Initially, SMO was the principal target for the development of SHH inhibitors, and the first clinical trial performed using an SMO inhibitor to treat a brain tumor was conducted in 2008 in a male patient with metastatic medulloblastoma who was treated with a novel HH pathway inhibitor, GDC-0449 (vismodegib) [86]. The gene discussed is SHH; the disease is brain neoplasm.