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FDA Science: Protecting America's Health

By Linda Bren

Science and sound regulation are inseparable at the Food and Drug Administration. Science is the foundation for daily decisions on a wide range of products that affect human health--from the most common food ingredients, to complex medical and surgical devices, to lifesaving drugs and radiation-emitting consumer and medical products.

The science behind the regulatory decisions also is wide-ranging. It is not just the solitary investigator at the laboratory bench doing original research. And it's not just the physician asking questions about the safety and effectiveness of new drugs. Nor is it just the inspector assessing the safety of imported foods. Science at the FDA is all of that--and much more.

For those who want to know what science at the FDA is all about and want to meet the scientists who work there, they will have an opportunity in late April when agency researchers and reviewers gather at the ninth annual FDA Science Forum.

More than a meeting, the Science Forum is a training ground, a marketplace for the exchange of ideas, and a venue at which collaborative relationships can be created and nurtured.

As a training ground, the forum helps scientists learn new technologies and methods to better assess the safety and effectiveness of medical products. As a marketplace for ideas, it presents different perspectives on important public health issues, particularly as they might affect scientific and regulatory concerns. And as a venue for collaboration, it encourages scientists to work together toward finding better ways to address the nation's emerging health needs.

But the Science Forum is not just for scientists--consumers can benefit too.

"It's a one-stop shop for consumers to meet FDA research and review scientists from across the agency," says Susan Bond, an FDA senior science policy analyst. "It's an opportunity for consumers to voice their concerns, and for the FDA to increase public awareness about its initiatives and activities. This event is the only one of its kind that has this much openness and opportunity for public participation."

This year's forum, scheduled for April 24-25 at the new Convention Center in Washington, D.C., offers a mix of presentations, scientific posters, exhibitions, and breakout sessions--all of which allow consumers and scientists to exchange ideas and insights. Each presentation is followed by a question-and-answer period, during which attendees can ask scientists about their topics. An exhibition hall is the perfect setting for consumers to speak with scientists whose posters depict the FDA's recent scientific achievements. Industry representatives with their displays of products and services also will be on hand. And the final breakout session will feature a discussion by representatives from a consumer group and the media on how they partner with the FDA to better convey health information to the public.

Among those invited to speak at the forum are Health and Human Services Secretary Tommy G. Thompson, Centers for Disease Control and Prevention Director Julie L. Gerberding, M.D., M.P.H., and FDA Commissioner Mark B. McClellan, M.D., Ph.D. Others scheduled to make presentations include experts from the National Institutes of Health, Environmental Protection Agency, and Department of Defense, as well as scientists from major universities and industry labs.

This year's Science Forum program highlights three priority areas in which the FDA is working to protect America's health: risk assessment and management, novel science initiatives, and science in the aftermath of the events of Sept. 11, 2001.

Risk Assessment and Management

Emerging technologies, such as blood substitutes, genetically modified food and transgenics, translate into products with new complexities and uncertain risks. Part of the FDA scientist's job is to judge whether a new product's benefits will outweigh its risks. "Scientists can never get enough information to answer all the questions about a product or a technology," says Norris Alderson, Ph.D., the FDA's senior associate commissioner for science. Scientists must gather as much information as possible, but also must determine when they have enough data to safely assess risk and deem that a product won't cause harm. "It's a continuing balance," adds Alderson.

Get a first-hand look at how FDA scientists screen chemicals for their cancer-causing potential, assess the risk of foodborne bacteria and viruses, and work to minimize the risk of regulated products and technologies at the Science Forum.

Novel Science Initiatives

As technology moves forward at an extraordinary pace, the science gets increasingly more difficult. FDA scientists face new public health threats, such as tougher strains of antibiotic-resistant bacteria, more dangerous foodborne illnesses, and life-threatening biological weapons that may be used to contaminate products regulated by the FDA.

"Staying current with novel science and technology represents a continuing educational challenge for every scientist at the agency," says Alderson. New technologies affect a product's characteristics, chemistry, mode of action, and the way it's manufactured. "When a new technology comes along, the scientists have to get retooled. They must develop and apply new standards and techniques in reviewing the product."

Learn how FDA scientists use the latest technology to detect cancer in early stages, test for antibiotic resistance, investigate food and drug tampering, and more, at the Science Forum.

