Background:
-Laboratory-based investigations have contributed to an improved understanding of the
biology of cancer and to the development of new therapies for pediatric malignancies.
Objectives:
-Systematic Molecular, Genomic, Proteomic, Metabolomic ("Omic") and other profiling for
enrolled subjects where omics refers to mechanistic studies using a set of advanced
technologies to capture comprehensive aspects of biological systems providing insights
into structure, function and interactions to understand these complex processes and
diseases better.
Eligibility:
Pediatric or Adult subjects of any age with one of the following:
- Diagnosis of any tumor or malignancy, pre-malignant disorder or suspected cancer
susceptibility familial syndromes, regardless of age
- Individuals without malignancy undergoing surgery, other treatment such as apheresis
or normal well visit or routine blood draw as part of standard of care.
- Biological relatives of a subject with a tumor or malignancy (pediatric or adult) or
with suspected familial cancer syndrome. Patients enrolled in an approved companion
protocol
- Blood and/or tissue specimens that have been previously collected and are available
for research analysis
- Biospecimens can be collected with minimal additional risk to the subject during
sampling or procedures required for routine patient care.
Design:
- This study will allow for the collection of specimens for a Tissue Repository, and
for designated sample investigations including systematic molecular, genomic and
proteomic (Omic) profiling, and growth factor and cellular profile investigations.
- Testing activities may include:
- DNA, RNA and protein extracted from a section of tumor and normal samples; the
remainder will be stored.
- Germ line DNA and RNA extracted from lymphocytes or other normal uninvolved
tissue
- Germ line DNA will be extracted from lymphocytes or other normal uninvolved
tissue of relatives of the subject.
- Xenografts, explant and cell lines established from tumor, pre-malignant and
normal samples
- Tumor samples and samples for circulating tumor cells sent for the
establishment of Xenografts and single cell suspension of tumor for drug
testing or for monitoring cell free DNA over time
- Omics (Genomics and Proteomic) studies will be performed
- Growth factor and cellular profile investigations of bone marrow-derived cell
populations to include quantification of hematopoietic progenitor cells (HPCs),
endothelial progenitor cells (EPCs), and mesenchymal progenitor cells (MPCs),
levels of matrix metalloprotease 2 and 9 (MMP2) and (MMP9), gene expression,
growth factor and microvesicle analysis and bone marrow analysis of progenitor
cells in blood and tissue.
- Research tests described in active IRB approved protocols.
- Immune profiling and stromal profiling of blood, tumor, and normal tissues
- Immune function studies from blood and normal tumor tissues.
- Utilizing an oversight committee to oversee the receipt and the distribution of
unlinked tissues to other investigators.
- Qualitative methodologies will be used to ascertain knowledge, attitudes, beliefs,
and behaviors in 25-35 parents/caregivers at NIH concerning the anticipated use of
NGS for diagnosing and directing therapy for pediatric cancer and how incidental
findings might be returned.
- Expected accrual of 100-150 patients per year.
