Scientists at the University of Edinburgh's Anne Rowling Regenerative Neurology Clinic and the Euan MacDonald Centre for Motor Neuron Disease Research, have developed a human myelin model using skin samples donated by volunteers, enabling them to see myelin developing spontaneously around the axons between cells within the organoids. Their findings were published in Developmental Cell.
The research team then created an organoid using stem cells from a patient with a rare gene mutation that affects myelination—Nfasc155 deficiency. The model showed that key aspects of this cell bundle were consistent with the disease. These new models will allow scientists to compare the differences between the cells of healthy individuals and those with different neurological diseases and to test drugs of interest in human cells before using them in a full clinical trial with patients.
The approach complements animal models, which can be limited in how they reflect human disease and the way that drugs interact with human cells. The authors say the model is a significant step forward in the study of human myelination and drug development, but caution that treatments tested on this model are still some way from being offered to patients.
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Lead researcher Gwydion James, concluded, "Demyelinating disorders have a profound effect on the quality of life for patients. Now we have the capability of studying human myelination experimentally, a major goal is to identify drugs that can promote myelination. We believe that this new approach could be a huge boost to the toolbox that allows us to do this effectively."