&ER& United States Environmental Protection Agency Office of Exploratory Research Washington DC 20460 Research and Development EPA-600/S8-81-020 Dec. 1981 Project Summary Assessment of Future Environmental Trends and Problems: Industrial Use of Applied Genetics and Biotechnologies Robert H. Zaugg and Jeff R. Swarz This study represents a portion of an overall EPA/ORD assessment of future environmental trends and prob- lems. The focus of this summary is the industrial use of applied genetics. The pharmaceutical, chemical, energy, mining, and pollution control indus- tries are examined. Following a brief historical review of the important developments in basic biological research that heralded the advent of modern biotechnology, the summary describes the variety of experimental and commercial tech- niques encountered in this field. These methods include recombinant DNA technology, mutagenesis, cell fusion procedures, immobilized bioprocesses, and fermentation technology. The section entitled "Interested Parties" lists those who are actively involved in the commercialization of applied genetics. Over 100 U.S. busi- ness firms and about 50 foreign concerns are identified as having substantial commercial interest in biotechnology. This section also describes the role of U.S. government agencies in examining progress in this field. Much of the summary consists of an industry-by-industry analysis of current R&O activities in biotechnol- ogy, an estimate of future prospects. and an assessment of potential environmental and health hazards associated with these activities. Trends are identified and, wherever possible, schedules for the appearance of new applications are predicted. The final section summarizes the findings and makes recommendations to the EPA regarding future action in the field of applied genetics. This summary is submitted in fulfill- ment of Contract No. 68-02-3638 by Teknekron Research, Inc., under the sponsorship of the U.S. Environ- mental Protection Agency. This summary covers the period October 1, 1980 to February 28, 1981 and the work was completed as of March 31, 1981. This Project Summary was develop- ed by EPA's Office of Strategic Assessment and Special Studies, Office of Exploratory Research, Washington, DC, to announce key findings of the research project that is fully documented in a separate report of the same title (see Project Report ordering information at back). Introduction Applied genetics has recently emerged as an exciting technology that: ------- (1) may contribute significantly to the solution or alleviation of world-wide problems such as dwindling food and energy supplies, infectious diseases, cancer and environmental pollution; (2) has stimulated over one billion dollars in capital investment by private business in the United States; and (3) has occasioned a heated and contro- versial debate within academic and government circles regarding issues of safety, academic freedom, and societal ethics. These events have arisen largely from scientific advances made in basic biological research during the past 10 years. Scientists are now able to manipulate the genetic material (DNA) of living cells, thus making possible the transfer of specific genes from the cells of one organism to the cells of another. Consequently, for the first time, man is able to make fundamental changes in the inherent structure of living things, thereby endowing organisms with characteristics that were previously unavailable to them. This new technology, referred to as "recombinant DNA," is the most pub- licized and controversial issue in the applied genetics field, but it represents just one of several biological techniques that are increasingly being applied to commercial processes in the industrial sector. This summary describes these techniques and summarizes their appli- cation to industrial processes in phar- maceuticals, chemicals, energy, mining, and pollution control. Future uses of these technologies are also projected within each industrial sector and, most importantly, potential environmental and health hazards associated with either current or future applications of biotechnology are identified. Finally, the EPA is provided with several recommendations as to those areas requiring additional study, as well as the advisability of promul- gating regulations designed to oversee commercial activities. Technology of Applied Genetics The technology encompasses a variety of procedures and processes. • Non-recombinant DNA methods for inducing genetic alterations in cells include: (1) mutagenesis, in which physical or chemical agents (mutagens) induce mutations in the DNA; (2) cell fusions, in which two cells of differing types are fused together into a single unit that manifests characteristics of both parental cell types; "hybrid- omas" that produce monoclonal antibodies represent a very useful application of this technique; and (3) simple genetictransformations of cells can be