The immune system’s role in ALS remains puzzling—it appears both harmful and protective. The goal of this project is to validate preliminary findings that autoimmune responses directed against the so-called C9orf72 protein are associated with longer survival in ALS patients.
2026 Tullie and Rickey Families Spark Awards finalist
Tanner Michaelis
Can we develop a vaccine to slow down the progression of ALS?
Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease that progressively weakens muscles, leading to paralysis and respiratory failure. With no cure available, most patients survive only four years after diagnosis.
Research shows ALS patients have increased immune cell infiltration in the brain and elevated activated immune cells in the blood. Much attention has focused on T lymphocytes, specialized immune cells that recognize specific protein fragments. While some T cells can damage neurons, recent evidence suggests others may actually protect against disease progression.
The key question is: What’s the difference between protective and harmful immune responses?
In a small-scale experiment, we found that autoreactive T cells targeting the human protein C9orf72 correlate with extended survival in ALS patients. Building on these preliminary findings, an in-depth characterization of this protective immune response will be performed in a larger cohort to substantiate the initial observation.
Understanding these protective immune mechanisms could revolutionize treatment approaches, shifting from broad immunosuppression to targeted enhancement of beneficial immune responses that slow disease progression.
“This project represents a shift in how we view the immune system’s role in ALS, not only as a source of harm, but potentially as a key to protection. By identifying immune responses linked to slower disease progression, I hope to uncover new therapeutic opportunities.”
