Project ALS funds critical drug testing at Columbia University and Massachusetts General Hospital/Harvard Medical School.

The Project ALS Pre-Clinical Core at Columbia is currently testing four drugs, including an all-new anti-inflammatory, and an FDA-approved cancer drug. The Core, led by Drs. Serge Przedborski and Hynek Wichterle, screens potential therapies in a wide range of ALS models, before delivering the most promising of them to human trial. “We’ve been able to test new, outside-the-box ideas that would otherwise have been difficult to fund. We used stem cell-derived motor neurons to screen a small library of FDA-approved drugs, and we’re now testing several promising candidates from that screen in a mouse model of ALS” said Dr. Emily Lowry, one of the researchers at the Pre-Clinical Core.

At Massachusetts General East Campus/Harvard Medical School, neuroscientist Dr. Ghazeleh Sadri-Vakili, one of Project ALS’ newest researchers, is using drugs to target neuroinflammation and innate immunity pathways. Her lead ALS drug is cromolyn sodium, which was originally marketed to treat asthma. Dr. Sadri-Vakili is aggressively testing whether cromolyn sodium extends survival in ALS mice, and demonstrates neuroprotection in other cell-based models of ALS. One of the big pros of this drug is its delivery: through an inhaler.