In a small clinical trial, a CAR T-cell therapy—a type of immunotherapy that uses a patient’s own immune cells to fight cancer—shrank tumors in several children and young adults with diffuse midline gliomas. This fast-growing form of brain and spinal cord cancer typically causes death within a year of diagnosis.

In the trial, several participants were still alive 2 years or more after receiving the treatment.

Patients in the trial had a type of diffuse midline gliomas known as H3K27M mutant, a genetic change that is found in about 80% of younger patients with these cancers Exit Disclaimer. Researchers at Stanford University, who led the study, designed the experimental CAR T-cell therapy to target a molecule called GD2 that is produced in large amounts by H3K27M-mutant diffuse midline gliomas.