CAR T–cell therapy shows promise for ependymoma though barriers remain

Ependymoma is a type of brain cancer that is chemotherapy-resistant, causing physicians to rely on surgery and radiation treatments that can affect neurocognitive abilities. Even when treated, 10-year survival for the disease after diagnosis is less than 50%, indicating a need for more effective and less disruptive therapies. A study led by Giedre Krenciute, PhD, St. Jude Department of Bone Marrow Transplantation and Cellular Therapy, and Stephen Mack, PhD, St. Jude Department of Developmental Neurobiology, found that immunotherapy for ependymoma using chimeric antigen receptor (CAR) T cells holds promise for improving treatment.

 Findings show existing CAR T cells targeting the B7-H3 cancer-related protein could kill brain tumors in model systems without an immune system. 

However, the approach did not work as well if the immune system was left fully functional or for larger tumors, indicating that more research is needed to optimize the approach. The findings were published in Clinical Cancer Research. 

“Understanding why mouse B7-H3-targeting CAR T cells fail in immunocompetent systems could be key to unlocking their potential in human patients,” Mack said.

“This is the first preclinical study that tests the efficacy of human and murine B7-H3-CAR T cells against ependymoma,” Krenciute said.

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