According to SciTechDaily, researchers at Northwestern University have published a study on December 18 in Alzheimer’s and Dementia detailing a new experimental drug called NU-9. The small-molecule compound targets a previously unrecognized, highly toxic subtype of amyloid beta oligomer, dubbed ACU193+, which appears inside neurons and on brain cells called astrocytes very early in Alzheimer’s disease. In a pre-symptomatic mouse model, a daily oral dose of NU-9 for 60 days markedly reduced this toxic subtype, sharply cut early inflammatory responses, and decreased abnormal TDP-43 protein. The team, led by Daniel Kranz and professors William Klein and Richard Silverman, believes this approach could prevent or slow the disease’s chain of damage if given early, long before memory loss begins. The drug, now called AKV9 by startup Akava Therapeutics, is already FDA-cleared for human trials in ALS.
The Prevention Paradigm Shift
Here’s the thing that makes this research so compelling: it’s all about timing. As first author Daniel Kranz pointed out, Alzheimer’s pathology begins decades before symptoms show up. And by the time someone gets a diagnosis, the brain is already a mess. That’s probably why so many late-stage drug trials have failed miserably. They’re basically trying to put out a forest fire with a garden hose.
So this study flips the script entirely. They’re treating mice before any signs of trouble, modeling what it would be like to intervene in a 50-year-old with early biomarkers but zero cognitive issues. William Klein called the results “stunning,” specifically pointing to the drug’s effect on reactive astrogliosis—that’s the inflammatory activation of star-shaped brain cells that’s a core part of early disease. If you can stop that inflammatory cascade before it spirals, you might just stop the whole neurodegenerative process in its tracks.
The Hidden Culprit and a Familiar Strategy
The discovery of the ACU193+ oligomer subtype is a big deal. We’ve known amyloid beta oligomers are bad news, but this suggests there’s a specific, extra-toxic version acting as a key instigator. It seems to jump from stressed neurons to the surface of astrocytes, potentially kicking off that harmful inflammatory chain reaction. NU-9 appears to specifically lower levels of this bad actor.
Now, the comparison the researchers make is brilliant. Richard Silverman, who also invented Lyrica, likens it to managing cholesterol. You don’t wait for a heart attack to treat high cholesterol; you take a statin to prevent it. The vision is that future blood tests could spot Alzheimer’s biomarkers, and someone could start taking NU-9 as a prophylactic. It’s a shift from treating a devastating disease to managing a lifelong risk factor. That’s a huge conceptual leap.
Market Impacts and the Long Road Ahead
Let’s be real, though. This is promising preclinical mouse data. The path from a successful mouse study to an approved human drug is long, expensive, and littered with failures. But the players involved give it a serious pedigree. Silverman’s startup, Akava, is behind NU-9/AKV9. And William Klein is a co-founder of Acumen Pharmaceuticals, which is already in clinical trials with an antibody (ACU193) targeting this same oligomer subtype. So there are two parallel shots on goal from the same lab targeting this newly identified culprit.
If this early-intervention approach pans out, it could completely reshape the Alzheimer’s market. The current crop of drugs, like Leqembi, are approved for early symptomatic disease. A true preventive pill would be in a different league entirely, potentially prescribed to millions of at-risk adults. It would also place massive importance on the development and widespread adoption of cheap, reliable early diagnostic blood tests—another area seeing furious competition. The winners wouldn’t just be the drugmaker, but the diagnostics companies that get there first.
Cautious Optimism Is The Key
So, should we get excited? Cautiously, yes. The science is elegant, and the prevention analogy is powerful. But mice aren’t people, and Alzheimer’s is a beast of a disease. The team knows this—they’re already planning studies in a late-onset mouse model that better mimics human aging and tracking treated animals for much longer to see if symptoms truly are prevented.
It’s also worth noting that NU-9’s mechanism—rescuing a cellular pathway that clears toxic proteins—is being explored for ALS too. That suggests a broader potential for neurodegenerative diseases. Basically, we’re looking at a potential platform technology, not just a one-disease drug. The next few years of clinical trials will be critical. But for now, this is exactly the kind of innovative, early-stage thinking that the field desperately needs. You can follow more science news on Google News.
