Our society has become increasingy short-term focused, perhaps most exemplified by quarterly earnings. But the solution to many of the biggest challenges facing our country – from the economy to the environment, to improving health – requires long-term thinking. The same principle can be applied to funding for basic research.
Our story of a recent success in gene therapy underscores the need to take a long-term view. Anyone who has followed the 20-year-old field of gene therapy knows that the road has been extremely rocky. Initially heralded as a means to cure many genetically based diseases, much like an overhyped stock offering, gene therapy fell sharply out of favor after some troubling clinical failures.
But as the New York Times reported on Nov. 5th 2009, successes in giving new genes to correct specific inherited diseases suggests the technology may have a bright future after all. A French research team used gene therapy based upon a tool developed at the Salk Institute to effectively treat adrenoleukodystrophy (ALD) in two Spanish children. The French scientists used a safe version of the deadly AIDS-causing virus to deliver healthy genes into two young boys – children who otherwise faced likely death before adolescence from the progressive brain disease.
Our own Professor Inder Verma first imagined this unexpected use of the fearsome AIDS virus back in 1993. After several years of experiments employing the Salk's highly creative and collaborative brand of science, he published seminal papers on his viral vector discovery. And 11 years after Verma's inspiration, the viral vector was used to treat two kids half a world away. Their deteriorating condition appears stabilized.
The history of Inder's scientific feat and its ramifications is the subject of our Inside Salk cover story. But it is the 2009 science policy context of that discovery that I am most eager to underscore. You see, a basic science breakthrough has a life cycle, and that cycle is typically far longer than one would expect. It took about 15 years from Inder's first Aha! moment to the report of encouraging treatment of these children. No one could have accurately predicted the precise, convoluted path from original idea to human use.
When Salk scientists publish their groundbreaking work, the implications can take years or even decades to become manifest. Thus, my take-away message is that basic science requires long-term investment and long-term perspective.
Unfortunately for the Salk Institute and other basic science bastions, federal funding for fundamental research is flattening. Increasingly, there is pressure on the National Institutes of Health (NIH) to funnel more dollars toward translational science in the belief it may provide more immediate application to patients. While this may be true in some cases, the history of breakthrough treatments of various diseases is that the road is generally long and full of many unanticipated detours.As a member of the Scientific Management Board of the NIH, it is my role to persuade our federal policymakers to once again more fully embrace the long-term view and not short-change basic research. I believe wholeheartedly that the knowledge base and discoveries developed through superb basic science are essential to the American innovation and leadership we have all come to expect. Limiting the opportunity for scientific ingenuity to flourish is not a smart strategy.
At the same time, under the most optimistic scenarios we cannot anticipate the same kind of growth in federal funding for biomedical research we experienced in the last decade. Hence, there is a growing and urgent need for private foundations and individual philanthropists to invest in basic research, particularly in the type labeled high-risk and potentially high-reward. Exceptionally valuable are unrestricted gifts and contributions to our endowment that give us the flexibility to invest in the bright ideas of the future Inder Vermas – those whose out-of-the-box schemes will ultimately lead to innovative and effective treatments for serious disease.
Thanks to our many private contributors who understand the long-term nature of basic research, the Salk continues to move science forward. Inder Verma's breakthrough in gene therapy is just one compelling example of how your support of the Salk Institute is, in the long run, a valuable and extremely wise investment in improving human health.