Development of the ganglioside GD2 as a biomarker and clinical trial endpoint for childhood cancers

Email Principal Investigator
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Frank M. Balis

CBTN Samples Used


CBTN Participants


CBTN Samples




About this


Tumor biomarkers (TB) that are validated surrogate endpoints of a drug's therapeutic effect can improve the accuracy and sensitivity of assessing tumor response in phase 2 trials and could also substantially shorten the time line of phase 3 trials if they are predictive of subsequent relapse or survival. Our goal is to identify and validate TB to serve as surrogate endpoints in clinical trials to measure tumor burden and tumor response and to predict relapse. GD2 has many of the characteristics of an ideal biomarker. The overall objective of this proposal is to initiate the validation of GD2, which is expressed on the surface of neuroblastoma and other tumors of neuroectodermal or mesenchymal origin, as a TB for neuroblastoma using existing sample sets. This request is for an exploratory set of serum samples taken at diagnosis from patients with high grade glioma or medulloblastoma to assess the specificity of GD2 as a TB for neuroblastoma and determine whether GD2 is present in the serum of children with CNS tumors.

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Ask the scientists

What are the goals of this project?

THe goal of this project is to quantify serum GD2 concentrations in patients with a variety of common childhood cancers, including brain tumors, and in children without cancer to assess the specificity of GD2 as a tumor biomarker for neuroblastoma or other neuronal tumors.

Our hypothesis is that GD2 will not be quantifiable in the serum of patients with most common types of childhood cancer and children without cancer and that GD2 is a tumor-specific biomarker for neuroblastoma and brain tumors.

What is the impact of this project?

Although our focus is on development of GD2 as a surrogate endpoint, regulatory approval of GD2 as a clinical test7 could provide substantial patient benefit and facilitate the development of GD2 as a research tool by making it widely available. For a TB to be recognized as a valid surrogate endpoint it must be biologically plausible, have prognostic value for a clinical outcome, and predict for treatment effect on the clinical outcome in prospective clinical trials.

This is an exploratory study to screen for the presence and amount of GD2 in the serum of patients with the common forms to childhood cancer, including brain tumors, to evaluate the specificity of GD2 as a biomarker for neuroblastoma as part of the clinical validation process for GD2 as a tumor biomarker for neuroblastoma, and to determine whether it is present in the serum of patients with other types of neuronal tumors.

This pilot study is designed to determine whether GD2 is measurable in the serum of children with common types of cancer other than neuroblastoma, including CNS tumors



Specimen Data

The Children's Brain Tumor Network contributed to this project by providing plasma samples.