The twisted and tangled proteins Many of them People to die with Alzheimer’s disease disease.
Some scientists believe that these neural knots Damages and kills brain cellsbut so far Clinical TrialsHowever, drugs targeting tangles have shown limited efficacy.
An international team led by researchers at the University of Texas has unveiled a promising new drug: a nasal spray that can bypass the blood-brain barrier and destroy tau protein tangles in live mice. The drug also works in the lab to remove tangles within and between human neurons.
The drug was specifically designed to recognize and destroy the most toxic forms of tau protein, such as those found in some cases of Alzheimer’s disease and dementia.
But that wasn’t the only challenge: researchers also had to figure out how to get the drugs across the blood-brain barrier and into brain cells.
Neuroscientist Sagar Gaikwad and his team decided to package the drug in tiny bubbles that could slip through cell membranes, and by squirting them up the noses of mice, they were able to bypass the blood-brain barrier.
In the case of old mice with tau-related brain disease, one spray was all that was needed to clear toxic tau from the brain. After two weeks, the mice’s cognitive function had improved.
Of course, this doesn’t mean the drug has the same effect on humans.
A review of the survey results showed that Science Translational MedicineNeuroscientists Soraya Mehta, Claire Durant and Tara Spires-Jones of the University of Edinburgh, who were not involved in the study, said: Note Although many of the tau-based therapies have shown promise in animal models, they have “so far failed to translate into effective drugs for humans.”
Although much work remains to be done, the early results from Gaikwad and his colleagues are promising.
The new study was conducted using postmortem human brain tissue donated by patients with Alzheimer’s disease, Lewy body dementia and Pick’s disease (a type of frontotemporal dementia). antibody The drug not only dismantled the tau protein tangles, but also prevented the release of “tau protein seeds” that travel through connected neurons to tangle proteins in other parts of the brain.
“Many open questions remain, including whether administering this treatment intranasally would allow the antibodies to penetrate in effective amounts throughout the much larger human brain, and whether there would be any potentially dangerous side effects, such as inflammation, that are a concern in all trials of amyloid-targeting immunotherapy.” write Mehta, Durant, and Spiers-Jones.
“Despite these limitations, this is an important piece of work.”
Gaikwad and his colleagues hope that their technique will spur further research into treating tau-related diseases.
This study Science Translational Medicine.