Finding treatments for ALS
Genome editing manager Chang Tong and his colleagues generated several lines with a fluorescent marker inserted into the genome of an induced pluripotent stem cell (iPSC) line derived from a patient with amyotrophic lateral sclerosis (ALS). The fluorescent marker indicates when the iPSCs differentiate into motor neurons, which undergo the disease’s signature degeneration. This provides an efficient tool for USC Stem Cell researcher Justin Ichida to screen for drug-like compounds to cure ALS, also known as Lou Gehrig’s disease.
In ALS, patients suffer from the death of the cells that transmit signals from the brain to the muscles, called motor neurons, leading to paralysis and usually resulting in fatal respiratory failure within three to five years of diagnosis.
This work is part of a $1.5 million project funded by the U.S. Department of Defense in collaboration with two biotech companies, Icagen and DRVision, to discover and screen 43,000 compounds to slow or stop ALS. Although they are still in the early stages of the project, they have already found compounds that keep motor neurons alive in Petri dishes—and could eventually do the same in patients.