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
Next Steps for Prosetin
Prosetin is moving to Phase I clinical trials. View the recording of our live conversation with clinical trial specialist Jinsy Andrews, MD, MSc and regulatory
Masitinib Voluntary Hold Reinforces Need for ALS-Focused Drug Development
On June 3rd, AB Science announced that they were putting all clinical studies of their lead compound masitinib—including a Phase 3 trial in ALS—on voluntary
The Hunt for ALS Biomarkers: Fat?
Updated October 2021: Check out new results from Dr. Estela Area-Gomez in Nature Scientific Reports The entire ALS community—patients, researchers, biotechs, doctors, regulators alike—agree on one thing:
CORE Update: Drug Discovery Progress at the Kirchhoff Family Drug Screening Unit
The core of THE CORE—its primary drug assessment platform—is the Kirchhoff Family Drug Screening Unit, where CORE researchers develop new cellular models of ALS, and
Neuroscience Publishes Special Issue Commemorating Project ALS Research Leader’s Legacy
The current issue of Neuroscience journal is dedicated to the staggering contributions to science made by longtime Project ALS Research Advisory Board Chair Thomas M. Jessell, PhD, before his death from the rare neurodegenerative disease progressive supranuclear palsy on April 28, 2019, at 67.
Project ALS – Winter 2020 News
While COVID-19 profoundly affected its day-to-day operations, The Project ALS Therapeutics Core at Columbia generated new approaches to disease modeling and genetic engineering technologies in 2020.