New Drug Targets

The Core is the culmination of 20 years of Project ALS research achievements in genetics, stem cell research, and basic research studies. These breakthroughs allow the Core to target ALS more accurately.

Recent discoveries in ALS genetics provide new therapeutic targets for the Core and beyond. For example, when Project ALS incorporated in 1998, only two genes had been identified as contributors to ALS disease. Now there are well over 50 ALS genes in play. Breakthroughs in genetics have also generated helpful new laboratory models of the disease. Project ALS piloted many of the first experiments to “turn off” gene mutations in ALS.

Additionally, the identification of new “constellations” or combinations of ALS genes across thousands of ALS patients has given researchers major clues about why the brain goes wrong in ALS, and exactly where to intervene therapeutically. For example, genetics findings led Project ALS research scientists to focus recently on the autophagy pathway—a new target in ALS treatment. Several drug candidates—including autophagy modulators–are now being tested in the Core.