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La Jolla Scientists Make Breakthrough Discovery in ALS Research

For decades, amyotrophic lateral sclerosis (ALS) has remained one of medicine’s most devastating mysteries. But now, La Jolla scientsts are shedding new light on this progressive disease, offering hope to thousands of patients and their families.

Researchers at the La Jolla Institute for Immunology, working alongside colleagues at Columbia University’s Irving Medical Center, have discovered compelling evidence that ALS—also known as Lou Gehrig’s disease—may actually be an autoimmune condition. This groundbreaking finding could revolutionize how we understand and treat the disease.

Understanding ALS

ALS is a progressive neurodegenerative disease that attacks nerve cells in the brain and spinal cord, gradually stripping away the brain’s ability to control muscle movement. As the disease advances, patients may lose the ability to speak, eat, move, and ultimately breathe.

The statistics are sobering. Approximately 5,000 Americans receive an ALS diagnosis each year, and about half of patients succumb to the disease within 14 to 18 months, typically due to respiratory failure. While roughly 10% live with ALS for a decade or longer—as physicist Stephen Hawking did for an remarkable 55 years—most patients face a rapidly progressing condition. Baseball legend Lou Gehrig himself died just two years after his diagnosis.

The Breakthrough Discovery

What makes this research from La Jolla scientists so significant? The team discovered that in people with ALS, inflammatory immune cells called CD4+ T cells mistakenly attack proteins that are essential to the nervous system. It’s a case of friendly fire within the body.

“An immune response can target a disease or allergen, but in some cases it can target something in the body,” explains La Jolla Institute for Immunology Professor Alessandro Sette, who co-led the study. “That can lead to things like diabetes or rheumatoid arthritis, so there are a number of situations in which the immune response can target the body, and that is not what you want.”

This type of “self-attack” is the hallmark of autoimmune diseases—conditions where the body’s defense system turns against itself.

Two Responses, One Disease

The research revealed something particularly intriguing: ALS patients actually experience two simultaneous immune responses. One is inflammatory and harmful, while the other is anti-inflammatory and protective.

“The first thing was determining there is an autoimmune response with ALS,” Sette notes. “Next was that there are two different flavors of immune responses going on at the same time in the same individual.”

Perhaps most importantly, the La Jolla scientists found that patients with stronger anti-inflammatory responses had longer projected survival times. This discovery could prove invaluable for developing new treatments.

What This Means for Treatment

While Sette cautions that we’re still in the “early days” of this research, the implications are profound. Understanding the specific target of these immune cells opens the door to designing more effective therapies.

Tanner Michaelis, a research technician at the La Jolla Institute for Immunology and the study’s first author, suggests that future ALS therapies might work by boosting protective CD4+ T cell responses while reducing harmful inflammation.

“Hopefully, now that we know the specific target for these immune cells, we can make more effective therapies for ALS,” Michaelis says.

Sette envisions other research groups and pharmaceutical companies building on this foundation, launching additional studies and clinical trials. However, he’s careful to manage expectations about timelines.

“This is not something that is going to be translated into a treatment in the immediate future,” he acknowledges. “But if our research will translate into something that can help ALS patients, I would be so delighted and so happy.”

A Beacon of Hope

For the ALS community, this research represents more than just scientific progress—it’s a beacon of hope. As La Jolla continues to establish itself as a global hub for biomedical innovation, discoveries like these remind us why this work matters so profoundly.

While a cure may still be years away, understanding ALS as a potential autoimmune disease fundamentally changes the conversation. It transforms the disease from an unknowable enemy to one that can be studied, understood, and ultimately targeted with precision therapies.

For the families touched by ALS, and for the researchers working tirelessly in La Jolla’s laboratories, that shift from mystery to mechanism represents the first real step toward a future where ALS is no longer a devastating diagnosis, but a treatable condition.