Research and Development for Central Nervous System Therapeutics: Overcoming the Blood-Brain Barrier and Disease Heterog
The development of treatments for disorders affecting the brain and spinal cord—collectively the Central Nervous System (CNS)—is perhaps the most challenging and high-risk area within all of medicine
. Despite decades of intense research, a profound unmet need persists for conditions ranging from neurodegenerative diseases like Alzheimer's and Parkinson's to psychiatric disorders, chronic pain, and spinal cord injuries. The obstacles are inherent to the biology of the CNS and encompass issues of access, target identification, and the complexity of the diseases themselves.
The Formidable Biological Obstacle: The Blood-Brain Barrier (BBB)
The primary physical barrier to therapeutic success is the Blood-Brain Barrier (BBB), a highly selective interface composed of specialized endothelial cells that tightly regulate the passage of substances from the bloodstream into the CNS tissue. This biological security system, while essential for protecting the brain from toxins and pathogens, simultaneously prevents the vast majority of promising therapeutic agents—including nearly all large-molecule biologics and most small-molecule drugs—from reaching their intended neural targets…
