The present invention provides the use of a TRPML1-specific
small molecule agonist ML-SA1 in the
pharmaceutical industry. Through single and multiple
uranium exposures to simulate acute and chronic
uranium-exposed mouse models, it was confirmed that delayed administration of the
small molecule compound ML-SA1 can significantly promote the urinary
uranium excretion of acute and chronic uranium-exposed mice, reduce renal uranium accumulation, alleviate the
pathological damage and
apoptosis of uranium-induced renal proximal tubular epithelial cells, protect
renal function, and is closely related to the significant improvement of lysosomal
exocytosis; through the uranium-exposed human renal proximal tubular epithelial HK-2
cell model, it was confirmed that delayed administration of the
small molecule compound ML-SA1 significantly promotes the
excretion of uranium accumulated in HK-2 cells by promoting lysosomal
exocytosis and accompanying compensatory lysosomal
biogenesis, and significantly reduces uranium-induced
cell death. Since the small molecule compound ML-SA1 excretes uranium deposited in lysosomes out of the
cell through lysosomal
exocytosis, thereby effectively reducing the
intracellular uranium content, it can also be used for the enhanced
excretion and detoxification of other
heavy metal poisonings deposited in lysosomes.