Scientists Find Protein That Shields Brain Cells From Death

For the first time, scientists have found a way to stop brain cells from self-destructing in diseases like ALS, offering real hope for conditions that currently have no cure.

Researchers at the University of Utah's Pulst-Scoles Laboratory discovered that targeting a single protein called STAUFEN-1 protects neurons from a deadly process called apoptosis. When they reduced levels of this protein, brain cells survived attacks that would normally kill them.

"We can prevent DNA damage and they survive," said Dr. Mandi Gandelman, the study's lead author. The findings appear in the journal Cell Death & Disease.

The discovery matters because over 90% of neurodegenerative diseases aren't inherited. They develop when brain cells receive signals telling them to die, even though they shouldn't. Understanding how to block those signals could help millions of people.

The research team tested their approach using human neurons grown in laboratory dishes from reprogrammed adult cells. These lab-grown brain cells behave like real neurons, forming connections and sending signals just like cells inside our heads.

When the researchers exposed these neurons to compounds that cause DNA damage, the cells normally died in massive numbers. But with STAUFEN-1 levels reduced, the neurons survived even severe damage.

The team then tested mice carrying a mutation that causes ALS in humans. These mice naturally accumulate DNA damage as they age. When researchers bred them with mice that had lower STAUFEN-1 levels, the deadly p53 pathway that kills neurons became less active.

"This works in human neurons and that this also works with the triggers of neuronal death in the mouse," Gandelman explained. "That gives us a good answer as to how this could be valuable for therapeutics."

The Ripple Effect

The implications stretch far beyond ALS. STAUFEN-1 levels increase in many neurological diseases, including Parkinson's and Alzheimer's, and all of them show increased DNA damage. By targeting this one protein, researchers might be able to help people with multiple devastating conditions.

The lab is already developing therapeutic molecules for clinical trials. While there's still work ahead, the path from laboratory discovery to patient treatment is now clear.

The research represents a fundamental shift in how scientists think about treating neurodegenerative diseases. Instead of trying to fix what's already broken, they're learning how to prevent the breakdown in the first place.

For families watching loved ones struggle with these diseases, this discovery transforms "someday" into a timeline they can see.