JDRF Round-Up with Aaron Kowalski

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As a parent of a child with type 1 diabetes, it can be hard to keep track of all the developments in the field, and to get a realistic sense of whether these developments will be available in the relatively near future, or years from now. So my husband and I were glad to get the chance to hear Aaron Kowalski, JDRF’s chief mission officer and vice president for research, speak in Boston last week. He gave a useful round-up of JDRF’s work: what’s almost here, what’s on the horizon, and what he’s most excited about. We’re at a “golden moment” in diabetes research, he said, with progress on many different fronts. Kowalski is following this progress not just out of professional interest but out of personal interest too, since both he and his brother Steve have type 1.

Kowalski described life with the disease as the “diabetes happiness seesaw—a teeter totter between glucose control and quality of life.” So one area of research that JDRF is funding are ways to help people get better glucose control. Kowalski shared some sobering statistics: 4 out of 5 children in the U.S. are not achieving their A1C goal of below 7.5%, as recommended by the ADA. Two thirds of adults are also not reaching their A1C goal. And the average A1C for a seventeen-year-old in the U.S. is 9%—equivalent to an average blood sugar of 210.

On one end of the spectrum, DKA, resulting from prolonged high blood sugar, remains common in teenagers and young adults. On the other end of the spectrum, episodes of severe hypoglycemia rise over time, meaning that older people are the ones who most need CGMs—yet they’re not covered by Medicare (Kowalski encouraged people in the room to pressure Congress to address this coverage gap). Meanwhile, even while using some of the best technology currently available, insulin pumps and CGMs, many people with diabetes still frequently experience lows at night.

JDRF is looking to help people improve their glucose control in a few different ways:

By funding the development of the artificial pancreas, a version of which will be on the market next spring. (Kowalski described looking forward to a time when people using an artificial pancreas could wake up “every single morning with a good blood sugar number.”)

By supporting the development of new infusion sets, a technology that has not improved for a decade. An infusion set that’s not prone to occlusions would be “a big deal,” said Kowalski.

By supporting improvements in CGM technology. Kowalski explained that JDRF has been talking with Google and Dexcom about developing a sensor that would be very small (the size of a penny), last for two weeks, and would render blood tests unnecessary. “It’s not a cure, but that’s pretty nice.” (Kowalski mentioned that even current CGM technology has been transformative for his family. His brother has been to the hospital more than a 100 times, due to hypoglycemia unawareness. “We shared a bedroom as kids and I could hear him go low, based on his breathing changing. I could hear him having a seizure. But he hasn’t had a severe low in three years. That has been such a huge transformation for our family.”)

By supporting development of glucose-responsive insulin, which would respond to changes in blood sugar by releasing more or less of the hormone as necessary. Kowalski described a bit of an arms race going on in this field, where Merck has already started human trials on its version of glucose-responsive insulin (GRI). JDRF, partnered with Sanofi, is supporting the early development of several different GRI technologies. “This would be transformative, the ultimate insulin. The good news is, the fact that Merck is moving on this has caused a groundswell of interest in the field. Companies are trying to outdo each other, and that competition will help.” (As a side note in terms of improvements to insulin, Kowalski mentioned how much he likes using Afrezza, an inhaled insulin. “It’s almost a miracle drug for me.” But the company that created it, MannKind, “is having a hard time staying afloat. If MannKind fails, that’s a big deal for other companies spending money on better diabetes treatments.”)

Glucose-responsive insulin is one of the technologies on the horizon that Kowalski says he’s most excited about. The other is encapsulation, where insulin-producing cells would be protected from the body’s auto-immune attack. Kowalski used the metaphor of a shark cage. “Instead of trying to kill all the ‘sharks,’ we physically protect the cells from their attack.” JDRF is providing funding to several different groups working on encapsulation, including ViaCyte, which is in human trials testing an implanted device that protects cells, [https://asweetlife.org/feature/documenting-viacytes-stem-cell-diabetes-trial/] and Daniel Anderson’s lab at MIT, which is working on an alginate coating for beta cells. [https://asweetlife.org/feature/the-road-to-a-type-1-diabetes-cure-encapsulation/]. “There are so many smart people working on this—it’s going to happen. I can’t wait to see the encapsulation data, I think we’re going to see really rapid progression.”

Still, JDRF’s ultimate goal is the “holy grail” of a cure. Kowalski pointed out that a study at the Joslin Diabetes Center has shown that even people with long-term T1D can still produce insulin—but not enough to regulate blood glucose, since the immune system of someone with T1D continues to kill off insulin-producing cells (he compared it to a game of whack-a-mole, with the beta cells being “whacked” by the immune system). To this end, JDRF is supporting research into immune therapies that would end the autoimmune attack characteristic of type 1. “It’s going to take time, but this is the end-game.”

 

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