First U.S. Trial of Immunotherapy Deemed Safe for Type 1 Diabetes

In the first large U.S. study of its kind, researchers from UC San Francisco found that immunotherapy is a safe approach that may help to improve the body's response to insulin.

In the Phase 1 trial, researchers used regulatory T cells (Tregs) to help "retrain" the immune system. These cells can help to stop the immune response that causes type 1 diabetes, but many therapies that use this approach leave patients with an increased susceptibility to infection or disease.

In the new trial, however, the Tregs used were able stop the body's attack on beta cells without compromising the immune system. The modified Treg cells used in the study were derived from the patients' own cells using a technique called fluorescence-activated cell sorting (FACS), which enabled researchers to create therapeutic Treg cells.

"This could be a game-changer," said first author Jeffrey A. Bluestone, PhD. "For type 1 diabetes, we've traditionally given immunosuppressive drugs, but this trial gives us a new way forward. By using Tregs to 're-educate' the immune system, we may be able to really change the course of this disease."

A breakthrough for autoimmune diseases?

The trial included 14 adult patients who all received large infusions of the Treg cells. Results showed the cells were tolerated well by all the patients, and that up to 25 percent of the cells that were infused were still detectable in the patients' bodies a year after they had received the therapy.

Due to the positive safety results - the patients' immune systems did not seem to be compromised by the infusions - a Phase 2 trial is being planned for potential drug development.

If successful, this type of treatment could prevent and treat type 1 diabetes, as well as other autoimmune diseases like rheumatoid arthritis or lupus.

The work has largely been funded by a recent $10 million donation from Silicon Valley tech entrepreneur Sean Parker, who founded the Sean N. Parker Autoimmunity Research Laboratory at UCSF.

"Using a patient's own cells - identifying them, isolating them, expanding them, and infusing them back into the patient - is an exciting new pillar for drug development," said Bluestone, "and we expect Tregs to be an important part of diabetes therapy in the future."

Source: UCSF