Francis S. Collins is director of the National Institutes of Health.
Biomedical research is at a critical juncture — a moment of exceptional opportunities that demand exceptional attention if their promise is to be fully realized. Many of the most exciting possibilities stem from the convergence of several factors: innovative tools and technologies arising from the Human Genome Project; advances in computers and biomedical imaging that are fueling a generation of complex digital data sets known as “big data”; and increased interest by both public and private sectors in finding ways to accelerate the rate at which research findings are turned into treatments and cures.
The National Institutes of Health (NIH) is responsible for turning scientific discoveries into better health, but a combination of sequestration-mandated spending cuts and budgets that have not kept pace with biomedical research inflation over the past 10 years has weakened NIH’s ability to carry out its mission.
One transformative program that hangs in the balance is the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative, part of a focus aimed at revolutionizing understanding of the human brain. By catalyzing development and application of new technologies, researchers plan to produce a dynamic picture of the brain that shows, for the first time, how individual cells and complex neural circuits interact in both time and space. This could mean enormous advances for efforts to treat and possibly even prevent epilepsy, schizophrenia, Alzheimer’s disease, autism, Parkinson’s disease, traumatic brain injury and many other neurological conditions.
Another area of great promise is the ability to assemble very large data sets of medical research information. The advent of electronic health records will dramatically accelerate the “big data revolution.” Details can be understood by focusing on one disease: cancer. Taking advantage of breathtaking advances in DNA-sequencing technology, NIH-funded researchers working on the Cancer Genome Atlas have conducted comprehensive analyses of more than 20 different types of cancer and plan to study dozens more. Besides expanding understanding of the molecular roots of different cancers, this pioneering work has opened doors to new therapeutic targets, as well as to new and more precise uses of chemotherapy drugs. Unfortunately, this mountain of data will be of limited use to cancer patients if researchers and clinicians lack the tools necessary to manipulate and mine it effectively.
Another fascinating frontier is the microbiome. The human body contains trillions of microbes, outnumbering human cells 10 to 1. So far, work to gather information on the genetic makeup of these microbial communities has yielded important insights into their roles in a wide range of chronic diseases, including obesity, diabetes, allergies, cancer and heart disease. The opportunity to expand and build on that knowledge to develop evidence-based treatments, such as probiotics for obesity, is just one of the exciting possibilities.
Vaccine development is also poised for rapid progress, especially in the areas of AIDS and influenza. Some researchers are exploring the production of “broadly neutralizing” antibodies that work against all strains of a virus. In the case of influenza, that could mean an end to the annual flu shot and a vaccine that would profoundly reduce the global risks of the next influenza pandemic.
In recent years, researchers have learned how to reprogram human skin cells or white blood cells into induced pluripotent stem cells, which have the amazing ability to develop into different types of cells. This opens the door to unprecedented opportunities for the advancement of basic research into human development, acceleration of drug screening for a host of diseases, and design of new “cell-based” therapies to cure some of medicine’s toughest challenges, such as sickle cell anemia and inherited forms of blindness.
Clearly, NIH is not lacking for ideas. And the institutes will continue to support innovative research across the country. But without sustained investment, many high-priority efforts would move at a substantially slower pace, and years of effectively flat funding for biomedical research have left scientists facing the lowest chances in history of having their research funded by NIH. Many young scientists are on the verge of giving up, taking with them the talent needed to make tomorrow’s medical breakthroughs.
The budget deal reached last week, however, would give appropriators the chance to provide some much-needed relief for biomedical research. The economic benefits of NIH funding include a return-on-investment of research grants to local economies and cost savings from decreased disease burdens. But beyond that, for the millions of sick people awaiting treatments and cures, the investment in NIH’s mission is priceless. If an investment in hope is not worth supporting, I don’t know what is.