The purpose of this study is to prospectively collect specimens from pediatric patients with diffuse intrinsic pontine glioma or brainstem glioma, either during therapy or at autopsy, in order to characterize the molecular abnormalities of this tumor.
High grade diffuse intrinsic pontine glioma (DIPG) accounts for approximately 80% of pediatric brainstem tumors and 10% of pediatric brain tumors, and is the most lethal form of brainstem gliomas in children. There is currently no effective therapy to treat these tumors. We hypothesize that this tumor exhibits unique molecular abnormalities leading to altered RNA and protein expression. The aim of this trial is to collect specimens from pediatric patients with diffuse intrinsic pontine glioma including serum, cerebrospinal fluid, urine, brain tumor and other constitutional tissue, during therapy and/or at autopsy. Our goal is to study this tissue to characterize the genetic abnormalities that lead to tumor formation in order to identify key molecules as biomarkers which we can target to design and test new and more effective treatments.
Study Type
OBSERVATIONAL
Enrollment
100
Children's National Medical Center
Washington D.C., District of Columbia, United States
RECRUITINGGenome-wide expression patterns of RNA in tumor samples, normal brainstem tissue and cerebrospinal fluid using Affymetrix gene expression profiling
Collected tumor and normal samples will potentially be used for RNA genome-wide expression pattern profiling.
Time frame: 5 years
Validation of results of the genome-wide analysis
The molecular analysis done on collected samples will be validated through whole genome sequencing.
Time frame: 5 years
Proteomic profiling of tumor, normal brainstem tissue and cerebrospinal fluid
To obtain full characterization of collected samples, proteomic profiling will be done on tumor and normal samples collected.
Time frame: 5 years
Protein expression patterns as assessed by immunohistochemistry and western blot compared to normal brainstem tissue
Collected tumor and normal samples will have the immunochemistry and western blot compared to assess protein expression variation.
Time frame: 5 years
Genome-wide analysis of tumor samples and normal brainstem tissue
To obtain full characterization of collected samples, whole genome sequencing will be done on tumor and normal samples collected.
Time frame: 5 years
In vitro and in vivo molecular analysis of collected samples
Collected samples will potentially be used for in vitro analysis and generation of animal models of this tumor.
Time frame: 5 years
Assess aspects associated with specimen acquisition, including potential benefits and drawbacks
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
In an effort to continue to draw knowledge from samples collected, potential benefits and drawbacks from specimen acquisition will be continuously accessed.
Time frame: 5 years