Development of Preclinical Models of Pediatric Ependymoma

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Ching Ching Lau

CBTN Samples Used


CBTN Participants


CBTN Samples


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About this


Development of targeted therapy against ependymomas has been hampered by the lack of robust preclinical models. In addition, earlier genomic profiling of ependymomas failed to identify recurrent mutations in these tumors. With the discovery of the recurrent C11orf95/RELA translocation in supratentorial ependymoma (ST-EPN), it opens the door to finding novel targeted therapy. But first we have to generate the appropriate models to better understand the underlying biology including the pathogenesis of C11orf95/RELA+ ependymomas. The properly designed models based on proper biologic knowledge of the tumors will be reliable tools for development of novel therapies.

Previously we reported the successful development of orthotopic PDX models of various pediatric brain tumors (Shu et al, 2008) and also an orthotopic PDX model of ependymoma (Yu L et al., 2010), demonstrating the feasibility of and advantages of generating orthotopic PDX. After my recent relocation to the Jackson Lab, we are now ready to expand this work to generate a panel of at least 10 supratentorial ependymomas in order to represent the heterogeneity of these tumors.

Ependymomas are also notoriously difficult to grow in vitro. We have recently adopted a novel technique of establishing 3D cultures of neural tissues (Tang-Schomer et al, 2014) in growing ependymoma cells. This has resulted in the successful establishment of long term cultures of ependymoma from 3 consecutive cases. Again, we plan to expand this work to create a large panel of in vitro models of ependymomas for studying the biology of these tumors and also preclinical drug screening.

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What are the goals of this project?

This project aims to generate up to 10 PDOX models from fresh/cryopreserved surgical specimens or autopsy materials of supratentorial ependymomas (ST-EPN) and 2D and 3D cultures from the same tumors.

What is the impact of this project?

With the successful establishment of the models by the complementary methods outlined above, we will have a comprehensive panel of models for studying the biology of pediatric ependymomas as well as preclinical testing of novel therapeutic interventions. Because it is our intention to distribute these models widely through the established pipeline of the Jackson Lab, this work will benefit other investigators in the field as well.

Why the CBTN request is important to this project?

The incidence of pediatric ependymoma is low. In order to expedite the development of these preclinical models so that they could also be used for preclinical drug development, we are trying to identify as many existing cases as possible. Since CBTN currently has the largest collection of childhood brain tumor specimens that are available for collaborative research, we are therefore very interested in exploring the possibility of utilizing what are currently banked and also that our request to use live or cryopreserved tissues be considered as one of the priorities for future specimens that will be submitted.

In addition to CBTN, we have collaborative relationship with several other institutions that will also provide fresh tissues for this study including Connecticut Children’s Medical Center where Dr. Lau is currently Chief of Hematology and Oncology.

Specimen Data

The Children's Brain Tumor Network contributed to this project by providing tissue in freezing media for the generation of these models.