House Energy and Commerce Committee Testimony

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 earlier this year 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. It took over 270 attempts before Dolly was successfully cloned. 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. Rogue and grandstanding so-called scientists who claim they can - and will - clone humans for reproductive purposes insult the hundreds of thousands of responsible, reputable scientists who are working hard to find new therapies and cures for millions of individuals suffering from a wide range of genetic diseases and conditions.

The Food and Drug Administration (FDA) has publicly stated that it has jurisdiction over human reproductive cloning experiments and that it will not approve them. BIO supports that view and hopes that the next FDA commissioner - whoever that might be - will assert FDA's current statutory authority forcefully.

There are also many ethical concerns raised by the specter of cloning. As noted in BIO's letter to the President, "Cloning humans challenges some of our most fundamental concepts about ourselves as social and spiritual beings. These concepts include what it means to be a parent, a brother, a sister and a family.

"While in our daily lives we may know identical twins, we have never experienced identical twins different in age or, indeed, different in generation. As parents, we watch with wonder and awe as our children develop into unique adults. Cloning humans could create different expectations. Children undoubtedly would be evaluated based on the life, health, character and accomplishments of the donor who provides the genetic materials to be duplicated. Indeed, these factors may be the very reasons for someone wanting to clone a human being."

As you can see, Mr. Chairman, 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 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 will describe today - that could lead to cures and treatments for some of our most deadly and disabling diseases.

Beneficial Uses of Cloning Technology

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 many diseases. They 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.

Let me briefly explaining a cloning technology - somatic cell nuclear transfer - and how it is used for research purposes. First, the nucleus of an egg cell is removed. In its place, we insert the nucleus of an already differentiated cell (a cell that performs a specific function in the body). Chemicals are added to stimulate the egg to start dividing. At about 3-5 days, a blastocyst is formed which contains an inner cell mass comprised of undifferentiated, pluripotent cells. These cells are removed and used for research. The research value of these cells is enormous. These stem cells have the potential to form any cell in the body and can replicate indefinitely. Studies in animals demonstrate that this could lead to cures and treatments for millions of Americans who suffer from diseases and disabilities such as diabetes, stroke, Parkinson's Disease, heart disease, and spinal cord injury.

As exciting as that is - it's only a part of the story. The full potential of this technology comes from its use in regenerative medicine.

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.

Somatic cell nuclear transfer research is essential if we are to achieve our goals in regenerative medicine. We must understand the biological properties of the egg cell (and the transferred nucleus) that cause a differentiated cell to turn into a pluripotent cell. This process is called "re-programming" - and we're still not sure how it works. That's why we need to continue to perform research.

At Geron, our aim is to harness and therapeutically apply the power of this biology. Once we fully understand re-programming we will be able to develop specific cells for transplantation without immune rejection. We'll do that by taking a differentiated cell from a particular individual and re-programming it to form a pluripotent cell from which we can produce the differentiated cells we need for transplantation back into that individual. By using the patient's own cells as starting material, we will avoid complications due to immune response rejection.

However, this is precisely the research that would be banned by the Weldon bill. Because the Weldon bill does not distinguish between reproductive cloning and use of cloning for research purposes, it will cut off this work and prevent its therapeutic applications from reaching patients. In contrast, the bi-partisan bill introduced by Reps. Greenwood, Deutsch, and others bans reproductive cloning but allows the continuation of research. BIO supports Greenwood/Deutsch because it strikes the appropriate balance between prohibiting acts that are unsafe and unethical, while promoting vital medical research.

It is important to emphasize that once we understand the molecular biology of re-programming, we will no longer need to use egg cells or create blastocysts. Therefore, this technology is likely to be used only for a short, finite period of time. Moreover, understanding the biology re-programming is a critical step to improve the usefulness of adult stem cells. Ironically, therefore, the Weldon bill will also be a setback to adult stem cell research.

Conclusion

As the current Congress pursues legislative prohibitions on human reproductive cloning, we urge caution and a distinction between reproductive and therapeutic cloning. We all agree that given the current safety and social factors, human reproductive cloning is repugnant. However, it is critical that in our enthusiasm to prevent reproductive cloning, we not ban vital research, turning wholly legitimate biomedical researchers into outlaws, and thus squelching the hope of relief for millions of suffering individuals.

Our nation is on the cusp of reaping the long dreamed of rewards from our significant investment in biomedical research. The U.S. biotech industry is the envy of much of the world, especially our ability to turn basic research at NIH and universities into applied research at biotech companies and in turn, into new therapies and cures for individual patients. Using somatic cell nuclear transfer and other cloning technologies, biotech researchers will continue to learn about cell differentiation, re-programming, and other areas of cell and molecular biology. Armed with this information, they can eventually crack the codes of diseases and conditions that have plagued us for hundreds of years, indeed, for millennia.

In conclusion, Mr. Chairman, human reproductive cloning remains unsafe, and the ethical issues it raises have not been reasonably resolved. It should be prohibited. However, as Congress seeks to outlaw reproductive cloning, it must not write legislation that will stop research using cloning technology. Unfortunately, the Weldon bill fails that test. Simply put, enactment of the Weldon bill will stop critical therapeutic research in its tracks. Only Greenwood/Deutsch strikes the right balance.

Thank you for the opportunity to testify. I'll be happy to answer any questions.