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Breakthrough: Drug for rare nerve disease ALS shows promise for Alzheimer’s

by Ana Zirojević
April 9, 2025
in Research, Science
Breakthrough: Drug for rare nerve disease ALS shows promise for Alzheimer’s

Breakthrough: Drug for rare nerve disease ALS shows promise for Alzheimer’s

As medical professionals and researchers continue to struggle with finding an effective treatment for one of humanity’s most devious conditions – Alzheimer’s disease – a medicine for another, equally malicious illness might be a major step forward.

Indeed, experimental drug NU-9 – a molecule compound approved by the United States Food and Drug Administration (FDA) for clinical trials for the treatment of amyotrophic lateral sclerosis (ALS) – has evidently improved neuron health in animal models of Alzheimer’s disease, per a report from April 8.

Specifically, a new Northwestern University study has discovered that the NU-9 acts on a common underlying mechanism at work in both neurodegenerative diseases, giving scientists hope that they could use it to address the cause instead of just treating the symptoms of these diseases.

The mechanism in Alzheimer’s addressed by the ALS medication

As it happens, both ALS and Alzheimer’s are the results of misfolded proteins that clump together inside brain cells and damage brain health, and the NU-9 acts by inhibiting the buildup of these proteins, called amyloid beta oligomers – small peptide aggregates that instigate neurodegeneration.

In a previous study, Silverman discovered that the NU-9 helped cells eliminate the protein clumps to restore neuron function in animal models with ALS, so he wanted to explore whether the NU-9 could have a similar effect on Alzheimer’s disease – and succeeded.

During the most recent study, they administered an oral dose of NU-9 to a mouse model of Alzheimer’s, discovering that the animals’ performance on memory tests actually improved, as well as finding that the NU-9 reduces brain inflammation associated with the disease.

Commenting on the breakthrough, Northwestern’s Richard B. Silverman, the Patrick G. Ryan/Aon Professor of Chemistry at Northwestern’s Weinberg College of Arts and Sciences who invented the NU-9, explained:

“This drug is quite remarkable that it works in these multiple systems. (…) We need to test it in humans before we know how effective it is in treating Alzheimer’s disease. But how well upper motor neurons function in mice is similar to how well they function in humans. So, it seems to me, NU-9 really should work.”

In the words of Northwestern’s William Klein, the study’s co-corresponding author and a professor of neurobiology at Weinberg, the study shows that the same mechanism affects two completely different proteins in two entirely separate diseases, in which cells sustain a toxic protein aggregation, and NU-9 is “rescuing the pathway that saves the cell.”

Meanwhile, as patients are waiting for the ALS medication to treat their Alzheimer’s, there’s a collection of trivia games that helps those with dementia keep their minds active by turning their memories into simple and engaging quizzes, creating opportunities for them to reminisce on topics like history, music, places, food, pets, and so on.

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