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
New ALS Drug Screening Platform Identified; Leads to Early-Stage Drugs of Promise
Q&A with Project ALS Researcher Dr. Sebastian Thams, Karolinska Institute, on His Recent Discovery, Published in Molecular Therapy Can you describe your discovery that was
Kirchhoff Family Fellow Proposes New Therapeutic Strategy in ALS
Joesph Klim, PhD, a Kirchhoff Family Fellow at the Lab of Kevin Eggan, PhD at Harvard University has discovered that restoring expression of the gene
Doctors Track Relatives of ALS Patients for Clues on Disease: Project ALS and Columbia Launch ALS Families Project; Goal of Early Detection and Treatment
Project ALS and Columbia University’s Eleanor and Lou Gehrig ALS Center have opened the ALS Families Project to study relatives of ALS patients who carry
Autophagy Team Update
Last year, we announced the formation of a six-lab collaboration at the direction of Project ALS to focus on the cell process known as autophagy,
Promising Drugs Emerge from Project ALS Pre-Clinical Core at Columbia
Let’s be honest, for the most part, experts have failed to deliver potent drugs to clinical trial in ALS. Part of the reason is we
Scientists Achieve Next Step in ALS Stem Cell Research
Project ALS scientists in the Wichterle Lab at Columbia University have made a crucial breakthrough in stem cell research. The recent study, published in Neuron,