by the molecular characteristics of their tumors rather than where the tumors originated in the body, collaboration among children’s cancer centers and a strong national clinical research program are essential to ensure that trials enroll sufficient numbers of participants to produce meaningful results.

In addition, more efficient ways to curate and share research knowledge—from genomic data to clinical outcomes—need to be developed to speed progress against childhood cancers.

NCI’s Rare Cancer Clinics Fostering Collaboration

Clinics bring together clinicians, patients, and advocates.

Another challenge is that, although most cancers in children (and adults) are thought to develop as a result of genetic changes that lead to uncontrolled cell growth and eventually cancer, the causes of most of these genetic changes in children are unknown. A small percentage of cancers in children can be linked to inherited genetic changes or to exposure to diagnostic or therapeutic radiation. But environmental causes have not been identified for most childhood cancers. As a result, identifying opportunities to prevent childhood cancer may be difficult.

In addition, the types of cancers children develop, and the biology of those cancers, generally differ from those of cancers diagnosed in adults. For example, tumors that originate in developing organs and tissues (such as retinoblastomas in the eye and osteosarcomas in bone) are more common in children.

Moreover, most childhood cancers have relatively few genetic alterations , and they often lack the genetic targets for treatments that have been developed and approved for cancers occurring in adults. And drugs that target signaling pathways that are active in some adult cancers might be difficult to use in children, given that many of these signaling pathways are essential for normal development.

Investigating Fusion Proteins in Childhood Cancers

The work of NCI’s FusOnc2 Consortium may one day lead to new treatments for children with cancer.

In fact, the genetic changes that drive childhood cancers are often distinct from those in adult cancers. For example, chromosomal translocations that fuse parts of different genes together to form fusion oncoproteins are common in childhood cancer. Although fusion oncoproteins are also found in some adult cancers, those found in children have proven particularly difficult to target. Another contributing factor to the small number of targeted therapies for childhood cancers is that the rarity of these diseases has been an impediment to commercial drug development.

Developing new treatments that are less toxic and cause fewer adverse effects (both acute and late) than current treatments and developing interventions to mitigate the adverse effects of both current and future treatments are additional challenges in childhood cancer research. The late effects of some childhood cancer therapies can have profound physical, emotional, and other consequences for survivors, including a shortened life expectancy. Finding ways to minimize and address these late effects to improve both the quality and the length of life of survivors is a research priority.

More information about cancer drug metabolism in children, which varies with developmental age, is also needed, as are better laboratory and animal models for screening and testing drugs for potential use in children and adolescents. The optimal use of radiation therapy in treating childhood cancers also needs to be defined so that efficacy is maintained or increased while long-term side effects are reduced.

Basic Research Drives Progress against Childhood Cancer

Virtually all progress against cancer in both children and adults has its origins in basic research, often in areas that are not directly related to the disease.

As an example, the discovery of CRISPR-Cas9 for gene editing has revolutionized the study of genes that control cancer cell growth and survival in both childhood and adult cancers. This discovery came from basic research in microbiology on how bacteria resist infections by viruses.

Another example had its origins in basic research on proteins called histones , which are DNA-binding proteins that provide structural support for chromosomes and help control the activity of genes. Scientists spent years investigating how these proteins are modified in the cell nucleus and the role of histone modifications in controlling when and to what extent genes are expressed.

The findings of this research became immediately relevant to a type of pediatric brain tumor called diffuse intrinsic pontine glioma (DIPG)  when it was discovered that most DIPG tumors have a mutation in the gene for the histone protein H3.3 that prevents a specific modification of the protein. This mutation in H3.3 is thought to be a driver mutation for DIPG and is associated with aggressive disease and shorter survival.

Promising Areas of Research on Childhood Cancers

Although our understanding of the biology underlying cancers that occur in children has increased tremendously in the past decade, there are still critical gaps in our knowledge. NCI has identified several areas in which more research is needed and has identified opportunities to use new approaches to gain additional insights into childhood cancers.

