Genetics in the New Millennium - Stem Cells
By Ronald Bachman, MD Chief, Department of Genetics

Those of us in Genetics believe that research into the basic science of stem cells will lead to treatment for genetic disorders, including not only rare conditions but also common conditions not often thought of as genetic. These will include neurologic disorders (Parkinson's disease, Alzheimer's disease, etc.), diabetes, heart disease from atherosclerosis, and diabetes. In addition to these genetic conditions, stem cells will be used to treat spinal cord injuries and leukemia.

There are many sources of human stem cells. Most feel that embryonic stem cells have the best chance of success, but the concern is that they must be obtained from a human embryo. Other sources of stem cells are from umbilical cord (and placenta) blood collected at birth and adult stem cells. Adult stem cells can be harvested from blood or bone marrow. In actuality stem cells have been found in every organ (e.g. liver, brain, and even skin), but their numbers are small and they are difficult to collect.

Another source of stem cells are called cloned stem cells. This was the technology of "Dolly" and is referred to as "somatic cell nuclear transfer or SCNT" or cell cloning.

This technology includes removing the genetic material (DNA) from the nucleus (the center) of a cell and then introducing the genetic material from another individual. A stimulus such as an electrical current of a chemical then is used to induce the egg cell to begin to divide. This reengineered cell grows into a group of cells that have the potential to become any tissue of the body with the appropriate growth conditions. An example would be inducing these cells to become insulin producing cells (the beta cells of the pancreas) to treat diabetes.

The concern about the SCNT technology is that if the group of cells is implanted into a woman's uterus, it could develop into a baby. This is known as "reproductive cloning". In genetics we are interested in "theraputic cloning" which is producing stem cells for the treatment of disease and not for a new way of producing babies. It is also known from experimentation using this technology to produce animals like Dolly, that all were born with some type of birth defect, immune problem, or a neurologic deficit. It is for this reason that theraputic cloning and not reproductive cloning is our goal.

On August 9, 2001 President Bush limited Federal funding for embryonic and cloned stem cells to a small number of established embryonic stem cell lines. Private funding still could be utilized for research on embryonic and cloned stem cells. Federal funding has been available for research on umbilical cord and adult stem cells, but has been limited to about $25 million per year. Because of this most of the basic embryonic stem cell research is taking place in other countries (Great Britain, Israel, South Korea).

On November 2, 2004 the California electorate approved Proposition 71 with an astounding 59% of the voters. This proposition was the California Stem Cell Research and Cures Initiative, which provides $300 M per year (for ten years) for stem cell research and the development of medical therapies. It is hoped that this will eventually decrease health care costs, create more jobs, and stimulate the growth of the states biotechnology sector. The concerns of opponents of Proposition 71 are the ethical issue of the use and eventual loss of embryos, the uncertainty of how useful stem cells will be, and the increase of the California debt burden since it will be funded by tax free state bonds. On September 23, 2004 in a New York Time's editorial stated, "in effect, the proposal would have California pick up the leadership role that President Bush abdicated".

We are all excited about the possibilities that stem cells will be the break through for previously non-treatable genetic disorders. Let me conclude with a quote, "How'd you like to have your own personal biological tool repair kit, staying behind, waiting for you at the local hospital" (Ron Reagan, Jr. at the 2004 Democratic National Convention).