A phase II clinical trial of a vaccine that will track down tumor stem cells “like a bloodhound” has begun at Wake Forest Baptist Medical Center, one of 20 facilities to participate in the randomized, placebo-controlled study.

Wake Forest will test the novel cancer vaccine (ICT-107) on at least 25 patients with newly diagnosed glioblastoma multiforme (GBM), the most aggressive and highest grade malignant glioma. Participants will first receive standard treatment for GBM, including surgery, radiation and chemotherapy.

The vaccine’s approach is unique, said Glenn Lesser, principal investigator of the study at Wake Forest, because it targets the antigens or proteins that are present on glioma stem cells, whereas other treatment approaches mostly target differentiated tumor cells.

 “Most of the cells we kill with standard treatment are likely not the ones driving the tumor growth. If the stem cells aren't targeted, they keep generating more tumors,” explained Lesser. "[This] is a way of presenting antigens or proteins normally found on the surface of the cancer cells to the immune system so that immune cells can seek out and kill those cancer cells anywhere in the body. This is not unlike giving a piece of clothing to a bloodhound and then letting it loose to find a missing person.”

In the phase I clinical study of ICT-107 in GBM, the 16 patients achieved a one-year survival rate of 100%, and a two-year rate of 80%. Although the study was conducted with such a small number of patients, it compares favorably with historic results of 61% and 26%, respectively, of standard care alone.