Tang et al. first described the use of BET inhibitors in Hedgehog-driven cancers, including medulloblastoma. They found medulloblastoma as well as atypical teratoid/rhabdoid tumors (ATRT), another pediatric brain tumor with non-canonical Hedgehog signaling, to respond to treatment with BET inhibitor JQ1 [48]. This evidence concerns the gene DNER and cancer.