REGALS: Regulatory T Cells in Amyotrophic Lateral Sclerosis
Abstract
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease affecting motor neurons (MNs) with the typical time from symptom onset to death of 2-3 years. The majority of ALS cases are sporadic. ALS, once considered to involve MNs exclusively, is now known as a multi-cellular and multi-systemic disorder in which MN degeneration is driven not only by cell autonomous mechanisms, but also by glial cell pathology and a confluence of other pathogenic mechanisms such as inflammation. Pathological studies have found immune abnormalities in the central nervous system (CNS) and also in the blood and cerebrospinal fluid (CSF) of subjects with ALS. Additionally, inflammation and immune abnormalities are also found in animal models of ALS. However, how the immune system is activated and what are the targets of these cytotoxic immune cells are not clear. Currently there are no meaningful treatments for ALS. In an effort to treat this aggressive form of ALS, we have organized an expanded access, compassionate use program on four sporadic ALS patients at the Eleanor and Lou Gehrig ALS Center at Columbia University in collaboration with Cellenkos Inc. This pilot clinical trial leverages cord blood-derived (CB) regulatory CD4+ T cell (Treg) therapeutic approach designed to halt ALS progression. Preliminary studies in this pilot clinical trial reported a slowing of the ALSFR-S slope decline rate from 3.0 per month to 0.7 per month associated with a decrease in inflammatory cytokines in the CSF.
Document Details
- Document Type
- Technical Report
- Publication Date
- Oct 01, 2023
- Accession Number
- AD1223451
Entities
People
- Angelica Ortiz
- Hanien Amer
- Jess Singleton
- Julia Yasek
- Marisha Liliah
- Neil Shneider
- Samuel J Levy
- Wassim Elyaman
Organizations
- Columbia University