Basic Research of the Brain Unlocks Myriad Discoveries and Gives Us a Path Toward New Treatments

Bill Martin

More than 100 million Americans live with the devastating effects of a debilitating neurological or psychiatric disease each year. In addition to producing severe hardship for millions of families, such suffering costs the U.S. economy at least $760 billion annually. The Society for Neuroscience (SfN), the world’s largest organization of scientists and physicians devoted to understanding the brain and nervous system, believes that the only way to foster the scientific breakthroughs needed to alleviate that suffering is with strong, consistent and reliable public funding of biomedical research through agencies including the National Institutes of Health (NIH) and the National Science Foundation (NSF).

Nearly every important drug and breakthrough therapy succeeds because scientists following their curiosity have increased our knowledge and understanding of the biological underpinnings of a disease or disorder at its most elementary level. As we’ve improved our understanding of the brain through decades of research, we’ve already seen profound advances in human health.  While researchers continue to investigate the many unknown aspects of Parkinson’s disease, we know the brain chemical system that’s been disrupted as well as the affected brain regions. This knowledge is the basis for the therapies that have been developed from levodopa to deep brain stimulation. Basic research is integral to uncovering new paths to pursue, providing unexpected biological targets to treat disease and a wider repertoire of molecules to test as potential drugs. The more options basic researchers can provide the more likely we will find the best drug options and cures. 

Without a rich federal investment in basic science, we starve the drug development pipeline because of the staggering lag-time between discovery and profitability. This means that the pharmaceutical, biotechnology and medical device sectors build off federally-funded basic research and provide a multiplier effect to that investment as those industries develop new therapies and treatments.

The cross-disciplinary Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative is focused on this type of basic research and is developing new technologies and tools that will support the efforts of many scientists to understand the brain. Drawing on knowledge from the life sciences, physical sciences and engineering, the BRAIN Initiative has begun to enable new and important insights.

We are gaining insight into brain chemical systems with DREADD 2.0, developed with both BRAIN Initiative funds and long-term NIH funding. An upgrade to the widely used technology called DREADD -- Designer Receptors Exclusively Activated by Designer Drugs -- this technology allows researchers to probe neuronal communication by turning specific neurons “on” and “off.” Using DREADD 2.0 researchers can directly control specific neurons in mice and alter the animals’ behavior as a result. The technology will spur a deeper, more thorough exploration of the brain and behavior. Such research will seed the discovery of potential treatments for disorders caused by the breakdown of neuronal communication like Alzheimer’s disease and schizophrenia.

Additionally, we are gaining insight into brain circuitry with novel imaging techniques. Two-photon microscopy of the mouse brain has generated images of activity in nerve cells, dendrites and synapses, allowing scientists to more precisely examine single neurons and signaling in the brain. Photoacoustic microscopy has provided three-dimensional high-resolution, high-speed imaging of a mouse brain.  All of these technologies provide the groundwork for neuroscientists to move the field forward in ways that may ultimately identify new therapies for neurological and psychiatric diseases. I invite you to learn more about these and other discoveries at www.BrainFacts.org.

We live at a time of extraordinary opportunity in neuroscience. If we are to make the most of it, we must ensure we maintain public support for a robust investment in research.

Bill Martin is the Chief Scientific Officer and Head of Research and Development, BlackThorn Therapeutics and Chair, Government and Public Affairs Committee, SfN.

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