Harvard researcher Doug Melton is on a mission to cure type 1 diabetes. He retooled his own lab to pursue this mission when both of his children developed the disease.
For nearly a century, treating diabetes has remained pretty much the same—an injection of insulin to balance the body’s blood sugar supporting the conversion of food to fuel, reports Karen Weintraub of WBUR. Some believe material progress towards a cure is closer. Doug Melton knows that a cure probably will depend on the ability to restore the body’s own ability to make and deliver insulin.
Melton notes that “Evolution has made a cell whose job is to measure sugar and secrete out insulin… I believe that the biological solution—sort of nature’s solution—is the right answer to the problem.” After years of methodical research Melton has identified how to make beta cells—the cells in the pancreas that measure glucose levels and squirt out insulin as needed. He has even manufactured these beta cells for testing.
Moreover, Melton has taken the process of producing the beta cells in the laboratory and conducted experiments with animals—demonstrating that his manufactured beta cells can measure glucose and secret insulin when the body requires it.
Enter a Well-Funded Biotech Venture
Semma Therapeutics has taken notice. Apparently, the venture will launch a clinical trial to determine if Melton’s manufactured beta cells can work in humans. Semma is a serious player—founded in 2015 they have raised $163 million, and Melton happens to be a co-founder.
Nature Showing the Way
Melton is convinced nature shows us a way toward the type 1 diabetes cure. And each step along the way he can’t find any reason why there isn’t a cure. Obviously, Melton is restless and wants faster results—but from one perspective it is pretty amazing what he has pulled off thus far. However, he knows that even if his cells worked perfectly that is only half the problem.
Type 1 Diabetes Autoimmune Attack Mode Problem
Melton’s cells could work perfectly, but we still face the problem that type 1 diabetes creates a condition in which the immune system attacks the beta cells that produce insulin. This doesn’t change with the beta cells. The other half of solving the problem requires protecting these manufactured beta cells.
So, Melton and expanding team must figure out how to make the human beta cells (from multi-purpose stem cells) and protect them—like putting the beta cells into some form of protective “tea bag.” In this analogy, Melton notes, “The insulin and the glucose can go across, but the cells stay in… and importantly the immune cells can’t go in and attack it.” Semma Therapeutics will test this concept next year.
Enter Gene Editing
Melton and the Joslin Diabetes Center are looking at ways to edit genes in the manufactured beta cells to enable them to be invisible to the immune system—they can be thought of as designer beta cells. Researchers have found that by editing and testing the genes one by one, they have found that only about a dozen seem to hide the beta cells from the immune system when studied in mice with a version of type 1 diabetes.
Stephan Kissler, an assistant professor at Harvard and an investigator at Joslin reports, “We think that when you mutate that one gene, you protect the cells against the autoimmune attack without really changing the beta cell and without preventing it from being recognized by the immune system.
The goal: gene-edit manufactured beta cells before they are delivered to a patient—they end up working like regular beta cells but don’t suffer from the same immune attack, reports Weintraub.
Research Costs and Economics
Melton has run up tens of millions in costs over the last 15 years—supported by federal grants and private foundations. This doesn’t compare to the $200 billion the U.S. government spends each year to treat and manage diabetes.
Melton believes that if it takes even $100 million to cure the disease it is a great deal. TrialSite News couldn’t agree more. The cost of diabetes is staggering.
Dr. David Nathan, Director Diabetes Center at Massachusetts General Hospital is a little more skeptical in outlook. “They’re coming along but I don’t see that on the near horizon.” Dr. Nathan believes we are at least a decade out from any kind of cure—if not longer. Dr. Nathan is pursuing various ways to make treatment easier—follow the link below to read the entire story.
In the meantime Melton continues his quest. We are thankful for brilliant researchers such as Doug Melton—it is their commitment to solve the problem, crack the code and develop the cure that promotes progress.