A new experimental drug designed by the team from the US Albert Einstein School of Medicine is capable of reversing the key symptoms of Alzheimer’s disease in mice, according to scientists.
The drug, designed by researcher Ana Maria Cuervo and her team, works by revitalizing a cellular cleaning mechanism that cleans unwanted proteins by digesting and recycling them.
The study is published today in the journal ‘Cell’.
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“Findings in mice do not always translate into humans, especially Alzheimer’s disease,” the corresponding author, Cuervo warns, but “we were encouraged to find in our study that the decrease in cellular cleanliness that contributes to Alzheimer’s in mice also occurs in mice. people with the disease, suggesting that our drug could also work in humans.”
Although cautiously, she notes that she is more optimistic because they also have data with patients that show us that this same mechanism exists in humans.
In this article, and in another that we recently published in ‘Nature Communications we have seen that the molecular failure that occurs in mice is the same. These give us “more peace of mind in the sense that the compound should also work in humans as it has the same defect.
In the 1990s, the Spanish researcher discovered the existence of this cellular cleaning process, known as chaperone-mediated autophagy (CMA), and has published 200 articles on its role in health and disease.
This April, Dr. Cuervo’s team reported in Nature Communications that, for the first time, they had isolated lysosomes from the brains of Alzheimer’s disease patients and observed that the reduction in the number of LAMP2 receptors causes the loss of CMA in humans, just as it does in animal models of Alzheimer’s.
The researchers were unable to start treating the animals before they developed symptoms, making the model more similar to what is produced in humans since most patients start treatment when the disease is already in its symptomatic phase.
Patients arrive already with cognitive impairment, memory loss … We thought it was too late
the researcher acknowledges.
Researchers used two mouse models: pure Alzheimer’s and fronto-temporal dementia. “Many Alzheimer’s patients aren’t properly Alzheimer’s patients,” she recalls.
The designed compound is for oral consumption and the animals received the pill every day.
“One of the advantages of our compound is that it is for oral use and that it is capable of crossing the blood-brain barrier,” something that is important for future use in humans.
The results were so good that “we could not believe them”, acknowledges the expert.
“When you do an experiment you expect your results to be good, but when they are, you sometimes doubt.” For this reason, the brains of the second mouse model, those with frontotemporal dementia, “were sent to an external laboratory. And they confirmed our results.”
The study was designed for three months, but “we extended them to 6 months to see if there were toxic effects,” says Cuervo.
“Six months of the life of a mouse is like 20 years in humans, and at that time there were no toxic effects,” she highlights.
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This fact, she stresses, is very important because it is a pill, which, in theory, is going to be of chronic use, that is, “every day” and that is why we must be sure that it has no toxic effects. Research continues and they hope that the drug can be given on a weekly basis because, “once the proteins are cleaned, it takes a while for the accumulations to occur.”
The ability to walk improved significantly in the animal model in which it was a problem. And in brain neurons of both animal models, the drug significantly reduced levels of tau protein and protein groups compared to untreated animals.
“It is important to note that animals in both models already showed symptoms of disease and their neurons were clogged with toxic proteins before the drugs were given,” explains Ana María Cuervo.
Another important aspect is that aging processes, such as Alzheimer’s, are accompanied by an inflammation process called gliosis. In this case, and unexpectedly, “there was a decrease in inflammation”.
“Gliosis is associated with toxic proteins and is known to play an important role in the perpetuation and worsening of neurodegenerative diseases. And we think inflammation is what gives the process chronicity.”
“This means that the drug can help preserve neuronal function even in the later stages of the disease. We were also very excited that the drug significantly reduced the gliosis, inflammation, and healing of the cells surrounding the neurons in the brain,” she explains.
The next step is to start clinical trials in Alzheimer’s, something very complex. Fortunately, the researcher advances, there is a disease, macular degeneration, which also causes an accumulation of garbage in the retina, and it can be used as a model. In fact, the team has a collaborative trial with Patrica Boya’s group to test this compound.