DEC 16, 2017 02:23 PM PST
Using Gene Therapy to Treat Type 1 Diabetes
WRITTEN BY: Carmen Leitch
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Some diseases are directly related to dysfunction in the immune system, like type 1 diabetes. T cells can function as important regulators of the immune system, and scientists have been trying to engineer them for use in disease therapeutics; in some disorders, hyperactive T cells attack healthy tissue in the body, which can have devastating consequences on organ function.

"Instead of stimulating the immune system to seek and destroy cancer cells, treating autoimmune conditions will require programming a patient's own T cells to tell rogue immune cells to calm down," explained Dr. David Rawlings; he is Director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute and Chief of the Division of Immunology at Seattle Children's Hospital. Learn more from the video.

Utilizing existing gene-editing tools, Rawlings and colleagues have begun the process of creating T cells that may reverse the effects of type 1 diabetes, in which the pancreas is destroyed by immune cells. They are aiming for human clinical trials.

There are several kinds of T cells, and while regulatory T cells play a role in immune regulation, effector T cells are defensive responders to stimuli. In previous work by this group, gene editing successfully created T cells that could regulate other, dangerous effector T cells; those cells worked both in cells in culture and animal models. For this work, they are seeking to identify the deleterious effector T cells, and turn them into regulatory T cells.

"These engineered regulatory T cells, when returned to a diabetic's body, would have the potential to stop effector T cells from destroying the insulin-producing cells," explained Rawlings. "We believe these cells could be used to treat type 1 diabetes as it begins, possibly leading to a long-term cure."

The scientists wanted to hone in on the immune cells of the pancreas only. They turned to Dr. Jane Buckner, president of Benaroya Research Institute, and her colleagues for assistance.

Dr. Jane Buckner of the Benaroya Research Institute at Virginia Mason and Dr. David Rawlings at Seattle Children's Research Institute are leading research to develop an immunotherapy for type 1 diabetes. / Credit: Seattle Children's

"The benefit of using regulatory T cells for therapy is that the T cells can focus immune suppression to a specific organ without impairing the entire immune system," noted Buckner. "For type 1 diabetes, we want to ensure the engineered T cells travel to the pancreas and stop any T-cell attacks in the pancreas, leaving healthy immune cells untouched."

The scientists want to see this work eventually applied to patients so their lives will be improved. "The ultimate goal of this research is to bring an engineered regulatory T-cell immunotherapy to patients," said Rawlings.

Cures are rare in medicine, but delivering modified regulatory T cells to patients could free them from a lifetime of monitoring insulin levels if it works as well as could be expected.

"It's hard dealing with type 1 diabetes every day," said thirteen-year-old Juliana Graceffo, who has type 1 diabetes and must be constantly vigilant. "A cure would mean that I don't have to think about diabetes all the time, and I can just be normal."

Sources: AAAS/Eurkealert! Via Seattle Children’s

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