TESTIMONY OF THOMAS OKARMA, MD, PhD PRESIDENT AND CEO OF GERON CORPORATION ON BEHALF OF THE BIOTECHNOLOGY INDUSTRY ORGANIZATION (BIO) BEFORE THE SUBCOMMITTEE ON OVERSIGHT AND INVESTIGATIONS COMMITTEE ON ENERGY AND COMMERCE US HOUSE OF REPRESENTATIV

Summary of Testimony of Thomas Okarma, MD, PhD

• BIO opposes human reproductive cloning. It is too dangerous technically and raises too many ethical and social questions. The voluntary moratorium on human reproductive cloning should remain in place and no federal funds should be used for human reproductive cloning.

• The Food and Drug Administration (FDA) has publicly stated that it has jurisdiction over human reproductive cloning experiments and that it would not approve them. BIO supports that view.

• It is critical to distinguish use of cloning technology to create a new human being (reproductive cloning) from other appropriate and important uses of the technology such as cloning specific human cells, genes and other tissues that do not and cannot lead to a cloned human being (therapeutic cloning). These techniques are integral to the production of breakthrough medicines, diagnostics and vaccines to treat heart attacks, various cancers, Alzheimer's, diabetes, hepatitis and other diseases.

• My company, Geron, as well as many other companies and academic laboratories, use cloning technology for many beneficial purposes. They include: regenerative medicine; predictive toxicology and drug discovery; and agriculture.

• In addition to the scientific obstacles, human reproductive cloning raises numerous ethical and social concerns. Many of these issues strike at the heart of beliefs and values that are inherent in the human condition. Reproductive cloning could be perceived to devalue human beings by depriving them of their own uniqueness.

TESTIMONY OF THOMAS OKARMA, MD, PhD
PRESIDENT AND CEO OF GERON CORPORATION
ON BEHALF OF
THE BIOTECHNOLOGY INDUSTRY ORGANIZATION (BIO)
BEFORE THE
SUBCOMMITTEE ON OVERSIGHT AND INVESTIGATIONS
COMMITTEE ON ENERGY AND COMMERCE
US HOUSE OF REPRESENTATIVES
MARCH 28, 2001

Good afternoon. My name is Thomas Okarma. I am the President and CEO of Geron Corporation in Menlo Park, California. Geron is a biopharmaceutical company focused on discovering, developing, and commercializing therapeutic and diagnostic products for applications in oncology, drug discovery and regenerative medicine. Geron's product development programs are based upon three patented core technologies: telomerase, human pluripotent stem cells, and nuclear transfer.

I am testifying today on behalf of my company and the Biotechnology Industry Organization (BIO). BIO represents more than 950 biotechnology companies, academic institutions, state biotechnology centers and related organizations in all 50 U.S. states and 33 other nations. BIO members are involved in the research and development of health care, agricultural, industrial and environmental biotechnology products.

Mr. Chairman, and members of the Subcommittee, thank you for the opportunity to testify today at this important hearing on cloning. Let me start by making our position perfectly clear: BIO opposes human reproductive cloning. It is simply too dangerous technically and raises far too many ethical and social questions.

That's why BIO wrote to President Bush last month and urged him to extend the voluntary moratorium on human reproductive cloning which was instituted in 1997. I would respectfully ask for this letter to be included in the hearing record.

It would be extremely dangerous to attempt human reproductive cloning. In fact, in most animals, reproductive cloning has no better than a 3-5% success rate. That is, very few of the cloned animal embryos implanted in a surrogate mother animal survive. The others either die in utero - sometimes at very late stages of pregnancy - or die soon after birth. Only in cattle have we begun to achieve some improvements in efficiency. However, scientists have been attempting to clone many other species for the past 15 years with no success at all. Thus, we cannot extrapolate the data from the handful of species in which reproductive cloning is now possible to humans. This underlines that this would be an extremely dangerous procedure.

It is simply unacceptable to subject humans to those risks.

The Food and Drug Administration (FDA) has publicly stated that it has jurisdiction over human reproductive cloning experiments and that it would not approve them. BIO supports that view.