accomplished by merely exposing the cells to puri- fied DMA which, under appropriate conditions, is taken up by the cells and incorporated into cellular DNA. • Immobilized bioprocesses refer to various methods of confining, or immobilizing, intact cells or cellu- lar enzymes within an inert matrix, followed by passage of substrate materials through this "bioreactor." Applications of this technology will become popular in all industrial sectors; some pollu- tion control practices already employ versions of this technology, such as rotating biological discs and trickling filters for waste water treatment. • Fermentation technology, although practiced for centuries, will be increasingly in demand owing to the requirement of mass-producing microorganisms and microbial products. Traditional batch fer- mentations will be supplanted increasingly by modern fermenta- tion techniques, such as continuous flow and solid phase processes. • Gene therapy in humans, al- though still highly experimental, may soon permanently cure tragic genetic diseases such as sickle cell anemia and thalassemia, by providing patients with DNA that replaces the defective genes. Interested Parties The immense excitement generated by applied genetics arose from findings made over the past decade in university laboratories. These scientific advances quickly burgeoned into a rapidly growing multi-million dollar industry. Meanwhile, various government agencies have become interested in this area owing in part to concerns about the overly fast commercialization of tech- nologies whose safety has not been established absolutely. University labs have provided most of the fundamental advances in both the science and engineering aspects of bio- technology. Many university scientists have become affiliated with genetic engineering firms. This situation has occasioned some rivalry among univer- sity scientists who now view their research as potentially lucrative. As a result, the qualities of cooperation and intercommunication that once charac- terized academic research have been seriously compromised. Over 100 U.S. companies are engaged in some aspect of modern applied genetics. Many large corpora- tions, particularly those that emphasize research and development, have initi- ated in-house programs in biotechnol- ogy. These include virtually all pharmaceutical firms, most energy and chemical companies, and several agriculture and food product firms. Meanwhile, dozens of new, small genetic engineering firms have sprung up in recent years. These entrepreneur- ial ventures, which are exemplified by Cetus Corp. and Genetech Inc., have used risk-taking investors for initial support but can now count on revenues generated through research contracts negotiated with large companies, such as Dow Chemical, Eli Lilly, and Hoffmann- LaRoche. The federal government became involved with applied genetics primaril} in response to concerns, first expressec by research scientists in the mid 1970's, that the continued use o recombinant DNA techniques couU produce new organisms that migh escape from the laboratory am endanger the human population and thi environment. The National Institute a Health (NIH) has set forth recommendei procedures for constructing and hand ling recombinant DNA molecules These guidelines are mandatory fo federally-sponsored research and ar voluntary for commercial firms engage in recombinant DNA research an development. Since their first publice .tion in the Federal Register in Summe 1976, the guidelines have bee amended considerably and now reflei a more confident and relaxed attituc about the potential risks inherent i these activities. The most recent versic of the guidelines appeared in tr November 21, 1980, issue of tr Federal Register. Other federal agencies involved wit recombinant DNA issues include: ------- • The Food and Drug Administration (FDA), whose interest stems from the fact that the first commercial products emerging from this new technology are intended for human use; namely, human insulin, growth hormone, and interferon. As of June 1980, the position of the FDA was that drugs produced by recombinant DNA techniques could not be marketed under existing INDs or NDAs as simply changes in manufacturing technique. • The Occupational Safety and Health Administration (OSHA), which announced (during the Carter Administration) that it will develop a recombinant DNA regulatory policy over the next two years. • The National Institute for Occu- pational Safety and Health (NIOSH), which is interested in examining several worker safety issues related to commercial recombinant DNA activities. The question of patent protection for products and processes evolving from applied genetics research and develop- ment is both controversial and important to commercial firms anxious to protect their investment. Two recent events are noteworthy. • On June 16, 1980, the Supreme Court ruled that patent protection cannot be denied io living things