T-regulatory Cells in ALS

Based on data collected in a previous study with a small group of patients, evidence was found to show that interfering with the immune system using Treg cells slowed ALS disease progression. It is known that Treg cell numbers and function are reduced in patients with ALS and in some patients with lower Treg cells, they have a more marked rapid progression of their ALS. For this study, there are two sites (in Houston, Texas and Boston, Massachusetts) in which Tregs will be taken from participants, increased or expanded outside the body, and then re-administered back to the participants from which the Tregs came. This study has two parts and due to the pandemic two groups \[Group 1 and Group 2\]: 1. The first period is a 6-month, randomized, placebo-controlled part of the study to study the biological activity, safety, and tolerability of the monthly expanded Tregs administered intravenously (IV) OR placebo (saline) administered intravenously plus subcutaneous low-dose Interleukin-2 (IL-2) OR subcutaneous placebo (saline) injections in 12 adults with ALS. IL2 helps regulate the immune system's white blood cells. \[Group 1 only.\] 2. The second period is a 6-month OPEN-LABEL part of the study (no placebo) in which all participants will receive their own expanded Treg cells administered intravenously in combination with subcutaneous low-dose IL-2 at the following schedule: single dose of Treg cells twice (once per month with three IL2 injections per week); followed by double the dose of Treg cells twice (once per month with three IL2 injections per week); followed by triple the dose of Treg cells twice (one per month with three IL2 injection per week); followed by a 1 month follow-up safety visit after the last triple dose of Tregs with was received and the last set of IL2 injections three times per week injections. \[Group 1 and Group 2.\] Groups: GROUP 1 will go through the double-blind part of the study (receive either Tregs infusions and IL2 injections OR saline infusions and saline injections) for six months and open label part of the study (receive Treg injections and IL2 injections) for six months. GROUP 2 will receive only the open label (Tregs infusions and IL2 injections) for six months. This study is studying whether the enhancement of Treg numbers and function will slow disease progression. In the first study of Tregs, we completed a single-center, open-label phase I study of Tregs from people with ALS. Tregs were increased outside the body and returned back to the individual Treg owners in multiple doses every 2 to 4 weeks. This early study provided evidence in a small group of patients that treatment with autologous Tregs may be effective in slowing ALS progression.