CNS Therapeutics: The Next Blockbuster or Bust?

The BRAIN Initiative¹ sponsored by the NIH has pushed neuroscience and technology to center stage with President Obama supporting advancing neuroscience. But the central nervous system has been on the minds of pharmaceutical companies for the past decade with development pipelines heavily invested in CNS therapeutics.² The prospects for drugs in this space have seemed promising, but the many challenges facing the pharmaceutical field in general and specific caveats for neuroscience have created several setbacks for the field. Surprisingly, CNS therapeutics are still an area with high interest. In this series we discuss: why the field is prepared to develop these therapeutics; the specific challenges for developing drugs for neurobiology applications; and the futures for CNS therapeutics.

Part of the impetus for the BRAIN Initiative is that this is the era of neuroscience. Oftentimes certain scientific questions must wait for the right technology in order to address them. This is certainly the case for neuroscience. Neuroscience requires an interdisciplinary approach that involves cell and molecular analysis of neurons and their function, electrophysiology to understand the influx and efflux of ions controlling the cells, large scale data collection and analysis, and of course, techniques to analyze the genetics underlying normal and disease functions. The complexity of the nervous system needs to considered as CNS therapeutics are developed.

Disease understanding and biomarkers associated with disease must also be clearly understood. Elegant examples of this have been developed over the past decade. Generous families with genetic lesions associated with neurodegenerative disorders have participated in long term studies where their blood, brain, and other physiology has been monitored well before any onset of symptoms. As a result, valuable indicators of disease precursors are being developed. These long term studies are infinitely valuable as the biomarkers become diagnostics that lead treatment and serve as measurable outcomes during the development of CNS therapeutics.

The extensive investigations that have lead to the state of the neuroscience field today have prepared the way for the development of CNS therapeutics to treat an unmet medical need. In the next posts for this series, we discuss specific challenges facing the development of CNS therapeutics and future prospects for the field.