provided that the principal criteria for patentability (namely, that the item be new, useful, and non- obvious) are met satisfactorily. • On December 2, 1980, Stanford University and the University of California were jointly awarded a patent dealing with techniques and processes commonly used in recombinant DNA experiments. The protection afforded by this patent will permit the universities to license the technology to any company that wishes to employ the techniques (i.e., every firm engaged in recombinant DNA research and development) and they will collect royalties on its use. The practice of applied genetics in foreign countries has generally proceeded in a fashion similar to that in the United States. In contrast to U.S. activities, however, some foreign governments have supplied consider- able financial backing to fledgling genetic engineering enterprises. The governments of both Britain and France have established nationally owned genetic engineering companies. In Japan, more than a dozen large chem- ical and pharmaceutical firms are actively pursuing biotechnology programs with government support. Likewise, private firms in Canada and Israel have undertaken ambitious projects in genetic engineering applied to agriculture, industrial chemicals, and waste management. Industrial Applications, Trends, Potential Hazards Pharmaceutical Industry The largest efforts to date toward commercial application of modern bio- logical techniques have taken place in the pharmaceutical industry. The fol- lowing new or improved drugs and vaccines are likely to be the first marketable products stemming from recombinant DNA technology; • Interferon, a protein made by most cells of higher organisms in response to virus infections, may prove to be a valuable anti-viral drug as well as a potential anti- cancer agent. • Insulin, a hormone made in the pancreas, is a necessity of life for most diabetics. Currently, insulin is obtained from cows and pigs, but recombinant DNA technology will make possible a steady supply of human insulin that will replace the animal insulins as the prefer- red drug for treating diabetes. • Human growth hormone, or somatotropin, is produced in the pituitary gland and mediates growth and stature, particularly in children. Its value as a therapeutic in humans is still speculative, but growth hormone may be useful in the treatment of ulcers, burns, bone fractures, and bone deterior- ation, as well as dwarf ism in children. • Somatostatin is a hypothalamic hormone that may have thera- peutic potential in the treatment of diabetes. • Thymosin, a thymus hormone, regulates the development of a portion of the immune system and may have application in cancer therapy. • Beta-endorphin is a naturally occurring opiate that mimics the action of morphine. It has consid- erable therapeutic potential as a safe, non-narcotic pain-killer. • Urokinase, a kidney enzyme, dis- solves blood clots and may reduce the likelihood of heart attacks and strokes. The second major pharmaceutical area in which recombinant DNA tech- niques are finding considerable applica- tion is in the development of new, safe vaccines. Vaccines to combat the following viral and bacterial disease- causing agents are now under develop- ment: • Hepatitis virus, which causes a serious liver disease that has reached epidemic proportions in some parts of the world. • Influenza virus, the many forms of which have made reliable vaccines unobtainable using con- ventional techniques. • Foot-and-mouth disease virus, which causes a life-threatening disease among domesticated live- stock. • Gonococcus bacteria, which cause a type of venereal disease. • Pathogenic E. coli, which cause severe diarrhea in children. • Oral bacteria, which are respon- sible for tooth decay. Future developments within the pharmaceutical area are likely to be involved with plants and sea creatures as sources of new and powerful drugs, human monoclonal antibodies for use as in vivo diagnostics and therapeutics, and techniques for effectively trans- planting genes into human recipients. ------- This last advance, although 10 to 15 years distant, will bring about many political and ethical issues whose seriousness will outweigh any technical obstacle. Applied genetics, especially recom- binant DNA technology, has been promoted to a greater extent in the pharmaceutical industry than in other industrial sectors. For this reason, the potential risks arising from this new technology have been assessed in the context of laboratory and industrial practices pertinent to the pharmaceuti- cal sector. Several risk assessments have been conducted to evaluate the safety of using £. coli K12 as a host bacterium for the manufacture of human proteins via recombinant DNA techniques. The viewpoints reached by these evaluations are: • It is virtually impossible for K12 to colonize the human gut or to be communicated between individ- uals. • The insertion into K12 of gene sequences