Frontotemporal dementia is the most common type of early-onset dementia.
It typically begins between ages 40 and 65 and affects the frontal and temporal lobes of the brain, which leads to behavior changes, difficulty speaking and writing, and memory deterioration.
A subgroup of patients with frontotemporal dementia has a specific genetic mutation that prevents brain cells from making a protein called progranulin.
Although progranulin is not widely understood, its absence is linked to the disease.
In a recent study from the University of Kentucky and elsewhere, researchers have found that a class of antibiotics called aminoglycosides could be a promising treatment for frontotemporal dementia.
The study is published in Human Molecular Genetics. One author is Haining Zhu, a professor in UK’s Department of Molecular and Cellular Biochemistry.
In the study, the team found that after aminoglycoside antibiotics were added to neuronal cells with this mutation, the cells started making the full-length progranulin protein by skipping the mutation.
They found two specific aminoglycoside antibiotics – Gentamicin and G418 – were both effective in fixing the mutation and making the functional progranulin protein.
After adding Gentamicin or G418 molecules to the affected cells, the progranulin protein level was recovered up to about 50 to 60%.
These results could be promising for drug development. Currently, there are no effective therapies for any type of dementia.
After this preclinical proof of concept study, the next step is to study the antibiotics’ effects on mice with the mutation that causes frontotemporal dementia.
Another plan is to develop new compounds from Gentamicin and G418 that could be safer and more effective.
Although Gentamicin is an FDA-approved drug, its clinical usage is limited as it is associated with a number of adverse side effects.
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