FDA Consumer

Harvesting the fruits of biotechnology

Harvesting the fruits of biotechnology

Frank E. Young

Harvesting the Fruits of Biotechnology

One thing that attracted me to the Food and Drug Administration three years ago was the opportunity to help bring the fruits of the new biotechnology–with which I had been so involved in the research lab–into the marketplace. My whole career was in microbiology and genetics and the exciting research that led to a revolution in cloning techniques. Now, as FDA commissioner, I’m involved in the equally exciting work of overseeing the review of the growing number of biotechnology products as they pass from laboratory to commercial application. It is a rare privilege to have been in on the ground floor of a new field scientifically and now to be responsible for its safe introduction to the marketplace.

The early therapeutic products of biotechnology–the drugs and vaccines approved by FDA–have generally been paragons of purity, safety and effectiveness. And FDA review times have averaged less than half that ordinarily required to approve other new products. A major reason is that FDA reviewers are scientists whose research expertise and interests are closely related to the types of products they review. Since FDA functions as a gatekeeper for a wide variety of biotechnology products, this science-based approach is essential to efficient technology transfer. Because we have the expertise to work closely with industry sponsors and clinical investigators engaged in biotechnology, FDA has been able to actively collaborate in the process of developing safe and effective products.

One question in particular arose early on as FDA prepared to confront the explosion of new products derived through biotechnology: Were new regulations needed? We soon concluded that the answer was no; that the products of biotechnology are fundamentally similar to those produced by conventional techniques, and they are adequately controlled by existing regulations. We do not need new rules. The best course is to judge each product on its own merits.

When we speak of biotechnology, it sounds like something brand new and, possible, foreboding. Actually, biotechnology has been used since the beginnings of civilization. Broadly defined, biotechnology means any technique that uses living organisms (or parts of organisms) to make or modify products, to improve plants or animals, or to develop microorganisms for specific uses. For centuries, simple forms of biotechnology have been used to crossbreed plants and animals and to make cheese, beer and wine.

What’s so newsworthy today are the advances being made possible through the “new’ biotechnology of genetic engineering and monoclonal antibodies. Genetic engineering (a type of biotechnology), also known as recombinant DNA technology, involves manipulating an organism’s genetic material. (Genes are segments of DNA; they are often described as the “blueprint’ for a living organism.) In genetic engineering, genes for specific functions, such as making insulin, are taken from one organism and combined with the genes of a second organism. In this way, microbes such as bacteria can be induced to make human proteins such as insulin or growth hormone. Genetic engineering has also improved penicillin production by the organism Penicillium chrysogenum, resulting in an increased output of pencillin of more than a hundredfold.

Monoclonal antibody production harnesses cells of the immune system–rather than bacteria or yeast–to manufacture medically useful products. An immune cell, stimulated so that it will secrete a specific antibody, is fused with a long-lived cancer cell. The resultant group of hybrid cells, or hybridoma, produces the single desired antibody, and continues to do so indefinitely. This technique permits the production of purer and less expensive materials for research and for diagnostic and therapeutic applications.

Much of the pioneering commercial application of the new biotechnology is taking place in the pharmaceutical, food, and medical device industries–all product areas regulated by FDA. In fact, FDA has already approved many products of new biotechnology, including human insulin, two human growth hormones, two forms of interferon to treat leukemia, and a new vaccine for hepatitis–all derived through recombinant DNA techniques. Also, some 200 monoclonal antibodies are being used as diagnostic tests to detect conditions from pregnancy to venereal disease. Another monoclonal antibody, to reverse kidney transplant rejection, is the first approved for use inside the human body–a major breakthrough.

One of the next breakthroughs in biotechnology may well be in a field called gene therapy. This is a technique that may one day allow doctors to cure inherited diseases, such as sickle-cell anemia or even some forms of heart disease, by replacing absent or defective genes with normal ones. We can also look forward to the possibility of a malaria vaccine achieved through recombinant DNA techniques. One such vaccine is now being tested; if it proves successful, it could alleviate massive human suffering in the less developed nations of the world, where malaria afflicts an estimated 200 million people.