from human pathogens would not create a pathogenic K12 strain and would present fewer risks than those pathogens existing freely in nature. • The ingestion of a K12 strain that synthesizes and secretes a human hormone, such as insulin, would not contribute significantly to the hormone levels that occur naturally. • The bacterial synthesis of human proteins in the Gl tract (or elsewhere in the body) would not likely trigger an auto-immune response to the human substance. Apprehensions regarding the use of other microorganisms, such as Bacillus subtilis or Saccharomyces cerevisiae, as host organisms for recombinant DNA have been far less than fears attending the use of E. co//" K12. NIH and NIOSH have examined the issue of worker safety in the pharma- ceutical industry within the context of recombinant DNA activities. NIH has established a set of recommendations for large-scale fermentation of recom- binant DNA organisms with which industrial firms are expected to comply voluntarily. NIOSH has conducted walk- through surveys of two commercial fermentation plants (Eli Lilly and Genetech). Lilly operates an eminently safe fermentation plant, which serves to set the standard of practice in this area. The pharmaceutical area as a whole appears to be well equipped to deal with the experimental and engineering safety issues that are posed by the advent of recombinant DNA technology in particular, and by the various applica- tions of biotechnology in general. Chemical Industry The fruits of applied genetics are less immediate in this industry than in the drug sector, but biological processes and renewable resources will eventu- ally replace the physical-chemical transformations of petroleum feed- stocks upon which the chemical industry is currently based. Some projects underway or planned include: • An immobilized bioprocess for the oxidation of alkenes to their corresponding alkene oxides. • The commercial production of microbial enzymes for use in various industrial processes. These enzymes include amylases used to break down polysacchar- ides for biomass conversions, proteases for use as meat tender- izers, oxidases and isomerases that perform highly specific chem- ical transformations of interest to specialty chemical manufacturers. • The direct production of simple organic compounds by microbial fermentation. These compounds include acetone, acetic acid, butanol, citric acid, ethanol, gly- cerol, isopropanol, lactic acid, methanol, propionic acid, and numerous amino acids. • The production of surfactants or detergents from microbial fer- mentation, particularly from certain species of photosynthetic algae. • The isolation and commercial production of several complex organic compounds from higher plants. Examples include natural rubber from guayule and euphor- bia, terpenoids and insecticides from the juniper and scorpion flowers, and long-chain fatty acids from Lunaria and Vernonia. The future applications of biotech- nology in the chemical industry will reside largely in the manufacture of certain high-priced specialty chemicals rather than in the production of bulk commodity substances. The economics favoring the future use of bioprocess in this industry will depend substantially on process design and engineering characteristics, rather than on the biotechnology involved. Potential risks involved in the use of applied genetics in the chemical industry include the following: • The species of microorganisms likely to be utilized in the chemical industry differ from those in the drug industry. These microbes include Pseudomonas. Acineto- bacter. and Flavobacteria, about which little is known regarding pathogenicity in man. • The chemical industry is unaccus- tomed to the application of biological processes as a business enterprise and individual firms may be unaware of potential hazards inherent in these activ- ities. • The chemical industry has a poorer record than the pharmaceutical industry in areas related to worker safety and envi- ronmental protection, causing apprehension as to the introduc- tion of new technologies for which industry-wide experience is limited. Energy Industry Biological processes may someday supply a major proportion of work energy needs. Current activities in thi: industry, however, are limited to tw< general areas: • The conversion of biomass inu more useful fuels, such as ethane or methane gas. • Microbiologically enhanced oi recovery, in which microorgan isms injected into a spent oil we may serve to rejuvenate the resei voir by degrading a portion of th residual oil (thereby lowering th viscosity), repressurizing the we by producing carbon dioxide ga or by manufacturing chemical su 4 ------- factants that would tightly bound oil. mobilize The range of potential uses of biotechnology in the energy industry is very wide, although most of these possi- bilities lie far in the future. These prospects include: • The mass-production of hydro- carbons from microbes (such as