Science After 9/11

The events of Sept. 11, 2001, revealed the vulnerability of our nation and underscored the need for countermeasures to terrorist threats. FDA scientists are working to ensure the availability of safe and effective countermeasures. They are researching vaccines and treatments in case of biological attack, developing new methods for detecting toxins in foods, and improving blood collection and testing procedures to ensure the continuing safety of the nation's blood supply.

See what the agency is doing to detect biological and security risks, protect the nation's food supply, and communicate risks to the public at the FDA Science Forum.

For more information on the FDA 2003 Science Forum, including fees and how to register, visit or call 1-757-423-8823. Early registration is recommended to assure seating. The forum will be available by webcast beginning May 16 at the same Web page.

Science in the Aftermath of 9/11

The FDA is used to dealing with emergencies. Every week, FDA personnel investigate threats to public health ranging from foodborne illness to drug tampering to malfunctioning medical gas cylinders. But never has the agency been challenged with a national emergency of the magnitude of the attacks on Sept. 11, 2001.

"Immediately after the World Trade Center and the Pentagon were hit, science at FDA played a role," says Bernard A. Schwetz, D.V.M., Ph.D., the FDA's senior advisor for science. "We immediately thought of the supply of blood."

While firefighters doused flames and rescue workers searched for victims amid the rubble, FDA scientists worked behind the scenes to enable the rapid collection of blood from thousands of people near the disaster sites. "We reviewed which parts of the information collection process and the blood testing process are nice but not essential in these circumstances," says Schwetz. After weighing the risks and benefits carefully, the FDA streamlined the processes and provided guidance to the blood community to help make blood available while still ensuring the safety of the supply. "We were better off tolerating a little more risk at that time to save lives," says Schwetz.

Another demand placed on FDA scientists was the review of drugs and burn wound dressings to determine their usefulness in treating 9/11 victims. FDA scientists, along with those from the Centers for Disease Control and Prevention, also checked mobile hospitals to ensure that they had adequate reserves of drugs and medical supplies that were current and ready for use. The FDA contacted manufacturers to see if reserve products could be transported quickly to hospitals in New York City and Washington, D.C.

"Good science and good judgment were part of responding to the emergency," says Schwetz, who adds that the FDA's administrative component is also a crucial part of emergency response. "In order to have good science be really effective, we've put an administrative process in place that allows us to respond as an agency more quickly in an emergency."

Through participation in simulated disaster exercises, FDA scientists and emergency operations personnel continue to sharpen their response skills and gain experience working in concert with other federal and state agencies to address an emergency.

Schwetz is scheduled to speak at the Science Forum on "Did Events of 9/11 and Later Make Us a Better FDA?"

A New Kind of Science: Bioinformatics

The need to make sense of the cold reality of disease and death is as old as mankind itself. Equally old are the questions: Why are some people more susceptible to disease than others? What is the risk that an individual will get a certain disease? How would a person respond to a particular treatment?

Scientists may be getting closer to the answers. In June 2000, researchers announced the first complete sequencing, or ordering, of the human genome-the total genetic content of a human. With this new knowledge, researchers are learning more about disease susceptibility and treatment responses and are working to develop new drugs and vaccines targeted to individuals based on their genetic code.

Along with the excitement generated by the human genome sequencing is an explosion of data about each of the components of the more than 30,000 genes that are expressed by this genome.

"Only a few years ago, we were evaluating a single gene at a time," says Daniel Casciano, Ph.D., director of the FDA's National Center for Toxicological Research. But scientists can now evaluate tens of thousands of genes at the same time in one experiment by using platforms, or microarrays, consisting of many thousands of genes. Unfortunately, different research laboratories use different microarrays, says Casciano. "Comparing data from these different platforms is a big problem. There's a lot of confusion because we don't have standardized tools that would be useful across any platform."

The need for tools to make sense of all the data has spawned a new kind of science: bioinformatics.

"Bioinformatics involves biology, computer science, mathematics, and statistics to analyze biological sequence data, genome content and arrangement, and to predict the function and structure of molecules," says Casciano. Applying bioinformatics, a team of FDA scientists is working to produce a standardized set of tools that will aid in evaluating biological data. Using computer algorithms, statistical models, and visualization and filtering tools, these scientists are screening out information in the genome that isn't useful ("junk DNA"), reducing the data to useful and manageable information, and portraying it in such a way that it can be interpreted by any scientist anywhere.

