Singapore: A study conducted by Dr Steven Goldman and his team at the University of Rochester Medical Center (URMC), US, has attempted to employ human induced pluripotent stem cells (hiPSC) from human skin to produce a population of cells that are critical to neural signaling in the brain, by transplanting them into animal models of myelin disease.

The first-of-its-kind research was published in the February 7 issue of the journal Cell Stem Cell and shows that human brain cells created by reprogramming skin cells are highly effective in treating myelin disorders, which is a family of diseases that includes multiple sclerosis and rare childhood disorders called pediatric leukodystrophies.

The source of the myelin cells in the brain and spinal cord is a cell type called the oligodendrocyte. Oligodendrocytes are, in turn, the offspring of another cell called the oligodendrocyte progenitor cell or OPC. Myelin disorders have long been considered a potential target for cell-based therapies. Scientists have theorized that if healthy OPCs could be successfully transplanted into the diseased or injured brain, then these cells might be able to produce new oligodendrocytes capable of restoring lost myelin, thereby reversing the damage caused by these diseases.

Dr Goldman's team was the first to successfully master the complex process of using hiPSCs to create OPCs. This process proved time consuming. It took Dr Goldman's lab four years to establish the exact chemical signaling required to reprogram, produce, and ultimately purify OPCs in sufficient quantities for transplantation and each preparation required almost six months to go from skin cell to a transplantable population of myelin-producing cells.

Once they succeeded in identifying and purifying OPCs from hiPSCs, they then assessed the ability of the cells to make new myelin when transplanted into mice with a hereditary leukodystrophy that rendered them genetically incapable of producing myelin.