ALS, also known as Lou Gehrig’s disease, is a brain disease that targets brain cells called motor neurons.Motor neurons send messages from the brain to muscles throughout the body. In ALS, as motor neurons die, a person progressively loses the ability to walk, speak, swallow, and breathe. ALS affects adults of all ages, from teens to seniors, and is usually fatal within 2-5 years of diagnosis. The Core and others are working together to develop the first effective treatments.
ALS is complicated—but decades of dedicated research, and advances in technology, have allowed us to understand more about the genetic and biological drivers of this devastating brain disease. That’s why the time is right for The Core: we can now target specific pathways that we know to be important in ALS with specific, rational therapeutics:
- More than 60 genes have been implicated in the ALS disease progress—and we can target these genes with specific therapeutic approaches
- While 90% of ALS cases are “sporadic”—with no known genetic mutation—we know more about the disease’s biology, and can develop drugs to hit common pathways of ALS
- We’ve built better tools for drug screening, including patient-derived “ALS in a Dish”—so drugs tested at The Core have a better chance of working