Scientists find a new treatment for early-onset dementia

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Frontotemporal dementia (FTD) is the most common form of dementia that occurs in people younger than 65. It typically begins between the ages of 40 and 65 and affects the brain’s frontal and temporal lobes.

These areas are responsible for controlling behavior, speech, and memory. As a result, people with FTD may experience significant changes in their personality, have difficulty speaking or writing, and eventually lose memory.

One subgroup of FTD patients has a genetic mutation that stops their brain cells from making a protein called progranulin. While scientists don’t fully understand all the functions of progranulin, its absence is strongly linked to the disease. Without this protein, the brain cells cannot function properly, which contributes to the progression of FTD.

A team of researchers from the University of Kentucky, along with others, recently discovered that a certain class of antibiotics called aminoglycosides might offer hope as a potential treatment for FTD.

Their study focused on how these antibiotics could help brain cells overcome the genetic mutation that prevents progranulin production.

In the laboratory, the researchers added aminoglycoside antibiotics to human neuronal cells with this genetic mutation. Remarkably, the cells began producing full-length progranulin protein. This happened because the antibiotics helped the cells bypass the genetic error that blocked the protein’s production.

Two specific antibiotics, Gentamicin and G418, were particularly effective in restoring progranulin levels. The cells produced about 50% to 60% of the normal protein levels after treatment.

This discovery is significant because there are currently no effective treatments for any type of dementia, including FTD. Restoring progranulin production might slow or stop the progression of the disease in patients with this specific mutation.

While these results are promising, the research is still in its early stages. The current findings are based on tests conducted in a laboratory setting using human cells. The next step is to test the antibiotics on mice that carry the same genetic mutation.

This will help researchers understand how the treatment works in a living organism and whether it could be safe and effective for humans.

However, there are challenges to overcome. Gentamicin is already approved by the FDA for treating infections, but its use is limited due to potential side effects, such as kidney damage and hearing loss.

To address this, the researchers also plan to develop new compounds based on Gentamicin and G418 that could be safer and more suitable for long-term use in FTD patients.

The study, led by Haining Zhu and colleagues, was published in Human Molecular Genetics. While it will take more time and research to determine whether this approach can become a practical treatment, the findings offer a new direction in the search for therapies for frontotemporal dementia and related conditions.

For patients and their families, this research provides a glimmer of hope in the fight against this devastating disease.

If you care about dementia, please read studies about low choline intake linked to higher dementia risk, and how eating nuts can affect your cognitive ability.

For more information about brain health, please see recent studies that blueberry supplements may prevent cognitive decline, and results showing higher magnesium intake could help benefit brain health.

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