Begun in 2017, the Project ALS Pre-Clinical Core (the Core) is the first rational, aggressive effort to rapidly and thoroughly test promising therapies for safety and potential efficacy in ALS before they reach human clinical trial.
Located at the Columbia University Motor Neuron Center in New York, in partnership with leading research universities and drug companies, the facility utilizes the most predictive laboratory models of ALS disease to fast-track the best drug candidates to human trial. Directed by Drs. Serge Przeborski, Hynek Wichterle, and Neil Shneider, the Core subjects drug candidates to a rigorous battery of testing to measure for such outcomes as dosing and delivery methods, brain penetrance, and, ultimately, target engagement and efficacy.
In its first year, the Core tested four potential ALS therapies: (1) An FDA approved drug called sunitinib, originally developed for the treatment of cancer (2) AMYLYX, a drug already being tested in ALS patients (3) alsterpaullone, a powerful molecule that may be neuroprotective (4) PB Compound, a novel drug developed by Core scientists that shows early signs of promise. This year the Core is testing eight additional drug candidates.
In 2020, Cathy and Richard called Project ALS after hearing about the development of a novel antisense oligonucleotide (ASO) therapy for people living with FUS ALS. So was born NextGen ALS, a Weber family initiative in collaboration with Project ALS that is committed to raising funds for targeted research toward treatments and cures for genetic forms of ALS.