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.

 ......

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