After much effort, a team of scientists, led by the University of Coimbra, "managed to generate human stem cells from skin cells, which showed long-term therapeutic potential for Machado-Joseph disease, a rare pathology with a high incidence in Portugal", according to a press release.

This is a research project, available in Scientific Report, capable of opening "the way for the development of cells that could be used in the treatment of this neurodegenerative disease" that "affects movement and speech articulation".

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More specifically, the team "shows that it is possible to create stem cells from cells extracted from people with Machado-Joseph disease with therapeutic potential", explains Liliana Mendonça, a researcher at the Center for Neurosciences and Cell Biology of the University of Coimbra (CNC-UC) and the Center for Innovation in Biomedicine and Biotechnology (CIBB).

In case you don't know, Machado-Joseph disease is a pathology that currently has no treatment, with the cerebellum being one of the most affected regions of the brain. It is generally characterized by "extensive neuronal death, difficulties in motor coordination, swallowing and speech articulation". It is a condition with "a high prevalence in the Azores, especially on Flores Island, which has the highest incidence of the disease worldwide", warns the researcher.

For the research, the team "created cells that have shown the ability to generate neurons in cell cultures (a set of techniques to test the behavior of cells in an artificial environment) and also in brain organoids (tissues generated in vitro, that is, outside living organisms, that simulate human neurodevelopment, functioning as "mini-brains")".

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Simultaneously, "in this study we also observed that human stem cells survived up to six months after transplantation into the cerebellum of the animal model, having differentiated into glial cells [cells of the central nervous system that perform various functions, supporting, namely, neurons] and neurons", explains researcher. advances Liliana Mendonça. This means that cells can help control neurodegenerative diseases.

So, now, the researchers plan to continue investigating how these cells can improve the motor coordination problems of the disease, using an animal model.

In addition, they are "developing strategies to improve cell migration and, subsequently, their differentiation into cerebellar neurons, after transplantation into the brain, something that can significantly increase the therapeutic effects of these cells".

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