algae) and from higher plants. • The production of hydrogen gas from water using enzymes iso- lated from plant chloroplasts. • The development of a solar battery in which immobilized photosyn- thetic biosystems mediate a direct conversion of sunlight into electricity. • The use of iron-oxidizing Thio- bacilli to mobilize the inorganic mineral content of oil shale or coal, thereby generating porous zones that may expedite in situ retorting or gasification schemes. The large-scale commercial application of biotechnology to the energy sector is at a very early stage of development. Nevertheless, some speculations can be made regarding potential hazards of these developments. • The production in the U.S. of suf- ficient ethanol to have significant impact on domestic fuel supplies will require the diversion of enormous quantities of food crops, particularly corn, for use as biomass feedstock. • The use of cellulosic feedstock in ethanol production may entail large-scale deforestation. • The use of municipal or agricul- tural wastes as raw materials for ethanol or biogas production may entail risks associated with the transport of these wastes to a central fuel generating plant. • The species of microorganisms likely to be utilized in the energy sector for enhanced oil recovery schemes (Pseudomonas and Acinetobacter, for example) are potentially serious pathogens in man. • Established energy companies are not accustomed to dealing with biological systems as a means of producing energy and, thus, may be unaware of potential hazards resulting from their use. Mining Industry The impact of biotechnology in the mining industry is currently quite limited in scope, consisting largely of bacterial leaching operations in which metals are solubilized from low grade ores by acidophilic, iron-oxidizing bacteria of the Thiobacillus, Leptospiril- lum, and Sulfolobus genera. Approxi- mately 12% of U.S. copper production stems from bacterial leaching. In addi- tion, these microbes are useful in leaching any mineral containing adequate quantities of reduced sulfur, or sulfide, such as pyrite (FeS2>, chalco- pyrite (CuFeS2), and zincblende (ZnS). Bacterial leaching is also used extensively to recovery uranium from ores that are rich in pyrite. , Microorganisms of numerous types are capable of accumulating metal ions from dilute solutions. This activity can be exploited to concentrate valuable metals from tailings ponds or waste streams. Genetic engineering applied to leaching bacteria may be used to increase the microbe's resistance to the high concentrations of metals being leached, to improve the efficiency of the leaching process, or to develop anaerobic strains of leaching bacteria to permit their use in the interior of huge slag heaps of low-grade ore where oxygen-free conditions exist. The limited scope of biotechnology in the mining industry confines the range of environmental concerns that demand consideration. However, all foreseeable applications of biological processes in this industry involve microbial systems operating in relatively open environ- ments, such as slag heaps or tailings ponds. Consequently, there are risks that microorganisms or their metabolic products will inadvertently contaminate the local ecology. Specifically: • Bacterial leaching operations generate large quantities of sul- furicacidwhichcouldcontributeto the acidification of U.S. fresh water supplies. • Thiobacilli and related species are not known to be pathogenic in man, but their increased use and greater exposure to human popu- lations may select for bacterial strains that have acquired the ability to infect humans. • The use of bacteria to concentrate metals from dilute mine waters entails the risk that such metals will accumulate in the food chain. • The mining industry has very little experience with biological pro- cesses. This lack of familiarity could result in a failure to recognize impending environ- mental hazards or in an eagerness to carry out biological processes before their safety has been firmly established. Pollution Control Industry Bioprocesses have long been instru- mental to the practice of waste manage- ment, but modern advances in applied genetics may greatly facilitate the remediation or elimination of some present-day, intractable pollution prob- lems. Current activities in the field of biotechnology applied to pollution control fall under three general head- ings: (1) biodegradation of organic sub- stances; (2) biological denitrif ication and desulfurization; and (3) biological concentration of toxic heavy metals. Microbes capable of degrading various organic pollutants, such as petroleum hydrocarbons, pesticides, herbicides, and lignocellulosics, have been identified and are under investiga- tion. A better understanding of the biochemical nature of these organisms will be required befaregeneticengineer- ing can have an impact in this area. Microbial systems capable of metabolizing sulfur-containing and