Novel therapy to increase beneficial immune cells shows promise in pilot type 1 diabetes clinical study

Type 1 diabetes (T1D) is caused when a person’s immune system mistakenly destroys their insulin-producing beta cells. How and why this misguided attack begins remains a mystery, but JDRF research has helped shed light on the disease process, opening the door to potential therapies. Previous research has shown that an important feature of T1D is the imbalance between two critical immune cells; T-regulatory cells (Tregs) and T-effector cells (Teffs). In T1D, the destructive Teff cells multiply unchecked, and the outnumbered Tregs are unable to rein them in, allowing a continuous attack on beta cells.

In previously conducted mice studies, an infusion of Tregs successfully treated T1D by reestablishing the balance between Teff and Treg cells. Now an important JDRF-supported translational study has tested this concept for the first time in people with T1D. The study results showed that increasing a person’s Treg cell count could rebalance the immune system and may provide benefit for people with T1D. The therapy involved collecting a person’s Treg cells, growing them in the lab, and infusing the new cells back into the same person to increase their Treg cell counts.

The pilot study included 14 people newly diagnosed with T1D and was led by Dr. Stephen Gitelman at University of California, San Francisco, and Dr. Kevan Herold at Yale University. The main goal of this small initial study was to evaluate the safety and tolerability of the experimental infusion, but it also captured some information on benefit. The infusions were well tolerated by the study participants. In addition to monitoring the overall health of participants, each person’s C-peptide level (an indicator of beta cell activity) was tracked to determine if the infusion accelerated or worsened their disease. The average C-peptide levels of the participants remained steady for the two-year study period, suggesting a stabilization of beta cell function. Clinical studies to confirm these preliminary findings and determine the practical utility of such a procedure for people with T1D are being planned by a company called NeoStem, Inc.

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Conceptual Illustration of Treg Therapy for T1D

Source: NeoStem, Inc.