jueves, 3 de septiembre de 2020

Experimental ALS drug, dreamed up in a dorm room, offers patients hope

Experimental ALS drug, dreamed up in a dorm room, offers patients hope

The Readout

Damian Garde & Meghana Keshavan

ALS drug shows a modest (but welcome) benefit in a clinical trial

A treatment for amyotrophic lateral sclerosis, dreamt up in a college dorm, slowed neurological decline in a mid-stage study, demonstrating a small but promising benefit in a disease with few other options.

As STAT’s Meghana Keshavan reports, on a 48-point scale of disease severity, ALS patients who took the experimental drug saw their condition decline on average about 2.9 points less than those taking placebo over that six-month period. The results, published yesterday in the New England Journal of Medicine, point to a modest benefit but will be welcome news for the ALS community.

The data are also a major milestone for Cambridge, Mass., biotech Amylyx, which is developing the drug. The company, formed in 2013 by two Brown University undergraduates, has raised about $75 million to get to this point.

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