The PBS documentary Cancer: The Emperor of All Maladies provides a vivid illustration of how essential research is to improving cancer care. It also demonstrates how long and difficult the road to effective cancer care is, and how far along that road we have come. For decades, doctors and scientists had only a vague idea of what cancer was and how it might be possible to treat it. Advances in our understanding of the basis of cancer have come from a wide variety of sources, often from very basic research that, at the time, seemed unrelated to cancer and was only later understood for its therapeutic potential.
In the early 1960s, when the biochemist Stanley Cohen stumbled upon a previously unknown molecule that caused newborn mice to precociously open their eyes, he probably had no idea that he had just uncovered what turns out to be one of the most important growth signaling pathways in cancer. Cohen wasn’t even working on cancer at the time, but the effects he observed were due to the Epidermal Growth Factor (EGF) that binds to a protein on cells called the EGF receptor (EGFR). The discovery won him the 1986 Nobel Prize in Physiology and Medicine and today, we understand that activation of EGFR is found in several types of cancer, including lung and brain cancer, and the FDA has approved cancer drugs such as Tarceva (erlotinib) and Erbitux (cetuximab) that block the function of EGFR. These therapies are widely used every day around the globe. Importantly, by testing tumor specimens before treatment, we can identify who is most likely to benefit from treatment with drugs that target EGFR. This is an example of precision medicine, in which medical care is tailored to an individual’s specific disease profile. Many current efforts, including President Obama’s recently announced Precision Medicine Initiative, are geared to achieving this objective.
At the core of most advances in precision medicine are discoveries that have come from fundamental basic research. Indeed, Cohen was neither the first nor the last scientist to make a finding that was pivotal to cancer treatment while working on a seemingly unrelated subject, and the effects of these discoveries have been incalculable. Yet, over the past decade, there has been an attrition of resources dedicated to our nation’s scientific endeavor, and the diminution of funding has negatively affected basic research that relies heavily on government funding to survive. By de-emphasizing fundamental research, we risk jeopardizing future discoveries that could be transformative for human health and that can make precision medicine routinely accessible to all patients. Cancer patients, families, doctors, and the general public all need to pressure Congress to provide scientists with the support and funding from the National Institutes of Health that they need to do their important work exploring the basic mechanisms occurring in living organisms.
There are countless examples of basic laboratory discoveries that have illuminated our path towards precision medicine. For instance, the ability to identify genetic alterations in cancer, a pillar of precision medicine, is the consequence of 40 years of technological development that began when Nobel Prize winner Fred Sanger first sequenced the DNA of a virus. Now, we can sequence tumor DNA and compare it to normal DNA from the same individual. This allows us to find genetic alterations for which there may be therapies, and to study the evolution of a cancer during treatment so that we can intervene with the most appropriate therapies. New sequencing technologies can even be used to sequence tumor DNA in blood samples, providing a non-invasive manner of monitoring cancer progression and response to drugs.
Immunotherapy, a new buzzword in the cancer field, refers to treatments that target the immune system, several of which are showing great promise in clinical trials. This field is still in its infancy, but thanks to basic research that has increased our fundamental understanding of how the immune system functions, we are beginning to understand which tumors are most likely to respond to these immunotherapies.
The tools and information available to scientists today are unprecedented, and allow us to do research at an incredible pace. Nevertheless, scientists need the time and the funding to pursue routes of investigation where the clinical relevance is unclear. Accidents and serendipity often yield enormously important findings. Science is, after all, about experimentation. This can only happen with increased government funding for basic research, as well as philanthropy directed towards fundamental research. Moreover, we must remember that the next new groundbreaking precision cancer therapy that could save our lives 20 years from now might come from the biologist studying how cells communicate with each other in worms, or the chemist trying out a new type of reaction, or the geneticist who identifies a new way in which DNA is modified. Tomorrow’s cures depend on today’s discoveries, and today’s discoveries depend on basic research, which in turn relies on federal funding. The future of our own healthcare, as well as that of our children, and our grandchildren depends on ensuring that scientists have the resources to make those discoveries.
Moreno is a cancer researcher at Emory University; Politi is a cancer researcher at Yale University. They are Public Voices fellows in The OpEd Project.