The research team then created an organoid using stem cells from a patient with a rare gene mutation that affects myelination. 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.
Lead researcher, Dr Owen Gwydion James, said:
“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.”
The authors added that 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.
The study was funded by the Euan MacDonald Centre and carried out in collaboration with the UK DRI and the MS Society Centre for MS Research at the University of Edinburgh.
James, O.G., Selvaraj, B.T., Magnani, D., Burr, K., Connick, P., Barton, S.K., Vasistha, N.A., Hampton, D.W., Story, D., Smigiel, R. and Ploski, R., 2021. iPSC-derived myelinoids to study myelin biology of humans. Developmental Cell, 56(9), pp.1346-1358.
Article published: 20 May 2021
Banner image: Human stem cell-derived myelinating oligodendrocyte can be seen with many myelinating processes wrapped around unstained neurons.
In-line image: Myelin moon
Both images courtesy of Dr Owen Gwydion James, Prof Siddharthan Chandran’s Lab, UK DRI at Edinburgh.