REGALS: Regulatory T Cells in Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS) is a disease in which patients suffer continuous loss of neurons that control muscle, leading to an inability to voluntarily move, speak, chew, or breathe. Over 5,000 people in the U.S. are diagnosed with ALS each year, but because of the fatal nature of this neurodegenerative disease, only about 16,000 Americans are living with ALS at any given time. Currently, there is no meaningful treatment for ALS. Human and experimental studies reported a detrimental role of the immune system in neurodegeneration observed in ALS brain and spinal cord. In an effort to treat sporadic forms of ALS, we have organized an expanded access, compassionate use program at the Eleanor and Lou Gehrig ALS Center at Columbia University to harness the neuro-protective effects of cord blood-derived regulatory T cell (Treg) therapy in collaboration with Cellenkos, Inc. to ultimately slow and halt the progression of ALS. We will take advantage of this clinical trial to study the role of the immune system in sporadic ALS. The overall goal of our study is to test the safety and efficacy of third party cord blood-derived Treg therapy in ALS and to identify the type of toxic immune cells circulating in the blood and found in the brain and the spinal cord of sporadic ALS patients. We are leveraging cutting-edge technologies that are available to us through Columbia University Medical Center's genetic and human immunology cores to dissect the immune system cell by cell. This analysis will provide novel information about the type of immune cells that are generated in the blood and migrate to the site of neuronal injury in the brain and the spinal cord.