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TESTIMONY OF ALISON TAUNTON-RIGBY, Ph.D. PRESIDENT AND CEO, AQUILA BIOPHARMACEUTICALS WORCESTER, MASSACHUSETTS ON BEHALF OF THE BIOTECHNOLOGY INDUSTRY ORGANIZATION (BIO) BEFORE THE SUBCOMMITTEE ON TECHNOLOGY COMMITTEE ON SCIENCE U.S. HOUSE OF REPRESENTATIVES LEGISLATIVE PROPOSALS REGARDING CLONING OF HUMAN BEINGS JULY 22, 1997 TESTIMONY OF ALISON TAUNTON-RIGBY, Ph.D. PRESIDENT AND CEO, AQUILA BIOPHARMACEUTICALS WORCESTER, MASSACHUSETTS ON BEHALF OF THE BIOTECHNOLOGY INDUSTRY ORGANIZATION BEFORE THE SUBCOMMITTEE ON TECHNOLOGY COMMITTEE ON SCIENCE U.S. HOUSE OF REPRESENTATIVES LEGISLATIVE PROPOSALS REGARDING CLONING OF HUMAN BEINGS JULY 22, 1997 .... "Cloning Dolly" The procedure used to develop "Dolly" is called "nuclear transfer technology." It is one type of cloning technology which involves transferring the nucleus from a donor cell into an egg from which the nucleus has been removed, and implanting the resultant embryo into a surrogate mother for gestation and birth. What makes Dolly so special is that the cell which supplied her genetic material was taken from a mature adult sheep, cultured in vitro and frozen in liquid nitrogen. This procedure resulted in the birth of a lamb which is a clone of its mother, who was the source of the genetic material. Thus, the lamb's genetic material is identical to the adult sheep, with the exception of mitochondrial DNA which is inherited through the enucleated egg. Dolly is the first sheep to be born using an adult cell and "somatic cell nuclear transfer" technology. It is important to note that this process is extremely difficult and inefficient at this time. This experiment was carried out on 277 eggs before the one success that resulted in the birth of Dolly. We believe that the term, "nuclear transfer cloning," as used by NBAC, represents two different types of procedures. "Nuclear transfer" involves transferring the nuclei from one cell to another; "cloning" is merely copying biological material, such as cells. Nuclear transfer is one type of procedure that results in a clone or copy of an organism. We believe the concerns surrounding the cloning experiment revolve around the application of this technology to human beings, rather than on the types of technologies used to achieve this result. We prefer to precisely identify the type of technology at issue throughout this statement. Cloning of Animals Since the announcement of the birth of Dolly, the sheep produced using nuclear transfer technology with the genetic material of an adult sheep cell, the potential benefits to be derived from cloning procedures in agriculture and laboratory animal species have been widely discussed and recognized. For example, cloning technologies are already used in agriculture to produce higher yield, better quality fruits and vegetables. While livestock animals have been cloned using embryo splitting techniques, the use of nuclear transfer technology is very likely to bring major improvements to the production of transgenic livestock animals. The development of transgenic animals has been occurring for many years. Already, the proteins produced in the milk of these animals are being tested for use in people in clinical trials. Nuclear transfer technology offers the potential for improving the efficiency of developing large numbers of transgenic animals with a wide range of medical benefits. Examples of ways in which nuclear transfer technology could enhance the development of transgenic animals include the following: Animal Models for Testing. Biotechnology companies are developing genetically engineered animal models to test potential treatments for various life-threatening diseases. For example, mice are designed to be susceptible to a specific human disease. Medicines to treat that disease can be tested on these mice, thereby increasing the speed with which human therapeutics can be developed. Through the use of nuclear transfer technology, all-not just a portion-of the animals born would be transgenic. This could significantly enhance our ability to test new treatments for human diseases. Organ Transplantation. Animals are being bred today whose organs may be transplanted into human patients suffering from organ failure or other diseases. These animals have been bred, using transgenic technology, to minimize the risk that the altered animal organ will be rejected by the patient. If a particular animal, such as a pig, is developed which has optimal characteristics as an organ donor, cloning that specific animal will produce identical animals. This could dramatically increase the supply of available organs, including hearts, kidneys and livers, for life-saving transplantation to humans. We are working with the Food and Drug Administration to address concerns regarding this technology. Production of Medicine in Animal Milk. Nuclear transfer technology could enhance the efficient production of human medicines in the milk of transgenic dairy animals, such as sheep, goats and cattle. Today, transgenic technology is used to produce therapeutic human proteins in animals' milk. The therapeutic protein is then extracted from the milk, purified, and used as a pharmaceutical product. Nuclear transfer technology could enable