ML318 is primarily utilized in research settings as a potent and selective inhibitor of the Niemann-Pick C1 (NPC1) protein. This protein plays a crucial role in cholesterol transport and homeostasis within cells.

By inhibiting NPC1, ML318 is used to study the mechanisms underlying Niemann-Pick Type C disease, a rare genetic disorder characterized by abnormal cholesterol accumulation in cells. Researchers employ ML318 to explore potential therapeutic strategies and to better understand the cellular pathways involved in lipid metabolism.

Additionally, it serves as a valuable tool in pharmacological studies to investigate the effects of NPC1 inhibition on cellular processes and disease progression.