In a new study, researchers find that a novel drug reverses memory deficits and stops Alzheimer disease pathology (AD) in an animal model.
The research is conducted at the Lady Davis Institute (LDI) at the Jewish General Hospital.
Importantly, this drug has already proven to be non-toxic for humans in a clinical setting. Therefore, it could be brought quickly to trials in humans against AD.
For years, the research team has strived to find early neurodegenerative condition responsible for age-related memory loss.
They discovered that the Caspase-6 enzyme is highly activated in Alzheimer disease brain lesions and associated with loss of memory.
The researchers, therefore, pursued the hypothesis that stopping Caspase-6 might provide relief from memory loss and stop progressive dementia.
Since there are no specific Caspase-6 inhibitors, the team moved upstream, ultimately discovering that Caspase-1 was responsible for activating Caspase-6.
The work showed that VX-765 has an unprecedented beneficial effect in Alzheimer mice.
The drug rapidly reverses memory loss, eliminates inflammation, and stops Alzheimer’s prototypical amyloid peptide accumulation in the mice brains.
In addition to being safe for humans at relatively high doses for extended periods of time, it is capable of reaching the brain, a significant challenge in the development of drugs against disorders of the brain.
While the team cautions that there is a considerable bridge to cross between the mouse brain and that of a human, they believe that there is a chance that this drug will work just as well in humans as it did in mice.
Nevertheless, a clinical trial is needed to determine whether the drug will be beneficial against Alzheimer disease in humans.
Currently, there are no effective treatments to significantly treat Alzheimer disease, the major affliction in a group of dementias that affect 47.5 million individuals worldwide.
These findings are published in Nature Communications.
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Source: Nature Communications.