More than any other scientific field, with the possible exception of climate change, embryonic stem cell research is subject to the ups and downs of politics and trouble may lie ahead for scientists in Connecticut and across the country.
Derived from early embryos, embryonic stem cells can become any cell in the body. Since the discovery of human embryonic stem cells in 1998, scientists have explored their potential use as therapies for diseases and injuries. Embryonic stem cell derivatives, for example, could replace the pancreatic cells lost in Type I Diabetes or the neurons lost in Parkinson's Disease. But just as this approach begins to show promise, a new threat appears on the horizon.
U.S. Rep. Tom Price, R-Ga., Donald Trump's nominee to head the Department of Health and Human Services with oversight over the National Institutes of Health, is on record opposing embryonic stem cell research. As stem cell researchers, we fear that this appointment would endanger human embryonic stem cell research in the United States and reverse the substantial progress made in recent years. There are promising clinical trials underway for macular degeneration, spinal cord injury and diabetes with more possible, including for Parkinson's disease.
Connecticut has recognized the importance of human embryonic stem cell research and funded first the Connecticut Stem Cell Program, and now the Regenerative Medicine Research Fund. This brought Connecticut to the forefront of stem cell research. Continued support at the national level is also needed, however, if we wish to continue making progress toward effective cell-based therapies.
What makes this field of research so controversial is that an early stage human embryo (five days after fertilization) called a blastocyst is used to produce a human embryonic stem cell line. Federal funds may not be used to produce a new human embryonic stem cell line becausethe money cannot supportresearch that directly uses human embryos. At this point, however, federal funds can be used to work on human embryonic stem cells. Despite this, a minority in the government strive to further limit federal funding so that it cannot be used even for studies on lines generated using alternative financial sources.
Many claim we can achieve our therapeutic goals using other stem cell sources, but as stem cell scientists we are keenly aware of the limitations of these alternatives.
Adult stem cells, which have limited capacity for generating the high number of cells needed for human transplants and can only produce certain cell types, will likely work for some applications, but not others.
Another type of stem cell, induced pluripotent stem cells, can be generated from adult cell types such as skin, without the need to start with a human embryo. These cells share many properties with embryonic stem cells, including the ability to become virtually any cell in the body. Work using these cells has exploded since their discovery 10 years ago. Induced pluripotent stem cells are useful for modeling human disease in a culture dish and for drug screening. For clinical application, however, these cells have several limitations. Virtually all the cell lines made to date are genetically modified, and this modification could potentially cause cancer, which precludes their use in humans. Most important, as described by many stem cell researchers, embryonic stem cells behave most consistently and therefore remain the gold standard against which other research is compared.
While this is not the place for a full discussion of the moral status of early human embryos, it is worth making some observations. The blastocyst forms 5 days after fertilization, prior to implantation in the uterus, and consists of a couple of hundred cells. All human embryonic stem cell lines that are approved for federally funded research are derived from blastocysts leftover from infertility treatment, with the informed consent of the donors. The alternative futures for these embryos are to be kept frozen indefinitely or to be destroyed. Given these options, many would agree that a future of producing a cell line that could eventually reduce suffering and save lives is a preferred fate.
The United States is a leader in embryonic stem cell research, from basic science to clinical application. This achievement has been fueled by successful collaborations between government-funded academic laboratories and the private sector. A skilled workforce and state-of-the-art infrastructure has been established. New restrictions could well lead to a brain drain and likely provide a serious roadblock to numerous cures.
Laura Grabel, Ph.D., is the Lauren B. Dachs Professor of Science and Society and a professor of Biology at Wesleyan University and president of the Connecticut Academy of Science and Engineering. Diane Krause, MD, Ph.D., is a professor at the Yale School of Medicine, associate director of the Yale Stem Cell Center, and director of the Clinical Stem Cell Processing Laboratory.
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