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Mateusz Koptyra

Children’s Hospital of Philadelphia
Philadelphia, PA, USA

CBTN Participants

CBTN Pre-clinical Models Used


Internal funding

National Institute of Health

About this


Brain cancer has become the leading cause of death in children with cancer. Brain tumor research generated little advances that have translated into meaningful clinical benefit for pediatric patients. One of the main reasons which significantly slowed down the progress of the scientific development in pediatric brain tumor field was limited access to tumor tissue, and research tools like cell lines, xenograft transplant or genetic mouse models. Over the recent years, CBTN developed and banked about 150 cell lines from numbers of pediatric brain tumor types. We are constantly striving working on growing cell line cohorts but also looking to explore both concept studies and high-throughput studies utilizing the developed models to accelerate the research to provide better therapeutic options for pediatric brain tumors.

Here we propose the collaboration effort with the National Center For Advancing Translational Studies (NCATS) to generate the atlas of drug screening response utilizing the preclinical CBTN cell line models and beyond. This work will utilize the high throughput screening approach allowing to find novel drug possibilities for the tested model. NCATS experts use qHTS, a process in which each compound of a large chemical library is tested at multiple concentrations. Through automated robotic screening, the technology integrates the pharmacologic dose-response relationship with the speed and accuracy of automated HTS. We propose to utilize the NCATS platforms for the high-throughput analysis for existing CBTN cell line cohorts.

Ask The


Ask the scientists

What are the goals of this project?

The goals of this project are selection of disease specific cohorts of cell lines for the high throughput testing, delivery of the cell lines to NCATS for drug testing, generation of the transcriptomic and methylation analysis data along the drug testing process in collaboration with NCATS and NCATS-D3b data processing, and analysis which will occur with deposition of data on Cavatica, PedcBioPortal with KidsFirst data integration.

What is the impact of this project?

As the ultimate goal, this project will provide extensive in vitro drug response information across multiple pediatric tumor diagnoses and allow to build the pediatric brain tumor cell line drug response atlas. The generated data will create a baseline to explore new tumor specific biomarkers/signaling pathways as direct drug targets providing preclinical information for potential future therapeutic strategy for analyzed tumor cohorts. The data will be returned to CBTN and available for researchers without restrictions.

Specimen Data

The Children's Brain Tumor Network is contributing to this project by providing cell lines.