The National Institutes of Health (NIH), which traces its roots as far back as 1887, has long been the primary driver for medical research in not only the United States, but the world. Work at NIH has led to a host of important cures, from life saving vaccines and miracle drugs to the use of fluoride to fight tooth decay.
Over the past 20 years, research at NIH has been making slow progress against cancer, increasing survival rates by about 1% per year. Yet now, through a new initiative called 21st Century Cures Act, we can accelerate that progress, perhaps drastically, and finally cure cancer as well as other chronic diseases such as Alzheimer’s and diabetes.
As Ron DePinho, President of MD Anderson, told me, “We have a confluence of major discoveries that have occurred across a wide range of fronts, which allow us to understand life and disease at a basic level and use those insights to influence its processes. We are now able to make a decisive assault on the cancer problem, if we have the resources.
Building A Cancer Map
In 1987, the Reagan administration proposed a fifteen-year, $3.8 billion project to decode the human genome. At the time, it seemed an unlikely project. The science of genomics was still in its infancy and many believed that not only was it was too ambitious, but even if it succeeded the benefits were unlikely justify the cost.
As it turned out, thanks to improved technology and a push from a private effort led by Craig Venter, the project was completed two years ahead of schedule. Since then, gene sequencing has outpaced Moore’s law, with efficiency doubling faster than every two years. Many believe that a $100 genome will soon be within reach.
The incredible success of the Human Genome Project led to another US government effort, the The Cancer Genome Atlas, approved in 2005 and the results have been no less impressive. To date, tens of thousands of tumors have been sequenced, casting new light on how we understand cancer and pointing the way to some revolutionary new treatments.
Yet there is still much work to be done. A gene map is merely the basis for a strategic plan. To find a cure, you need to attack cancer at the middle management level— the proteins that make up cell structures and support cellular processes, the RNA that transcripts those proteins and the cellular processes they support—to create safe and effective treatments.
Teaching The Body To Fight Cancer
One of the things that makes cancer such an insidious disease is that our bodies are effectively attacked by our own cells whose transcription machinery has run amok. Unlike an infectious disease, our immune system often does not recognize cancer cells as a foreign antigen, taking away our first line of defense.
Traditional cancer therapies like chemotherapy and radiation therapy do not only eradicate cancer cells, but also rapidly dividing normal cells, which causes harmful side effects, such as nausea, heart problems and infections that arise from weakening the immune system. Lives are saved, but at great cost, both financially and in terms human suffering.
Yet James Allison, a legendary immunologist, may have unlocked a key answer. He found that a molecule called CTLA-4 inhibits the activation of T-cell antibodies that attack disease. Later, he showed that by neutralizing CTLA-4, those same T-cells could be unleashed to attack cancer cells. It was an amazing discovery that Science magazine named its breakthrough of the year in 2013.
Allison’s discovery, along with parallel approaches developed by other researchers, has achieved unprecedented results, extending survival of patients with advanced disease beyond what anyone thought possible. Still, we need to understand why some patients respond to immunotherapy, while others didn’t, why some patients had side effects, while others didn’t.
These are questions that desperately need to be answered. More research is required.
Medical Science Powered By Data
When Ron DePinho first arrived at MD Anderson in 2011, research data was strewn across fifty databases and even more databases captured the clinical records of more than a million patients. Exacerbating the problem further, these two classes of data lived in largely separate universes, making it hard for clinical doctors to access the work of researchers and vice versa.
Scale that problem up to the level of the American healthcare system, which includes over 5,000 hospitals and 2500 research institutions, and you have a vast sea of information that remains largely untapped. Today, as computing power and big data techniques are advancing at blazing speed, there is an enormous opportunity to improve care.
Much like the immunotherapies that James Allison pioneered, data has the potential to transform science. At MD Anderson, DePinho and faculty leaders backed a project called the Oncology Expert Advisor that not only integrates data sources, but also uses cognitive computing to derive insights and share knowledge with clinical doctors, improving the care of everyday patients.
Yet, again, we have merely scratched the surface of what’s possible. To truly power medicine with data, we need to expand technology’s reach from the research center to the community level, allowing patients to connect in real time to their own physiology through mobile devices and enabling their doctors to access that data to provide more effective care.
To Win The Future, You Have To Invest In It
As medical science advances, the nature of its challenge evolves. Over the last century, we’ve largely conquered infectious disease and doubled life expectancy, but now must deal with a new set of problems. As people age, the chronic diseases, such as cancer, Alzheimer’s and diabetes are becoming endemic, creating a healthcare—as well as a fiscal—crisis.
Funding medical research would seem to be a no brainer. For a relatively small investment in finding new cures, we can eliminate terrible suffering and greatly decrease the nation’s medical bill. Yet in their zeal to appear fiscally responsible, politicians often seek to defund the NIH and the research it supports, which results in greater costs down the road.
President Reagan’s efforts to derail AIDS research in the 1980’s likely cost thousands of lives. More recent cuts resulted in decreased funding for an Ebola vaccine, just before a major outbreak inspired a panic throughout the country. These decisions seem ridiculously foolish now, but at the time many hailed them for their prudence.
Now, another such decision point is at hand. The 21st Century Cures Act, which aims to restore funds to the NIH for life saving research recently passed the House by a large majority. It now goes to the Senate where it will be submitted to debate and must compete with other priorities. If history is any guide, the chance that it passes is probably 50/50.
Yet also using history as a guide, investing in the future is always a smart bet. The Human Genome Project, for example, has had an economic impact of $796 billion, despite its relatively meager $3.8 billion price tag. The $31.2 billion invested in NIH during 2010 created nearly 488,000 jobs and generated more than $69 billion for the U.S. economy.
So today we are at another crossroads, similar to the ones we faced with AIDS, Ebola and other scientific initiatives. By restoring funding for medical research, we can win the race to cure cancer and other terrible diseases at a fraction of the cost that we will incur if we do not act. The right path forward would seem to be clear.
image credit: https://energycommerce.house.gov/cures
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