The NYSCF diabetes team, led by Dr. Dieter Egli, is generating diabetes-specific stem cell lines from patients with type 1, type 2, monogenic diabetes, and specific genetic variations leading to obesity that will enable NYSCF to overcome the fundamental challenges that have kept scientists and clinicians from fully understanding this debilitating autoimmune disease. This greater understanding will in turn lead to the discovery of cell-based treatments and new drugs for diabetes patients.
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A NYSCF Public Outreach Panel on diabetes and stem cell research Click here to learn about the event » |
Diabetes is a chronic disease that affects nearly eighteen million Americans (one million with type 1 diabetes) and requires a very demanding treatment regimen to preserve the health of affected individuals. In type 1 diabetes, cells of the immune system attack the insulin producing (beta) cells of the pancreas and kill them. In order to effectively stop this cell death and prevent it starting, researchers need to understand what triggers this attack and to find out which genes are involved.
We believe that human pluripotent stem cells carrying the genes for diabetes will provide an accurate human cell-based model for this disease. The ability of human pluripotent stem cells to grow indefinitely in culture, while retaining the capacity to differentiate into all the cells of the body, will allow us to produce a limitless quantity of the actual insulin-producing beta cells that are destroyed by diabetes. Because these cells will carry the genes responsible for the disease, they may degenerate over time in the same way they would within the pancreas.
Diabetes-specific stem cells will provide diabetes investigators with a renewable system for investigating the molecular and cellular events that underlie diabetes and, possibly, be used in cell replacement (transplantation) to modify or cure diabetes.
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NYSCF Senior Research Fellow Dr. Dieter |
Why Stem Cells?
Stem cell research holds the promise for scientists to overcome one of the most challenging problems in studying human disease: the fact that by the time the disease is diagnosed, many of the events that led to it have already occurred, preventing scientists from understanding its precise origin. It also gives us an unprecedented opportunity to create the cells needed for cell replacement therapy and for screening drugs on the actual cells that are getting sick in diseases.
The problem encountered by scientists attempting to study disease or injury is analogous to that faced by investigative teams trying to understand the causes of a plane crash before the advent of the "black box," when understanding the often subtle events that led to the incident was nearly impossible. The cell-based models of disease created in the NYSCF laboratory will serve as "data recorders" for the study of disease. Just as flight recorders allow the Federal Aviation Administration to replay air accidents over and over again to understand the underlying causes of a crash, these cell models will enable us to repeatedly replay the development of disease until it is fully understood.
Scientific research has been further hampered by the inability to obtain adequate numbers of the actual type of cells needed for research in specific diseases or other medical conditions. As a result, scientists have traditionally turned to the study of rodent models, which often poorly recapitulate human disease. NYSCF scientists have shown that human pluripotent stem cells carrying the genes causing a specific disease can replicate many of the degenerative processes that occur in humans with that disease. The ability of these cells to grow indefinitely in culture, while retaining the capacity to differentiate into all the cells of the body, will allow us to produce a limitless quantity of these degenerating cells for use in disease studies and drug identification.
Further Reading:
National Institutes of Health: National Diabetes Information Clearinghouse
New York Times Health Guide on Diabetes
Recent Diabetes Research:
“Sugar fuels growth of insulin-making cells”
Recent studies reveal a surprising regenerative capacity of insulin producing beta cells in mice, suggesting that regenerative therapy for human diabetes could be achieved. Scientists have shown that beta cell proliferation rates are controlled to a large extent systemically (rather than by local factors such as tissue damage) and that glucose induces beta cell replication via metabolism by glucokinase, the first enzyme involved in glycolysis. This finding suggests that drugs that boost the activity of glucokinase can increase beta cell growth while still lowering circulating blood sugar levels.
Read the article in Science News
"Control of Pancreatic B Cell Regeneration by Glucose Metabolism"
A spoonful of sugar may be a remedy for diabetes. The more glucose that insulin-producing cells in the pancreas use, the faster those cells reproduce, a new study in mice shows.
"Generation of pluripotent stem cells from patients with type-1 diabetes"
Researchers have shown that induced pluripotent stem cells can be generated by reprogramming the adult fibroblasts of patients with type-1 diabetes. These stem cells are type-1 diabetes specific, have the hallmarks of pluripotency, and can be differentiated into insulin-producing cells. Though the traditional rodent model for diabetes has yielded valuable insights, it rarely fully represents the human disease. These results are a large step towards developing a more complete human model for type-1 diabetes.
Read more in the Proceedings of the National Academy of Sciences
“Company’s stem cells treat diabetes in mice”
When implanted into mice, human stem cells transformed into functional insulin producing cells. The finding that human embryonic stem cells can serve as a renewable source of functional insulin producing cells for diabetes patients represents a large step towards treating or perhaps even curing diabetes.
"Pancreatic endoderm derived from human enbryonic stem cells generate glucose-responsive insulin-secreting cells in vivo"
Human stem cells transformed into nearly normal insulin-producing cells when implanted into mice, possibly offering a way to treat diabetes long-term.
