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Can a safe, cheap pill prevent type 1 diabetes?

Type 1 diabetes, an autoimmune disorder once known as juvenile diabetes because it tends to strike during adolescence or earlier, affects one in every 300 people. With the diagnosis comes the certainty of a lifetime of insulin injections, made necessary due to the destruction of insulin-producing cells in the pancreas by a misguided immune system.

Insulin is a hormone that alerts the body to the presence of glucose in the blood, typically after a meal. In insulin's absence, the body's tissues fail to take up glucose, a key energy source. Without several-times-daily insulin shots, type 1 diabetes patients' blood sugar levels can shoot up to dangerous heights - a condition called hyperglycemia.

There's never been any way to prevent type 1 diabetes, although it can be predicted based on the detection of self-targeting antibodies in a blood test. But screening for type 1 diabetes this way hasn't been particularly useful, because there's been nothing to be done for patients diagnosed in the asymptomatic phase except wait for them to become hyperglycemic and put them on insulin.

Now, an elaborate mouse study by Stanford immunologist and structural biologist Paul Bollyky, MD, PhD, shows that it might be possible to intervene during the asymptomatic stage of type 1 diabetes - using a pharmaceutical compound that's been on the global market for more than 40 years and has a terrific safety record - thereby stopping the immune system's stupid but relentless destruction of the pancreas's vital insulin-producing cells, and stave off hyperglycemia indefinitely.

Bollyky and his colleagues first showed that a particular substance, hyaluronan, builds up near insulin-producing cells in mice developing the murine equivalent of type 1 diabetes, confirming earlier findings in postmortem human pancreatic tissue that had been supplied to Bollyky's team by the Juvenile Diabetes Research Foundation.

"We wondered what would happen if we prevented that buildup," Bollyky told me when I interviewed him for my news release on the study. "And we knew a drug that does that."

As I wrote in our press release:

The drug was hymecromone, or 4-methylumbelliferone (4-MU for short). Prescribed in many European and Asian countries for painful, gallstone-associated spasms and sold by about 60 companies worldwide for research purposes, 4-MU inhibits hyaluronan synthesis. It is inexpensive, can be given orally and, over four decades of use, and has what Bollyky described as an "extremely boring safety profile": a very low rate of associated adverse events. "It's even approved in Europe for kids," he said.

The drug worked. Hyaluronan deposits near insulin-producing cells were greatly diminished, the mice's immune systems stopped attacking those cells, and blood sugar levels remained normal as long as the mice stayed on 4-MU.

The Food and Drug Administration has not licensed 4-MU for any indication in the United States. But Bollyky has received preliminary funding and is in discussions with the FDA for the initiation of a clinical trial of 4-MU for preventing type 1 diabetes in people. Its success would be a massive incentive for the introduction of a national screening program geared to catching type 1 diabetes early enough to derail it.

Previously: High blood sugar linked to reduced brain growth in children with type 1 diabetes, Can a series of DNA vaccine shots halt type-1 diabetes progression? and Researchers struggle to explain rise of type 1 diabetes
Photo by formatbrain

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