NIH Director Francis Collins spoke at a Newsmaker Briefing at Health Journalism 2011.
By Jeanne Erdmann
Independent journalist
At the start of his Newsmaker Briefing at Health Journalism 2011, Francis Collins, M.D., Ph.D., director of the National Institutes of Health, told a tale of discovery. The tale began with an observation made in one HIV-positive person who was cured of the virus following a bone marrow transplant for leukemia. The tale continued with the discovery that a rare gene variant may protect people from AIDS and ended with a small trial of HIV-infected people in Philadelphia using discoveries stemming from that research.
The story underscored Collins's desire for research efforts that bring together an assortment of approaches to help understand health and disease. Knowing the molecular basis for disease offers the greatest potential for therapeutics, he said.
Collins gave many examples of other genetic discoveries that led to advances in, for example, heart disease and breast cancer. Such genomic research is possible because sequencing costs have come down dramatically allowing for an explosion of research findings, especially from The Cancer Genome Atlas project, (a name that Collins said he worked on especially hard so the acronym would use the first letter of each DNA nucleotide: TCGA, for thymine, cytosine, guanine, and adenine). Research coming from this project has already led to a test that tells women with a common type of breast cancer whether they need or don't need chemotherapy. That predictive test, called the Oncotype DX breast cancer assay spares some women the toxicity and cost of chemotherapy and saves the healthcare system about $100 million dollars, said Collins.
"Don't let anybody tell you that new technology always costs more," said Collins.
The NIH hopes to take opportunities from what Collins calls the "golden era of discovery in the molecular basis for disease" and help researchers cross the gap between fundamental knowledge to FDA approved therapeutics. Most potential therapeutics fail in the pre-clinical stage; however, Collins sees that failure as an opportunity for something new and innovative.
For the past five years, the NIH has been helping researchers develop assays for potential therapeutics and then screen those assays for activity through the Molecular Libraries Program, which encompasses four large-scale screening centers. Farther down the therapeutic pipeline, physicians conduct Phase I and Phase II trials at the NIH Clinical Center in Bethesda, Maryland; a 240-beds facility that's also the largest research hospital in the world. There are also 55 clinical centers across the country. "The NIH is not all about test tubes," Collins told attendees.
Another new program aids in clinical pre-clinical animal testing through Therapeutics for Rare and Neglected Disease (TRND); this two-year old program is a congressionally mandated effort to speed development of new drugs for diseases where economic motivation is limited. One of the pilot projects could lead to the first sickle-cell therapy in many years.
The NIH is also partnering with FDA to obtain the regulatory science necessary for FDA to make wise decisions. The NIH and FDA now have a joint leadership council to help get compounds through the pipeline more quickly and to encourage creative clinical trial designs.
Collins is not stopping there. On Oct. 1, the NIH will unveil the National Center for Advancing Translational Sciences, a program he calls innovate and unprecedented." The effort is aimed to reengineer the effort to bring drugs discovered through basic science to the market more quickly and at lower costs.
One worrisome note is the recently approved 2011 budget. The NIH will receive $30.924 billion dollars, which is down 1 percent. This is highly unusual, said Collins, because there was only one time in the last 30 years that the NIH budget has dropped.
Where others see problems, though, Collins will always see opportunity. "We feel that NIH has a great story to tell and we love the chance to tell it."
To Contact Francis Collins:
francis.Collins@nih.gov
John Burklow, Chief of Communication
burklowj@od.nih.gov
o 301-496-5787
c 240-478-3141
