For the First Time, Stem Cells Were Created Specifically to Treat Disease

Researchers have used adult body cells to create stem cells, and eventually a human embryo, that might have an actual therapeutic use in fighting disease—in this case, type I diabetes.

It’s a huge breakthrough in stem cell research in what has already been an exciting year. Earlier this month, researchers reported making a human embryo using an adult’s body cells and a donor egg. Now, researchers at the New York Stem Cell Foundation have taken body cells from a diabetic patient, transplanted the nucleus from those cells into a donor egg that has had its genetic material stripped, and allowed it to begin dividing. The researchers, led by Dieter Egli, report that the new cells not only began dividing normally, but also began producing insulin naturally—a breakthrough that could eventually lead to a cure for the disease, in which patients are normally reliant on daily insulin injections.

“We show for the first time that we are able to derive diploid, patient-specific stem cells and are able to induce these stem cells into becoming cells that produce and secrete insulin, showing that this technique should be useful for the development of cell-replacement therapies for diabetes,” Egli said in a conference call with reporters.

The work was published in the journal Nature. Although not noted in the paper, Egli says that the cells work just as well as normally-functioning pancreas cells in non-diabetic humans.

“In independent work, the cells we make secrete just as much insulin on a cell-to-cell basis as cells in the human pancreas,” he said. “This is quite remarkable.”

In additional tests in mice, his team was able to essentially cure the disease. He says that they have taken these cells and transplanted them into mice, where they functioned normally and secreted insulin into the bloodstream. This isn’t early, theoretical research. The team is actively looking to translate this into a real-world stem cell therapy, and is hoping to get Food and Drug Administration approval to begin human trials as soon as possible, though he would not put a timeline on when that might happen.

In an accompanying commentary about the research, Case Western Reserve University professor Insoo Hyun, who was not affiliated with the study, wrote that “repeated cloning of embryos and generation of stem cells, now using cells collected from adults, increases the likelihood that human embryos will be produced to generate therapy for a specific individual.”

Susan Solomon, a cofounder of the New York Stem Cell Foundation, said that the work has taken nearly a decade and that the research is an “important step forward” to turning the promise of stem cell therapy—which could eventually offer treatments for diseases such as Alzheimer’s Parkinson’s, and cystic fibrosis—into a reality.

Despite all the promise that stem cells offer—in 2001, when George W. Bush banned federal funding for stem cell research, it seemed like cures for these diseases were right around the corner—progress has been slow. Scientists have become much better at actually deriving stem cells (and reprogramming them into different types of body cells), especially from adult donors, but actual therapies have taken much longer to come along. This seems like a huge step. From the start of this experiment, the goal was to create insulin-producing cells from a diabetic donor.

“This is a day that has long been sought,” Solomon said. For diabetes, “there have not been any new treatments or cures and advancements have been slim … it’s important to pursue all avenues to develop new, effective therapies.”