nitrogen-containing inorganic and organic substances have also been identified. These organisms will find considerable utility in eliminating these pollutants from industrial waste streams and from fossil fuels prior to combustion. The greatest research and development effort involving near-term applications of biotechnology to pollu- tion control will be in developing improved microbial strains for decon- tamination of polluted waste waters and for in situ detoxification of contaminated soils and sediments. However, a better understanding must be acquired of the types and activities of microorganisms capable of degrading toxic chemicals. ------- The follow! ng list outlines some aspects of applied genetics and waste manage- ment that will be under development. • Cataloging the types of chemical transformations performed by microbes. • Isolating and characterizing the genetic material and enzymes responsible fortheobserved trans- forming activity. • Conducting genetic engineering on organisms that occur naturally in a particular polluted environ- ment in order to improve the effi- ciency or survivability of the organisms. • Developing specific biotreatment systems for dealing in situ with toxic wastes. * • Designing bioreactors for on-line waste stream treatment that will reduce or eliminate toxic wastes at their source. Conclusions and Recommendations The U.S. economy is on the verge of a "biology boom." Excitement over the commercial potential of genetic engi- neering has been very high, as exempli- fied by the considerable media attention to this area, as well as the enthusiasm shown by financial investors. One aspect of applied genetics, recombinant DNA technology, has received the bulkof public attention, in regard to both the favorable and hazardous results of its application. But the risks inherent in the practice of recombinant DNA techniques are surely much fewer than originally feared. As with other technological advances, biotechnology will be applied where it will yield substantial commercial pay- off. Only the pharmaceutical industry is likely to realize near-term returns on investments in this new technology. Other industrial sectors will thoroughly investigate naturally occurring biologi- cal systems for potential commercializa- tion prior to making significant investments in recombinant DNA technology. Biotechnologies other than recombi- nant DNA have received less public attention but, nevertheless, are expected tocontribute to the commercial success of the "biology business." Modern fermentation technologies will be applied to relevant operations in all industrial sectors, as will immobilized bioprocesses, such as on-stream bioreactors for waste stream detoxifica- tion. Cell fusion techniques will undergo further development as an alternative to recombinant DNA methods for pro- ducing genetically altered organisms. Specific recommendations to the EPA are as follows: • Any environmental risks arising from industrial uses of applied genetics are speculative. At this time, there exists no compelling reason for the EPA to establish regulations in this area. • Should environmental hazards emerge in the future, it is probable that they can be handled within the existing regulatory framework (most notably Toxic Substances Control Act). • TheEPAshouldcontinuetotakeaTi active role in promoting applied research and development of biological waste management processes and techniques, with particular emphasis on acquiring a better understanding of the biology of relevant systems rather than on process design and engineering. • The EPA should sponsor further investigation into the generation, dispersal, and control of biological aeosols. aerosols. • The EPA should endeavor to monitor commercial and scientific developments in the field of applied genetics with the aim of identifying both imminent envi- ronmental hazards and areas where this technology might be applied to pollution control problems. ------- Robert H. Zaugg and Jeff R. Swarz are with Teknekron Research, Inc.. 1483 Chain Bridge Road, McLean, VA 22101. Morris A. Levin is the EPA Project Officer (see below). The complete report, entitled "Assessment of Future Environmental Trends and Problems: Industrial Use of Applied Genetics and Biotechnologies, "• (Order No. PB 82-118 951; Cost: $15.00, subject to change) will be available only from: National Technical Information Service 5285 Port Royal Road Springfield, VA 22161 Telephone: 703-487-4650 The EPA Project Officer can be contacted at: Office of Strategic Assessment and Special Studies Office of Exploratory Research U.S. Environmental Protection Agency y Washington. DC 20460 •A- US GOVERNMENT PRINTING OFFICE, 1982— 559-092/3393 ------- ., . _ f _ „ Postage and United States Center for Environmental Research pees paj(j Environmental Protection Information Environmental Agency Cincinnati OH 45268 Protection Agency EPA 335 Official Business Penalty for Private Use $300 ------- |