CNSdirected

CNS-directed is an adjective used in pharmacology and neuroscience to describe drugs, therapies, or interventions that are intended to act on the central nervous system (the brain and spinal cord) or to modulate CNS function. The term is often used in research and development to distinguish central nervous system–targeted approaches from therapies with primarily peripheral effects.

CNS-directed strategies include small molecules engineered to cross the blood–brain barrier, biologics or enzymes modified for CNS delivery, gene therapies delivered to neural tissue, antisense oligonucleotides, and cellular therapies such as neural progenitor cells or engineered immune cells. Delivery methods may involve systemic administration with BBB penetration, intrathecal or intraventricular injection, and local intracerebral administration, depending on the therapeutic goal and safety considerations.

Development of CNS-directed therapies faces challenges such as restricted drug exposure in the CNS, regional heterogeneity, and potential CNS-specific adverse effects. Applications span neurodegenerative diseases (for example, Alzheimer's and Parkinson's disease), CNS cancers, psychiatric and mood disorders, pain with CNS involvement, and other conditions where central nervous system pathology is central. Efficacy is typically assessed with CNS biomarkers, imaging, and functional or cognitive endpoints, with regulatory considerations reflecting CNS safety and efficacy.