Ability to produce embryonic stem cells will allow researchers to push faster toward cure
Harvard stem cell researchers announced today that they have made a giant leap forward in the quest to find a truly effective treatment fortype 1 diabetes, a disease that affects an estimated 3 million Americans at a cost of about $15 billion annually.
With human embryonic stem cells as a starting point, the scientists were for the first time able to produce, in the kind of massive quantities needed for cell transplantation and pharmaceutical purposes, human insulin-producing beta cells equivalent in most every way to normally functioning beta cells.
Doug Melton, who led the work, said he hopes to have human transplantation trials using the cells under way within a few years. Twenty-three years ago, when his infant son Sam was diagnosed with type 1 diabetes, Melton dedicated his career to finding a cure for the disease.
“We are now just one preclinical step away from the finish line,” said Melton, whose daughter Emma also has type 1 diabetes.
A report on the new work is being published today by the journal Cell.
“You never know for sure that something like this is going to work until you’ve tested it numerous ways,” said Melton, Harvard’s Xander University Professor and a Howard Hughes Medical Institute investigator. “We’ve given these cells three separate challenges with glucose in mice, and they’ve responded appropriately; that was really exciting.
“It was gratifying to know that we could do something that we always thought was possible,” he continued, “but many people felt it wouldn’t work. If we had shown this was not possible, then I would have had to give up on this whole approach. Now I’m really energized.”
The stem cell-derived beta cells are undergoing trials in animal models, including non-human primates, Melton said.