Therapeutic Strategies in Neurodegenerative Diseases

Neurodegenerative diseases are characterized by progressive loss of specific populations of neurons giving rise to clinical syndromes of neurological dysfunction. The World Health Organization declared neurodegenerative diseases the biggest health issue in the twenty-first century and predicted the number of people with neurodegenerative diseases to triple over the next three decades adding to the mortality and morbidity risk and the associated economic cost to society. Most of the clinically available therapeutics are symptomatic and supportive leaving a tremendous need for therapeutics that lead to slowing of disease progression rate (disease-modification). In this chapter, we discuss key therapeutic strategies in neurodegenerative diseases including gene therapy, cell therapy, and immunotherapy. These strategies address various components of the pathogenic mechanisms thought to underlie neurodegeneration including proteinopathy, neurotransmitter and key molecule deficiencies, neuroinflammation and systemic inflammation, and neuronal degeneration. We also summarize symptomatic therapies that are currently available in the clinic.