Before pharmaceutical companies invest billions of dollars and more than a decade researching and developing a single FDA-approved drug, the process starts with a discovery — a distinct “eureka!” moment — in the lab of a basic scientist.
Basic science research lays the foundation for every new drug that appears on the shelves of our local pharmacies, and neuroscientists play an invaluable role in the lengthy and complex process through which these drugs are created. Before pharmaceutical companies invest billions of dollars and more than a decade researching and developing a single FDA-approved drug, the process starts with a discovery — a distinct “eureka!” moment — in the lab of a basic scientist.
To accomplish the mission of improving human health and wellbeing, we need to find the optimal balance of basic and translational research in the drug development pipeline, and that means valuing and understanding every step in the process. Neuroscientists must serve as advocates for the pipeline, and specifically the importance of funding basic science research to feed this process.
The research and discovery phase of drug development begins with basic science researchers striving to understand the biology underpinning a disease or condition at its most elementary level. As our knowledge of biology increases, so does our ability to identify and validate biological targets, allowing researchers to then seek molecular compounds that could influence these targets and potentially become new medicines. After researchers winnow down the options to the most promising compounds, preclinical trials follow, with extensive testing in lab and animal models to determine whether the compounds can be studied in humans. Finally, a single drug candidate must survive many, many phases of clinical trials in humans to determine whether it is worthy of FDA approval.
Neuroscientists play crucial roles in every stage of drug development, from basic science to preclinical to clinical. This process is an opportunity for us to bring our work beyond the pages of scientific journals and presentations at conferences, helping us to positively influence patients’ lives.
Unexpected Discoveries Can Play a Critical Role
Basic science research feeds the drug development pipeline, sometimes in unanticipated ways. By its nature, basic science is more curiosity-driven than translational research, allowing for greater experimentation. By employing the wide range of experimental systems and animals models not used elsewhere in the drug development pipeline, basic scientists have the ideal platform for making unexpected discoveries that lead us to a better understanding of biological processes. Increasing our basic understanding of the human brain and the diseases that affect it affords neuroscientists the best opportunity to identify new biological targets and then find and test compounds that could potentially become medicines to treat brain disorders affecting countless people around the world.
Basic science holds a critical role in developing these medicines because after drug candidates hit preclinical trials, the goal from that point forward is to exclude candidates from the list that will be tested in clinical trials, because clinical trials are the most expensive part of the pipeline. Therefore, basic science needs to provide numerous options for translational researchers to sort through and narrow down to the best drug option. Without a rich investment in basic science, we will starve the drug development pipeline.
Nearly every important drug and breakthrough therapy succeeds because it is based on the understanding of basic science. Ultimately, increasing the amount of funding for basic science is important for the entire scientific enterprise.
Communicating the Process
In order to gain the support of the public and policymakers for funding basic science research, we as neuroscientists must convey the importance of basic science in fueling the drug development process. For some scientists, communicating that role can prove difficult. Sometimes we struggle to explain and advocate for our work because we are unsure how to draw the connection for a lay audience between the scientific details and the big picture. SfN can help with this.
The Society is committed to supporting basic science and helping others understand that it is the foundation for the drug discovery pathway. Members can access training and advocacy resources on SfN.org and Neuronline, SfN’s home for learning and discussion. They can also attend live training events at the annual meeting that teach scientists how to communicate our work to non-scientists.
In addition, SfN organizes an annual Capitol Hill Day in the United States as well as other advocacy activities throughout the year, such as social media and letter-writing campaigns, focused on communicating to policymakers the value of funding basic science, particularly through the budgets of NSF and NIH.
But we can’t stop there. NIH and NSF are key collaborators in forwarding this cause, but truly supporting innovative sciences means we must work with private funding sources and philanthropic organizations such as the McKnight Foundation, the Howard Hughes Medical Institute, the Wellcome Trust, and the Science Philanthropy Alliance, which serve as valuable partners in supporting open-ended, basic discovery science.
SfN also promotes basic science through BrainFacts.org, a public information initiative of SfN, the Gatsby Charitable Trust, and The Kavli Foundation. The website provides the public with accurate, reliable content about the basics of neuroscience and the importance of brain research. For instance, SfN’s Research & Discoveries series provides examples of curiosity-driven basic science research that resulted in important advances in the field and led to medical innovations for treating neurological disorders.
Although the work of the basic scientist often remains behind the scenes, this research is absolutely critical to scientific progress and the development of new drugs that will save improve and save lives of people with debilitating brain diseases and disorders. Therefore, neuroscientists must continue to advocate for robust investment in basic research as an essential part of ensuring medical progress for future generations.
Message From SfN President Hollis Cline: Basic Science in the Pipeline
@SfN Society for Neuroscience