Liver GSL levels in the Npc1−/− mice were significantly reduced in Npc1−/− mice treated with ADLL (26.8%, P = 0.03), ADL (26.9%, P = 0.0253) and 45.5% for ALL (P = 0.0003) (Supplementary Fig. 2F). Here, NPC1 is linked to acute lymphoblastic leukemia.