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STEM CELL RESEARCH
Global Conference Highlights Stem Cells' Role as Models for Disease Study and Treatment
New York, NY, July 28, 2004 — Reflecting the growing worldwide interest in stem cell research, 1,435 scientists from 28 countries attended the annual meeting of the International Society for Stem Cell Research in Boston recently, co-sponsored by JDRF. More than 650 scientific abstracts were submitted at the event, which is the premier gathering of stem cell scientists.
Among important trends in stem cell research relevant both to type 1 diabetes and to the study of disease in general, researchers reported on the first stem cell lines derived from embryos with genetic diseases.
A Window into the Biology of Disease
Although embryonic stem cells are most often heralded for their potential to develop into cell therapies for a host of serious diseases, the cells could also shed light on the various afflictions themselves, because these cells can develop into any type of human cell.
For example, many human disorders, such as muscular dystrophy, arise from a defect in a single gene. In order to understand how the defect contributes to disease, and how to develop ways to correct it, scientists benefit from the ability to study cells carrying the defective gene from the earliest stage of development. This is especially useful in cases where animal models for a disease do not faithfully simulate the symptoms and conditions that occur in humans.
Some couples who undergo in vitro fertilization treatment have a family history of genetic disorders that cause disease. Because of recent technological developments, these couples can now use pre-implantation genetic diagnosis to identify which of their embryos created in the laboratory are unaffected by such disorders and thus appropriate for reproductive use. Embryos identified as having serious genetic disorders, while not usable for reproductive purposes and normally discarded, can, however, be used as the source for human embryonic stem cell lines that will have these disease characteristics.
At the ISSCR meeting, research teams from institutions in Chicago and in Israel reported developing 14 such lines, and described their usefulness in studying specific diseases and overall human biology.
The Israel team, from Technion - Israel Institute of Technology, a science and technology institute in Haifa, derived stem cell lines from genetically compromised embryos. For example, one line was derived from an embryo with Van Waardenburg disease (a disorder characterized by varying degrees of hearing loss and changes in skin and hair pigmentation). Another line originated from an embryo with myotonic muscular dystrophy (a disease in which the muscles contract but have decreasing power to relax).
The Chicago researchers, from the Reproductive Genetics Institute, a clinic and research institute focused on genetic disorders and infertility problems, reported producing 12 new human embryonic stem cell lines with a total of seven different genetic defects, including two forms of muscular distrophy.
The creation of these lines provide a proof of principle and illuminate a use for embryonic stem cell lines not previously a center of focus in stem cell research. The scientists note that the lines they created may have important potential in developing new treatments for the diseases in question. The lines will be made available to other researchers, and the scientists report they plan to create more cell lines from embryos affected by other genetic diseases.
Type 1 Diabetes and Embryonic Stem Cells
Although type 1 diabetes is not a single-gene disease (meaning many genes contribute to a risk of developing the disorder, rather than one genetic mutation causing the disease), researchers can learn about what triggers the disease by studying embryonic stem cells derived from a type 1 patient. For example, as embryonic stem cells develop into immune cells, researchers could look for differences between these immune cells and those of someone without type 1 diabetes. These differences could provide insights into the mechanism of type 1 diabetes and offer new way to test drugs or therapies in their earliest stages, thereby speeding the process toward human clinical trials.
Researchers also might look for biological characteristics not present in the beta cells of people without type 1 diabetes. That might provide answers to what stimulates the autoimmune attack that causes the disease.
A number of research findings relevant to type 1 diabetes were presented during the ISSCR meeting:
