Over the past several years, neurotrophic factors have made considerable progress from the laboratory into the clinic. Evidence from preclinical and clinical studies indicates that it may be possible to use neurotrophic factors to prevent, slow the progression of, or even reverse the effects of a number of neurodegenerative diseases and other types of insults in both the central and peripheral nervous system. Their potential importance in the development of therapeutic agents against neurodegenerative disorders and nerve injury has led to a flurry of activity towards understanding their structure, function and signalling mechanisms. Approaches to develop pharmacological agents that target neurotrophic factors, their receptors or neurotrophic factors signalling pathways have been attempted. This review focuses on some of the major themes and lines of mechanistic and therapeutic advances in this fast-moving field of neuroscience.