Cancer and sphingolipid storage disease therapy using novel synthetic analogs of sphingolipids

► Novel sphingolipid analogs inhibit the hydrolysis of ceramide or its conversion to complex sphingolipids. ► Synthetic analogs were effective in killing cancer cells in vitro and in vivo. ► Analogs were tested for two therapeutic approaches for sphingolipidoses: substrate reduction therapy and chaperone therapy. ► Synthetic fluorescent sphingolipids were developed for the analysis of sphingolipid metabolism.