Our Research

what is the core?

The Core is the world’s first and only partnership between a world-class academic institution and a leading nonprofit organization dedicated to a full-spectrum approach to ALS drug development. Our goal is to develop the first effective treatments, and ultimately a cure, for ALS.

CORE ACCOMPLISHMENTS

THE CORE has made significant progress toward better therapeutic options for ALS. These accomplishments are increasing the number and testing the feasibility of potential ALS therapies, improving possible diagnostic tools in the clinic, and expediting the transition of promising drug candidates into patient populations. Among its achievements, CORE researchers have:

Tested >1700 chemical compounds and FDA approved drugs for other indications in our in vitro screening unit,
Collaborated with >20 other academic groups and biotechs to assess their ALS compounds of interest in a range of pre-clinical models,

Developed a novel blood-based biomarker for ALS, which we are now validating for diagnostic and prognostic purposes,

Moved two potential ALS drugs, jacifusen and CK0801, to people with ALS, and

Developed an in-house drug, Prosetin, which can penetrate the brain through straightforward oral administration, measurably rescue stressed motor neurons in all of our ALS models, provably engage a cellular pathway of interest across neurodegeneration.

“For the first time, ALS patients can directly participate in research that will move us toward therapies that actually work…The Core provides an immensely exciting opportunity to capitalize on decades of ALS advances and translate them into meaningful treatments now.”

Neil Shneider, MD, PhD, Director of the Eleanor and Lou Gehrig ALS Center at Columbia