In the current environment of fiscal austerity, some in Washington have wondered aloud whether or not federal investment in basic research -- the benefits of which are often only realized many years down the road – is a luxury that the nation can afford. In an article in the May 28 issue of the journal Neurology, Nina Schor, M.D., Ph.D., the William H. Eilinger Chair of Pediatrics and pediatrician-in-chief of URMC’s Golisano Children’s Hospital, argues that the basic research conducted in our nation’s labs fuels a creative process that leads to medical breakthroughs upon which the cures of tomorrow will depend.
Schor notes that, despite the climate in Washington, the public have long supported investment in research and development. Recent surveys by the National Science Foundation and the Pew Charitable Trusts show that 82 percent and 73 percent of participants support federal investment in research and agree that these investments have long-term benefit for the nation and society.
While the desire for quicker results have increasingly led corporations and foundations to shy from research that is higher risk or whose potential benefits are a long-term proposition, the pressure for a faster “return on investment” is also now being felt by the federal agencies that provide the bulk of support for basic research.
This is a fundamentally shortsighted perspective, argues Schor, as basic research over the long term provides the foundation for advances in medical care. The reason, she writes, is due to “the complexity of the human organism and its interaction with it environment.” The understanding necessary to unravel the multiple variables – genetic, molecular, proteomic, environmental, etc. – that contribute to diseases are not possible without the fundamental investigation that occurs in basic research labs. The clinical research that ultimately leads to new treatments must, she argues, be informed by a mechanistic understanding that can only be obtained through basic science.
As an example, Schor cites the evolution of our understanding of the role of RNA from that of a simple fabricator of proteins to an active regulator of cellular activity to (when produced in abundance) a toxic agent of disease. These findings have enabled scientists to look with a fresh eye at diseases in which RNA dysfunction plays a role, such as muscular dystrophy, and explore new treatments. Schor observes that these discoveries would not have been possible without the fundamental work done in fruit flies, worms, and mice to discover the mechanisms of RNA function. “Without this basic science exploration… no one would have imagined that the RNA, rather than the protein, produced from a gene could be responsible for the clinical pathology of genetic aberrancy.”
The need for continued investment in basic research is essential to the creation of new knowledge – either actively pursued or serendipitously discovered. “Because scientists only ask questions to which they do not know the answers, science often takes us in unanticipated directions.” Many of the key advances in medicine of today are the result of investments made 20 years ago. Consequently, the cures of the future, writes Schor, will depend upon the investments made today. “To build a bridge that extends from the laboratory to the clinic to the community and to simultaneously close the entrance to that bridge is to forfeit the future of translational medicine.”