Biotech Revolution

Challenges for the 21st Century
by Food and Drug Administration (FDA)

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The CBER's major challenge for the 21st century is to expedite approval of biological products for use by the public while, at the same time, maintaining high levels of safety and quality.

"So many of our products are important for new emerging threats," says Goodman, "especially infectious diseases such as pandemic flu, West Nile Virus, SARS, antibiotic-resistant tuberculosis, and the agent that causes the human form of mad cow disease, variant CJD. There's an incredibly important need for us to be prepared for these threats and to help get products developed rapidly to be able to meet them." The CBER is currently overseeing clinical studies for new vaccines to protect against HIV, hepatitis, WNV, and bird (avian) flu.

"We're no longer just talking about the United States of America," adds Goodman. "Infectious diseases do not respect national boundaries. The world needs to face these emerging threats, and the world needs to leverage resources to meet them." Drugs and biologics, including vaccines for use in the United States, are increasingly made all over the world, he says. The CBER continues to work with the World Health Organization and other international bodies to foster safety and availability of vaccines, blood, and tissues.

In the past, the CBER's role in regulating vaccines against smallpox helped to eradicate this dread disease. Now, with the potential threat of a biological attack, the CBER's attention is turned once again to smallpox. The CBER's work with manufacturers and the CDC has boosted the national stockpile of investigational smallpox vaccine by hundreds of millions of doses. The CBER is also aiding the development of new-generation anthrax vaccines.

"We need to be ready not only to produce vaccines that can prevent these threats, but to preserve the safety and integrity of blood and blood products," says Goodman. "It's a huge challenge we live with every day." The CBER has taken strong measures to ensure that blood and tissue products continue to be safe, and is helping to produce diagnostic tests to detect bioterrorist agents in blood donations.

The rapidly advancing field of biotechnology presents additional challenges for the CBER. New technologies involving genetically manipulated cells introduced into the body offer the potential to fight disease, restore normal function, repair injuries, replace lost cells, or regenerate failing organs. Stem cell-based treatments could someday repair many different body tissues. And gene therapy products can replace faulty or missing genetic material, potentially treating or curing a disease. The CBER must continue to make sure that these products are as safe as possible while studies of these promising therapies continue.

The study of gene structures (genomics) and proteins in living cells (proteomics) is leading to potentially effective treatments for a variety of serious diseases, including cancer, diabetes, and heart disease. The CBER is collaborating with the NIH on proteomics research that has the potential to provide early diagnosis of disease and early warning of drug toxicity. This work could potentially revolutionize cancer detection and care.

The need for human organs for transplantation far exceeds the supply. Xenotransplantation, the transplant of animal cells, tissues, or organs into a human, offers new hope for an added source of organs. The CBER must advise researchers conducting clinical trials involving xenotransplantation while safeguarding transplant recipients from new infectious agents that animal tissues and organs may harbor.

CBER scientists must continually improve their scientific expertise in order to ensure the safety and effectiveness of new products. "Some products don't fit in the traditional box," says Goodman. "They are not a drug or a biologic or a device. They are oriented toward individual patients or treatment for a rare disease. We are called upon to develop innovative ways to review and to regulate these innovative products."

While keeping pace with cutting-edge science, the CBER must also keep the public informed of new medical products and emerging threats. "It's important to communicate health information and the potential benefits and potential risks of a treatment or product," says Karen Midthun, M.D., the CBER's deputy director for medicine. "We need to get the public's input and we need to address their concerns," she adds. "We must be able to give them the information they need so they can think through the benefits and risks and make the best choices for themselves and their families."

The Center for Biologics Evaluation and Research (CBER) regulates

• more than 14 million units of blood and blood components transfused each year in the United States
• more than 235 million vaccinations given each year to prevent serious diseases
• more than 1 million human tissues transplanted each year to repair injury, restore function, and improve quality of life
• more than 800 active human trials studying experimental cell, gene, vaccine, or blood products for serious diseases such as AIDS, cancer, diabetes, and heart disease.

Legal Action Against Blood Banks

The first prosecution of a licensed blood bank occurred in 1962. The Division of Biologics Standards, the predecessor to the FDA's Center for Biologics Evaluation and Research (CBER), brought suit against John Calise and the Westchester Blood Bank in New York for altering the expiration dates on blood. Calise pleaded guilty and, in 1964, was convicted on three counts of misbranding, three counts of false labeling, two counts of shipping an unlicensed biological product, and one count of conspiracy. He was placed on probation for five years and was forbidden to take part in the manufacture, distribution, or sale of any biologics, including blood products. Today, the CBER develops and enforces quality standards and regularly inspects blood establishments.

Screening Blood for HIV

The appearance of AIDS in the United States in 1981 threatened the safety of the U.S. blood supply. The human immunodeficiency virus (HIV) that causes AIDS is found in the blood of people with the disease, as well as in the blood of those who have been exposed to the virus but who are not yet ill. HIV was not identified as the cause of AIDS until 1984. In 1985, the CBER licensed the first test kit to screen donated blood for antibodies to HIV-1, the most common type of AIDS. Since then, screening tests for both HIV-1 and HIV-2 have been continually improved. In 1985, the risk of HIV infection from a blood transfusion was 1 in 2,500. In 2002, it was only 1 in 2 million.

Edible Vaccines

The FDA regulates a variety of vaccine types, and is preparing to regulate new products now under study, such as "edible vaccines." Edible vaccines have some advantages over traditional vaccines. They are unable to cause infection, are relatively easy to produce in large quantities, and are stable during storage. Researchers produce edible vaccines by genetically altering the edible parts of plants. Substances that stimulate an immune response (antigens) to rabies have been produced in tomato plants, and antigens to hepatitis and cholera have been produced in potatoes. Bananas are being investigated as possible vaccine delivery foods because they can be eaten raw and appeal to children.

About the Author

www.fda.gov
FDA is A United States government body that oversees medical devices, including contact lenses, intraocular lenses, excimer lasers and eyedrops. In the US, these products must be approved by the FDA before they can be marketed.