Growth factor treatment of neurodegenerative disorders: new developments pave the way for clinical success

Neurotrophic factors have received significant attention in recent years for their potential use in the treatment of neurodegenerative and other nervous system disorders. However, the first attempts to apply factors such as nerve growth factor, glial-derived neurotrophic factor and ciliary neurotrophic factor failed to demonstrate any significant benefits in the clinic, despite a wealth of positive preclinical data. Recent advances in our understanding of the biology of neurotrophic factors in combination with the development of several new and improved delivery methods, for example, genetically engineered cells and viral vectors, have revitalized the interest in these potential nervous system protein therapeutics, and influenced the design of new clinical trials.