T Cell Therapy Safe but Ineffective for Slowing T1D Progression

A clinical trial for a novel treatment for type 1 diabetes has concluded that while the therapy is safe for children, it does not appear to slow the disease's progression. The study, known as the Sanford Project T-Rex Study, was a Phase II trial that included 113 young patients aged 8 to 17 who had been recently diagnosed with the condition. The participants were administered either a high or low dose of the experimental cell therapy, named CLBS03, or a placebo.

The primary objective of the trial was to evaluate the safety and tolerability of CLBS03, an autologous regulatory T cell therapy developed by Lisata Therapeutics and Sanford Health. The therapy involves the use of the patient's own regulatory T cells, which are expanded and functionally enhanced in vitro using a proprietary method. The cells are intended to regulate effector T cells, potentially preventing the destruction of β-cells and restoring immune balance.

The results, published in Science Translational Medicine, indicated that the therapy was well-tolerated, with transient activation of memory regulatory T cells observed in the high-dose group one week post-treatment. However, the treatment did not significantly impact C-peptide levels, a key indicator of β-cell function and insulin production. Neither the high nor low doses demonstrated a significant preservation of β-cell function, with p-values of 0.21 and 0.94, respectively.

Despite the lack of therapeutic effect on β-cell function, the study provided valuable insights. Jay Skyler, deputy director at the University of Miami’s Diabetes Research Institute, highlighted the safety of the therapy and the process of expanding and transporting the cells for treatment. "It shows us that these cells can be expanded, that you can ship cells that you collect at clinical sites to a central facility and expand them, ship them back, they can be improved. And this can be done safely," he said.

The CLBS03 therapy was originally developed by Caladrius Biosciences, which merged with Cend Therapeutics in 2022 to form Lisata Therapeutics. The findings of the study, while not leading to a breakthrough in treatment, contribute to the understanding of cell therapy's potential and limitations in managing type 1 diabetes.