Principal Institution: Memorial Sloan Kettering Cancer Center – This Phase 1 clinical trial tests the safety and efficacy of combination checkpoint inhibitors in the treatment of children with brain tumors. Checkpoint inhibitors are not showing the same kind of side-effect profiles or toxicities seen in the standard treatments kids with brain tumors currently receive like high-dose chemotherapy and radiation, which can be critical for increasing quality long-term survival.

Checkpoint inhibitors are humanized antibodies that take the “brakes” off immune cancer cells to effectively and efficiently kill cancer cells. This combination treatment targets the immune system rather than the cancer itself.

In this case, children will be treated with a combination approach leveraging two agents, an anti- PD1 antibody (nivolumab), and another antibody targeting CTLA-4 (ipilimumab), a combination that has been highly effective in several high-risk adult cancer trials already. The hope is that children with malignant brain tumors will have similar results while reducing the toxicities that these kids usually receive from treatment.