Targeting Primary Ciliogenesis with Small-Molecule Inhibitors

The primary cilium is generally a non-motile solitary organelle that protrudes from a basal body at the cell surface in various cell types in multicellular organisms. This microtubule-based structure acts as a cell signaling platform to control key cellular processes, including cell proliferation and differentiation in development and in adult tissues. Elongated and/or dysfunctional primary cilia cause developmental disorders termed ciliopathies and cancers. The genetic inhibition of ciliogenesis inducers can block the progression of these diseases in model organisms. Thus, pharmacological inhibition of primary ciliogenesis has emerged as a potential strategy to treat these pathological conditions. Pharmacological inhibitors that affect cilium assembly, and have an impact on other cellular processes, have been identified. Here, we review some of these tools and discuss their value and limitations in the study of primary cilium biology, as well as for the treatment of some ciliopathies and cancers.