Frontotemporal dementia, the most commonly diagnosed form of early-onset dementia, usually starts between the ages of 40 and 65.
This condition affects the brain’s frontal and temporal lobes, leading to changes in behavior, challenges with speaking and writing, and a decline in memory.
A particular group of patients with this disease has a genetic mutation that stops their brain cells from producing a vital protein called progranulin.
Though the full role of progranulin is not entirely understood, its absence is closely linked to the development of the disease.
Excitingly, researchers from the University of Kentucky and other institutions have discovered that a certain type of antibiotic, known as aminoglycosides, could offer a new treatment avenue for frontotemporal dementia.
In their study, they found that when aminoglycoside antibiotics were introduced to neuronal cells affected by the genetic mutation that causes a reduction in progranulin, the cells began to bypass the mutation and started producing the full-length progranulin protein again.
Specifically, two aminoglycoside antibiotics, Gentamicin and G418, were identified as capable of correcting the mutation and restoring functional progranulin protein levels.
Treatment with either of these antibiotics led to a recovery of progranulin protein levels to about 50 to 60% of normal levels in the affected cells.
This groundbreaking research could pave the way for developing new treatments for frontotemporal dementia, a condition for which there are currently no effective therapies.
The findings from this “preclinical proof of concept” study are a crucial first step, with plans to further explore the effects of these antibiotics in mouse models of the disease.
The researchers are also looking into developing new compounds derived from Gentamicin and G418 that could potentially be safer and more effective for human use.
While Gentamicin is already approved by the FDA, its use has been limited due to various adverse side effects. The goal is to create a treatment option that maximizes benefits while minimizing risks.
This study, led by Haining Zhu and published in Human Molecular Genetics, opens up a new potential pathway for treating not only frontotemporal dementia but possibly other forms of dementia as well.
As the search for effective dementia treatments continues, this research shines a light of hope for patients and families affected by these challenging conditions.
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