BIOTECHNOLOGY AND HEALTH CARE

Biotechnology has thus far had its greatest impact in the area of human health through the development of new pharmaceuticals, diagnostics and other medical products.

During the past few years a number of new biotechnology-produced pharmaceuticals have been introduced.

Vaccines for hepatitis

Antianemia drug

Progress in the science underlying these advances will ensure that biotechnology's impact on health will continue during the next decade. Prospects include new drugs, diagnostics and vaccines as well as gene therapy. In the future we can expect specific cancer vaccines as powerful weapons against this deadly group of diseases, as well as cures for many inherited diseases based on the use of genetically engineered human cells.

Equally important, biotechnology is likely to lead to significant reductions in health care costs, particularly through advances in prevention, early diagnostics and treatment.

A remarkable number of Federal agencies, recognizing biotechnology's potential for wide-spread fundamental breakthroughs, have initiated or expanded biotechnology research effort in support of their respective missions:

Department of Agriculture (USDA)

Department of Commerce (DOC)

Department of Defense (DOD)

Department of Energy (DOE)

Department of Human and Health Services (DHHS)

Department of Interior (DOI)

Department of Justice (DOJ)

Department of Veterans' Affairs (DVA)

Agency for International Development (AID)

Environmental Protection Agency (EPA)

NASA

National Science Foundation (NSF)

Research in the area of health is directed towards improving, restoring and preserving human health.

The contributions of biotechnology to health care are extremely diverse, ranging from elegant techniques of molecular biology used to diagnose disease to the development of more rational approaches for designing new therapeutic agents. As a result of the demonstrated applications of biotechnology, molecular mechanisms of disease can now be elucidated, diagnostic methods improved, therapeutic modalities increased, and safer, more effective vaccines developed.

An understanding of the molecular basis of disease represents the ultimate answer to the fundamental question of disease causation, and by implication ultimate specific target for disease prevention and treatment.

Applications of rDNA technology, Monoclonal antibodies, the production of transgenic animals, gene amplification using the polymerase chain reaction, and the new methods for protein production and purification can all now be used to improve the prevention, diagnosis and treatment of disease.

Examples of applications of biotechnology to molecular medicine:

- Gene manipulation to construct vectors for targeted gene delivery

- Characterization of molecules such as hormone receptors

- Studies of mechanisms of cellular communication

- Development of diagnostics such as molecular probes for diseases

In the area of therapeutics, recombinant DNA technologies make possible new classes of agents used as drugs; in particular there are proteins such as cytokines and growth hormones. Equally important is the development of monoclonals which can be targeted to tumor cells or infectious organisms. There are also rapid and exciting advances in the creation of chimeric toxin molecules which can be directed to kill specific cell populations such as cancer cells, while sparing normal cells Gene therapy is now a reality with five human trials under way and more to follow in the near future. While the immediate focus is on diseases caused by a defect in a single gene. The techniques of gene transfer can be used to augment drug delivery systems and target gene delivery cells such as skin or liver cells.

Drug discovery is being enhanced by the use of molecular modeling and the development of cell receptor based agents. With the discovery that the genes associated with cancer are also present in non-humans, there has been considerable effort and real promise in using laboratory organisms such as yeast to screen various types of anticancer drugs.

The use of biosensors to measure metabolites, enzymes, and other cellular products in small biological samples is increasing. Some types of sensors can generate an electronic signal in response to minute quantities of sugars. This property suggests applications for drug delivery involving insulin, for example.

New techniques are emerging for constructing artificial organs. For example, efforts are underway to improve methods of culturing human liver cells and placing them in a matrix that could serve as an "artificial liver." Similarly, there are important advances in developing artificial blood and artificial platelets.

Vaccines: Advances in molecular biology now provide unique opportunities to produce safe and effective vaccines by rDNA technology. Vaccine development has targeted diseases for which current vaccines have proven ineffective because of genetic changes in prevalent circulating strains. New research is directed toward developing new kinds of improved vaccines (polysaccharide-protein conjugates, immunogenic proteins) for preventing bacterial diseases. Another high priority is the development of genetically engineered vaccines against HIV, Hepatitis A and C and other viruses.

In 1993 The Federal Government invested ~$1,680 million in health related biotechnology research and development.

Providing good health for all is the aim of every nation in the world especially in U.S. One dollar invested in vaccines saves $10 in future costs of health care.

Prevention of disease utilizing new and improved vaccines will prevent the need for hospitalization and may prevent death.

Some Products:

Natural Therapeutics Development: Proteins that are found in living systems. It is believed that therapy based on such products will result in more specific and effective treatment, with fewer side effects, than conventional drugs can provide.

Therapeutic proteins are divided into several categories:

Hormones

Lymphokines

Blood Proteins

Vaccines

Miscellaneous

Examples: Tissue-type plasminogen activator (TPA)

A blood clot dissolving agent used to treat heart attacks and other clot related disorders such as deep vein thrombosis. The time of application is critical as the product appears to be ineffective 4-6 hours after the onset of symptoms. In the U.S. there are approximately 1.5 million heart attacks each year, half of which are treated in the hospital. Treatment costs $1,500-2,000.

Interleukin-2 (IL-2)

Stimulates production of T-cells in the immune system and helps certain T-cells destroy foreign matter such as tumors. In U.S. approximately 900,000 new cases of cancer are reported each year with a pool of nearly 2 million cancer victims living at any given time. About 1/3 are estimated to have a response to IL-2.

Factor VIII

Hemophiliacs with type A disease are deficient in the blood coagulation agent called factor VIII. Injection of the factor can help reduce bleeding episodes. In the U.S. there are approximately 20,000 hemophiliacs receiving facto VIII with another 30,000 living outside U.S. There are over 450,000 additional hemophiliacs who could use factor VIII.