A compound initially crafted for a rare motor neuron disease may be stepping into the Alzheimer’s spotlight. Early findings from Northwestern University have scientists buzzing.
Originally intended to treat ALS—a brutal condition known for progressively paralyzing the body—this drug now shows signs it could help tackle memory loss and brain damage tied to Alzheimer’s.
Same Drug, Different Fight
It started with ALS. NU-9, a small molecule born in a Northwestern chemistry lab, was designed to protect upper motor neurons—those crucial cells in the brain and spinal cord that falter in ALS.
Fast-forward to this year, and researchers have stumbled upon something unexpected. NU-9 doesn’t just help in ALS models—it’s doing something pretty remarkable in Alzheimer’s models too.
It’s not just another symptom-targeting med. NU-9 works deeper. Instead of patching things up, it goes after the root problem: misfolded proteins gumming up the brain.
Why That Protein Buildup Is a Big Deal
In Alzheimer’s, these sticky protein clusters—especially amyloid beta oligomers—are bad news. They form toxic clumps, stick to neurons, mess with brain signals, and eventually lead to cell death.
And those proteins? They start out good. Normal, even. But when they lose their shape, they’re like guests overstaying their welcome—causing trouble wherever they go.
In studies, NU-9 not only stopped the buildup of these proteins in mouse brains but also helped clear out the gunk that was already there. One quick win: it protected the dendrites—those branch-like parts of neurons that keep the lines of communication open.
One researcher summed it up: “These are good proteins gone bad.”
What It Actually Did to Mice
The drug was tested on mouse models of Alzheimer’s—and the results were eye-opening. In one set of experiments, NU-9 improved memory performance. That’s huge. Because in Alzheimer’s, memory loss is one of the earliest and most devastating symptoms.
But it didn’t stop there.
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NU-9 reduced inflammation in the brain
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It stopped harmful protein clumps from forming
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Even after the drug was removed, benefits stuck around
That kind of lasting effect is rare—and impressive.
A Closer Look: How It Works Inside Cells
What’s going on under the hood? That’s where it gets interesting. NU-9 seems to activate lysosomes—those little recycling centers inside cells. They break down and dispose of waste like misfolded proteins. This isn’t just cellular housekeeping—it’s vital. It also uses an enzyme called cathepsin B to help digest unwanted proteins.
Here’s a simple comparison:
| Component | Function | Role in NU-9 Action |
|---|---|---|
| Lysosome | Breaks down cell waste and toxic proteins | Activated to remove amyloid clumps |
| Cathepsin B | Enzyme that helps lysosomes digest material | Helps NU-9 digest sticky proteins |
| Proteasome | Another cellular cleanup system | Not involved, surprisingly |
So while other drugs might aim at breaking up plaques after they form, NU-9 appears to help prevent them from showing up in the first place.
Familiar Faces Behind the Science
This isn’t some no-name startup operation either. The inventor, Richard B. Silverman, is no stranger to big-impact meds. He developed Lyrica, a blockbuster drug that treats nerve pain and seizures. Now he’s back with NU-9, working alongside William Klein, a neurobiologist known for his work on Alzheimer’s.
Both researchers have biotech startups aimed at bringing their work from the lab to real-world treatments. And they’ve got a history of getting things done.
Silverman’s company, Akava Therapeutics, is moving NU-9 forward. Klein co-founded Acumen Pharmaceuticals, which is also knee-deep in Alzheimer’s trials with a different drug. This isn’t their first rodeo.
A Possible Link Between Brain Diseases?
One of the more intriguing ideas to come out of this research is that maybe—just maybe—brain diseases like ALS, Alzheimer’s, Parkinson’s, and Huntington’s aren’t as disconnected as once thought.
Silverman said it plainly: “It has long been thought that every neurodegenerative disease is a completely separate disease, but our findings suggest that common mechanisms might connect them.”
If that’s true, then NU-9 isn’t just a one-trick pony. It could be the beginning of a whole new class of drugs that work across multiple diseases. Of course, they’re not popping champagne yet. Human trials still need to happen.
What’s Next for NU-9?
It’s early days. The drug isn’t in clinical trials for Alzheimer’s yet. It has been approved for ALS trials by the FDA, which means it’s cleared some important safety and regulatory hurdles.
The team at Northwestern wants to run more memory tests. They also want to fine-tune NU-9 to see if they can boost its effects even more. And yes, they’re already eyeing other diseases where protein clumps wreak havoc—like Parkinson’s and Huntington’s.
But the fact that NU-9’s protective effect lingers even after it’s stopped? That’s not something researchers see every day. One sentence here—just to let that sink in. This is the kind of breakthrough that gets scientists cautiously optimistic.
