Researchers at the Washington University School of Medicine in St. Louis and Harvard have produced insulin-secreting cells from stem cells derived from patients with type 1 diabetes (T1D), suggesting a potentially novel approach to treating T1D is not too far off in the future.
Type 1 diabetes and the potential for personalized treatment
Type 1 diabetes (T1D) is a chronic condition in which the body’s immune system destroys insulin-releasing pancreatic cells – the beta cells in the islet tissue – thus rendering the pancreas incapable of producing enough insulin to maintain normal blood sugar levels.
Although there are pre-existing treatments available to help patients cope with this condition – such as exercise and diet changes, regular insulin injections, and blood pressure monitoring – there are currently no cures.
However, this new discovery suggests that personalized T1D treatments may become available in the not-so-distant future: it relies on the T1D patients’ own stem cells to produce new beta cells that make insulin to help control sugar levels, therefore essentially becoming a self-sustaining treatment for the patient and eliminating the need for regular insulin shots.
Research and success of cell differentiation in laboratory In Vivo and In Vitro testing
Researchers at the Washington University School of Medicine demonstrated that the new cells derived from stem cells could produce insulin when they encountered glucose sugar. The new cells were tested in vivo on mice and in vitro in cultures, and in both scenarios, researchers found they secreted insulin in response to glucose.
The scientists’ research was published in the Nature Communications journal on May 10, 2016:
"In theory, if we could replace the damaged cells in these individuals with new pancreatic beta cells -- whose primary function is to store and release insulin to control blood glucose -- patients with type 1 diabetes wouldn't need insulin shots anymore," said Jeffrey R. Millman (PhD), first author and assistant professor of medicine and biomedical engineering at the Washington University School of Medicine. "The cells we've manufactured sense the presence of glucose and secrete insulin in response. And beta cells do a much better job controlling blood sugar than diabetic patients can."
Similar experiments have previously been conducted but only used stem cells from individuals without diabetes. The breakthrough occurred when the researchers used beta cells from the skin tissue of patients with T1D and discovered that it is, in fact, possible for the stem cells of T1D patients to differentiate into insulin-producing cells.
"There had been questions about whether we could make these cells from people with type 1 diabetes," explained Millman. "Some scientists thought that because the tissue would be coming from diabetes patients, there might be defects to prevent us from helping the stem cells differentiate into beta cells. It turns out that's not the case."
Implementation of T1D patient stem-cell differentiated beta cells to treat diabetes
While the research and discovery shows great promise in the near-future, Millman says further research is needed to ensure that tumours do not form as a result of using T1D patient-derived stem cells. Tumours sometimes develop during stem cell research, although the researcher's trials in mice did not show evidence of tumours up to a year after the cells were implanted.
Millman says the stem cell-derived beta cells could be ready for human trials in about three to five years. The minimally invasive surgical procedure would entail implanting the cells under the skin of patients, allowing the cells access to the blood supply to regulate blood sugar levels.
"What we're envisioning is an outpatient procedure in which some sort of device filled with the cells would be placed just beneath the skin," stated Millman.
Millman also notes that the new technique could be used in a variety of ways to treat other diseases. Since Millman and his colleagues’ experiments have proven that it is possible to differentiate beta cells from stem cells in T1D individuals, Millman says there is a likely possibility this technique would also function in patients with other forms of the disease – including (but not limited to) type 2 diabetes, neonatal diabetes (diabetes in newborn children), and Wolfram Syndrome.
Not only would it be possible to treat T1D in a few years’ time, but it may also be possible to develop novel treatments for related diseases and to test the effect of diabetes drugs on the stem-cell differentiated cells of these patients.
This research and its implications could potentially help treat a large fraction of the world’s population affected by T1D and related diseases upon its public release. According to the American Diabetes Association, in 2012, approximately 1.25 million adults and children had T1D in the United States alone. Though this number is on the rise, the promise of the research of stem cell-differentiated insulin-producing cells paints a brighter future in not only T1D patients, but the future of health, technology, and medicine itself.