Published on 10 October 2014

Researchers at Harvard University in the USA have developed a method for producing new cells that could make for a giant leap in the search for a cure for type 1 diabetes.

By using human embryonic stem cells as a starting point Harvard stem cell researchers have found a way 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 (cells in the pancreas responsible for storing and releasing insulin.

Human stem cell-derived beta cells that have formed islet-like clusters in a mouse. The cells were transplanted to the kidney capsule. This photo was taken two weeks later. The beta cells are making insulin, curing the diabetes in the mouse.

It is estimated that around 400,000 people in Britain have type 1 diabetes, with treatment costing the NHS £1 billion annually.

The research, published in medical journal Cellwas led by Harvard’s Xander University’s Professor Doug Melton, who 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, Dr Melton dedicated his career to finding a cure for the condition. His daughter Emma also has type 1 diabetes.

Doug Melton, Harvard’s Xander University Professor, and his team have announced that they have made a tremendous gain on the type 1 diabetes front.

Dr Melton said: “We are now just one preclinical step away from the finish line. You never know for sure that something like this is going to work until you’ve tested it numerous ways.

“We’ve given these cells three separate challenges with glucose in mice, and they’ve responded appropriately; that was really exciting.”

He added: “It was gratifying to know that we could do something that we always thought was possible, 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 energised.”

The stem cell-derived beta cells are undergoing trials in animal models, including non-human primates.

Elaine Fuchs, the Rebecca C. Lancefield Professor at Rockefeller University, and a Howard Hughes Medical Institute investigator who is not involved in the work, hailed it as “one of the most important advances to date in the stem cell field, and I join the many people throughout the world in applauding my colleague for this remarkable achievement.”

She added: “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.”

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. Therefore the final preclinical 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.

Dr Melton is collaborating to develop an implantation device to protect the cells and said that he and his colleagues at Massachusetts Institute of Technology are testing has thus far protected beta cells implanted in mice from immune attack for many months. “They are still producing insulin,” Melton said.

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.

DRWF Research Manager Eleanor Kennedy agreed that this was an important piece of research but added a note of caution. "It is exciting to see these results," she said. "Professor Melton’s group has worked in the field for many years and to see this research come to fruition is great news. However, these results are still based in animal models and it could take several years before we will know how effective this potential treatment is for the treatment of type 1 diabetes in people"

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