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June 15, 2021

New Drug Aims to Retrain the Immune System in T1D

Carla J Greenbaum

What if doctors could treat type 1 diabetes (T1D) by teaching the immune system to stop attacking cells in the pancreas? BRI researchers are working with the pharmaceutical company Novo Nordisk and global research consortium T1D TrialNet, testing a new approach that aims to do just that.

“We are trying to retrain the immune system, by telling certain immune cells to stop attacking the pancreas,” says Carla Greenbaum, MD, director of BRI’s Diabetes Research Program and director of TrialNet Hub. “This is the first time that industry has partnered with TrialNet to test a brand-new drug and recognizes that TrialNet sites like BRI are key to moving therapies to clinical practice.”

Testing a New Therapy

This trial, the TOPPLE Study, is testing a treatment that uses a plasmid-based approach. Plasmids are tiny rings of DNA that can be engineered to carry information into cells to change what they do. Scientists splice genes encoding proteins and other information into plasmids, which can teach immune cells not to target cells that produce insulin. This could preserve those cells and enable the body to keep producing insulin.

TOPPLE is a Phase I trial, which is the first time a therapy is tested in people after comprehensive testing in the lab. This type of trial tests a therapy in a small number of people to assess its safety, look for side effects and determine the right dosage. The TOPPLE team will give four groups of participants slightly different doses of the plasmid treatment or a placebo, to study potential side effects and the right dosage.

If the therapy meets the criteria set in the Phase I trial, future trials will test the therapy in larger groups of people. Those trials would continue to monitor for potential side effects and study how the therapy impacts immune cells’ attack on the pancreas.

“We're extremely grateful for our research participants,” Dr. Greenbaum says. “They play a crucial role in pushing science forward and help us move closer to better treatments and prevention for this debilitating disease.”

BRI has many trials for people living with T1D and their family members. To learn more or get involved, contact us at 1-800-888-4187 or

The TOPPLE study is currently recruiting participants

You may be eligible if you: 

  • Are 18-45 years
  • Have been diagnosed with T1D in the last four years

To learn more about or get involved with the study, please contact us: 800-888-4187 or

Originally printed in the the Summer issue of the Powering Possibility Newsletter

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