Plaudits for Harvard beta-cell regeneration
Data showing that robust human pancreatic beta cells can be produced in vitro is being described as a breakthrough for modern science and specifically for the future treatment of Type 1 diabetes
On 9 October, the journal Cell published the results of a study by Douglas Melton and colleagues from the Harvard Stem Cell Institute in the US which showed that it is possible to generate insulin-producing pancreatic beta cells from human pluripotent stem cells in the laboratory. These stem-cell-derived beta cells express the same markers found in mature beta cells and are able to secrete similar quantities of insulin to that of adult beta cells. Moreover, when transplanted into mice, the cells secreted human insulin in a glucose-regulated manner.
The study is the first time that a scientific team has produced mature beta cells that are glucose sensing and insulin-secreting and therefore suitable for potential use in patients. “It is the maturity and quality of the cells that Doug Melton’s lab has been able to produce which is so impressive. Five years ago people thought this was hardly possible,” said Cord Dohrmann, chief scientific officer of Germany’s Evotec AG. Evotec has two diabetes collaborations with Dr Melton’s laboratory, though it is not involved in the research reported in Cell.
Type 1 diabetes is an autoimmune metabolic condition in which the human body kills off all the pancreatic beta cells that produce the insulin which is needed for glucose regulation. Currently patients with the disease have to rely on injections of artificial or human insulin to control blood glucose levels. Some patients with Type 1 diabetes receive transplants of islets, or the hormone-producing regions of the pancreas, from cadavers. But the supply of these donor islets is limited.
The holy grail of diabetes research therefore has been to generate insulin-producing beta cells de novo to treat millions of patients with the disease.
“There is no question that the ability to generate glucose-responsive, human beta cells through controlled differentiation of stem cells will accelerate the development of new therapeutics,” said Daniel Anderson of the Koch Institute at the Massachusetts Institute of Technology. “In particular this approach opens the door to an essentially limitless supply of tissue for diabetic patients awaiting cell therapy,” he added.
The stem cell-derived beta cells produced in Dr Melton’s laboratory are currently undergoing trials in animals including in non-human primates. Provided they are shown to be safe, the first human trials would start thereafter.
One hurdle still to be overcome is delivery. When administered to humans, the cells will need to be protected from attack by the immune system. Encapsulation devices are already in development with this application in mind, Dr Dohrmann commented.
Dr Melton is reportedly collaborating with MIT’s Daniel Anderson on the development an implantation device.
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