A CAR-T full of promise

Atypical teratoid/rhabdoid tumors (ATRT) are rare and aggressive tumors of the brain and spinal cord found in fewer than 10% of children with brain tumors, and most often seen in toddlers. Median survival for this cancer, even with treatment, is only about 17 months. In a Nature Medicine paper published in April, Ludwig Stanford’s Crystal Mackall and colleagues reported their development of a chimeric antigen receptor T (CAR-T) cell therapy to treat ATRT that virtually eliminated the tumors in mice. The CAR-T cells were engineered to target a surface protein on ATRT cells known as B7-H3, which Crystal and her team found is abundantly expressed by this cancer and drives its growth. Infusing the CAR-T cells into the cerebrospinal fluid that bathes the brain of mice was safer and more effective than administering the cells into a blood vessel. The approach curtailed dangerous brain inflammation because it required about 10 times fewer CAR-T cells than intravenous injection. The researchers plan to conduct clinical trials of the CAR-T cells beginning next year, first in adult patients with the brain cancer glioblastoma and—if all goes well—later in kids with ATRT.

This article was published in the August 2020 issue of Ludwig Link. Click here to download a PDF (2 MB).


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