The potential capacities of FTY720: Novel therapeutic functions, targets, and mechanisms against diseases

Fingolimod (FTY720), an antagonist of sphingosine-1-phosphate (S1P), functions by binding to S1P receptors (S1PRs), excluding S1PR2. It received approval from the Food and Drug Administration (FDA) for the treatment of multiple sclerosis (MS) in 2010. As the first non-selective oral agonist for S1PRs, FTY720's diverse and systemic receptor expression often leads to alterations in various signaling pathways and multiple systems, making it a subject of intense research. Recent studies have identified a wide range of novel or potential functions for FTY720 beyond its application in MS. These include effects on the blood-brain barrier (BBB), vascular system, organelles, and cell death, as well as potential applications in organ transplantation, immune disorders, oncological conditions, neurological and psychiatric disorders, viral infections, and hypersensitivity diseases. This paper reviews the novel roles, targets, and mechanisms of FTY720 that hold promise for clinical utility. Additionally, it summarizes FTY720's derivation and development process, the characterization and mechanism of the structure of FTY720-P bound to S1PRs, the clinical safety profile, future challenges, and potential strategies to address them. These insights aim to guide future research and applications of FTY720, maximizing its therapeutic potential.