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The Buffalo News

By Peter Pitts

December 24, 2007

Once again, politics seems to have gotten in the way of efforts to save lives. Congress’ latest omnibus spending package blocks the U. S. Food and Drug Administration from transferring any money to a special non-profit foundation aimed at finding new treatment for deadly diseases.
The FDA Amendments Act, which implemented a slew of new policies, stipulates that FDA provide between $500,000 and $1.25 million per year to the Reagan-Udall Foundation, which is designed to modernize drug development and accelerate innovation. But the omnibus appropriations bill passed by Congress on December 20 specifically blocks the agency from transferring any money to the foundation.

Who could be against such advances? Representative Rose DeLauro, a Democrat from Connecticut, for one. DeLauro, who chairs the appropriations subcommittee that funds the FDA, is willing to put politics ahead of public health in her effort to cease work on the foundation because of the pharmaceutical industry’s involvement in it and her concerns that it would unduly influence its work. The foundation was created by Congress to support public-private partnerships that could translate biomarker research into new treatments, and new treatments into hope for patients.

Consider the story of Eric Lester, an oncologist in St. Joseph, Michigan, who was trying to find a way save the lives of his patients with advanced lung cancer. Instead of waiting for someone else to develop diagnostic tools, Lester developed his own test to see what kind of genetic factors were causing the cancer to grow. It turned out that epidermal growth factor receptor was the culprit and that a drug called Tarceva, designed to shut off that receptor, would work.

Instead of waiting to use Tarceva when all else was failing, he decided to use it as part of a tailor-made drug cocktail for his patient based on the information from the diagnostic he developed while staying within the bounds of the FDA. The patient fared better than most of the patients Lester sees.

It’s not a cure, but the results of Lester’s experiment—and others like it—are being shared with academic researchers and drug companies to accelerate the development of cancer drugs tailored to variations of growth factors causing lung cancer. Patients like Lester’s would receive tests that look for biomarkers—biochemical indicators of a disease—to see which drug would work best with fewest side effects. Such collaboration could bring about a new generation of cancer-killing treatments in years.

The Reagan-Udall Foundation was part of the FDA reform bill signed into law recently. The foundation’s mission (under the chairmanship of Mark McClellan) is to provide scientists at the FDA tools for evaluating drugs and devices that are based on the same scientific advances that allowed Lester to determine that genetic variations in people are linked to specific responses to drugs or changes in disease. Most of the work involves setting standards, conducting experiments, and gathering data.

The foundation is designed to refine and standardize the use of new methodologies in the drug development process and find ways to apply them regularly to the prevention and treatment of cancer—just like Lester is doing. But apparently that is the sort watered-down standard Congresswoman DeLauro is protecting us against.

Biomarkers are being improved by genetic science that shows how certain genes trigger disease and drug response.

This will allow scientists to predict how individuals will react differently to treatments. Nearly 90 percent of adverse drug reactions and serious side effects are the result of these genetic "glitches." For instance, most of the dangerous and costly problems associated with antidepressants and blood thinners are the result of genetic variations in dose response to common medicines. Developing regulations for product development around such biomarker-based knowledge is at the heart of the foundation’s effort.

But according to DeLauro, the Reagan-Udall Foundation "has the potential of endorsing the approval of drugs and devices based on lower standards for safety and efficacy, and without appropriately designed clinical trials."

Those who would slow down the work of the Reagan-Udall Foundation are only slowing down the work of real scientists who wish to use sound 21st century science to extend and enrich life—and make drugs safer and more targeted. We cannot afford having 21st century medicine stalled by politics and ignorance. Twenty-first century public health cannot afford to only inherit the wind.

Peter J. Pitts is President of the Center for Medicine in the Public Interest, a former FDA Associate Commissioner, and an original member of the FDA’s Critical Path Taskforce. Dr.Robert Goldberg is vice president, Center for Medicine in the Public Interest.