by CHRIS ROGERS
Living with diabetes takes constant vigilance: counting carbs, checking blood sugar, and taking insulin. Even so, serious complications — heart conditions, kidney disease, and vision loss — are not uncommon. A full 10 percent of Americans have diabetes, according to Centers for Disease Control, (CDC) and in 2017, it was the seventh leading cause of death in the U.S. But, thanks to support from the Wanek family of Arcadia, researchers at the California-based City of Hope have developed a vaccine against type 1 diabetes, which is in clinical trials with very promising results so far.
Where diet and exercise play a large role in causing or preventing type 2 diabetes, the root causes that lead some people to get type 1 diabetes are unknown, according to the Mayo Clinic. Type 1 diabetes is believed to be the result of a person’s immune system attacking their own pancreas and destroying beta cells, which produce insulin, the Mayo Clinic and National Institutes of Health report. Insulin enables cells to absorb blood sugar, aka glucose. Without it, blood sugar levels can soar, with terrible effects.
Some researchers have focused on trying to prevent type 1 diabetes by using drugs that suppress the immune system and stop it from destroying beta cells. However, suppressing the immune system has some serious downsides. Dr. Bart Roep, the director of the City of Hope Wanek Family Project for Type 1 Diabetes, pointed out, “You want your immune system to be prepared to fight cancer. You want your immune system to be prepared to fight COVID.”
Instead, Roep and his team are pioneering just the opposite approach: working with patients’ immune systems and teaching them not to attack beta cells. The treatment is a form of cell therapy, in which doctors take some of the patient’s cells, recalibrate them, and return them to the patient’s body. “We take cells of the patient, immune cells from the blood, and we train them outside the body in the laboratory with vitamin D3, believe it or not,” Roep explained in an interview. “This is a vitamin that is capable of doing many things, including training the immune system. This is not an immunosuppressant; it balances the immune system … When we do that, those cells become anti-inflammatory, and then we add the magic, which is a vaccine we created from insulin. And that we take together and inject under the skin of the patient.” That vaccine teaches the immune system to stop attacking the pancreas and the insulin-producing beta cells, he explained.
This approach has been dubbed an “inverse vaccine” because, whereas normal vaccines try to kick the body’s defense systems into high gear, this treatment aims to deactivate the destructive tendencies of overzealous immune cells. It could have applications for all kinds of autoimmune diseases, Roep said.
Major contributions from the family behind Ashley Furniture made the Wanek Family Project for Type 1 Diabetes possible. “Thanks to the Wanek gift, we can actually accelerate the ideas that we have rather than having to wait for pilot studies to be finished, because I’m in a hurry and so are people with type 1 diabetes,” Roep said in a City of Hope video.
Last summer, the Wanek Family Project team published the results of this vaccine’s first clinical trial. It was a small-scale experiment — just nine patients — but in those treated, the vaccine was both safe and functional. Six months later, the diabetes patients’ blood sugar control and insulin needs were stable. The treatment “protects [beta] cells from autoimmune destruction and can act as curative therapy for type 1 diabetes,” researchers Tatjana Nikolic, Jaap Zwaginga, Roep, and their colleagues wrote in The Lancet.
Roep was even more thrilled by what trial participants’ doctors told him about how the participants were doing long after the trial. “What we’ve seen is that although these patients have had disease for 10-15 years, all the patients were at an all-time low of HbA1c,” he said. HbA1c is a sign of how much blood sugar someone has; so, for diabetes patients, lower levels are a good thing. “That basically means their blood glucose control has never been better,” Roep said. “I didn’t know what to make of this. I was totally blown away.”
The Wanek Family Project is currently enrolling patients in California in another trial. Roep said it is the final phase I trial before the treatment could move to phase II trials, possibly in a year or two. After phase III trials, the vaccine could be approved by the U.S. Food and Drug Administration.
The cell-therapy vaccine is not the only treatment the Wanek Family Project is working on. One of the drawbacks of cell therapies is that they require highly specialized labs to retrain the immune cells for each individual patient, making them expensive and hard to scale up. That won’t work for every patient, Roep said. So, his team is also studying medicines that could bolster beta cells, even in people who have had diabetes for a long time. By contrast to the tailored cell therapy, he explained, “Here we have a drug that we could make in unlimited amounts and offer to an unlimited number of patients.” Additionally, the Wanek Project is also researching treatments to stop and potentially reverse the complications of diabetes.
While these treatments could still be years away from the finish line, this life-changing research is happening far faster than it otherwise would. “Without the Wanek family, we would have been so much slower,” Roep said. “We would not be anywhere near where we are now.”
The next trial of the type 1 diabetes vaccine is slated to start this fall and focus on patients who’ve only had diabetes for a few years, to see if the vaccine can stop beta-cell destruction before it gets too far along.