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December 19, 2022

A Promising New Approach to Stopping Type 1 Diabetes

What if the cure to type 1 diabetes (T1D) is hiding in your own cells? 

A team of researchers from BRI and Seattle Children’s Research Institute have been asking this question for years – and it led them to develop a new type of therapy that could someday become a groundbreaking treatment for T1D and other autoimmune diseases.

T1D starts when immune cells go haywire and start attacking the pancreas. To try and stop these attacks, the researchers used gene-editing technology to create a special kind of cell called an engineered t-regulatory cell, or Treg (pronounced “tee-reg”). These cells mimic natural cells that keep the immune system in check. A new study, led by a BRI researcher and published in Science Translational Medicine, put these engineered cells to the test. In lab tests, the research team found that the engineered cells controlled the cells that attack the pancreas in T1D patients. What’s more, the engineered cells prevented T1D in preclinical models.

“It’s an important step because it shows that, by copying a natural process that protects the body, we might be able to stop T1D at the source,” says Soo Jung Yang, PhD, a BRI researcher who led the study.

Creating a Defense Mechanism Against T1D

To understand this new approach, it helps to step back and understand how the immune system regulates itself in healthy people. Everyone’s has Tregs in their immune system. These cells are like traffic cops that help control T-effector cells, which fight bacteria and viruses.

Soo Jung Yang

When T-effector cells get out of line and start attacking the wrong thing, Tregs blow their whistles and yell, “Stop!” But this defense mechanism is altered in people with T1D. The T-effector attacker cells turn into out-of-control truck drivers barreling toward the pancreatic islets – the parts of the pancreas that create insulin. And no one is there to stop them.

Enter Dr. Yang, who has spent the past five years as a member of the Buckner Lab. She was one of the first to use gene editing technology – developed in partnership between BRI and Seattle Children’s Research Institute – to turn healthy immune cells into Tregs. This made it possible to create large numbers of Tregs that could be used as therapies. The new study built on this advance, by developing Tregs specifically designed to fend off the attacks that cause T1D.

“To create a new type of therapy, you have to build a lot of the methodology and technology from the ground up because no one has ever done it before,” Dr. Yang says. “For us, that meant finding ways to engineer Tregs and then figuring out how to instruct them to protect the pancreas.”

Bringing Hope to Millions of People with Autoimmune Disease

The study found that the engineered Tregs did exactly what the researchers hoped. In lab models, the cells traveled to the pancreas, stood guard and stopped T-effector cells. 

“The Tregs prevented T1D by blocking the cells that attack the pancreatic islets,” Dr. Yang says.

This is a key advance because it shows that the engineered Tregs have potential as real-world therapies. That would mark a huge improvement over today’s T1D therapies, which treat some of T1D’s symptoms but don’t address what causes the disease.

Now Dr. Yang and her colleagues plan to continue studying the mechanisms that allow the engineered Tregs to beat back the T-effectors. This will enable the researchers to keep refining their approach.  

“We believe it’s possible to create not only Tregs that are designed to stop T1D, but also to develop other versions that work against everything from rheumatoid arthritis to multiple sclerosis,” Dr. Yang says, “and we’re eager to keep refining our approach until it can be carried out of the lab and be applied in ways that could improve the lives of people around the world.”

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