Development of CAR-T Cell Therapy for Atypical Teratoid/Rhabdoid Tumors

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Peter Madsen

Children’s Hospital of Philadelphia
Philadelphia, PA, USA

CBTN Specimen

About this


Atypical teratoid rhabdoid tumors (ATRT) are an aggressive pediatric brain tumor that affects young children, especially those less than 2 years old. There are some treatment options such as surgical resection and chemotherapy, however these approaches have done little to change the poor survival outcomes of ATRT patients. Researchers on this project hope to explore new avenues of treatment for ATRT. They will be investigating chimeric antigen receptor T-cell (CAR-T) therapy. T-cell receptors are used in immunotherapy and CAR-T therapy involves engineering a special receptor that can target specific proteins associated with a given cancer. In the case of ATRT, researchers are hoping to target the protein claudin-6 (CLDN-6) which is known to be found in abundance on the cell surface of ATRT tumor cells. ATRT samples provided by CBTN will be used to test this novel therapy. Researchers will also explore differing delivery methods for this therapy in the hopes of informing future clinical trials. If successful, this work will expand treatment options for ATRT patients.

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Ask the scientists

What are the goals of this project?

Researchers will attempt to use a novel therapy, chimeric antigen receptor T-cell therapy to treat the difficult treat ATRT.

What is the impact of this project?

The options currently available for the treatment of ATRT are slim with poor outcomes and this study could help expand options for patients.

Why is the CBTN request important to this project?

CBTN has a biorepository with the samples necessary for researchers to test this novel therapy.