The creation of stem cells from the skin of people with Machado-Joseph disease raises hopes of improving the lives of these patients. The disease is rare, inherited from the parents and “highly disabling”, explains the researcher at the Center for Neuroscience and Cell Biology at the University of Coimbra (UC), Liliana Mendonça.
Currently, the disease still has no treatment. The creation of stem cells implanted in the cerebellum of animal models allows speculation about the possibility of this type of therapy helping human patients with the disease. Liliana points out concerns and possible side effects.
The extraction of cells from patients’ skin could result in “greater acceptance of the transplant”, since the new cells will be implanted in the patients’ cerebellum. However, there is still no forecast for the start of clinical trials in patients with Machado-Joseph. The investment in human and financial resources will allow us to move forward, explains Liliana Mendonça.
In the study, it was observed “that human stem cells survived up to six months after transplantation into the cerebellum of the animal model, having differentiated into glial cells [células do sistema nervoso central que desempenham diversas funções, apoiando, nomeadamente, os neurónios] and neurons”, says UC in a statement.
The researcher adds that this discovery could boost other neurodegenerative diseases.
The next steps include “developing strategies to improve cell migration and then their differentiation into cerebellar neurons, after transplantation to the brain”.
Other UC researchers participated in the study: Luís Pereira de Almeida, Daniel Henriques, Vanessa Fernandes, Ricardo Moreira, João Brás and Sónia Duarte.
The investigation also included the collaboration of the researcher at the Luxembourg Center for Systems Biomedicine, Jens C. Schwamborn.
Illustration: UC@Rita Félix
Tags: research creates therapy improve lives patients MachadoJoseph disease
