ALS Super Models: The 2024 Edition 

Change is coming for ALS drug discovery. New and improved laboratory models of ALS now allow researchers and drug companies to study potential therapies more thoroughly– before they reach the clinical trial stage. In the old days—before  patient stem cell models of ALS were developed—identifying effective ALS therapies was a crapshoot, a series of educated guesses. Drugs that “looked good on paper” didn’t work in people with ALS—and clinical trials failed over 95% of the time.

We are in a new generation of discovery. The Project ALS Therapeutics Core and its partners are changing the drug discovery game by prioritizing the development of better, more predictive laboratory models of ALS. It stands to reason: If promising drugs and therapies “fix” ALS in relevant laboratory ALS disease models, they are more likely to help slow down ALS in real people.

The Project ALS Therapeutics Core is a leader in next generation ALS disease modeling. In 2008, Project ALS pioneered the first induced pluripotent stem cell models of ALS—or ALS in a Dish—limitless functional motor neurons derived directly from patient DNA. In recent years, the Project ALS Core has developed next generation discovery platforms made up of motor neurons and other brain cells derived directly from people with ALS. While they represent an improvement, current laboratory models of ALS leave room for upgrades. Stay tuned for improvements, as the Project ALS Core, the Packard Center for ALS Research, and others, join in a community-wide effort to improve drug screening via better laboratory disease models.