Experimental drug for ALS offers patients glimmer of hope

An experimental ALS drug from Cambridge, Mass.-based biotech Amylyx slowed neurological decline in a closely watched trial. The drug — a combination of sodium phenylbutrate and taurursodiol — helped patients retain a higher level of motor function than those given a placebo. While the results of this Phase 2/3 trial of 137 patients with particularly fast-progressing illness were described by some experts as "tantalizing," they also emphasized the need for a Phase 3 trial to confirm findings. Still, the results are encouraging for a disease whose exact cause is still a mystery and for which there is no cure and few available treatments.