Drug Combination Slows Progression Of ALS And Could Mark ‘New Era’ In Treatment

Brain in motor neurone disease. FLAIR magnetic resonance imaging (MRI) scan of an axial section through the brain of a 32-old patient with motor neurone disease, showing hyperintensity of the pyramidal tracts. Motor neurone disease (also Charcot disease or amyotrophic lateral sclerosis), is a degenerative neurological disorder that involves the death of neurons (nerve cells).

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A combination of two experimental drugs appears to slow the decline of patients with amyotrophic lateral sclerosis, an illness often known as ALS or Lou Gehrig’s disease.

A six-month study of 137 patients with a fast-progressing form of the disease found that those who got daily doses of a two-drug combination called AMX0035 scored several points higher on a standard measure of function, a team reports in the Sept. 3 issue of The New England Journal of Medicine.

The difference was modest but meaningful to patients, said Dr. Sabrina Paganoni, the lead author and a researcher at Massachusetts General Hospital and Harvard Medical School.