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
Models Make Medicines: How Project ALS’ Focus on Multidisciplinary Models is Revolutionizing ALS Therapy Development
ALS has been a notoriously difficult disease to address therapeutically because of its heterogeneity in the clinic. The progression and symptoms of ALS differ broadly
Motor Neurons in a Dish: The Scientific Breakthrough that Fundamentally Changed ALS Research
In order to prevent, treat, or cure diseases, scientists rely on methods for identifying consistent mechanisms behind disease, and efficiently testing potential therapies on a
Viruses for Good, Not Evil: Tools for ALS Therapy Delivery
Motor neurons project out to the muscles in our limbs to drive muscle contraction, allowing us to speak, breathe, and move. These cells are housed
New Model and Gene Targeting Approach Show Promise for ALS Therapies
Researchers from the Project ALS Therapeutics Core at Columbia (the Core), in a groundbreaking collaborative effort with Ionis Pharmaceuticals, have developed a new tool for studying ALS mechanisms and therapies, as well as a novel approach for treating ALS caused by mutations…
Announcing the Project ALS Research Advisory Board
Project ALS Announces Research Advisory Board 2.0 Non-Profit ALS Research Leader Gathers a Diversity of Expertise to Guide Next-Phase Progress (New York, NY)
Amylyx, the Core, and the Power of Collaboration
On Wednesday, Amylyx Pharmaceuticals announced that they are planning to file a New Drug Application—the formal submission for a drug to be approved by the