Beneficial Uses of Cloning Technology – Therapeutic Cloning

It is critical to distinguish use of cloning technology to create a new human being (reproductive cloning) from other appropriate and important uses of the technology such as cloning specific human cells, genes and other tissues that do not and cannot lead to a cloned human being (therapeutic cloning). These techniques are integral to the production of breakthrough medicines, diagnostics and vaccines to treat heart attacks, various cancers, Alzheimer's, diabetes, hepatitis and other diseases. This type of therapeutic cloning could also produce replacement skin, cartilage and bone tissue for burn and accident victims, and result in ways to regenerate retinal and spinal cord tissue.

My company, Geron, as well as many other companies and academic laboratories, use cloning technology for many beneficial purposes. Let me explain how we use it to develop products that could revolutionize medicine and improve the lives of people suffering from serious illnesses.

Regenerative Medicine

Many diseases result in the disruption of cellular function or destruction of tissue. Heart attacks, strokes, and diabetes are examples of common conditions in which critical cells are lost to disease. Today’s medicine is unable to completely restore this loss of function. Regenerative medicine, a new therapeutic paradigm, holds the potential to cause an individual's currently malfunctioning cells to begin to function properly again or even to replace dead or irreparably damaged cells with fresh healthy ones, thereby restoring organ function.

The goal of Geron's regenerative medicine program is to produce transplantable cells that provide these therapeutic benefits without triggering immune rejection of the transplanted cells. This could be used to treat numerous chronic diseases such as diabetes, heart disease, stroke, Parkinson's Disease and spinal cord injury.

At Geron, therapeutic cloning technology is one of the techniques we use to create pure populations of functional new cells that can replace damaged cells in the body. For example, we are learning how to turn undifferentiated human pluripotent stem cells into neurons, liver cells and heart muscle cells. Thus far, these human replacement cells appear to function normally in vitro, raising the possibility for their application in the treatment of devastating chronic diseases affecting these tissue types. This would, for instance, allow patients with heart disease to receive new heart muscle cells that would improve cardiac function. Cellular cloning techniques are a critical and necessary step in the production of sufficient quantities of vigorous replacement cells for the clinical treatment of patients.

Predictive Toxicology/Drug Discovery

Geron is also developing research tools to facilitate the safe development of new drugs. The use of normal, cloned human liver cells to test new drugs under development for certain toxic metabolites would reduce the danger of human clinical trials by eliminating such compounds before human testing. This process could streamline and make safer the drug development process, thereby reducing by several years drug development time, bringing drugs to patients sooner and with greater safety, and reduce the reliance upon animal testing.

Agriculture

Geron uses cloning technology for applications in agriculture as well. These include producing animals with desirable qualities such as disease resistance, longevity, or improved product quality. Animals can also be cloned to produce proteins for human therapeutic use such as human antibodies, allowing for large-scale production of vaccines.

Ethical Concerns of Reproductive Cloning

In addition to the scientific obstacles, human reproductive cloning raises numerous ethical and social concerns. When the moratorium was imposed in 1997, scientists, ethicists, and policy makers believed that the various ethical issues raised by human cloning had not been resolved. At the time, the National Bioethics Advisory Commission (NBAC) called human cloning "morally unacceptable".

Mr. Chairman, that is still true. Not only is there no consensus in our society about how to resolve the ethical concerns implicated by human reproductive cloning, these issues have not yet even been adequately discussed. Many of these issues strike at the heart of beliefs and values that are inherent in the human condition. What does it mean to be an individual? How should we view our parents, brothers, sisters, and children? How does the world around us influence our intellectual, physical and spiritual development? These are just a few of the questions raised by human cloning. In my view, reproductive cloning would devalue human beings by depriving them of their own uniqueness.

To allow human reproductive cloning without a full and fair discussion of these and other moral issues would be irresponsible. Worse yet, it could lead to a backlash that would stifle the numerous beneficial applications of therapeutic cloning technology - some of which I have described today - that could lead to cures and treatments for some of our most deadly and disabling diseases.

Conclusion

Mr. Chairman, human reproductive cloning remains unsafe. Moreover, the ethical issues it raises have not been fully debated throughout our society. Therefore, the voluntary moratorium on human reproductive cloning should remain in place and no federal funds should be used for human reproductive cloning.

Thank you. I'd be happy to answer any questions.