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Transforming Discovery into Health, From NIH Director Dr. Francis S. Collins

Dr. Francis S. Collins, M.D., Ph.D., Director of the National Institutes of Health

Testifying recently before Congress, Dr. Francis S. Collins, M.D., Ph.D., Director of the National Institutes of Health, described advances in cancer therapy and obesity, and the future direction of biomedical research.

Science is not a 100-yard dash. It is a marathon — a marathon run by a relay team that includes researchers, patients, industry experts, lawmakers, and the public. Although we have accomplished much, biomedical research still has an enormous amount of ground to cover before discovery is turned into health for all Americans.

Meeting the Challenge of Cancer

Cancer still claims the lives of more than 500,000 Americans annually — about one every minute. But in 2007, for the first time in our nation's history, the absolute number of cancer deaths in the U.S. went down. And, over the past 15 years, cancer death rates have dropped 11.4 percent among women and 19.2 percent among men, which translates into some 650,000 lives saved — more than the population of Washington, D.C. These are very encouraging milestones, but they are not nearly enough.

Revolution in Cancer Research

NIH-funded research has revolutionized how we think about cancer. A decade or two ago, cancer diagnosis was based on the organ involved and treatment depended on broadly aimed therapies that often greatly diminished a patient's quality of life. Today, basic research in cancer biology is moving treatment toward more effective, less toxic therapies tailored to the genetic profile of each patient's cancer.

Breast Cancer Success

Among the early success stories in this area is the drug trastuzumab (Herceptin) for breast cancer. An NIH-sponsored clinical trial found that when breast cancer patients whose tumors were genetically matched to trastuzumab received the drug, along with standard chemotherapy, their risk of cancer recurrence fell 40 percent. This is the best improvement ever reported in post-surgical treatment of breast cancer.

Personalizing Cancer Treatment

To accelerate development of more individualized strategies for more types of cancer, NIH has launched The Cancer Genome Atlas (TCGA). Over the next few years, TCGA researchers will build comprehensive maps of the key genomic changes in 20 major types and subtypes of cancer. The resulting information is being made rapidly available to the worldwide scientific community. It will provide a powerful new tool for developing better ways to diagnose, treat, and prevent cancer. Already, TCGA has produced a comprehensive molecular classification system for ovarian cancer and glioblastoma, the most common form of brain cancer.

"Today, basic research in cancer biology is moving treatment toward more effective, less toxic therapies tailored to the genetic profile of each patient's cancer."

Taking on Obesity

More than one-third of adults in the U.S. are obese, according to the latest data from the Centers for Disease Control and Prevention (CDC). And there are signs that the next generation may face an even greater struggle. Since 1980, obesity has more than doubled among U.S. children ages two through five, nearly tripled among young people over age six. This translates into tens-of-millions of Americans who are at increased risk for type 2 diabetes, as well as cardiovascular disease, high blood pressure, certain cancers, osteoarthritis, and other serious health problems associated with excess body fat.

To address our growing national obesity, NIH has undertaken a variety of innovative approaches for weight control. One is the National Collaborative on Childhood Obesity Research, which has pulled together experts from four NIH institutes, the CDC, and the Robert Wood Johnson Foundation. They have begun the Trial of Activity for Adolescent Girls, a national study to develop and test school- and community-based interventions to get girls more involved in gym class, organized sports, or recreational activities.

Another NIH program, called We Can! (featured on page 6), provides families with practical tools for weight control at more than 1,000 community sites nationwide. How to get more people to lose weight is also among the questions being explored by OppNet, a new trans-NIH behavioral and social sciences research initiative.

Shortening the Pathway to Health

Whatever the disease, be it depression, diabetes, or something much rarer, NIH's emphasis will be on translating basic discoveries into new diagnostic and treatment advances in the clinic. For many disorders, there are new opportunities for NIH to shorten and straighten the pathway from discovery to health.

This expectation is grounded in several recent developments: the dramatic acceleration of our basic understanding of hundreds of diseases, the establishment of NIH-supported centers that enable academic researchers to use such understanding to screen thousands of chemicals for potential drug candidates, and the emergence of public-private partnerships to aid the movement of drug candidates identified by academic researchers into the commercial development pipeline.

Envisioning the Future

In the world I envision just a few decades from now, we will use stem cells to repair spinal cord injuries; bioengineered tissues to replace worn-out joints; genetic information to tailor health outcomes with individualized prescriptions; and nano-technology to deliver therapies with exquisite precision. I also dream of a day when, in ways yet to be discovered, we will be able to prevent Alzheimer's, Parkinson's, and other diseases that rob us much too soon of family and friends.

Spring / Summer 2010 Issue: Volume 5 Number 2 Pages 2 - 3