Possible therapy against frontotemporal dementia

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Dimethyl fumarate (DMF) can significantly reduce neurodegeneration and brain inf
Dimethyl fumarate (DMF) can significantly reduce neurodegeneration and brain inflammation in a mouse model of TDP-43-associated frontotemporal dementia.

A study led by the Autonomous University of Madrid (UAM) reveals that repositioning the drug dimethyl fumarate reduces neuronal degeneration in a mouse model of TDP-43-dependent frontotemporal dementia. The finding opens the possibility of reusing an already approved drug to treat a neurodegenerative disease without a cure, potentially accelerating the development of new therapies for dementia patients.

A team of Spanish researchers has discovered that dimethyl fumarate (DMF), a drug already approved for multiple sclerosis, can significantly reduce neurodegeneration and brain inflammation in a mouse model of TDP-43-associated frontotemporal dementia. These findings could open the door to new treatments for one of the leading causes of dementia in people under the age of 65, which currently lacks effective therapies.

The team, led by Dr. Isabel Lastres Becker at the Autonomous University of Madrid (UAM), in collaboration with researchers from the Complutense University of Madrid (UCM) led by Dr. Javier Fernández-Ruiz and Dr. Eva de Lago, evaluated the neurodegenerative process, inflammatory markers, oxidative stress and TDP-43 accumulation. The results, published in the journal Antioxidants, reveal a protective effect of DMF, mitigating brain damage.

This breakthrough highlights the importance of exploring existing therapies to accelerate the development of effective treatments for neurodegenerative diseases.

Drug repositioning as a therapeutic alternative

Frontotemporal dementia (FTD) is a devastating neurodegenerative disease, and its association with the accumulation of TDP-43 in the brain makes it especially challenging for researchers. However, drug repositioning, as in this case with DMF, offers a fast and less expensive route to new treatments, as these are drugs whose safety profile is already known.

"The positive results obtained in the mouse model suggest that dimethyl fumarate could have a therapeutic effect on this form of dementia, improving the quality of life of those affected and alleviating the burden on their families and healthcare systems," the study authors point out. This would not only have a positive impact on patients, but would also reduce the burden on families and healthcare systems, given the debilitating impact and long-term costs associated with neurodegenerative diseases".

In sum, this work - carried out by researchers belonging to the Instituto de Investigaciones Biomédicas "Sols-Morreale", CIBERNED, Instituto de Investigación Sanitaria La Paz (IdiPaz), Instituto Universitario de Investigación en Neuroquímica, and Instituto Ramón y Cajal de Investigación Sanitaria (IRYCIS) - highlights the potential of DMF to treat FTD. "However, these results still need to be taken to human clinical trials to confirm its efficacy and advance towards a viable treatment for this devastating neurodegenerative disease," the authors conclude.

Bibliographic reference:

Silva-Llanes I, Martín-Baquero R, Berrojo-Armisen A, Rodríguez-Cueto C, Fernández-Ruiz J, De Lago E, Lastres-Becker I. Beneficial Effect of Dimethyl Fumarate Drug Repositioning in a Mouse Model of TDP-43-Dependent Frontotemporal Dementia. Antioxidants (Basel) . 2024 Sep 2;13(9):1072.

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