Sci. Transl. Med. 11, eaaw5680 (2019)

A locally injected version of the gene for the anti-cancer cytokine human IL-12 (hIL-12) can be safely and specifically activated in humans with an oral activator.

Recurrent high-grade glioma (rHGG) is an aggressive brain tumor for which treatment options are limited by inadequate understanding of the tumor and its microenvironment and the challenge of getting therapies across the blood–brain barrier. Although IL-12 is a potentially promising rHGG therapy that activates immune responses to target the tumors, its toxicity precludes systemic administration in humans.

Chiocca and his colleagues carried out a phase 1 study in individuals post tumor resection, who had the hIL-12 gene vector injected and also took an oral activator. They found that this approach was safe and showed some promising effects, including the passage of the activator across the blood–brain barrier.