If a father happens to be a scientist and his daughter afflicted with Diabetes 1 from birth, then there is no way he could sleep peacefully unless he finds a solution to it and that’s what happend with Harvard researcher Doug Melton, who had spent 23 years in his quest to fine one to develop a device that can insert a device to produce millions of beta cells inside the body.
His life-long dedication to find treatment for type 1 diabetes that hits about 3 million Americans costing about $15 billion in treatment finally led him to explore stem-cell production of insulin in massive quantity from the human insulin-producing beta cells like any normally functioning beta cells.
Type 1 diabetes is an autoimmune metabolic condition in which the body kills off all the pancreatic beta cells that produce the insulin needed for glucose regulation in the body. Thus the final pre-clinical step in the development of a treatment involves protecting from immune system attack the approximately 150 million cells that would have to be transplanted into each patient being treated. And Melton succeeded developing an implantation device to protect the cells.
He was helped in his research by Daniel G. Anderson, the Samuel A. Goldblith Professor of Applied Biology, Associate Professor in the Department of Chemical Engineering, the Institute of Medical Engineering and Science, and the Koch Institute at MIT. Melton also expressed gratitude to both the Juvenile Diabetes Research Foundation and the Helmsley Trust, saying “their support has been essential.”
While diabetics can keep their glucose metabolism under general control by injecting insulin multiple times a day, that does not provide the kind of fine tuning necessary to control metabolism, and it leads to devastating complications from blindness to loss of limbs.
Even 10 percent of the more than 26 million Americans living with type 2 diabetes are also dependent upon insulin injections, and would presumably be candidates for beta cell transplants, Melton said.
Now, he hopes to have human transplantation trials using the cells within a few years. “We are now just one pre-clinical step away from the finish line,” said Melton, whose daughter Emma has type 1 diabetes.
His work will be published in the journal Cell, with Felicia W. Pagliuca, Jeff Millman, and Mads Gurtler of Melton’s lab as co-first authors on the Cell paper.
The stem cell-derived beta cells are presently undergoing trials in animal models, including non-human primates, Melton said.
Elaine Fuchs at Rockefeller University hailed the research outcome. “For decades, researchers have tried to generate human pancreatic beta cells that could be cultured and passaged long term under conditions where they produce insulin. Melton and his colleagues have now overcome this hurdle and opened the door for drug discovery and transplantation therapy in diabetes,” Fuchs said.
Jose Oberholtzer, M.D., Associate Professor of Surgery, Endocrinology and Diabetes, and Bioengineering at the University of Illinois at Chicago, said, “Doug Melton has put in a life-time of hard work in finding a way of generating human islet cells in vitro. He made it. This is a phenomenal accomplishment.”
Cell transplantation as a treatment for diabetes is still essentially experimental, uses cells from cadavers, requires the use of powerful immunosuppressive drugs, and has been available to only a very small number of patients.