Immunotherapies for Childhood Cancers

Immunotherapy Drug Effective in Children with Relapsed Leukemia

In two studies, blinatumomab improved survival and was less toxic than chemotherapy.

Immunotherapies are treatments that restore or enhance the immune system’s ability to fight cancer. The field of cancer immunotherapy research has produced several new methods for treating cancer.

One example is chimeric antigen receptor (CAR) T-cell therapy , which is now used to treat some children with acute lymphoblastic leukemia . This therapeutic approach arose from decades of research on how the immune system works and how to manipulate it for clinical benefit.

The NCI Center for Cancer Research's Pediatric Oncology Branch (POB) conducts clinical trials of immunotherapy in pediatric and young adult patients, and the Children’s Oncology Group (COG) and the Pediatric Brain Tumor Consortium (PBTC) are evaluating immunotherapy treatments for selected childhood cancers. The Cancer Immunotherapy Trials Network (CITN) has a pediatric component that is developing clinical trials to test immunotherapies for children with cancer.

As part of the Cancer Moonshot, NCI has established the  Fusion Oncoproteins in Childhood Cancers (FusOnC2) Consortium , Pediatric Immunotherapy Discovery and Development Network (PI-DDN) , and Childhood Cancer–Data Integration for Research, Education, Care, and Clinical Trials (CC-DIRECT).

Learn about POB, COG, and the other programs mentioned above in How NCI Programs Are Making a Difference in Childhood Cancer .

Molecularly Targeted Therapies for Childhood Cancers

NCI Research and the National Cancer Plan

The broad variety of research NCI supports on childhood cancers aligns with the goals of the National Cancer Plan. Read about the plan and explore each goal.

Molecularly targeted therapies are drugs or other substances that kill cancer cells by targeting specific molecules that are necessary for cancer cells to grow and survive. These therapies can be small-molecule inhibitors , monoclonal antibodies , or antibody–drug conjugates .

POB conducts clinical trials of targeted therapy in pediatric and young adult patients, and COG and the PBTC are evaluating targeted therapies for selected childhood cancers.

For example, results from an NCI-sponsored clinical trial, conducted by COG and led by Alice Yu, M.D., Ph.D., of the University of California, San Diego, led to the approval of the monoclonal antibody dinutuximab (Unituxin) to treat high-risk neuroblastoma .

Additionally, the PBTC studied the targeted agent selumetinib in children with relapsed or refractory low-grade gliomas . Reductions in tumor size were observed in most patients. Based on these results, COG researchers are studying selumetinib in phase 3 clinical trials for children with newly diagnosed low-grade glioma .

In 2017, NCI and COG launched the NCI–COG Pediatric Molecular Analysis for Therapy Choice (Pediatric MATCH) trial, which is testing molecularly targeted therapies in children with advanced solid tumors that are not responding to treatment. Tumor DNA sequencing is being used to identify those children whose cancers have a genetic abnormality that is targeted by a drug being studied in the trial.

How NCI Programs Are Making a Difference in Childhood Cancer

NCI Fiscal Year 2025 Professional Judgment Budget Proposal

Each year, NCI prepares a professional judgment budget to lead progress against cancer.

NCI recognizes that children are not just small adults and that specialized treatments tailored to childhood cancers are needed. NCI engages with researchers, clinicians, policymakers, advocates, and other partners to address this critical area of research. NCI supports an array of programs specifically to advance childhood cancer care and has renewed these initiatives and programs over numerous funding periods. Some of these programs include:

