Teaching T cells to hunt cancer has spurred remissions in two kinds of leukemia. A new study suggests the technique may also work to treat glioblastoma, a type of brain cancer.

Today's study, published in Science Translational Medicine, describes a range of tests to determine a target for the immune system in glioblastoma. The protein, called EGFRvIII, occurs in about one-third of glioblastomas. On the basis of the study's results, the researchers are moving into human testing.

In this method of treatment, scientists harvest the blood of a person with cancer. Then, in the lab, they use a gene transfer to teach the T cells to target a specific protein. The engineered T cells are then transplanted back into the patient. In the case of blood cancers, the targeted protein is one expressed on the surface of B cells. In this example, the protein is EGFRvIII.

"We had complete tumor control" The patients in this case were mice, with human tumors, who were treated with human T cells as a model of how the treatment might work in people. While the T cells alone weren't enough to kill the cancer, when they were combined with chemotherapy, "we had complete tumor control," says Marcela Maus, one of the study's authors and the director of medical affairs for the translational research program at the University of Pennsylvania's Abramson Cancer Center. The next step is a new trial, which will enroll 12 people whose cancers have the EGFRvIII protein and has already started, she says.

The study, which was partially funded by Novartis, hints at the promise of the so-called chimeric antigen receptor T cells, or CAR-T for short. They can be used to target any sugar or protein on the surface of a cancer. So far, the method has shown promising results in chronic lymphocytic leukemia and acute lymphoblastic leukemia. Today's mouse work provides the justification for moving into human trials in glioblastoma.