Researchers at Northwestern University have announced new findings suggesting that an experimental drug used for ALS may effectively treat Alzheimer's disease in animal models. NU-9, a compound approved by the FDA for ALS clinical trials, has been shown to improve neuron health by addressing underlying disease mechanisms, potentially affecting multiple neurodegenerative diseases.
"This drug is quite remarkable that it works in these multiple systems," stated Richard B. Silverman from Northwestern University, who invented NU-9. He emphasized the need for human testing but expressed optimism in the way the drug functions.
The research, detailed in the Proceedings of the National Academy of Sciences, demonstrated NU-9’s ability to reduce protein buildup, a common factor in ALS and Alzheimer's. "What our study demonstrates is that the same mechanism affects two totally different proteins in two totally different diseases," explained William Klein, co-author of the study.
Silverman, also known for inventing Lyrica, leads the commercialization efforts of NU-9 through his company, Akava Therapeutics. Klein, a neurobiology professor and cofounder of Acumen Pharmaceuticals, highlighted the study's potential for treating Alzheimer’s by preventing toxic protein clumps.
Experiments involved neuron cultures and Alzheimer’s disease mouse models. Results showed that NU-9 reduced amyloid beta buildup and improved memory test performance, as well as decreased brain inflammation. "Inflammation of the brain was prevented or greatly reduced by the treatment of NU-9," Klein added.
NU-9 operates within cell processes involving lysosomes and the enzyme cathepsin B. "Cells have two important ‘junk compartments’: the lysosome and the proteasome," Silverman noted, as research continues to pinpoint the exact process NU-9 targets.
Despite promising results, the researchers acknowledge the need for further studies, including more rigorous memory testing and compound refinements. Klein and Silverman also plan to test NU-9’s effects on other neurodegenerative diseases like Parkinson's and Huntington's.
Silverman remarked, "It has long been thought that every neurodegenerative disease is a completely separate disease, but our findings suggest that common mechanisms might connect them."
This study was supported by the National Institutes of Health and the Chemistry of Life Processes Institute. Editor’s notes disclosed financial interests of Silverman and Klein in their respective companies, and Northwestern University’s involvement.