Neuroblastoma, Pediatric Cancer, Sarcoma
Conditions
Brief summary
To explore metabolic phenotypes of children with extra-cranial solid tumors and compare these with their histopathological and genetic alterations to discover potential novel biomarkers and therapeutic targets to improve outcomes in children with high risk disease.
Detailed description
The principal objective of this study is the metabolic characterization of pediatric solid tumors, with a particular focus on neuroblastoma (NBL) and fusion positive sarcoma (FPS), which will allow the detection of tumor specific metabolic alterations that can be exploited with the aim of developing novel therapeutic strategies and biomarkers. Cellular metabolism studies provide insight, in a complementary way to genomics, into processes acting downstream from oncogenes and oncogenic fusion proteins, and such insight may point toward previously unrecognized therapeutic targets or onco-metabolites that are traceable as robust biomarkers for response. The investigator's new approach to use an in-vivo comprehensive analysis of metabolic reprograming in FPS/NBL has never been performed in childhood FPS/NBL and will complement genomics studies for these cancers. For this study, the investigators plan to obtain tumor samples at time of surgical biopsy/resection and study their metabolic signatures.
Interventions
DRUG13C-glucose
Includes standard pre-operation nursing care, 13C-glucose infusion and finger stick/IV glucose checks and storage of blood sample approximately every 30 minutes
OTHERNo glucose infusion
Only includes standard pre-operation nursing care
Sponsors
University of Texas Southwestern Medical Center
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
No minimum to 26 Years
Healthy volunteers
Yes
Inclusion criteria
* Suspected malignancy * Age ≤ 26 years and being cared for at Children's Medical Center * Ability to undergo standard of care diagnostic procedure, including biopsy or resection of the tumor, in the OR or IR at CMC
Exclusion criteria
* Poorly controlled diabetes * Any other medical condition that prevents administration of glucose
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Metabolic phenotypes | 2 years | Upon collection of tumor samples, they will be processed and analyzed with mass spectrometry to learn how the tumor processes the labeled glucose by assessing enrichment of metabolites to identify the active metabolic pathways in each tumor (metabolic phenotype) |
| Compare the metabolic phenotype with the result of histopathological diagnosis and genetic alterations of the specific tumor | 2 years | We will collect data and information from the patient's medical record including pathologic diagnosis and genetic testing results throughout their treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Metabolic evolution of tumors over time | 2 years | Compare tumor metabolism at different points in therapy (diagnosis, metastasis, recurrence) if the family consents to further studies as their child's condition progresses. Will compare high risk samples to low risk samples within a diagnosis (IE: high risk neuroblastoma vs low risk neuroblastoma) |
| Metabolic change due to chemotherapy | 2 years | Compare tumor metabolism at different points in therapy (before vs after chemotherapy is given) if the family consents to further studies as their child's condition progresses. For example, tumor sample at time of neuroblastoma biopsy to resection a few months later prior to bone marrow transplantation. |
| Metabolic phenotypes and outcomes | 2 years | Assess for correlations between metabolism and patient outcome if applicable |
Countries
United States
Outcome results
None listed