The Sanford Project: T-Rex Study has reached its midway point through a Phase II, fast-tracked trial.
A potentially ground-breaking study into cell therapy treatment for type 1 diabetes (T1D) pediatric patients has reached its midway point.
The Sanford Project: T-Rex Study, a Phase II clinical trial conducted by Sanford Health and Caladrius Biosciences, is looking into Caladrius cell therapy CLBS03’s efficacy on patients’ T cells.
The trial’s therapy, which received Fast Track designation from the US Food and Drug Administration (FDA), seeks to prevent patients’ immune system from destroying insulin-producing beta cells while combating the condition. T cells are extracted from randomized patients, purified and multiplied, then re-administered to the patients’ blood. The other half of randomized patients are treated with placebo, for comparison.
T1D is an autoimmune disease, Kurt Grifin (pictured), MD, PhD, director of clinical trials for the Sanford Project said. It differs from the “run of the mill” type 2 diabetes (T2D) diagnosis in that supplemental insulin is needed for survival. Therefore, insulin is a treatment — not a cure.
The idea of bolstering T cells follows about 15 years of research into autoimmunity modulation for T1D, Griffin said. There’s been investigative results with treatments approved for conditions such as rheumatoid arthritis, but none as promising at CLBS03.
“They’re not cells off the shelf — they’re that person's own immune cells,” Griffin said. “It’s really a very exciting approach that no one’s done before.”
The research team opened the study to patients aged 8-17 years old who had been diagnosed within 100 days of the trial beginning. Griffin said children are more easy to find for the latter half of those qualifications, which coincides with T1D being a “classic child onset disease.”
It’s additionally important to find positive results in pediatric patients because of T1D’s far more immediate and critical impact on younger children than other age groups. Griffin noted a diagnosed toddler could be playing outside, then be in the Intensive Care Unit within 2 days.
The first half of the trial featured 56 of 111 participants having received treatment or placebo already. Researchers plan an interim analysis of CLBS03’s early therapeutic effects 6 months after treatment, with results to be announced in late 2017 or early 2018.
For the latter half of the study — which is still seeking enrollment — the minimal age qualification was lowered from 12 to 8 years old.
But if the cell therapy treatment proves efficacious and safe, it’s equally promising for adult T1D patients.
“The problem with working on cells is that an immune system could still kill them,” Griffin said. “Rebalancing the immune system is important in people with longstanding diabetes as well.”
A press release regarding the study's progress was made available.