(NewsNation) — Scientists have discovered what the immune system attacks in people with ALS, a breakthrough that could lead to new treatments for the fatal disease.
The study, published Wednesday in Nature, found that immune cells mistakenly target a protein called C9orf72 in patients with amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease.
It’s the first time researchers have identified a specific protein the body attacks in ALS.
The finding is especially important because mutations in the C9orf72 gene are the most common genetic cause of ALS, affecting 40% of people who inherit the disease and 10% of those who develop it without a family history.
Researchers at La Jolla Institute for Immunology analyzed blood samples from 40 ALS patients and 28 healthy people. The immune response against C9orf72 was four times stronger in ALS patients than in healthy people.
ALS destroys nerve cells that control muscles, causing progressive paralysis. Most patients die within four years of diagnosis. Doctors have long noticed inflammation in affected tissues, but couldn’t pinpoint what was triggering it.
The study offers hope for treatment.
Patients who lived longer had stronger anti-inflammatory immune responses, suggesting that boosting the body’s natural calming mechanisms might help slow the disease.
“These findings highlight the potential of therapeutic strategies aimed at enhancing regulatory T cells,” the researchers wrote. Regulatory T cells are immune cells that help prevent the body from attacking itself.
The discovery also changes how scientists think about brain diseases. Researchers once believed conditions like ALS, Parkinson’s and Alzheimer’s weren’t autoimmune disorders. Now, evidence suggests the immune system plays a major role in all three.
The research was funded by La Jolla Institute for Immunology, Kyowa Kirin North America, the Swedish Research Council and the Freedom Together Foundation.