HeConnection-Treatment-PreventionIf it works in humans, it could get prevent the need for #insulin injections…

This pill staved off #type1diabetes in mice, and it may one day prevent the disease in people, too.

The pill blocks the buildup of a specific acid in the #pancreas, which then stops the disorder from taking hold, according to research published this week in the Journal of Clinical Investigation. Doctors could screen patients for traces of the disorder, and give the medication to those at risk of developing the condition. The pill would then prevent the body from getting hurt significantly.

“Instead of taking #insulin for the rest of your life, it’d just be a pill, and you wouldn’t get [type 1] diabetes.”

People with type 1 #diabetes must regularly inject themselves with insulin for the rest of their lives in order to control the amount of glucose (sugar) in their blood. Otherwise, too much #bloodsugar can wreak havoc on their bodily systems. But if the disorder never takes hold, then there is no longer a need for lifelong shots. “Instead of taking insulin for the rest of your life, it’d just be a pill, and you wouldn’t get #diabetes,” said Dr. Paul Bollyky, a professor of infectious disease at Stanford.

The idea sprang from analyzing what happens in the pancreas before it stops producing insulin. This has been incredibly difficult for scientists to do, since pancreatic tissues can only be studied after a person dies. “You can’t biopsy the pancreas,” said Bollyky. “By the time you can look in the tissues, it’s after people die, so you’re years to decades out from what happened as far as when the disease started to form.”

To get around this, the researchers used pancreatic tissues from people who had died right after being diagnosed with type 1 diabetes. This collection of pancreases helped the team see what was going on in the early stages of the disorder. The pancreases had a significant buildup of something called hyaluronic acid, which is often found in areas of #inflammation. And the build-up was most notable near the pancreatic #betacells — the ones that make insulin.

This buildup may be helping the immune system do extra damage to the pancreas, Bollyky says. Hyaluronic acid is instrumental in causing sites to swell; it soaks up water and increases fluid buildup. It also makes inflammation worse by suppressing the body’s regulatory T cells (Tregs), which help to keep the immune system in check. “Think of your immune system as an army, and the regulatory T cells are the military police,” said Bollyky.

“If a marauding army conquers a new area, there’s lots of pillaging going on afterward, so you need military police to keep that from happening. They rein in the army and make them behave.”

But without the regulatory T cells, the immune system can damage otherwise healthy cells. Bollyky thinks the hyaluronic acid is preventing the #Tregs from showing up in the pancreas, so the immune system isn’t being told to stand down from a person’s own body. The Stanford researchers wondered if getting rid of the hyaluronic acid would allow the Tregs to restore balance

So they turned to a drug called #hymecromone, which is already used in Europe and Asia to treat complications associated with gallstones. It also has the side effect of shutting down hyaluronic acid production. The researchers tested out hymecromone’s abilities by giving it to mice that had immune systems engineered to attack the pancreatic beta cells.

#Hymecromone may be safe for use overseas, but it hasn’t been approved yet by the FDA.

In the mice taking the drug, the Tregs showed up in the pancreas and subdued the overactive immune response. The immune cells still stayed in the pancreas, but they didn’t touch the beta cells. “The invading army doesn’t go anywhere, but all of a sudden it becomes respectful,” said Bollyky. “It’s respectful inflammation that’s not destroying your beta cells anymore.”

Perhaps the biggest hurdle the researchers have to overcome is showing the drug works the same in humans. Bollyky says they’re moving on to human clinical trials soon. But only a small percentage of drugs make receive full FDA approval. Only a third of drugs that enter the second stage of clinical testing typically required by US regulators move on to final stage. Bollyky says he’s confident that the drug will at least be considered safe, since it’s already used abroad. However, rodents and humans aren’t the same, so what works for one animal may not work for the other.

And then there’s still a lot of work to be done before people can start using this drug to treat diabetes. While hymecromone may be safe for use overseas, it hasn’t been approved yet by the U.S. Food and Drug Administration. Additionally, Bollyky wants to rework the drug so that it’s something people can take once a week. Right now, to match the mouse dosing schedule, they would have to take it multiple times a day.

Health-e-Solutions comment: This could be a significant prevention strategy IF it is proven safe and effective for humans.  People would have to take the pill for the rest of their lives, though it might spare them #InsulinInjections if it works to prevent beta cell destruction by the body’s immune system. We won’t get too excited until we see human trials that show results similar to those in this mouse study. If they do we’d be satisfied to cal this prevention strategy a cure for type 1 diabetes before it progressed!

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Journal Reference: Nadine Nagy, et al. Inhibition of hyaluronan synthesis restores immune tolerance during autoimmune insulitis. J Clin Invest. doi:10.1172/JCI79271