the selective breeding of a large number of identical offspring from a single female animal chosen for its ability to produce higher concentrations of specific proteins in its milk. This could accelerate the production of a herd and facilitate clinical development of useful drugs. In addition, nuclear transfer technology would ensure that, within a herd, each animal would be genetically identical. By eliminating the variability in background genetic factors, this method of producing human proteins could be made more efficient and productive. As a result, products could be developed more quickly and would reach patients faster. We are pleased that the debate occasioned by the birth of Dolly has not involved any serious challenges to the cloning by nuclear transfer of agricultural and laboratory animals. NBAC has affirmed the benefits of animal cloning research and indicated its support for these technologies and we urge the Subcommittee to do the same. Medical Benefits of Cloning Cells and Genes Cloning techniques-the duplication of cells and genes-are integral to the process used to produce breakthrough medicines, diagnostics and vaccines to treat heart disease, many cancers, kidney disease, diabetes, hepatitis, multiple sclerosis, cystic fibrosis and a host of other diseases. More than 100 million people worldwide have already benefited from biotechnology medicines and vaccines. These techniques have been used in research and development for almost 20 years and are the cornerstone of the biotech industry and almost all modern biological research at academic institutions and the National Institutes of Health. Scientists also are conducting valuable research into cloning human cells, organs and tissue. This could produce replacement skin, cartilage and bone tissue for burn and accident victims. This avenue of study may produce cells useful for cancer therapy and result in ways to regenerate retinal or spinal cord tissue. As mentioned above, research is also underway to develop replacement internal organs in transgenic animals for human transplantation. Also, treatment and diagnosis of mitochondrial genetic disorders may be advanced by nuclear transfer technology. Perhaps even more important, cloning of human cells and genes is essential to research that will provide profound new insights into how genes control human development and contribute to human diseases. These fundamental insights, in the years ahead, will provide the basis for even greater biomedical advances in the service of humanity, such as in identifying treatments for many diseases associated with aging. People of all ages are awaiting the development of these diagnostic tools and cures; their suffering provides strong motivations for researchers pursuing these valuable studies. Existing Constraints on Cloning of Entire Human Beings We believe that there is little immediate risk of the application of nuclear transfer technology or other cloning techniques for the purpose of developing an entire human being for several reasons. Scientific Constraints: First, it may not be feasible to apply this technology to human beings. Second, there are immense scientific hurdles that would need to be overcome before any attempt could be made to apply nuclear transfer technology to humans. We believe it is not currently possible, and will not be possible for some time, to clone an entire human being using nuclear transfer technology. The reliability of nuclear transfer technology is far from established, as the cloning of a sheep from an adult sheep cell has occurred only once. The first successful use of nuclear transfer technology came in sheep in 1984. This was based on a large body of knowledge developed over many years by many researchers. It has taken an additional twelve years to yield the imperfect technology that resulted in Dolly. It may take a considerable period of time before this technology is perfected when applied to sheep and other farm animals, so that it can be performed with a greater success rate than this first experiment. Furthermore, there are risks associated with the technology which we might tolerate in the case of farm animals, but which we would never tolerate were the technology to be applied to entire human beings. Assuming all the scientific procedures were known and optimized, many eggs, as well as surrogate mothers, would be necessary to establish this technique as a reliable method of developing new human beings. This type of research is neither feasible nor ethically acceptable at this time. FDA and NIH Regulation of Research on Human Subjects: Regulations already exist to protect the individuals who participate in medical research studies. Under these regulations, the National Institutes of Health (NIH) and the Food and Drug Administration (FDA) require informed consent from participants and approval by a national or local review board for medical research. In addition to obtaining FDA or NIH review, research studies also must be approved by institutional review boards (IRBs), in compliance with NIH guidelines and FDA regulations, to assure patient safety. BIO recommends that medical research, including research using cloning and nuclear transfer technologies, continue to be governed by existing regulations