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The Path to Cancer Treatment Breakthroughs

A Report from the National Cancer Institute


Knowledge image

It’s a simple question with no simple answer: When are you going to cure cancer? At the National Cancer Institute, the component of the National Institutes of Health tasked with leading America’s research efforts against all forms of this disease, we understand the urgency of the question; we also understand the fear of cancer that drives the search for answers. NCI’s efforts to reduce cancer incidence and mortality cover a wide range of research areas, including prevention, screening, early diagnosis, treatment, and survivorship efforts.

Cancer is a disease of stunning complexity. Decades of impressive research progress have enabled experts to understand that cancers arise primarily from changes in our genes, our bodies’ chemical building blocks of who we are, and in the proteins they produce. As we grow and age, our genes can accumulate changes in a cell that can begin the process that eventually manifests itself in the form of a tumor or other form of cancer. Researchers have come to understand how the body supports and nourishes a growing tumor and how a small group of cells can split off to form a new tumor at a different location. It is this process, called metastasis, that most makes cancer lethal.

Importantly, it is the steady accumulation of knowledge about cancer – its earliest origins, its growth, and its spread – that makes possible new thinking about treatment that we hope will, in time, turn cancer from a killer to a curable condition or, at worst, a disease that can be successfully managed through a lifetime. While the news stories that draw the most attention are about new drugs and enhanced therapies that target tumors and have fewer side effects than traditional chemotherapy, each of those breakthroughs begins in a laboratory.

The foundation for most cancer breakthroughs has long been so-called basic research: studies at the laboratory bench. Bench scientists begin with an idea, a hypothesis. In many cases, science often relies on analogs to people, which may be yeast, fruit flies, zebrafish, or mice. A given laboratory may be testing a gene or testing a protein, which is the messenger chemical that carries out instructions of our DNA, and these seemingly simple stand-ins for people can give the earliest signs of whether an avenue of research is promising.

It is the steady accumulation of knowledge about cancer that makes possible new thinking about treatment that we hope will, in time, turn cancer from a killer to a curable condition.

Biomedical science is also a process that builds experiment on top of experiment. Researchers employ strict methods of study and of recording their processes and results. When experiments are conducted, repeated, and confirmed, the result is a scientific paper most often submitted to and published in a peerreviewed journal. It is then that others in the research community can review the published research and incorporate that science into closely related studies or, in some cases, collaborate with the authors and move on to next steps.

Cancer science, as performed in the laboratory, often looks at chemicals and processes that transcend many forms of cancer; for example, potentially finding commonalities between prostate cancer and breast cancer.

Some of today’s promising science is also performed more collaboratively, by researchers from many allied labs. For example, The Cancer Genome Atlas (TCGA) is an effort led by NCI and the National Human Genome Research Institute to catalogue all of the genes responsible for 20 cancers. TCGA requires large centers that sequence the DNA from 500 people or more per type of cancer, generating volumes of data that, even in an era of huge computer storage, is massive. Other facilities, principally large research universities, work to analyze that information and ultimately come up with a key to all of the changes in DNA that lead to cancer. Because TCGA is led and financed by governmental agencies, its results are available to the broader research community without charge.

In the end, research from laboratory benches and from large-scale research efforts like TCGA lead to targets, potential vulnerabilities in tumors that might be exploited by a new drug. The cascade of work that follows includes the efforts of medicinal chemists, computer scientists, and many others who attempt to develop new compounds or who may compare thousands of existing drugs or natural substances to a target, in order to see if there is any beneficial effect. Those studies may also take place using laboratory animals.

It is only at the end of a lengthy process that new drugs and therapies might first be tested in a person, a separate process that begins with small studies to verify safety.

When all goes well, the end of that process, which often began in a solitary laboratory, is the approval of a new, safe drug. When that occurs, we are used to turning on the news to see physicians in white coats talking about effectiveness, about people with cancer living better or living longer. But that story on the news is also about the culmination of a much broader effort, and each person in that chain of success has heard the questions to which cancer survivors most want answers. Indeed, most improvements in cancer science over the past decade have been the result of such efforts.

Soon is never soon enough, but with concerted efforts and advanced technologies, progress will continue.

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Since 1994, the National Cancer Institute has written an exclusive report for Coping’s July/August Celebration issue.

This article was published in Coping® with Cancer magazine, July/August 2012.