Assessing Commercial Potential of Central Nervous System Delivery Approaches

Publisher Summary This chapter focuses on the assessment of the approaches for commercial potential of central nervous system delivery. Advances in molecular neurobiology have provided potential breakthrough opportunities for therapies for neurodegenerative disease. Most developments have occurred in the area of neurotrophic factors, which affect various aspects of neuronal function, neurite growth, and cell division. The behavior of these molecules can be influenced by the test conditions used to evaluate them, such that their biological roles need to be assessed in in vivo studies. For this reason, it is critical that methods to deliver these protein agents into the brain be developed and refined. The principal problem that confronts their study and application is their limited ability to pass through the blood-brain barrier and their potential to produce systemic side effects when administered chronically. For this reason, strategies must be developed that will allow researchers, and later clinicians, to deliver protein agents chronically into well-defined regions of the brain. This chapter describes an approach for the assessment of the commercial potential of central nervous system delivery technology for selected chronic degenerative diseases. It discusses the interpretation of data from studies with animal models of human diseases and the development of general therapies for diseases of unknown etiology.