These tools will help FDA scientists to interpret and evaluate information from different companies, allowing the FDA to rapidly assess the safety of a new drug.

Casciano will be presenting an introduction to bioinformatics at the Science Forum.

Proteomics and Cancer Detection

Much like a grocery store cashier swipes a cereal box over a simple bar code reader to get a price, FDA scientists send blood and tissue through a sophisticated bar coder to read protein patterns. These patterns are helping researchers to detect early-stage cancers and changes in cancer cells that could aid doctors in choosing an appropriate treatment for their patients.

Using proteomics, the study of proteins in living cells, scientists at the FDA and the National Cancer Institute (NCI) have developed a finger-stick blood test that may help detect ovarian cancer before it has spread outside the ovary. Early detection dramatically increases the chances of survival, but currently available screening methods detect only 10 percent to 15 percent of ovarian cancers at this earliest stage.

The new test requires only one drop of blood to detect specific patterns of proteins that signify cancerous cells. Using a sensitive analytical method called mass spectrometry, the scientists sort the proteins by weight and electrical charge, resulting in what looks like a bar code, or a fingerprint, of each protein.

But whereas a store product bar code may have 30 lines, protein bar codes have hundreds of thousands of lines, says Emanuel Petricoin, Ph.D., co-director of the FDA-NCI Clinical Proteomics Program. Using computers to read the line patterns, scientists can discriminate a cancerous cell from a normal cell.

"We have several test models that have given us 100 percent accuracy in detecting ovarian cancer in small studies of several hundred women," says Petricoin. Thousands more women are being recruited for clinical trials at NCI this spring. "If we get good preliminary data, we'll look at other types of cancers, such as breast and prostate," adds Petricoin. Blood tests for these cancers could revolutionize screening tests, eliminating the need for many unnecessary biopsies.

In addition to detecting protein patterns in blood, FDA and NCI scientists have developed a new type of protein array technology for analyzing the cellular "wiring diagram" from only a few hundred cells. Using a laser microscope they developed to pluck cells from tissue and magnify them, the investigators can analyze the proteins by their protein arrays. "We found patterns that were changing as disease occurs," says Petricoin. These protein patterns also change when different drugs are used, which may one day help doctors determine which drugs and doses of those drugs might work best in treating individual cancer patients.

"For the first time, we can now gain insights into the functional state of the cellular circuitry in patients' tissue before, during, and after therapy," says Petricoin. This work is now leading to NCI-based clinical trials where proteomic tools are being used and evaluated in the clinic. "While DNA is the information archive, it is the proteins that do the work and are the targets for therapy," Petricoin adds. "The critical information we get here cannot be predicted by gene arrays, so this technology was sorely needed."

Petricoin will be leading the breakout session, "Serum Proteomic Pattern Diagnostics Using Artificial Intelligence Based Bioinformatics" at the Science Forum.

Blood Substitutes

Blood can be a life-saving fluid for trauma victims, heart surgery patients, organ transplant recipients, and many others. An average of 34,000 units of red blood cells are needed each day in the United States, according to the American Association of Blood Banks, and the demand for blood continues to increase.

Researchers have been searching for an effective blood substitute for more than half a century, and they appear to be getting closer to finding one. FDA scientists are currently reviewing several chemically and genetically modified hemoglobin solutions at different stages of development. Hemoglobin is the protein in red blood cells that carries oxygen throughout the body and gives blood its bright red color.

"These unique products present a research and regulatory challenge because there is no precedent and there has been very little research done in the field," says Abdu Alayash, Ph.D., a research chemist in the FDA's Center for Biologics Evaluation and Research. Human hemoglobin is encapsulated within red-cell membranes, unlike hemoglobin-based blood substitutes, which are derived from human or bovine outdated blood and are then chemically modified. "Since the hemoglobin is not encapsulated in protective gear, we must ensure that it's not toxic to tissues," says Alayash. Only a few animal species, such as the earthworm, have hemoglobin in free form, he adds.

For the last 12 years, FDA researchers have been studying hemoglobin toxicity. "We have defined several pathways of toxicity and we have suggested ways and means of controlling it," says Alayash. Through continued research and collaboration with other scientists, FDA researchers are contributing to the design of safe and effective blood substitutes.

Alayash will be leading the breakout session, "Blood Substitutes: Can We Tame Hemoglobin?" at the Science Forum.