Study uses child’s own immune system against type 1 diabetes

From the Summer 2017 edition of Vanderbilt Medicine Magazine

Grace Long, the first patient enrolled in the multi-site T-Rex Study, talks with Daniel Moore, M.D., Ph.D., far left, Faith Brendle, R.N., and Jordan Smith, R.N. Photo by Anne Rayner.

Eighteen-year-old Grace Long had just been accepted at the renowned United States Naval Academy, with plans to become a nuclear engineering officer. Then, she learned she had type 1 diabetes, an immediate disqualifier for military service.

Rather than allow the news to derail her, Long immediately began to investigate how she could spin her disappointment into a positive. She and her mother, a former family practice physician, searched for a way her diagnosis could benefit science through participation in a research study. Long is now part of a Sanford Research-led, multisite clinical trial that uses a child’s own immune system to create a therapy that has the potential to diminish the severity of type 1 diabetes. She is the first individual to be enrolled in the study at the Vanderbilt University Medical Center (VUMC) site.

“If I was going to be diagnosed with something like this, something good might has well come out of it,” Long said. “I live in Virginia, and there wasn’t really any study available in my area. I love the idea of this specific study, as opposed to many of the other studies that are looking at the use of immunosuppressant drugs. The idea of taking something my own body already has and making it better was interesting to me.”

The Sanford Project: T-Rex Study (NCT02691247), conducted in collaboration with Caladrius Biosciences Inc., is the first of its kind in the United States and is named after the body’s regulatory T cells, or Treg cells, that regulate the immune system. Treg cells are harvested from a study participant’s blood, then multiplied in a laboratory to create an investigational treatment called CLBS03. The CLBS03 is then given back to the same patient intravenously. Study participants are children, ages 8 to 17, who have recently been diagnosed with type 1 diabetes. Long was still 17 when she began the study, so she met the criteria.

Type 1 diabetes is an autoimmune disease in which a person’s body attacks and destroys the insulin-producing beta cells in the pancreas. Individuals with type 1 diabetes have been shown to have fewer Treg cells than others without the disease. Investigators hope the investigational treatment can prevent the immune system from continuing to destroy beta cells.