A new study from Northwestern University has found that an experimental drug called NU-9 can help brain cells recover in animal models of Alzheimer’s disease. Originally developed for treating amyotrophic lateral sclerosis (ALS), NU-9 appears to target a common root cause of several brain disorders—misfolded proteins that build up inside brain cells and cause damage.
Neurodegenerative diseases like Alzheimer’s and ALS involve different proteins, but they share a key problem: the proteins that are normally helpful start folding the wrong way, sticking together, and becoming toxic. These clumps of bad proteins disrupt brain function and eventually lead to brain cell death.
NU-9 is different from most drugs because it doesn’t just treat the symptoms of a single disease. Instead, it works on the basic process inside cells that causes damage in several brain disorders. The drug seems to help cells clear out these toxic protein clumps, especially in early stages, before the damage becomes permanent.
The new study, published in Proceedings of the National Academy of Sciences, showed that NU-9 worked well in both cell cultures and mice. In lab tests, when scientists added a toxic form of amyloid beta—the main harmful protein in Alzheimer’s—to brain cells, the cells quickly became unhealthy.
But when they gave the cells NU-9 first, much less protein buildup occurred, and the cells were healthier. Even after the drug was removed, its protective effects lasted.
In the next phase, the researchers gave NU-9 by mouth to mice bred to show signs of Alzheimer’s. These mice performed better on memory tests after treatment.
The drug also helped reduce inflammation in the brain, which is another major problem in Alzheimer’s disease. Inflammation can make brain damage worse, so stopping it is an important goal in treatment.
NU-9 works inside brain cells by helping them clean up harmful proteins. The researchers found that the drug activates lysosomes—tiny compartments in cells that act like garbage disposals. These lysosomes break down waste and old or damaged parts of the cell.
In Alzheimer’s disease, the lysosomes don’t work properly, leading to protein buildup. NU-9 seems to help restart this process, with the help of an enzyme called cathepsin B.
The team found that NU-9 specifically targets amyloid beta proteins inside cells but not outside. That’s important, because it suggests the drug is acting directly on a key step in how these toxic proteins are handled inside the brain.
The researchers believe NU-9 may help shuttle the harmful proteins through different “junk compartments” in the cell until they reach the lysosome, where they’re broken down. They’re still trying to figure out exactly how NU-9 starts this process.
The drug was invented by Professor Richard Silverman, who also developed the drug Lyrica, and is now being tested through his company Akava Therapeutics. The study was co-led by Professor William Klein, an expert in Alzheimer’s, and supported by scientists at Northwestern University.
While these early results are exciting, the researchers stress that much more work is needed before NU-9 can be used to treat people. More testing in animal models is planned, along with efforts to improve the drug’s strength and effectiveness.
The team is also interested in seeing if NU-9 might help with other brain diseases like Parkinson’s and Huntington’s, which also involve toxic protein buildup.
If NU-9 continues to show positive results, it could mark a big step forward in treating multiple brain diseases with a single drug. For now, it gives scientists hope that one drug may help fix a shared problem that lies at the heart of many devastating conditions that affect the brain.
If you care about Alzheimer’s, please read studies about Vitamin D deficiency linked to Alzheimer’s, vascular dementia, and Oral cannabis extract may help reduce Alzheimer’s symptoms.
For more information about brain health, please see recent studies about Vitamin B9 deficiency linked to higher dementia risk, and results showing flavonoid-rich foods could improve survival in Parkinson’s disease.
The research findings can be found in PNAS.
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