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Until recently, most scientific knowledge about amyotrophic lateral sclerosis (ALS), better known as Lou Gehrig’s disease, came from mouse studies. But new research is taking this incurable neurodegenerative condition to the dish, tapping induced pluripotent stem cells (iPS cells)—made from ALS patients’ skin cells—to create motor neurons. These motor neurons are being used not just to model how ALS works at the cellular level but also to screen potential drugs.
This work, taking place at the Harvard Stem Cell Institute (HSCI) in collaboration with Boston Children’s Hospital and Massachusetts General Hospital (MGH), has now paved the way for a clinical trial of a drug that might never otherwise have been thought of. …