There is a vast amount of evidence indicating that neurotrophic factors play a major role in the development, maintenance, and survival of neurons and neuron-supporting cells such as glia and oligodendrocytes. In addition, it is well known that alterations in levels of neurotrophic factors or their receptors can lead to neuronal death and contribute to the pathogenesis of neurodegenerative diseases such as Parkinson disease, Alzheimer disease, Huntington disease, amyotrophic lateral sclerosis, and also aging. Although various treatments alleviate the symptoms of neurodegenerative diseases, none of them prevent or halt the neurodegenerative process. The high potency of neurotrophic factors, as shown by many experimental studies, makes them a rational candidate co-therapeutic agent in neurodegenerative disease. However, in practice, their clinical use is limited because of difficulties in protein delivery and pharmacokinetics in the central nervous system. To overcome these disadvantages and to facilitate the development of drugs with improved pharmacotherapeutic profiles, research is underway on neurotrophic factors and their receptors, and the molecular mechanisms by which they work, together with the development of new technologies for their delivery into the brain.