Immunotherapy May Treat ALS, Says Study

A study led by scientists at Oregon Health & Science University (OHSU) has found that a type of monoclonal antibody already tested in certain forms of cancer may be a promising treatment in stopping the progression of amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease. The study, which involved a mouse model and was confirmed in the tissue of human brains affected by ALS and donated after death, revealed for the first time that modulating immune cells can slow the progression of the disease.

The researchers used a monoclonal antibody targeting a5 integrin, which has already been developed and used in treating certain forms of cancer. Using postmortem tissue from 139 brains donated for research, scientists confirmed the presence of a5 integrin within areas of the brain associated with motor function. Specifically, they found a5 integrin expressed by microglial cells and macrophages in blood — cells associated with the immune system — to be highly pronounced in the spinal cord, motor cortex and peripheral nerves during ALS. They then tested the monoclonal antibody targeting a5 integrin in mice genetically predisposed to carry ALS and found that it protected motor function, delayed disease progression and increased mouse survival.

“When we blocked its expression in mice, we were able to slow down the disease,” said senior author Bahareh Ajami, PhD, assistant professor of molecular microbiology and immunology and behavioral neuroscience in the OHSU School of Medicine. “We hope that it will get to the clinic very soon.” However, Dr. Ajami added, “At this point, we cannot say it’s a cure but it’s a very interesting start. It may be similar to what immunotherapy did for cancer or will do for Alzheimer’s by targeting immune cells.”