• The Pediatric Oncology Branch (POB) in NCI’s Center for Cancer Research conducts high-risk, high-impact basic, translational, and clinical research on childhood cancers. For example, POB investigators helped lead a team of international researchers who analyzed data from many patients with rhabdomyosarcoma , a rare childhood cancer that affects the muscles and other soft tissues, and found mutations in several genes that are associated with a more aggressive form of the disease . Genetic clues from the study could lead to more widespread use of tumor genetic testing to predict how children with this cancer will respond to therapy, as well as to the development of targeted treatments for the disease.
• NCI's Division of Cancer Epidemiology and Genetics (DCEG) conducts clinical, genetic, molecular pathology, and epidemiological studies of children at high risk of cancer. For example, DCEG researchers are leading a genome-wide association study of Ewing sarcoma to better understand the genetic architecture of the disease and to identify regions of the genome that may increase risk. DCEG researchers are also studying osteosarcoma to better understand the role that genetic variation plays in risk and patient outcomes and identify genes or genomic regions that may be important in osteosarcoma . The division also studies familial cancer syndromes, including Li-Fraumeni Syndrome , DICER1 syndrome , NF1 , and inherited bone marrow failure syndromes (IBMFS) , to better understand these disorders and investigate possible genotype/phenotype relationships that will improve clinical management and aid in genetic counseling.
• The T herapeutically Applicable Research to Generate Effective Treatments (TARGET) program uses genomic approaches to catalog the molecular changes in several types of childhood cancer to increase our understanding of their pathogenesis, improve their diagnosis and classification, and identify new candidate molecular targets for better treatments. For example, TARGET researchers performed a pan-cancer study of somatic alterations in nearly 1,700 pediatric leukemias and solid tumors and found major genomic differences when compared with adult cancers. The related Cancer Genome Characterization Initiative (CGCI) includes genomic studies of various pediatric cancers that often do not respond well to treatment.

Tailored Radiation for Kids with Medulloblastoma?

Study suggests the volume and dose could be tailored to the genetics of the patient’s tumors.

• The NCI–COG Molecular Analysis for Therapy Choice (Pediatric MATCH) precision medicine trial is a nationwide trial to explore whether targeted therapies can be effective for children and adolescents with solid tumors that harbor specific genetic mutations and have progressed during or after standard therapy. The trial, which is funded by NCI and conducted by COG, opened to patient enrollment in 2017. Germline testing is performed on all enrolled patients to assess whether the genetic aberrations identified in their tumors are inherited. The genomic data captured in the trial will serve as an invaluable resource for researchers seeking to understand the genetic basis of pediatric cancer.
• The Pediatric Brain Tumor Consortium (PBTC) is a multidisciplinary cooperative research organization devoted to identifying better treatment strategies for children with primary brain tumors.
• NCI participates in the Gabriella Miller Kids First Pediatric Research Program , which is building a rich data resource (sponsored by the National Institutes of Health) to increase knowledge about the genetic changes associated with childhood cancers and structural birth defects. The program allows investigators from different research communities to share data and collaborate, and it encourages new ways of thinking about childhood diseases.
• NCI supports several research projects for children, adolescents, and young adults (AYAs) with cancer as authorized by the Childhood Cancer Survivorship, Treatment, Access, Research (STAR) Act . The act enables NCI to enhance biospecimen collection, biobanking, and related resources for childhood and AYA cancers, with an emphasis on those cancer types, subtypes, and their recurrences for which current treatments are least effective.

The CCDI Molecular Characterization Initiative (MCI)

Learn what MCI is and how it’s providing state-of-the-art testing at no cost.

