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.”
RESEARCH NEWS
SNEAK PEEK: Research Progress Highlighted at the Tomorrow is Tonight Virtual Fall Gala
Project ALS, along with the rest of the world, has faced serious headwinds this year—but we regrouped, focused our priorities, and have taken significant strides
Math Matters at the Project ALS CORE
The key to finding better drugs at the Project ALS Therapeutics Core at Columbia (THE CORE) is developing better pre-clinical laboratory models of ALS. A
Prosetin Receives Orphan Drug Designation
Earlier this month, we reached a big regulatory milestone: Prosetin received Orphan Drug Designation, or orphan status, for the treatment of ALS from the Food
Improving Pharmacokinetic Properties of Promising Compounds
In our biweekly webinars, we have described the process of developing Prosetin—the first potential investigational drug to emerge from The Project ALS Therapeutics Core at
COVID-19 UPDATE FROM THE CORE
Last year, Project ALS launched The Project ALS Therapeutics Core at Columbia (THE CORE), a 3-year, $6.3M initiative toward the first meaningful therapies for ALS.
Project ALS Endorses New ALS Legislation
Project ALS is encouraged by the introduction of two bills that aim to provide people with ALS faster, more robust access to experimental therapies. These