Neurology – neuroimmunology Neuroinflammation (2022). DOI: 10.1212/NXI.0000000000200019″ width=”800″ height=”457″/>

Treg suppression function and numbers in the Open-Label Extension (OLE). (A) Treg suppressive function was assessed in each participant at screening, 4 weeks after the second infusion of 1× dose of Tregs (week 34), 4 weeks after the second infusion of 2× dose (week 42), and 4 weeks after the second infusion of 3× dose doses (week 50). (B) Treg numbers were assessed in each participant at screening, 4 weeks after the second infusion of 1× dose of Tregs (week 34), 4 weeks after the second infusion of 2× dose (week 42), and 4 weeks after the second infusion of 3× dose (week 50). Data were presented as visit-specific estimates ± standard error and were compared for progression of continuous endpoints using a common base linear mixed model. A p Neurology – Neuroimmunology Neuroinflammation (2022). DOI: 10.1212/NXI.0000000000200019

Removing ALS (amyotrophic lateral sclerosis) patients’ own dysfunctional cells, fixing them, and then putting them back into the patients’ bodies is a safe, well-tolerated process that has been shown to slow or stop disease progression in a small number of patients. study by Houston Methodist Research Institute and Massachusetts General Hospital.

A study published recently in the journal Neurology: Neuroimmunology and Neuroinflammation, was designed as a 12-participant, randomized, placebo-controlled phase 2a trial followed by an open-label study. But the unpredictability of the COVID-19 pandemic, which has interrupted and even stopped many research projects across the country, has reduced the number of participants to 7 participants in the double-blind trial and 8 participants in the six-month open-label trial.

Seven patients with ALS underwent a randomized, placebo-controlled study in Group 1, and eight participated in an open-label study in Group 2. Both groups had dysfunctional regulatory T cells, or Tregs, removed from their bodies. When functioning normally, Tregs help reduce inflammation. In all but the placebo control group, the researchers expanded the faulty Tregs to restore their function, then injected them back into the patients’ bodies along with a low dose of interleukin-2 to help boost the immune system.

The results showed that the Treg treatment was 100% safe and tolerable, and it remained biologically active in ALS patients for more than a year. The treatment also slowed or stopped disease progression in most participants.

Darcy Garcia and her family attended the Houston Astros’ recognition of Lou Gehrig Day at Minute Maid Park on June 6, 2022, where this video premiered. Author: Houston Methodist

Participants included Houston Methodist patients Darcy Garcia, who was part of Group 1, and John Lay, of Group 2, both of whom reported slowing the progression of ALS during and shortly after clinical trial treatment.

“We look forward to a much larger clinical trial that will adequately assess the clinical efficacy and further characterize the long-term safety of this therapy,” said Dr. Jason Tonhoff, principal investigator. “These early results in a few patients are promising.”

The open-label segment of this latest Treg trial led to an additional publication reporting the identification of two biomarkers that could potentially help track ALS disease progression and measure how well ALS treatments are working.

John and Jill Lay talk about their family’s journey with ALS and the importance of research. Author: Houston Methodist

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Additional information:
Jason R. Thonhoff et al., Combination Treatment of Regulatory T Cells and IL-2 Is Safe, Tolerable, and Bioactive for 1 Year in People with Amyotrophic Lateral Sclerosis, Neurology—Neuroimmunology Neuroinflammation (2022). DOI: 10.1212/NXI.0000000000200019

Citation: ALS patients’ own cells may be a safe way to slow or stop progression (2022, October 7) Retrieved October 7, 2022, from safe-pathway .html

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