Promising ALS Therapy Moves Closer to Clinic
An experimental drug first tried at Columbia University Irving Medical Center as a last-ditch effort to help a 25-year-old woman with juvenile ALS is now being tested in ALS patients in a global, phase 3 clinical trial, based on promising results from a new study at Columbia.
Results of a study published today found that the drug—informally named jacifusen—lowered levels of FUS, a toxic protein in the woman’s neurons and in mice with the disease.
The clinical trial will be pivotal in determining if the drug can slow the progression of the disease.
"Though the drug did not save the life of the young woman who first received it, the study found that “it had a profound effect, virtually eliminating the toxic proteins in the central nervous system and reducing the burden of FUS pathology dramatically,” says study leader Neil Shneider, MD, PhD, the Claire Tow Associate Professor of Motor Neuron Disorders in the Department of Neurology and director of the Eleanor and Lou Gehrig ALS Center at Columbia University Vagelos College of Physicians and Surgeons. [read more]
Source: CUIMC Newsroom