Background: Lysosomotropic molecules are taken up into lysosomes in vitro and in vivo. Many drugs
approved for clinical medicine are lysosomotropic agents, characterized by promoting particular effects including
cytoplasmic vacuolization, increase in number and size of lysosomes, inhibition of their enzymes and accumulation
of undegraded material, leading mainly to phospholipidosis. Despite lysosomotropism has been extensively described
and studied, the pathophysiological significance of this process is still not well understood.
Objetive: In this review, we focus on what is known about the effects of lysosomotropic drugs on specific lysosomal
functions and their similarities with the phenotypic features of lysosomal storage disorders (LSDs).
Conclusion: Some effects of lysosomotropic drugs are very similar to pathologic features of human genetic diseases
affecting lysosomal function, and therefore these drugs can be used as tools to understand the mechanisms underlying
such patho-pathways as well as to create pharmacologically-induced models of LSDs.