C9orf72 protein identified as target in ALS immune response

Published 01/10/2025, 16:38
C9orf72 protein identified as target in ALS immune response

Investing.com -- Scientists have identified how the body’s immune system may be attacking a protein in neurons, potentially driving the progression of amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease.

In a study published Wednesday in Nature, researchers from La Jolla Institute for Immunology and Columbia University discovered that immune cells in ALS patients target the C9orf72 protein, triggering inflammation that accelerates nerve cell loss.

The research team found that CD4+ T cells in ALS patients recognize the C9orf72 protein and preferentially release IL-5 and IL-10 cytokines. This represents the first identified target of an autoimmune response in ALS, potentially reshaping treatment approaches for the disease.

"Our results reinforce the previous hypothesis that neuroinflammation has an important role in ALS disease progression," the researchers wrote in their paper.

The study showed that patients with mutations in the non-coding regions of C9orf72 had approximately six times higher immune reactivity against the protein compared to ALS patients with other mutations.

Interestingly, researchers also found that higher levels of IL-10-releasing C9orf72-specific T cells were associated with longer predicted survival times in ALS patients, suggesting these T cells might have a protective influence on disease progression.

The findings highlight potential new therapeutic strategies aimed at enhancing regulatory T cells and identify a key target for antigen-specific T cell responses that could enable precision treatments for ALS.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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