An autologous and personalized regulatory T cell therapy is safe in patients with type 1 diabetes, but does not help preserve β-cell function.
An investigational autologous regulatory T cell therapy being developed by Lisata Therapeutics and Sanford Health is safe and tolerable when used to treat children with type 1 diabetes, but fails to slow or alter disease progression, according to Phase II data published Wednesday in Science Translational Medicine.
The mid-stage trial, called The Sanford Project T-Rex Study, enrolled 113 patients aged 8 to 17 years old who had recently been diagnosed with type 1 diabetes mellitus. Study participants were given a high or low dose of the cell therapy or a matching placebo. The goal of the study was to assess the tolerability of the investigational therapy and determine if it could alter disease course.
Wednesday’s results, which come from 110 patients, show that the experimental cell therapy was safe. Participants in the high-dose group showed a transient activation of memory regulatory T cells one week after treatment, which suggests that the transfer of the infused cell therapy was effective.
However, the treatment did not significantly influence C-peptide levels, a marker of pancreatic β-cell function that is indicative of insulin production. Both high and low doses failed to significantly preserve β-cell function, with p-values of 0.21 and 0.94, respectively.
This lack of therapeutic effect was consistent through one year of follow-up and was not influenced by age or baseline C-peptide concentrations.
“What is important is that it was demonstrated that this was safe,” Jay Skyler, deputy director at the University of Miami’s Diabetes Research Institute, told STAT News in an interview. The Institute was one of T-Rex’s study sites, but Skyler himself was not involved in the trial.
Skyler was not surprised that the investigational cell therapy showed no significant effect on β-cell function, but told STAT that the study’s findings were nevertheless valuable.
“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,” Skyler said.
The investigational personalized autologous cell therapy, dubbed CLBS03, was initially developed by Caladrius Biosciences and consists mainly of the patient’s own regulatory T cells. After harvesting, these cells are allowed to expand in vitro and are functionally enhanced using a proprietary method, which in turn allows them to regulate effector T cells.
This mechanism of action could prevent the self-targeting mechanism of these T cells, which destroy β-cells in type 1 diabetes, and restore immune balance.
In 2022, Caladrius merged with Cend Therapeutics and called the resulting entity Lisata Therapeutics.
Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.
