Autophagy is an intracellular lysosomal-mediated process that functions to degrade and recycle cellular components. In recent years, autophagy has been implicated in numerous human diseases, including cancer, neurodegenerative disorders, bacterial and viral infections, and bone disease. Depending on the disease and cellular context, autophagy may act to either promote or suppress human disease. In cancer, autophagy can act to promote tumorigenesis by providing cancer cells a means of survival. Autophagy also plays a cytoprotective role in tumors as a response to many anticancer agents. Genetic studies targeting autophagy pathway components indicate that inhibition of autophagy is a promising therapeutic strategy for some cancers. However, there are limited data and a lack of selective autophagy-modulating compounds available to provide validation. This symposium will bring together experts in cell biology, drug discovery, and clinical translation to help frame the state of our understanding of autophagy and the therapeutic potential for modulation with drugs or biological treatments. Topics will include human and mouse genetic analyses and modulation of autophagy genes, molecular target selection, and progress toward in silico design, screening, lead identification, and development of specific small molecule modulators of autophagy to provide proof-of-principle and, ultimately, potential therapeutic agents. Clinical evidence and implications will also be discussed.