Fig. 1.

The Smo inhibitor saridegib causes regression of mouse medulloblastoma and resolution of advanced clinical symptoms. Three-week-old mice symptomatic for medulloblastoma were randomized to receive daily i.p. saridegib (20 mg/kg per dose, n = 3) or vehicle (n = 2) for 19 d. (A) Compared with a representative vehicle-treated mouse with a large tumor (Left) and a WT littermate with no tumor (Right), a representative mouse treated with saridegib (Center) showed complete resolution of clinical symptoms after 19 d of saridegib treatment. Arrow denotes the bulging skull caused by tumor. (B–D) Response to saridegib was apparent by gross pathology (B), imaging with Tumor Paint (chlorotoxin:Cy5.5) (C), and H&E staining (D).