Stem cell research could lead to new therapies

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The issue of stem cell research is of great importance. The subject is being debated in Congress as well as in many state legislatures. Because of the differences in opinion, Prairie Fire believes that it is our responsibility to seek and explore thoughtful essays written by the scientists and ethicists who are actively engaged in this field. With this issue, we publish the first such essay and have extended an invitation to publish another.

By James Turpen, Ph.D.

The pace of new discoveries in science and biomedical research has increased dramatically. This is especially true in the field of stem cell biology where reports of significant findings appear almost weekly in the scientific literature. As with most scientific advances, there are numerous ethical implications that must be considered. At the same time, there also is a critical need to ensure that the public understands the promise that stem cell research holds for developing therapies for incurable diseases. Here, I would like to share why it is critical for the University of Nebraska Medical Center to develop and maintain a strong research program in embryonic stem cell research.

UNMC has been a world leader in the area of adult stem cell research and the translation of this research to patient care. We are nationally and internationally recognized for our bone marrow and peripheral stem cell transplant programs. This expertise has had a direct impact on the quality of health care available for citizens of Nebraska and the region.

However, the diseases that can be treated using adult stem cells are limited. Current stem cell therapies are effective in treating diseases associated with the blood-forming tissues, such as lymphomas and melanomas. In order to remain leaders in the field of stem cell research and treatment, UNMC must move forward in the area of embryonic stem cells and pursue all opportunities for developing new therapies.

To understand the promise of stem cell research and therapy, one must first understand the origin of embryonic stem cells and the technology used to generate embryonic stem cell lines that are used in research. The single-celled zygote, which is created at the time of fertilization, has the potential to give rise to all of the tissues and organs in our bodies. As that single cell multiplies - during the first five to seven days after fertilization - several additional cells develop which also have the potential to give rise to all the tissues and organs. When these cells are removed and placed in a plastic dish, they continue to divide, creating what we refer to as an embryonic stem cell line. Scientists throughout the world are busy learning how to coax these stem cell lines so they develop into specific types of tissues. As the technology for this work matures, these specific tissue types will form the basis for new therapies for a host of incurable diseases.

Progress already is being made toward the development of such therapies. For example, the loss of normal insulin-producing cells results in Type I diabetes, a life-changing disease. Recent breakthroughs have demonstrated that human embryonic stem cells can be directed to give rise to the pancreatic cells that produce insulin. Scientists have shown that when these modified insulin-producing cells are transferred to a diabetic mouse, the cells function normally, produce insulin and cure the disease.

In other research, scientists have shown that the transplantation of human embryonic stem cell-derived nervous tissue can lead to the repair of severed spinal cords in rats. FDA approval is pending for the start of a clinical trial in humans that will involve the use of human embryonic stems cells in the treatment of human spinal chord injuries. Another FDA approved clinical trial is investigating the transplantation of human embryonic stem cell-derived corneal cells as treatments for vision problems. Other promising uses for stem cell therapies are for the treatment of Parkinson’s disease and the regeneration of damaged tissues following heart attack. Therapeutic use of human embryonic stem cells is not some distant hope - it is real and moving forward with appropriate regulation and oversight.

Induced pluripotent stem cells, a promising line of research announced late last year, involves reprogramming the genetic information in adult cells in such a way that they behave as embryonic stem cells. Reprogramming of adult cells is in its infancy and the potential of these reprogrammed cells to give rise to a multitude of adult tissues is not known. Nor is it known whether techniques used for reprogramming would lead to genetic abnormalities that would make these cells unusable for therapy. But, if successful, the technique could eliminate the need for deriving new embryonic stem cell lines and silence the ethical controversy surrounding the research. In light of this research, many nonscientists suggest it is no longer necessary to pursue embryonic stem cell research, but nothing could be further from the truth. The consensus within the responsible scientific community is that the promise of reprogramming adult cells cannot be realized without continued research in embryonic stem cells.

Scientists at UNMC, and elsewhere, are currently limited to the number of stem cell lines that can be used in federally sponsored research. However, we are hopeful these restrictions will be relaxed in the near future and that new stem cell lines will become available for research and development of additional therapies. Scientists at UNMC have been highly successful in securing competitive funding from the National Institutes of Health and will seek new grants as they become available in this promising area.

Consequently, in addition to having direct health-care benefits, embryonic stem cell research significantly impacts the research enterprise within the state. With $80 million in research funding generated by UNMC alone, the economic impact of research cannot be ignored. It is an economic engine for the state, creating high-quality jobs, recruiting and retaining the best and brightest talent, and ensuring Nebraska’s position as a global leader.

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