ML-SA1 is a potent and selective agonist of TRPML1 (transient receptor potential mucolipin 1), a calcium-permeable ion channel located primarily in late endosomes and lysosomes. Its primary application lies in research settings where it is used to probe lysosomal function, autophagy, and endolysosomal trafficking. By activating TRPML1, ML-SA1 induces lysosomal Ca²⁺ release, which triggers lysosomal exocytosis and enhances the clearance of accumulated cellular debris. This property makes it a valuable tool in studying neurodegenerative diseases, such as Alzheimer's and Parkinson’s, where impaired lysosomal degradation contributes to pathology. ML-SA1 has also shown potential in promoting cellular repair mechanisms. It enhances autophagosome-lysosome fusion, supporting the clearance of protein aggregates and damaged organelles. In disease models involving lysosomal storage disorders, ML-SA1 improves lysosomal biogenesis and function, suggesting therapeutic relevance. Additionally, it is used to investigate cancer cell metabolism, as modulation of lysosomal activity can influence tumor cell survival and response to stress. Due to its cell permeability and specificity for TRPML1, ML-SA1 is widely used in cell-based assays to dissect signaling pathways linked to lysosomal ion channels and to evaluate pharmacological strategies aimed at restoring lysosomal homeostasis.