• The Childhood Cancer Survivor Study (CCSS) is examining the long-term adverse effects of cancer and cancer therapy on approximately 35,000 survivors of childhood cancer who were diagnosed between 1970 and 1999. The study was created to gain new knowledge about the long-term effects of cancer and its treatment and to educate survivors and the medical community about the potential impacts of a cancer diagnosis and treatment. The results obtained from CCSS are used to help design treatment protocols and interventions that will improve survival, while minimizing harmful late effects. This research is also used to develop and expand programs for early detection and prevention of late effects in children and adolescent cancer survivors. For example, to better understand the genetic risk of second cancers, DCEG and CCSS researchers are collaborating on studies that aim to identify both common and rare genetic variants that may be associated with second cancers or other late adverse effects among survivors of childhood cancer. In a related study, DCEG scientists also are studying the long-term health of survivors of retinoblastoma , following a cohort of individuals with hereditary or nonhereditary disease to understand how retinoblastoma treatments impact risk for second cancers and long-term mortality.
• The New Approaches to Neuroblastoma Therapy (NANT) Consortium consists of a multidisciplinary team of laboratory and clinical scientists focused on improving outcomes for patients with high-risk neuroblastoma by discovering mechanisms of resistance to therapies, discovering targetable vulnerabilities driving resistance, and translating these insights into clinical trials. NANT works closely with COG to translate their experimental therapy findings into COG phase 3 clinical trials. Their findings on the tumor microenvironment , tumor response to therapy, and the application of cellular therapies to solid tumors have implications beyond neuroblastoma.
• The RACE for Children Act requires that all new adult cancer drugs also be tested in children when the molecular targets are relevant to a childhood cancer. NCI launched the Pediatric Preclinical in Vivo Testing (PIVOT) program to systematically evaluate new agents in genomically characterized models of childhood cancer. The primary goal of the PIVOT program is to develop high-quality preclinical data to help pediatric cancer researchers identify agents that are most likely to show significant anticancer activity when tested in the clinic against selected childhood cancers. NCI also plans to partner with the Food and Drug Administration, academia, and pharmaceutical companies in the Pediatric Preclinical Testing Public-Private Partnership (PPTP3) , which will be established by the Foundation for the National Institutes of Health to accelerate the pace and broaden the scope of pediatric preclinical testing of these agents.
• The Pediatric Genomic Data Inventory (PGDI) is an open-access resource to help researchers access data from genomic sequencing projects for pediatric cancer. The inventory lists ongoing and completed sequencing projects from the United States and other countries, the type of cancer studied, molecular characterization data available, and points of contact for each project.
• The Hyperactive RAS Specialized Programs of Research Excellence (SPOREs) focus on developing better treatments for neurofibromatosis type 1 and related cancers in children, adolescents, and young adults.
• The Fusion Oncoproteins in Childhood Cancers (FusOnC2) Consortium is a multidisciplinary, collaborative network of investigators studying select fusion oncoproteins implicated in childhood cancers that have a high risk of treatment failure and for which there has been little progress in identifying targeted agents.
• The Pediatric Immunotherapy Discovery and Development Network (PI-DDN)  is a collaborative research network identifying and advancing research opportunities for translating immunotherapy concepts for children and adolescents with cancer toward clinical applications. Primary goals of the PI-DDN include the discovery and characterization of immunotherapy targets for childhood and adolescent cancers, the development of new immunotherapy treatment approaches, and an improved understanding of the immunosuppressive tumor microenvironment in order to advance new, more effective immune-based treatment regimens for high-risk pediatric cancers.

Mapping Tumors across Space and Time

A new NCI program will create maps of cancers with unprecedented detail.

• The Pediatric Cancer Immunotherapy Trials Network (CITN) is using the clinical trials infrastructure of the CITN to conduct clinical trials of immunotherapy agents of specific relevance to children and adolescents with cancer. Examples of the types of novel treatments to be investigated by the Pediatric CITN include cellular therapies (e.g., CAR T cells targeting pediatric cancer antigens) and antibody-based therapies, including antibody-drug conjugates, that target surface antigens preferentially expressed on childhood cancers.
• The My Pediatric and Adult Rare Tumor (MyPART) Network  of scientists, patients, family members, advocates, and health care providers is working together to help find new treatments for rare childhood, teen, and young adult solid tumors that have no cures. Working as a team, researchers share data and help design experiments and clinical trials, advocates discuss issues important to patients, and clinicians share their experiences treating rare cancers. MyPART is part of the larger NCI Rare Tumor Patient Engagement Network.
• DCEG researchers collaborate with the International Childhood Cancer Cohort Consortium (I4C) and the Childhood Leukemia International Consortium (CLIC) , collaborations that pool information from cohort studies from around the world to answer questions about childhood cancers. I4C brings together multidisciplinary teams of epidemiologists, basic scientists, and clinicians, to collaborate on investigations into the role of early-life exposures on cancer risk. CLIC includes more than 30 case–control studies and has identified associations between childhood leukemia and environmental risk factors.