A team of researchers from the Harvard Stem Cell Institute (HSCI) and Columbia University, in a collaboration catalyzed by the Project A.L.S./Jenifer Estess Laboratory for Stem Cell Research, has demonstrated that pluripotent stem cells generated from a patient with ALS (amyotrophic lateral sclerosis) can be directed to differentiate into motor neurons—the very brain cells destroyed by ALS. The results of the team’s study appear in today’s online issue of Science. This is the first published report to show that disease-specific stem cells may be derived from an individual patient.
In the study, led by Kevin Eggan, of the Harvard Stem Cell Institute, skin cells taken from a patient with a familial form of ALS were induced to become pluripotent stem cells. Scientists then differentiated the pluripotent cells into motor neurons and glia (support cells in the brain) that featured an ALS genotype.
“This is a seminal discovery,” said Valerie Estess, director of research for Project A.L.S. “The ability to derive ALS motor neurons through a simple skin biopsy opens the doors to improved drug discovery. For the first time, researchers will be able to look at ALS cells under a microscope and see why they die. If we can figure out how a person’s motor neurons die, we will figure out how to save motor neurons.”
