Portuguese scientists create human stem cells to treat Machado-Joseph disease. What to know
COIMBRA - A team of scientists led by the University of Coimbra has managed to create human stem cells from skin cells that have the potential for long-term treatment of Machado-Joseph disease, according to a study released on Tuesday.
University of Coimbra officials said this research paves the way for the development of cells that could be used in the treatment of the rare neurodegenerative disease, which affects muscles in the body, including those required for speech, and was originally described in families of Portuguese-Azorean ancestry.
“It has a high prevalence in the Azores, especially on the island of Flores, which has the highest incidence of the disease worldwide,” said Liliana Mendonça, the leader of the study.
She said this discovery demonstrates the feasibility of applying personalized therapies to people with the disease, by creating stem cells from the patients to be treated.
This will result in greater acceptance of the transplant, stressed the researcher from the University of Coimbra’s Centre for Neuroscience and Cell Biology (CNC-UC) and the Centre for Innovation in Biomedicine and Biotechnology (CIBB).
Considered very versatile, stem cells can give rise to specialized cells from various tissues and organs in the human body.
Machado-Joseph disease still has no treatment. The cerebellum is one of the regions of the brain most affected, leading to extensive neuronal death, difficulties with motor coordination, swallowing and speech articulation.
The research team created cells that proved capable of giving rise to neurons in cell cultures (a set of techniques for testing the behavior of cells in an artificial environment) and also in cerebral organoids (tissues generated ‘in vitro,’ i.e. outside of living organisms).
According to Mendonça, the researchers observed at the same time that the human stem cells survived up to six months after being transplanted into the cerebellum of the animal model, having differentiated into glia cells (cells in the central nervous system that perform various functions) and neurons, which means that they have the potential to act positively in the control of neurodegenerative diseases.
“There is a high need to develop therapeutic strategies that can treat neurodegenerative diseases, which robustly improve the quality of life of patients, thus contributing to reducing the health burden on health systems and the families of these patients,” she stressed.
Carried out by a team from CNC-UC’s Gene and Stem Therapies for the Brain Research Group, the research is being further developed.
One of the aims is to study how these cells can improve the motor coordination problems caused by the disease, using an animal model.
The research leader said the scientists are also going to develop strategies to improve the migration of the cells and then their differentiation into cerebellar neurons after they are transplanted into the brain, something that could significantly increase the therapeutic effects of these cells.
This article originally appeared on The Herald News: Human stem cells created in Portugal to treat Machado-Joseph disease
