Frontotemporal dementia, the most common form of early-onset dementia, typically begins between the ages of 40 and 65. This condition affects the frontal and temporal lobes of the brain, leading to changes in behavior, difficulties with speaking and writing, and memory loss.
For a subgroup of patients, the disease is linked to a genetic mutation that prevents brain cells from producing a protein called progranulin. While progranulin’s role in the brain is not fully understood, its absence is strongly associated with the disease.
Researchers at the University of Kentucky, along with collaborators, have discovered a potential treatment for this condition using a class of antibiotics called aminoglycosides.
Their study revealed that these antibiotics can bypass the genetic mutation, enabling brain cells to produce functional progranulin protein again.
This breakthrough offers hope for patients with frontotemporal dementia, as there are currently no effective treatments for this or any other type of dementia.
The researchers tested two specific aminoglycosides, Gentamicin and G418, on neuronal cells with the progranulin mutation. Both antibiotics successfully skipped over the mutation, allowing the cells to produce full-length, functional progranulin protein.
After treatment with Gentamicin or G418, progranulin protein levels in the affected cells recovered to about 50–60% of normal levels.
While these results are promising for drug development, much work remains before this approach could become a viable treatment. The next steps include testing the antibiotics on mice with the progranulin mutation to evaluate their effects in living organisms.
Additionally, researchers aim to modify Gentamicin and G418 to create new compounds that are safer and more effective for long-term use. Although Gentamicin is already approved by the FDA, its clinical use is limited due to significant side effects.
This study, led by Haining Zhu and published in Human Molecular Genetics, represents an important step toward finding a treatment for frontotemporal dementia.
If further research confirms these findings, it could pave the way for new therapies that improve the lives of patients with this challenging disease.
If you care about brain health, please read studies about Vitamin B9 deficiency linked to higher dementia risk, and cranberries could help boost memory.
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