Also of immense benefit to the developing world will be the application of the new biotechnology to revolutionize the way we grow and process foods. Such innovation even has the potential to produce totally new food products. Already, scientists are able to place genes in plants to make them produce more nutritionally complete proteins, like putting a beefsteak into a potato, if you will. Progress has also been made in introducing into various plants resistance to disease, pests, and spoilage after harvest.

The potential for biotechnology to cure disease and feed a hungry world is truly exciting. I believe we are just beginning to gather the first fruits of a harvest whose scope and abundance we can scarcely imagine.

But progress in biotechnology research has been accompanied by fears of unintended or unanticipated harm from genetic engineering experiments. I believe many of these fears have been greatly exaggerated. Experience has shown us that advances in knowledge are worth struggling for and are more likely to produce good than ill. In fact, the precise techniques of biotechnology in many ways ensure that its products are even safer than those produced through conventional methods. For example, genetically engineered human growth hormone has replaced growth hormone derived from cadavers, not only because it is easier and cheaper to produce, but also because it is more pure. Distribution of the cadaver-derived hormone was suspended in 1985 when some batches were thought to have been contaminated with the virus that caused Creutzfeldt-Jakob disease, an extremely rare and invariably fatal infection, in three patients who received the hormone.

The federal government has numerous safeguards to protect the public from any hazards of biotechnology through the existing regulations of FDA, the U.S. Environmental Protection Agency, and the U.S. Department of Agriculture. These agencies have for years overseen the safe use of products derived through the “old’ biotechnology, such as new plant species, animal breeds, and biological pesticides. Bacterial preparations for promoting the growth of soybeans, alfalfa and other legumes have been sold in this country since the beginning of the century. These products contain common Rhizobium bacteria that allow the plants to obtain nitrogen from the air. More than 75 microbial pesticides are approved by EPA, and these organisms are widely used in agriculture, forestry, and insect control. One of these is the Bacillus thuringiensis, more commonly known as milk spore, used to safeguard the nation’s forests from gypsy moths and tussock moths and by homeowners throughout the country against Japanese beetles.

There are innumerable other examples of “releases’ of live organisms into the environment that have been successful, beneficial and safe. For instance, live, weakened, genetically engineered viruses are used in human vaccines for mumps, measles, polio, and yellow fever. These are administered in tens of millions of doses annually throughout the world

The alternative to controlling nature is to allow nature to control us. Surely there can be no reason to prefer malaria to a genetically engineered vaccine; no reason to accept world hunger if we can improve crop yields and nutritional quality through biotechnology. And it’s worth noting that there has not been a single significant safety problem during the more than 10 years that new biotechnological techniques have been used in laboratories or applied by industry.

FDA and other health protection agencies must, and will, of course, acknowledge and respond to legitimate concerns. We must use scientifically sound regulation to govern the use of biotechnology products and to allow the underlying research to proceed. In such a fast-moving environment, our regulation must not to so rigid or excessive that it stifles innovation and blocks the public’s access to the fruits of biotechnology.

Along with its regulatory role, FDA is engaged in an educational effort to make sure the public understands and appreciates the safeguards that are in place for biotechnology products. I encourage others involved in biotechnology research, development and regulation to share in this effort, for–as for any scientific or engineering breakthrough–public understanding is vital to the success of the “gene revolution.’

Photo: Making a hybrid plasmid. To splice a human gene (in this case, the one for insulin) into a plasmid, scientists take the plasmid out of an E. coli bacterium, break the plasmid open at a specific site by means of a restriction enzyme, and splice in insulin-making human DNA. The resulting hybrid plasmid can be inserted into another E. coli bacterium, where it replicates together with the bacterium, making it capable of producing large quantities of insulin.

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