Among the many mysteries of autoimmune diseases, we know one thing for sure: These diseases are connected. They often occur when immune cells called T cells mistakenly attack healthy tissue — say the pancreas in type 1 diabetes (T1D) or the gut in inflammatory bowel disease (IBD). This understanding has led BRI scientists to an intriguing question: Could medicines proven to slow down IBD also curb T1D?

How exactly does this drug work?

This therapy treats IBD by keeping immune cells that cause disease from entering the gut.

“These cells can’t get to your gut, so they can’t attack it,” Dr. Speake says. “We’re asking if this therapy will block cells from accessing the pancreatic islets – and prevent inappropriate immune attacks there, too.”

How will BRI find out if this therapy helps people with T1D?

Researchers are examining two possible treatment approaches. They’ll give 10 people who were diagnosed with T1D in the past few years vedolizumab. And they’ll give a second group of people who were diagnosed with T1D in the same timeframe vedolizumab plus another type of therapy called anti-TNF. Anti-TNFs are therapies that help stop a specific type of inflammation. In clinical trials, anti-TNF therapies have been shown to slow down T1D, and they are commonly used in both IBD and rheumatoid arthritis.

Researchers will give participants a short course of each therapy and collect blood samples several times throughout the process.

“We’ll be able to look at immune cells in those blood samples and ask if vedolizumab changes the immune cell profile in the blood — and see if the two therapies together move it even more in the right direction,” Dr. Speake says.

What is the EMU?

The EMU is a BRI program that runs small human trials to better understand exactly how a medicine works. The research team examines the particular cells and mechanisms a medicine influences to treat a disease. These studies often look at “repurposing” therapies, which means testing to see if medicines that treat one condition might also treat another.

EMU studies are different from clinical trials, which typically focus on understanding if a medicine works to treat specific features of a disease. Unlike EMU studies, clinical trials often don’t examine the exact immune mechanisms a therapy uses in as much detail.

Why are these smaller EMU studies beneficial?

These studies give scientists deeper insight into exactly how a therapy works. 

“For example, two previous EMU studies looked at two therapies that slow down inflammation and are targeted against the same immune pathway – so people thought their immune mechanisms would be the same. We learned that these two drugs work in completely different ways,” Dr. Speake says. “Knowing that moves us closer to being able to give people therapies that target the exact mechanisms that drive their disease.”

They can also help scientists collect preliminary data before conducting larger trials.

“If we collect promising data from this pilot study about how the immune system changes after vedolizumab, that would be a strong case for testing it in a larger clinical trial,” Dr. Speake says. “And if we don’t, we’ll save a bunch of time and money on a larger-scale study testing a drug that wasn’t going to be helpful in T1D.”

How do philanthropy and foundation funding help advance this work?

A grant from the Helmsley Trust made this study possible, illustrating how foundations and donors play a key role in research by helping fund early-stage studies of new, innovative ideas.

“It can be challenging to fund new and innovative projects because larger funding sources like the NIH require lots of preliminary data to apply for larger grants,” Dr. Speake says. “Foundation funding and philanthropy help bridge that gap by funding early-stage studies and enabling us to test these different and exciting approaches.”