Research Questions

Research Questions


The initiative will prioritize scientific and clinical research data needs for addressing the burden of cancer in children, adolescents, and young adults. We must determine which research questions should be answered.


Answer one or more of the following questions in your response:

  • What research questions can be addressed through improved childhood cancer data sharing?
    • What common data would be needed to answer those questions?
(@snydercharlie)

Research Questions

Li–Fraumeni syndrome

Gain a better understanding of how p53 tumor suppression could potentially be restored in those with Li–Fraumeni syndrome using gene editing techniques or other mechanisms.

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(@ericzhou)

Research Questions

Sleep health in children with cancer

A considerable number of childhood cancer patients and survivors experience disrupted sleep, with known consequences to their health and well-being. Unfortunately, there has been a history of limited funding and research to understand and intervene upon the varied sleep issues experienced by this population. Rate limiting steps for researchers have been due to the relatively few childhood cancer diagnoses (relative to... more »

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(@dmitriy.sonkin)

Research Questions

Hereditary cancer syndromes, genetics of cancer progression and early tumor detection

There are number of hereditary cancer syndromes which may result in neoplasms in children and/or adolescents, for example: Li-Fraumeni syndrome, PTEN hamartoma tumor syndrome, Fanconi anemia, etc. Number of cancer screening/surveillance protocols have been established in order to decrease morbidity and mortality for individuals with pathogenic germline alterations known to cause hereditary cancer syndromes. For example,... more »

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(@jennifer.guida)

Research Questions

Assess aging trajectories in childhood and AYA cancer survivors

Emerging evidence suggests that childhood and AYA cancer survivors experience age-related conditions decades earlier in life due to cancer and its treatments. Having markers of biological age over time would allow researchers to assess aging trajectories in cancer survivors and determine whether cancer and its treatments cause an acceleration or accentuation of the aging process. Risk factor data would allow researchers... more »

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(@susanmcgovern)

Research Questions

Image-guided evaluation of the late effects of therapeutic childhood radiation

Parents of children who receive radiation for cancer treatment want to know about the long term effects of radiation on growth and development; robust data to answer these important questions are lacking. To address these questions, serial standard of care imaging could be collected from children undergoing radiation therapy at baseline and in followup. Using RTSTRUCT data in DICOM format, radiation dose to tumor volumes... more »

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(@herzog)

Research Questions

Optimize cancer outcomes with chronotherapy

Cancer treatment outcomes depend on the hospital environment. Research should evaluate the effects of ambient lighting (color, intensity, timing), and the best time of day for chemotherapy and feeding and other interventions which can disrupt a patient's normal circadian rhythm. This initiative was chosen as representative of the recommendations from members of the Society for Research on Biological Rhythms (SRBR).

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(@glassfish)

Research Questions

Addition of two data sets, 1. Zebrafish based PDX. 2. QOL data with FACT-G or EQ-5D-Y.

Dear committee, I would like to write a new plan for two date sets for cancer therapy of youth. One set of data is based on the pharmacological data set coming from the patient's cells embedded embryo of Zebrafish. Such PDX (Patient-Derived Xenograft Model; PDX) is now getting realistic and offer more precise medicine of cancer therapy. Because of the mechanism of Zebrafish based PDX, each embryo of Zebrafish needs... more »

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(@jaborandi)

Research Questions

Relapse Prediction

Dear Committee; - i would like to introduce you to my idea " relapse prediction using machine learning algorithms " - my topic is about turning the initial data collected from the patients when introduced for first time before starting the treatment into insights using data science. it depends on each disease type and according to the treatment protocol. - by using an Electronic Data Capture (EDC) system , we can turn... more »

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Research Questions

Understanding the biology of pediatric hematologic cancer development

Understanding the biology of pediatric hematologic cancer development through: • Insights from epigenetics: In all hematologic malignancies, including acute and chronic leukemias, and lymphomas, there are both inherited and somatic genetic alterations that contribute to predisposition, transformation, and disease progression. While genomics plays a significant role in the onset and progression of such malignancies,... more »

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Research Questions

Refining risk stratification protocols and enhancing risk prediction in the pediatric population

Refining risk stratification protocols and enhancing risk prediction in the pediatric population to identify children at risk of developing hematologic cancers: While the treatment outcome of children with acute leukemias (e.g., acute myeloid leukemia and ALL) has improved over the years, there remains a population of these patients who are unable to respond favorably to available therapies. Predicting poor response remains... more »

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Research Questions

Prioritizing novel therapeutic agents identified in pre-clinical research for testing in the clinical setting

Predictive disease models of pediatric hematologic cancers can inform efficient testing of possible novel therapeutic agents in the clinical setting. However, to ensure that these models are useful in prioritizing novel therapeutic targets, pre-clinical assessments must be conducted. Conducting such analysis efficiently and consistently will require investigators to share pre-clinical data generated from models (e.g.,... more »

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(@metayer)

Research Questions

Etiologic research and prevention

Incidence of several cancers continues to increase in children and adolescents. Individual studies and international collaborations have made progresses in understanding risk factors of certain childhood cancers, yet gaps remain. The CCDI should also invest in supporting data sharing for etiologic research (population-based and mechanistic studies).

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(@seis)

Research Questions

Understanding genomic switches of tumor progression in familial adenomatous polyposis

Familial adenomatous polyposis (FAP) is an autosomal dominant cancer predisposition syndrome. It is caused by germline mutations in the APC gene. Individuals affected with FAP are at increased risk of developing not only colorectal cancer but also other cancers, such as thyroid cancer, pancreatic cancer, hepatoblastoma, medulloblastoma, and desmoid tumors. Children, adolescents, and young adults with a family history... more »

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