to protect the safety of research subjects, specifically those regulations cited in the Code of Federal Regulations at 21 CFR 50 and 45 CFR 46. Ethical Obligations of Researchers and Physicians: The scientific and medical communities subscribe to ethical codes which make unlikely their participation in the cloning of entire human beings without an extended scientific, medical, and ethical review process which attains consensus regarding the benefits and risks of this procedure. Given the existing ethical and regulatory governance of science, a federal or state law to ban the cloning of entire human beings is not necessary. Rather, we recommended that NBAC extend the current moratorium and we urge the Subcommittee to do the same. We believe this moratorium will be sufficient to deter this type of experimentation, while allowing beneficial research to continue. History of Voluntary Moratoria in Biotechnology Research The history of strong adherence to moratoria in biotechnology research shows that this moratorium can and should be continued with regard to the cloning of entire human beings and that it will be effective. Early Biotechnology Research: In the mid 1970s, when the industry was very new, researchers wondered whether their work would pose hazards to human health, and they worried over the potential environmental impact of genetically engineered microorganisms. There were no easy answers to those early concerns, although most researchers thought their work could be extraordinarily beneficial and would pose no safety risks. Because of these questions, however, a moratorium was declared by academicians and industry itself on all experimentation until there could be a full debate of the issues. In 1974, the NIH began a program to consider the state of knowledge and what risks might be posed by biotechnology. In 1975, at the Asilomar Conference Center in California, a group of scientists drew up principles to govern biotechnology research and established the NIH Recombinant DNA Advisory Committee. This committee was given the responsibility to review experiments about which there was uncertainty or ethical concern. These guidelines have been modified over the years as scientific and regulatory experience has shown that most biotechnology procedures pose little or no risk. In fact, high school students now conduct recombinant DNA experiments involving cloning cells in their biology labs. BIO and its members support the research guidelines and the NIH advisory committee. Even though not specifically required to do so, BIO companies voluntarily adhere to these guidelines. Developing specific guidelines, rather than passing laws to try to determine the areas appropriate for scientific pursuit, allowed the biotechnology industry to grow and develop life-saving medications and vaccines. Hasty legislation could have halted the industry's growth in its infancy. The industry still maintains its promise of beneficial new medications that should not be swept aside. BIO also has formed a standing, Board of Directors'-level committee on bioethics to further examine bioethical issues as they arise. The industry has long been committed to abide by broad societal concerns as it strives to bring life-saving therapies to patients and their families. For example, BIO supports national legislation to protect the confidentiality of all individually identifiable medical information. We believe that this medical information must be respected, treated confidentially and safeguarded from discriminatory misuse. Industry's Commitment to Moratorium on Germ Line Gene Therapy: During the past ten years, research and development efforts to perfect somatic cell gene therapy procedures in humans and germ line gene therapy procedures in animals have proceeded at a vigorous pace. At the same time, the academic and industrial research communities have observed a voluntary moratorium on the development and practice of germ line gene therapy procedures in humans; a voluntary moratorium which, to our knowledge, has not been violated. If and when this moratorium is lifted for a particular medical therapy, it will be because an informed public debate has produced a consensus that the benefits of this technology outweigh any potential harms. We recognize that some may argue that voluntary moratoria will inevitably fail as safeguards and thus would seek to prohibit all research procedures which if taken to their final possible conclusion would result in the cloning of entire human beings. We must however, respectfully disagree. Over the last decade the biomedical research community in the U.S. has provided a salutary example of the power of responsible, voluntary restraint, illustrated by honoring this moratorium on germ line gene therapy. The industry has worked closely with the NIH Recombinant DNA Advisory Committee in upholding this moratorium. As a nation, we should take pride in this experience with a moratorium on germ line gene therapy in humans. We should follow this example in determining how to restrain the cloning of entire human beings, without undermining the use of nuclear transfer and cloning technologies to improve and save the lives of many Americans. ...... ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn